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Pembrolizumab monotherapy effective for rare melanoma
The findings could represent a new standard of treatment for this extremely rare tumor.
The study was inspired by a previous retrospective analysis which found an overall response rate of 77% and a complete response of 32% to anti–PD-1 monotherapy.
The ORR is about double what is seen in melanoma more generally, according to Kari Kendra, MD, PhD, who presented the study at the annual meeting of the American Association for Cancer Research.
“Our study was a positive study. Of note, in the retrospective study, they saw a complete response rate of 32%, which was amazingly similar to what we found. [The findings support] the use of single agent anti–PD-1 immunotherapy as first line treatment for most patients with unresectable desmoplastic melanoma. [There was 89% overall response and we saw] dramatic responses across the board,” said Dr. Kendra, who is a medical oncologist at Ohio State University Wexner Medical Center, Columbus.
The findings drew a strong reaction. “In a rare tumor session, to see response curves like that, it’s just outstanding,” said the session’s cochair Brian Van Tine, MD, PhD, who is a professor of medical oncology at Washington University in St. Louis.
“This really is one of the highest tumor response rates to immunotherapy that we are seeing in any cancer. And I think may also highlight the fact that we shouldn’t think of all cutaneous melanomas as one disease, given the heterogeneity in tumor responses based on some of the pathologic and molecular characteristics,” said Zeynep Aroglu, MD, who served as a discussant but was also one of the investigators who enrolled patients for the trial.
Desmoplastic melanoma represents about 4% of all cutaneous melanoma diagnoses, and its unique pathology can make it difficult to diagnose. That often leads to a late diagnosis, according to Dr. Aroglu. They typically occur in elderly patients, in the head and neck area, and are associated with sun exposure. DM also tends to have a high mutation burden, Dr. Aroglu said during the session.
It remains to be seen why there is such a high response rate in this tumor type, even among tumor types with mutation burdens that are nearly as high. DM tumors are often driven by neurofibromatosis type 1, but other tumors driven by NF-1 don’t have as high of a response rate to immunotherapy. The tumor environment could also play a role, she said.
“Is it a combination of all these factors? I think some of the ongoing analysis of tumor samples that Dr. Kendra mentioned may help to answer some of these questions,” Dr. Aroglu continued.
She also noted that the melanoma field is increasingly turning to combination of anti–PD-1 therapy with agents like that target LAG3 or CTLA4. Such combinations can achieve higher response rates, but at a cost of higher rates of grade 3-4 adverse events than anti–PD-1 inhibitors alone. “I wonder if for desmoplastic melanomas in light of this data, do we consider de-escalating therapy, given these very high response rates to PD-1 alone, given also the elderly age of many of these patients, because even the PD-1–LAG3 combo still has a higher rate of toxicity than PD-1 monotherapy. Perhaps the immunotherapy combinations can be reserved for those rare desmoplastic patients who are resistant to PD-1 alone,” said Dr. Aroglu.
Study details and adverse events
Twenty-seven patients were enrolled in the study; 93% were male, all were White, and 22% had elevated baseline lactate dehydrogenase. About 63% had disease located in the head and neck area, 33% experienced a complete response (P < .001), and 56% had a partial response for an ORR of 89%. The result surpassed the primary endpoint target of at least a 20% complete response rate.
The 2-year progression-free survival was 74%, and 2-year overall survival was 89%. The most common toxicities were fatigue (56%), diarrhea (33%), maculopapular rash (30%), pruritus (22%), anemia (19%), arthralgia (19%), and decreased lymphocyte count (19%). There were two grade 4 adverse events: a lipase increase and a lung infection accompanied by sepsis.
The researchers also carried out whole exome sequencing of biopsies and found that 67% had NF-1 loss of function mutations.
Dr. Aroglu has served on advisory boards for Pfizer, Array, Eisai, Genentech, Natera, Novartis, OncoSec, and Regeneron. She has received research support from Boehringer Ingelheim, Pfizer, and Novartis. Dr. Kendra has received institutional support from Bristol Myers-Squibb and trial support from CheckMate Pharmaceuticals, GlaxoSmithKline, Immunocore, Medspace, Merck, Novartis, and Varian Medical Systems. Dr. Van Tine has financial relationships with a wide range of pharmaceutical companies.
The findings could represent a new standard of treatment for this extremely rare tumor.
The study was inspired by a previous retrospective analysis which found an overall response rate of 77% and a complete response of 32% to anti–PD-1 monotherapy.
The ORR is about double what is seen in melanoma more generally, according to Kari Kendra, MD, PhD, who presented the study at the annual meeting of the American Association for Cancer Research.
“Our study was a positive study. Of note, in the retrospective study, they saw a complete response rate of 32%, which was amazingly similar to what we found. [The findings support] the use of single agent anti–PD-1 immunotherapy as first line treatment for most patients with unresectable desmoplastic melanoma. [There was 89% overall response and we saw] dramatic responses across the board,” said Dr. Kendra, who is a medical oncologist at Ohio State University Wexner Medical Center, Columbus.
The findings drew a strong reaction. “In a rare tumor session, to see response curves like that, it’s just outstanding,” said the session’s cochair Brian Van Tine, MD, PhD, who is a professor of medical oncology at Washington University in St. Louis.
“This really is one of the highest tumor response rates to immunotherapy that we are seeing in any cancer. And I think may also highlight the fact that we shouldn’t think of all cutaneous melanomas as one disease, given the heterogeneity in tumor responses based on some of the pathologic and molecular characteristics,” said Zeynep Aroglu, MD, who served as a discussant but was also one of the investigators who enrolled patients for the trial.
Desmoplastic melanoma represents about 4% of all cutaneous melanoma diagnoses, and its unique pathology can make it difficult to diagnose. That often leads to a late diagnosis, according to Dr. Aroglu. They typically occur in elderly patients, in the head and neck area, and are associated with sun exposure. DM also tends to have a high mutation burden, Dr. Aroglu said during the session.
It remains to be seen why there is such a high response rate in this tumor type, even among tumor types with mutation burdens that are nearly as high. DM tumors are often driven by neurofibromatosis type 1, but other tumors driven by NF-1 don’t have as high of a response rate to immunotherapy. The tumor environment could also play a role, she said.
“Is it a combination of all these factors? I think some of the ongoing analysis of tumor samples that Dr. Kendra mentioned may help to answer some of these questions,” Dr. Aroglu continued.
She also noted that the melanoma field is increasingly turning to combination of anti–PD-1 therapy with agents like that target LAG3 or CTLA4. Such combinations can achieve higher response rates, but at a cost of higher rates of grade 3-4 adverse events than anti–PD-1 inhibitors alone. “I wonder if for desmoplastic melanomas in light of this data, do we consider de-escalating therapy, given these very high response rates to PD-1 alone, given also the elderly age of many of these patients, because even the PD-1–LAG3 combo still has a higher rate of toxicity than PD-1 monotherapy. Perhaps the immunotherapy combinations can be reserved for those rare desmoplastic patients who are resistant to PD-1 alone,” said Dr. Aroglu.
Study details and adverse events
Twenty-seven patients were enrolled in the study; 93% were male, all were White, and 22% had elevated baseline lactate dehydrogenase. About 63% had disease located in the head and neck area, 33% experienced a complete response (P < .001), and 56% had a partial response for an ORR of 89%. The result surpassed the primary endpoint target of at least a 20% complete response rate.
The 2-year progression-free survival was 74%, and 2-year overall survival was 89%. The most common toxicities were fatigue (56%), diarrhea (33%), maculopapular rash (30%), pruritus (22%), anemia (19%), arthralgia (19%), and decreased lymphocyte count (19%). There were two grade 4 adverse events: a lipase increase and a lung infection accompanied by sepsis.
The researchers also carried out whole exome sequencing of biopsies and found that 67% had NF-1 loss of function mutations.
Dr. Aroglu has served on advisory boards for Pfizer, Array, Eisai, Genentech, Natera, Novartis, OncoSec, and Regeneron. She has received research support from Boehringer Ingelheim, Pfizer, and Novartis. Dr. Kendra has received institutional support from Bristol Myers-Squibb and trial support from CheckMate Pharmaceuticals, GlaxoSmithKline, Immunocore, Medspace, Merck, Novartis, and Varian Medical Systems. Dr. Van Tine has financial relationships with a wide range of pharmaceutical companies.
The findings could represent a new standard of treatment for this extremely rare tumor.
The study was inspired by a previous retrospective analysis which found an overall response rate of 77% and a complete response of 32% to anti–PD-1 monotherapy.
The ORR is about double what is seen in melanoma more generally, according to Kari Kendra, MD, PhD, who presented the study at the annual meeting of the American Association for Cancer Research.
“Our study was a positive study. Of note, in the retrospective study, they saw a complete response rate of 32%, which was amazingly similar to what we found. [The findings support] the use of single agent anti–PD-1 immunotherapy as first line treatment for most patients with unresectable desmoplastic melanoma. [There was 89% overall response and we saw] dramatic responses across the board,” said Dr. Kendra, who is a medical oncologist at Ohio State University Wexner Medical Center, Columbus.
The findings drew a strong reaction. “In a rare tumor session, to see response curves like that, it’s just outstanding,” said the session’s cochair Brian Van Tine, MD, PhD, who is a professor of medical oncology at Washington University in St. Louis.
“This really is one of the highest tumor response rates to immunotherapy that we are seeing in any cancer. And I think may also highlight the fact that we shouldn’t think of all cutaneous melanomas as one disease, given the heterogeneity in tumor responses based on some of the pathologic and molecular characteristics,” said Zeynep Aroglu, MD, who served as a discussant but was also one of the investigators who enrolled patients for the trial.
Desmoplastic melanoma represents about 4% of all cutaneous melanoma diagnoses, and its unique pathology can make it difficult to diagnose. That often leads to a late diagnosis, according to Dr. Aroglu. They typically occur in elderly patients, in the head and neck area, and are associated with sun exposure. DM also tends to have a high mutation burden, Dr. Aroglu said during the session.
It remains to be seen why there is such a high response rate in this tumor type, even among tumor types with mutation burdens that are nearly as high. DM tumors are often driven by neurofibromatosis type 1, but other tumors driven by NF-1 don’t have as high of a response rate to immunotherapy. The tumor environment could also play a role, she said.
“Is it a combination of all these factors? I think some of the ongoing analysis of tumor samples that Dr. Kendra mentioned may help to answer some of these questions,” Dr. Aroglu continued.
She also noted that the melanoma field is increasingly turning to combination of anti–PD-1 therapy with agents like that target LAG3 or CTLA4. Such combinations can achieve higher response rates, but at a cost of higher rates of grade 3-4 adverse events than anti–PD-1 inhibitors alone. “I wonder if for desmoplastic melanomas in light of this data, do we consider de-escalating therapy, given these very high response rates to PD-1 alone, given also the elderly age of many of these patients, because even the PD-1–LAG3 combo still has a higher rate of toxicity than PD-1 monotherapy. Perhaps the immunotherapy combinations can be reserved for those rare desmoplastic patients who are resistant to PD-1 alone,” said Dr. Aroglu.
Study details and adverse events
Twenty-seven patients were enrolled in the study; 93% were male, all were White, and 22% had elevated baseline lactate dehydrogenase. About 63% had disease located in the head and neck area, 33% experienced a complete response (P < .001), and 56% had a partial response for an ORR of 89%. The result surpassed the primary endpoint target of at least a 20% complete response rate.
The 2-year progression-free survival was 74%, and 2-year overall survival was 89%. The most common toxicities were fatigue (56%), diarrhea (33%), maculopapular rash (30%), pruritus (22%), anemia (19%), arthralgia (19%), and decreased lymphocyte count (19%). There were two grade 4 adverse events: a lipase increase and a lung infection accompanied by sepsis.
The researchers also carried out whole exome sequencing of biopsies and found that 67% had NF-1 loss of function mutations.
Dr. Aroglu has served on advisory boards for Pfizer, Array, Eisai, Genentech, Natera, Novartis, OncoSec, and Regeneron. She has received research support from Boehringer Ingelheim, Pfizer, and Novartis. Dr. Kendra has received institutional support from Bristol Myers-Squibb and trial support from CheckMate Pharmaceuticals, GlaxoSmithKline, Immunocore, Medspace, Merck, Novartis, and Varian Medical Systems. Dr. Van Tine has financial relationships with a wide range of pharmaceutical companies.
FROM AACR 2023
Cocaine damage can be misdiagnosed as nasal vasculitis
Nasal damage from cocaine use can be misdiagnosed as a rare, nonthreatening nasal disease, according to researchers from the United Kingdom.
Granulomatosis with polyangiitis (GPA), a disorder which causes inflammation in the nose, sinuses, throat, lungs, and kidneys, can have similar symptoms to cocaine-induced vasculitis, the researchers wrote. Drug testing can help identify patients who have cocaine-induced disease, they argued.
“Patients with destructive nasal lesions, especially young patients, should have urine toxicology performed for cocaine before diagnosing GPA and considering immunosuppressive therapy,” the authors wrote.
The paper was published in Rheumatology Advances in Practice.
Cocaine is the second-most popular drug in the United Kingdom, with 2.0% of people aged 16-59 years reporting using the drug in the past year. In the United States, about 1.7% of people aged 12 years and older (about 4.8 million people) used cocaine in the last 12 months, according to the 2021 National Survey on Drug Use and Health. The drug can cause midline destructive lesions, skin rash, and other vascular problems, and it can also trigger the production of antineutrophil cytoplasmic antibodies (ANCA) that lead to a clinical presentation that mimics GPA, which can make diagnosis more difficult. Treating cocaine-induced disease with immunosuppressant medication can be ineffective if the patient does not stop using the drug, and can have dangerous side effects, previous case studies suggest.
To better understand cocaine-induced disease, researchers conducted a review of patients who visited vasculitis clinics at Queen Elizabeth Hospital in Birmingham, England, and at the Royal Free Hospital in London between 2016 and 2021. They identified 42 patients with GPA-like symptoms who disclosed cocaine use or tested positive for the drug in urine toxicology test. The study included 23 men, 18 women, and 1 individual who did not identify with either gender. The median age was 41 years, and most patients were white.
Of those who underwent drug testing, more than 85% were positive. Nine patients who denied ever using cocaine were positive for the drug and 11 patients who said they were ex-users also tested positive via urine analysis. During clinical examinations, 30 patients had evidence of septal perforation, of which 6 had oronasal fistulas. Most patients’ symptoms were limited to the upper respiratory tract, though 12 did have other systemic symptoms, including skin lesions, joint pain, breathlessness, fatigue, and diplopia. Of the patients who received blood tests for ANCA, 87.5% tested positive for the antibodies.
The researchers noted that patients who continued cocaine use did not see improvement of symptoms, even if they were treated with immunosuppressant drugs.
“The experience in our two different centers suggests that discontinuation of cocaine is required to manage patients and that symptoms will persist despite immunosuppression if there is ongoing cocaine use,” the authors wrote.
“It can feel like chasing your tail at times if you’re trying to treat the inflammation but the real culprit – what’s driving the inflammation – is persistent,” Lindsay S. Lally, MD, a rheumatologist at the Hospital for Special Surgery in New York, said in an interview. She was not involved with the work.
Dr. Lally said the paper had a decent-sized cohort, and “helps us recognize that cocaine use is probably an under-recognized mimic of GPA, even though it’s something we all learn about and talk about.” She added that routine toxicology screening for patients deserves some consideration, though asking patients to complete a drug test could also undermine trust in the doctor-patient relationship. Patients who deny cocaine use may leave the office without providing a urine sample.
If Dr. Lally does suspect cocaine may be the cause of a patient’s systems, having a candid conversation with the patient may have a better chance at getting a patient to open up about their potential drug use. In practice, this means explaining “why it’s so important for me as their partner in this treatment to understand what factors are at play, and how dangerous it could potentially be if I was giving strong immunosuppressive medications [for a condition] that is being induced by a drug,” she said. “I do think that partnership and talking to the patients, at least in many patients, is more helpful than sort of the ‘gotcha’ moment” that can happen with drug testing.
The study authors disclosed no relevant financial relationships. Dr. Lally reported receiving consulting fees from Amgen.
A version of this article first appeared on Medscape.com.
Nasal damage from cocaine use can be misdiagnosed as a rare, nonthreatening nasal disease, according to researchers from the United Kingdom.
Granulomatosis with polyangiitis (GPA), a disorder which causes inflammation in the nose, sinuses, throat, lungs, and kidneys, can have similar symptoms to cocaine-induced vasculitis, the researchers wrote. Drug testing can help identify patients who have cocaine-induced disease, they argued.
“Patients with destructive nasal lesions, especially young patients, should have urine toxicology performed for cocaine before diagnosing GPA and considering immunosuppressive therapy,” the authors wrote.
The paper was published in Rheumatology Advances in Practice.
Cocaine is the second-most popular drug in the United Kingdom, with 2.0% of people aged 16-59 years reporting using the drug in the past year. In the United States, about 1.7% of people aged 12 years and older (about 4.8 million people) used cocaine in the last 12 months, according to the 2021 National Survey on Drug Use and Health. The drug can cause midline destructive lesions, skin rash, and other vascular problems, and it can also trigger the production of antineutrophil cytoplasmic antibodies (ANCA) that lead to a clinical presentation that mimics GPA, which can make diagnosis more difficult. Treating cocaine-induced disease with immunosuppressant medication can be ineffective if the patient does not stop using the drug, and can have dangerous side effects, previous case studies suggest.
To better understand cocaine-induced disease, researchers conducted a review of patients who visited vasculitis clinics at Queen Elizabeth Hospital in Birmingham, England, and at the Royal Free Hospital in London between 2016 and 2021. They identified 42 patients with GPA-like symptoms who disclosed cocaine use or tested positive for the drug in urine toxicology test. The study included 23 men, 18 women, and 1 individual who did not identify with either gender. The median age was 41 years, and most patients were white.
Of those who underwent drug testing, more than 85% were positive. Nine patients who denied ever using cocaine were positive for the drug and 11 patients who said they were ex-users also tested positive via urine analysis. During clinical examinations, 30 patients had evidence of septal perforation, of which 6 had oronasal fistulas. Most patients’ symptoms were limited to the upper respiratory tract, though 12 did have other systemic symptoms, including skin lesions, joint pain, breathlessness, fatigue, and diplopia. Of the patients who received blood tests for ANCA, 87.5% tested positive for the antibodies.
The researchers noted that patients who continued cocaine use did not see improvement of symptoms, even if they were treated with immunosuppressant drugs.
“The experience in our two different centers suggests that discontinuation of cocaine is required to manage patients and that symptoms will persist despite immunosuppression if there is ongoing cocaine use,” the authors wrote.
“It can feel like chasing your tail at times if you’re trying to treat the inflammation but the real culprit – what’s driving the inflammation – is persistent,” Lindsay S. Lally, MD, a rheumatologist at the Hospital for Special Surgery in New York, said in an interview. She was not involved with the work.
Dr. Lally said the paper had a decent-sized cohort, and “helps us recognize that cocaine use is probably an under-recognized mimic of GPA, even though it’s something we all learn about and talk about.” She added that routine toxicology screening for patients deserves some consideration, though asking patients to complete a drug test could also undermine trust in the doctor-patient relationship. Patients who deny cocaine use may leave the office without providing a urine sample.
If Dr. Lally does suspect cocaine may be the cause of a patient’s systems, having a candid conversation with the patient may have a better chance at getting a patient to open up about their potential drug use. In practice, this means explaining “why it’s so important for me as their partner in this treatment to understand what factors are at play, and how dangerous it could potentially be if I was giving strong immunosuppressive medications [for a condition] that is being induced by a drug,” she said. “I do think that partnership and talking to the patients, at least in many patients, is more helpful than sort of the ‘gotcha’ moment” that can happen with drug testing.
The study authors disclosed no relevant financial relationships. Dr. Lally reported receiving consulting fees from Amgen.
A version of this article first appeared on Medscape.com.
Nasal damage from cocaine use can be misdiagnosed as a rare, nonthreatening nasal disease, according to researchers from the United Kingdom.
Granulomatosis with polyangiitis (GPA), a disorder which causes inflammation in the nose, sinuses, throat, lungs, and kidneys, can have similar symptoms to cocaine-induced vasculitis, the researchers wrote. Drug testing can help identify patients who have cocaine-induced disease, they argued.
“Patients with destructive nasal lesions, especially young patients, should have urine toxicology performed for cocaine before diagnosing GPA and considering immunosuppressive therapy,” the authors wrote.
The paper was published in Rheumatology Advances in Practice.
Cocaine is the second-most popular drug in the United Kingdom, with 2.0% of people aged 16-59 years reporting using the drug in the past year. In the United States, about 1.7% of people aged 12 years and older (about 4.8 million people) used cocaine in the last 12 months, according to the 2021 National Survey on Drug Use and Health. The drug can cause midline destructive lesions, skin rash, and other vascular problems, and it can also trigger the production of antineutrophil cytoplasmic antibodies (ANCA) that lead to a clinical presentation that mimics GPA, which can make diagnosis more difficult. Treating cocaine-induced disease with immunosuppressant medication can be ineffective if the patient does not stop using the drug, and can have dangerous side effects, previous case studies suggest.
To better understand cocaine-induced disease, researchers conducted a review of patients who visited vasculitis clinics at Queen Elizabeth Hospital in Birmingham, England, and at the Royal Free Hospital in London between 2016 and 2021. They identified 42 patients with GPA-like symptoms who disclosed cocaine use or tested positive for the drug in urine toxicology test. The study included 23 men, 18 women, and 1 individual who did not identify with either gender. The median age was 41 years, and most patients were white.
Of those who underwent drug testing, more than 85% were positive. Nine patients who denied ever using cocaine were positive for the drug and 11 patients who said they were ex-users also tested positive via urine analysis. During clinical examinations, 30 patients had evidence of septal perforation, of which 6 had oronasal fistulas. Most patients’ symptoms were limited to the upper respiratory tract, though 12 did have other systemic symptoms, including skin lesions, joint pain, breathlessness, fatigue, and diplopia. Of the patients who received blood tests for ANCA, 87.5% tested positive for the antibodies.
The researchers noted that patients who continued cocaine use did not see improvement of symptoms, even if they were treated with immunosuppressant drugs.
“The experience in our two different centers suggests that discontinuation of cocaine is required to manage patients and that symptoms will persist despite immunosuppression if there is ongoing cocaine use,” the authors wrote.
“It can feel like chasing your tail at times if you’re trying to treat the inflammation but the real culprit – what’s driving the inflammation – is persistent,” Lindsay S. Lally, MD, a rheumatologist at the Hospital for Special Surgery in New York, said in an interview. She was not involved with the work.
Dr. Lally said the paper had a decent-sized cohort, and “helps us recognize that cocaine use is probably an under-recognized mimic of GPA, even though it’s something we all learn about and talk about.” She added that routine toxicology screening for patients deserves some consideration, though asking patients to complete a drug test could also undermine trust in the doctor-patient relationship. Patients who deny cocaine use may leave the office without providing a urine sample.
If Dr. Lally does suspect cocaine may be the cause of a patient’s systems, having a candid conversation with the patient may have a better chance at getting a patient to open up about their potential drug use. In practice, this means explaining “why it’s so important for me as their partner in this treatment to understand what factors are at play, and how dangerous it could potentially be if I was giving strong immunosuppressive medications [for a condition] that is being induced by a drug,” she said. “I do think that partnership and talking to the patients, at least in many patients, is more helpful than sort of the ‘gotcha’ moment” that can happen with drug testing.
The study authors disclosed no relevant financial relationships. Dr. Lally reported receiving consulting fees from Amgen.
A version of this article first appeared on Medscape.com.
FROM RHEUMATOLOGY ADVANCES IN PRACTICE
Study compares noninvasive treatments of cutaneous neurofibromas
PHOENIX – after only one treatment, according to preliminary results of an ongoing prospective trial that compared several treatment modalities.
“Neurofibromatosis type 1 is the most common single-gene disease of mankind, but there is so much we have yet to learn about it,” study author Patricia Richey, MD, who practices Mohs surgery and cosmetic dermatology in Washington, D.C., said in an interview in advance of the annual conference of the American Society for Laser Medicine and Surgery, where she presented the results during an abstract session. Dr. Richey also conducts research for the Wellman Center for Photomedicine and the Dermatology Laser and Cosmetic Center at Massachusetts General Hospital, Boston, and is working with R. Rox Anderson, MD, director of the Wellman Center, on this project. In his words, she said, “the lack of better treatments for cNF is a ‘problem worth solving.’ ”
“The accepted and widely available treatments for cNF result in scars and hypopigmentation. Our treatments do not,” she added. Since the epidermis overlying cNF is normal, “there is no reason to use nonselective or surgical methods and destroy a perfectly good epidermis when you don’t need to.”
Four treatments vs. controls
For the study, Dr. Richey and colleagues enrolled 19 adults with a total of 307 cNFs measuring 2-4 mm in size to receive one of four treatments: electrocautery with an insulated radiofrequency needle; 755-nm alexandrite laser with negative pressure (8-mm spot size, 100 J/cm2 fluence, 3-ms pulse duration); 980-nm diode laser (delivered via 8-mm sapphire skin-contact window), and intratumoral injection of 10 mg/mL deoxycholic acid at a volume approximately equal to that of the tumor. The average age of the participants was 49 years and 15 were female.
The investigators applied 5% lidocaine/prilocaine for 40 minutes to treatment sites before randomizing the tumors to treatment or to the control arm (no treatment). They compared safety, tolerability (including pain scores), and efficacy of each modality as measured by the change in cNF volume/height via three-dimensional imaging and clinical improvement via physician assessment at 6 months. All 19 participants have completed the 6-month assessment.
All modalities reduced or eliminated some of the cNFs by 6 months after treatment, with statistically significant reductions in height and volume across all four treatments. A wide variation of responses was observed. Specifically, the mean tumor volume changes for each modality, compared with controls, were –33.4% versus –5.1% among those treated with the 755-nm alexandrite laser; –24.9% versus –9.2% among those treated with the 980-nm diode laser, –23.3% versus –0.8% among those treated with insulated-needle radiofrequency coagulation, and –29.4% versus –3.7% among those treated with deoxycholic acid.
The variation in responses “may be due to histologic diversity of cNF or may indicate a need for more fine-tuned dosimetry, or a combination,” Dr. Richey said. “Our future trials will address this. We will also be treating all skin types in our upcoming trials.”
No adverse events categorized as higher than grade 2 occurred in any of the treatment groups, and no signs of regrowth or growth stimulation have been observed to date.
Tolerability of treatments
As for general tolerability, the 980-nm laser treatment caused moderate to severe pain; the alexandrite laser caused mild pain; insulated-needle radiofrequency coagulation caused mild pain, though more than deoxycholic acid injections or alexandrite laser, and pain associated with the deoxycholic acid injections was minimal.
When residual neurofibroma tumor was present histologically, its appearance was similar to that of untreated tumors in controls. There was no evidence of atypia, mitosis, or tumor inflammation, and mild fibrosis was present at the sites of prior tumor.
“It was surprising that all four modalities did work to some extent,” Dr. Richey said, noting that the lack of ulceration with deoxycholic acid injection “was pleasantly surprising.” Treatment with the 980-nm diode laser “was a bit more painful than we anticipated.”
The positive results of this trial has raised “more questions for us to answer. We have three additional trials in the works to fine tune these treatments and optimize dose/delivery, with the end goal of treating younger people.”
Dr. Richey said that she was “amazed” by how motivated the enrollees were to participate in the trial, noting that many patients with cNF undergo general anesthesia to have dozens of tumors surgically removed at once. “They pay $10,000-$20,000 on average out of pocket, as this surgery is considered cosmetic,” she said.
“This very important study could lead to effective, relatively noninvasive, therapy for small neurofibromas,” said Jeffrey S. Dover, MD, codirector of SkinCare Physicians in Chestnut Hill, Mass., who was not involved with the study and was asked to comment on the results.
“Remarkably, all four treatments worked to varying degrees, but of all the treatments, the selective alexandrite laser appeared to achieve the best results. Further study will be needed to see just how effective these treatments are, and to determine the best and safest treatment parameters. Given how common this autosomal dominant disease is, and how disfiguring neurofibromas become as they enlarge, a well-tolerated noninvasive nonsurgical treatment with limited side effects is highly sought after.”
The study, which was named the best clinical abstract at the meeting, was supported by the Neurofibromatosis Therapeutic Acceleration Program. Dr. Anderson is supported in part as the Lancer Endowed Chair in Dermatology at MGH. Dr. Dover reported having no relevant disclosures.
PHOENIX – after only one treatment, according to preliminary results of an ongoing prospective trial that compared several treatment modalities.
“Neurofibromatosis type 1 is the most common single-gene disease of mankind, but there is so much we have yet to learn about it,” study author Patricia Richey, MD, who practices Mohs surgery and cosmetic dermatology in Washington, D.C., said in an interview in advance of the annual conference of the American Society for Laser Medicine and Surgery, where she presented the results during an abstract session. Dr. Richey also conducts research for the Wellman Center for Photomedicine and the Dermatology Laser and Cosmetic Center at Massachusetts General Hospital, Boston, and is working with R. Rox Anderson, MD, director of the Wellman Center, on this project. In his words, she said, “the lack of better treatments for cNF is a ‘problem worth solving.’ ”
“The accepted and widely available treatments for cNF result in scars and hypopigmentation. Our treatments do not,” she added. Since the epidermis overlying cNF is normal, “there is no reason to use nonselective or surgical methods and destroy a perfectly good epidermis when you don’t need to.”
Four treatments vs. controls
For the study, Dr. Richey and colleagues enrolled 19 adults with a total of 307 cNFs measuring 2-4 mm in size to receive one of four treatments: electrocautery with an insulated radiofrequency needle; 755-nm alexandrite laser with negative pressure (8-mm spot size, 100 J/cm2 fluence, 3-ms pulse duration); 980-nm diode laser (delivered via 8-mm sapphire skin-contact window), and intratumoral injection of 10 mg/mL deoxycholic acid at a volume approximately equal to that of the tumor. The average age of the participants was 49 years and 15 were female.
The investigators applied 5% lidocaine/prilocaine for 40 minutes to treatment sites before randomizing the tumors to treatment or to the control arm (no treatment). They compared safety, tolerability (including pain scores), and efficacy of each modality as measured by the change in cNF volume/height via three-dimensional imaging and clinical improvement via physician assessment at 6 months. All 19 participants have completed the 6-month assessment.
All modalities reduced or eliminated some of the cNFs by 6 months after treatment, with statistically significant reductions in height and volume across all four treatments. A wide variation of responses was observed. Specifically, the mean tumor volume changes for each modality, compared with controls, were –33.4% versus –5.1% among those treated with the 755-nm alexandrite laser; –24.9% versus –9.2% among those treated with the 980-nm diode laser, –23.3% versus –0.8% among those treated with insulated-needle radiofrequency coagulation, and –29.4% versus –3.7% among those treated with deoxycholic acid.
The variation in responses “may be due to histologic diversity of cNF or may indicate a need for more fine-tuned dosimetry, or a combination,” Dr. Richey said. “Our future trials will address this. We will also be treating all skin types in our upcoming trials.”
No adverse events categorized as higher than grade 2 occurred in any of the treatment groups, and no signs of regrowth or growth stimulation have been observed to date.
Tolerability of treatments
As for general tolerability, the 980-nm laser treatment caused moderate to severe pain; the alexandrite laser caused mild pain; insulated-needle radiofrequency coagulation caused mild pain, though more than deoxycholic acid injections or alexandrite laser, and pain associated with the deoxycholic acid injections was minimal.
When residual neurofibroma tumor was present histologically, its appearance was similar to that of untreated tumors in controls. There was no evidence of atypia, mitosis, or tumor inflammation, and mild fibrosis was present at the sites of prior tumor.
“It was surprising that all four modalities did work to some extent,” Dr. Richey said, noting that the lack of ulceration with deoxycholic acid injection “was pleasantly surprising.” Treatment with the 980-nm diode laser “was a bit more painful than we anticipated.”
The positive results of this trial has raised “more questions for us to answer. We have three additional trials in the works to fine tune these treatments and optimize dose/delivery, with the end goal of treating younger people.”
Dr. Richey said that she was “amazed” by how motivated the enrollees were to participate in the trial, noting that many patients with cNF undergo general anesthesia to have dozens of tumors surgically removed at once. “They pay $10,000-$20,000 on average out of pocket, as this surgery is considered cosmetic,” she said.
“This very important study could lead to effective, relatively noninvasive, therapy for small neurofibromas,” said Jeffrey S. Dover, MD, codirector of SkinCare Physicians in Chestnut Hill, Mass., who was not involved with the study and was asked to comment on the results.
“Remarkably, all four treatments worked to varying degrees, but of all the treatments, the selective alexandrite laser appeared to achieve the best results. Further study will be needed to see just how effective these treatments are, and to determine the best and safest treatment parameters. Given how common this autosomal dominant disease is, and how disfiguring neurofibromas become as they enlarge, a well-tolerated noninvasive nonsurgical treatment with limited side effects is highly sought after.”
The study, which was named the best clinical abstract at the meeting, was supported by the Neurofibromatosis Therapeutic Acceleration Program. Dr. Anderson is supported in part as the Lancer Endowed Chair in Dermatology at MGH. Dr. Dover reported having no relevant disclosures.
PHOENIX – after only one treatment, according to preliminary results of an ongoing prospective trial that compared several treatment modalities.
“Neurofibromatosis type 1 is the most common single-gene disease of mankind, but there is so much we have yet to learn about it,” study author Patricia Richey, MD, who practices Mohs surgery and cosmetic dermatology in Washington, D.C., said in an interview in advance of the annual conference of the American Society for Laser Medicine and Surgery, where she presented the results during an abstract session. Dr. Richey also conducts research for the Wellman Center for Photomedicine and the Dermatology Laser and Cosmetic Center at Massachusetts General Hospital, Boston, and is working with R. Rox Anderson, MD, director of the Wellman Center, on this project. In his words, she said, “the lack of better treatments for cNF is a ‘problem worth solving.’ ”
“The accepted and widely available treatments for cNF result in scars and hypopigmentation. Our treatments do not,” she added. Since the epidermis overlying cNF is normal, “there is no reason to use nonselective or surgical methods and destroy a perfectly good epidermis when you don’t need to.”
Four treatments vs. controls
For the study, Dr. Richey and colleagues enrolled 19 adults with a total of 307 cNFs measuring 2-4 mm in size to receive one of four treatments: electrocautery with an insulated radiofrequency needle; 755-nm alexandrite laser with negative pressure (8-mm spot size, 100 J/cm2 fluence, 3-ms pulse duration); 980-nm diode laser (delivered via 8-mm sapphire skin-contact window), and intratumoral injection of 10 mg/mL deoxycholic acid at a volume approximately equal to that of the tumor. The average age of the participants was 49 years and 15 were female.
The investigators applied 5% lidocaine/prilocaine for 40 minutes to treatment sites before randomizing the tumors to treatment or to the control arm (no treatment). They compared safety, tolerability (including pain scores), and efficacy of each modality as measured by the change in cNF volume/height via three-dimensional imaging and clinical improvement via physician assessment at 6 months. All 19 participants have completed the 6-month assessment.
All modalities reduced or eliminated some of the cNFs by 6 months after treatment, with statistically significant reductions in height and volume across all four treatments. A wide variation of responses was observed. Specifically, the mean tumor volume changes for each modality, compared with controls, were –33.4% versus –5.1% among those treated with the 755-nm alexandrite laser; –24.9% versus –9.2% among those treated with the 980-nm diode laser, –23.3% versus –0.8% among those treated with insulated-needle radiofrequency coagulation, and –29.4% versus –3.7% among those treated with deoxycholic acid.
The variation in responses “may be due to histologic diversity of cNF or may indicate a need for more fine-tuned dosimetry, or a combination,” Dr. Richey said. “Our future trials will address this. We will also be treating all skin types in our upcoming trials.”
No adverse events categorized as higher than grade 2 occurred in any of the treatment groups, and no signs of regrowth or growth stimulation have been observed to date.
Tolerability of treatments
As for general tolerability, the 980-nm laser treatment caused moderate to severe pain; the alexandrite laser caused mild pain; insulated-needle radiofrequency coagulation caused mild pain, though more than deoxycholic acid injections or alexandrite laser, and pain associated with the deoxycholic acid injections was minimal.
When residual neurofibroma tumor was present histologically, its appearance was similar to that of untreated tumors in controls. There was no evidence of atypia, mitosis, or tumor inflammation, and mild fibrosis was present at the sites of prior tumor.
“It was surprising that all four modalities did work to some extent,” Dr. Richey said, noting that the lack of ulceration with deoxycholic acid injection “was pleasantly surprising.” Treatment with the 980-nm diode laser “was a bit more painful than we anticipated.”
The positive results of this trial has raised “more questions for us to answer. We have three additional trials in the works to fine tune these treatments and optimize dose/delivery, with the end goal of treating younger people.”
Dr. Richey said that she was “amazed” by how motivated the enrollees were to participate in the trial, noting that many patients with cNF undergo general anesthesia to have dozens of tumors surgically removed at once. “They pay $10,000-$20,000 on average out of pocket, as this surgery is considered cosmetic,” she said.
“This very important study could lead to effective, relatively noninvasive, therapy for small neurofibromas,” said Jeffrey S. Dover, MD, codirector of SkinCare Physicians in Chestnut Hill, Mass., who was not involved with the study and was asked to comment on the results.
“Remarkably, all four treatments worked to varying degrees, but of all the treatments, the selective alexandrite laser appeared to achieve the best results. Further study will be needed to see just how effective these treatments are, and to determine the best and safest treatment parameters. Given how common this autosomal dominant disease is, and how disfiguring neurofibromas become as they enlarge, a well-tolerated noninvasive nonsurgical treatment with limited side effects is highly sought after.”
The study, which was named the best clinical abstract at the meeting, was supported by the Neurofibromatosis Therapeutic Acceleration Program. Dr. Anderson is supported in part as the Lancer Endowed Chair in Dermatology at MGH. Dr. Dover reported having no relevant disclosures.
AT ASLMS 2023
Answers sought for mental health challenges in pediatric rheumatology patients
NEW ORLEANS – Pediatric patients with rheumatologic diseases experience a particularly high prevalence of psychological distress and depression and anxiety symptoms, according to research presented at the Pediatric Rheumatology Symposium. Although this finding is not necessarily surprising, the extent to which depression and psychological distress impacts these young patients’ quality of life has led to greater research and innovation in seeking ways to identify, address, and treat depression and anxiety in children and adolescents with diseases such as juvenile idiopathic arthritis (JIA) or systemic lupus erythematosus (SLE).
Accordingly, other studies presented at the conference examined more efficient ways to screen adolescent patients for depression and assessed programs designed to improve symptoms. In fact, the American College of Rheumatology award for the top Quality, Health Services, and Education Research abstract at this year’s symposium went to Lauren Harper, MD, a pediatric rheumatology fellow at Nationwide Children’s Hospital, Columbus, whose research examined the effects of automating depression screening during check-in for adolescent patients with SLE. Her findings revealed that automation of screening increased detection of depression and suicidality, thereby increasing interventions and ultimately resulting in a reduction in depression prevalence.
“The key clinical takeaway is that mental health screening is really important – it affects our patients in so many different ways – and it’s very doable in your rheumatology clinic,” Dr. Harper said in an interview. “It’s also important because they’re coming to us very frequently, but they don’t see their PCP [primary care provider] very often, so we can’t leave screening to the PCPs.”
Two other studies assessed the effectiveness of a 6-week cognitive-behavioral intervention for youth called Treatment and Education Approach for Childhood-Onset Lupus (TEACH). One study found that remote delivery of TEACH resulted in improved mood symptoms and reduced fatigue, and another found the program particularly effective in improving mood for patients deemed “high risk” because of greater depression and fatigue symptoms.
The impact of growing mental health problems has been enormous both in the pediatric rheumatology population and society at large, Daria Sosna, MSc, a research coordinator at the University of Calgary (Alta.), said in an interview as she visited the research posters related to psychological stress and depression.
“We need to do something,” said Ms. Sosna, whose department is currently applying for funding to develop a research project to improve mental health outcomes in adolescents with lupus. “This population, specifically, has higher numbers than anyone else does because they have chronic illness” – and those issues need to be addressed.
High psychological stress levels
The study looking at psychological stress in pediatric rheumatology patients, led by Natalie Rosenwasser, MD, of Seattle Children’s Hospital, relied on cross-sectional data from patients enrolled in two Childhood Arthritis and Rheumatology Research Alliance sites, one in Utah and one in Seattle. The average age of the 71 patients who completed the surveys was 13, and the researchers reported the findings in two separate age groups: those aged 13-17, who completed the surveys themselves, and those aged 8-12, whose parents completed the surveys. Nearly all the patients (94.4%) had JIA, but one had lupus and three had juvenile dermatomyositis.
The participants completed the Patient-Reported Outcomes Measurement Information System (PROMIS) for psychological stress, physical stress, and depressive symptoms. They also filled out the National Institutes of Health–Toolbox Perceived Stress survey, the 9-item Patient Health Questionnaire (PHQ-9), the Screen for Child Anxiety Related Disorders (SCARED), a visual analog scale for COVID-related distress, and a questionnaire asking about how receptive they were to mental health screening. The researchers determined that a score 1 standard deviation above the mean on the PROMIS and NIH-Toolbox assessments qualified as a high level of psychological stress.
“There are data that suggest that psychological stress can be a precursor to depression and anxiety, which raises the concern that not every patient who’s experiencing mental health symptoms is going to be picked up on traditional measures that meet that clinical threshold, but they may really need interventions to protect their mental health,” presenter Erin Treemarcki, DO, an assistant professor of pediatric rheumatology at the University of Utah, Salt Lake City, said in an interview. “Not every patient may necessarily need referral to a mental health specialist, but there are still potential interventions that we can do in the clinical setting to address mental health, which in turn can improve outcomes, including medication compliance and knowing how patients are feeling.”
More than one-third of the patients (39%) reported a high level of psychological stress, and 43% had elevated physical stress. Broken down by age, 26% of the teens and 15% of the younger patients reported high levels of perceived stress. The PROMIS only identified increased depressive symptoms in 26% of the participants, whereas more than half (54%) had a positive PHQ-9 depression screen. Furthermore, half the patients had SCARED scores (50%) that likely indicated anxiety disorder. Only 6% of patients reported severe stress specifically related to the pandemic, but most reported mild distress from the pandemic.
“Psychological stress was highly correlated with physical stress, perceived stress, depressive symptoms [PROMIS and PHQ-9], and anxiety,” the authors reported (P < .05). The authors next plan to expand their assessment to a third CARRA site and then explore the interaction between psychological distress and sociodemographic factors.
“There’s such an increase in mental health disorders right now, and we’re overwhelmed in general,” Ms. Sosna said in an interview. “There have to be interventions that approach this. We can use pharmacological approaches, we can use CBT, we can use a lot of these things that are very well established, and they’re absolutely fantastic, but we don’t necessarily have the resources or capabilities to do that all the time.”
Benefits of automated depression screening
To reduce the likelihood of depression screenings falling through the cracks during visits, Dr. Harper’s study assessed the impact of automating screens in an adolescent population. In her presentation, she noted previous research finding that nearly half of youth with lupus (47%) had depression, compared with 24% of adults with lupus. Pediatric patients have nearly three times the odds of depression and more than five times the odds of suicidal ideation, she told attendees. These mood disorders are correlated with greater physical disability, higher cardiovascular risk, more disease activity, higher risk of premature death, and decreased educational attainment, medication compliance, and quality of life.
Despite recommendations for depression screening from the U.S. Preventive Services Task Force and the American Academy of Pediatrics, only 2% of pediatric rheumatology patients are routinely screened for depression with a validated instrument, and only 7% of those with depressive symptoms are screened, according to a 2016 study that Dr. Harper cited. Yet the same study found that nearly all pediatric rheumatologists (95%) supported routine depression screening every 6-12 months. Hence her team’s decision to test whether automating screening improved their screening rates.
Their population included lupus patients aged 12 and older seen at Nationwide Children’s Hospital between 2014 and 2022. Initially, patients completed the PHQ-9 on paper, which was then transcribed into the electronic health record. The process became automated and administered on an iPad at every visit in 2022. Positive screens – those endorsing suicidality or with a score of at least 10 – caused an alert to pop up for clinicians during their workflow so that they would talk to the mental health team about the patient’s needs.
A total of 149 patients completed 529 screenings during the study’s 8 years. Only 1 patient completed a PHQ-9 in 2014, which increased to just 17 patients in 2017. Automation resulted in 225 screens (P < .01). Subsequently, positive screens increased from 0% in 2014 to 25%-30% in 2018-2021, but then fell to 12% in 2022 (P < .01). The median PHQ-9 score was 3; overall scores decreased as screening increased.
The overall incidence of positive screens during the study period was 20% and prevalence was 38%, the authors reported. Of the 10 automated alerts triggered by positive screens, 90% resulted in a meeting with a psychologist or social worker, and 90% completed a suicide risk assessment. The intrusive alert for clinicians requires them to acknowledge the alert, agreeing to initiate a risk assessment, before they can enter data into the patient’s chart.
The study findings reveal “that you can successfully screen a high-risk population using an automated, seamless process, and you can alert providers without too much disruption to their typical clinic flow,” Dr. Harper told attendees. “And all of these processes have led to sustainability for routine depression screening in our lupus clinic.”
Dr. Harper’s team next plans to expand the automated screenings to populations with other diseases, to add an automated screening for anxiety, and to explore how PHQ-9 scores correlate with disease activity.
Treating patients’ mental health
Another two other abstracts at the symposium looked at another option, the 6-week cognitive-behavioral TEACH program. Deborah Levy, MD, MS, an associate professor of pediatrics at the University of Toronto and the clinical director of rheumatology at The Hospital for Sick Children, and colleagues assessed the program’s success when delivered remotely to adolescent patients with lupus. Pilot testing with TEACH had already shown improvements in fatigue and mood, Dr. Levy told attendees, but barriers to in-person delivery limited its utility even before the pandemic, so this study aimed to determine a remote version’s feasibility and effects, compared with treatment as usual.
The randomized, controlled trial, led by Natoshia Cunningham, PhD, from Michigan State University, Grand Rapids, included 57 participants, aged 12-22, from seven U.S. and Canadian rheumatology sites. All had been diagnosed with childhood-onset SLE by age 18 and had elevated symptoms in fatigue, pain, or depression. A PROMIS Fatigue T score of 60 or greater indicated elevated fatigue scores, whereas a high pain score was at least a 3/10 on a visual analog scale, and a high depression T score was at least a 60 but not higher than 80 on the Children’s Depression Inventory–2 or the Beck Depression Inventory–II (depending on the patient’s age).
Patients with other chronic medical conditions, developmental delays, or untreated major psychiatric illness were excluded from the study, as were patients who were receiving overlapping treatment, such as cognitive-behavioral therapy for pain or mood. Thirty patients were randomly assigned to receive treatment as usual while 27 patients were assigned to participate in the remote TEACH program.
Nearly all the patients (94%) were female, but they were racially diverse, with 42% White, 28% Asian, 19% Black, 19% Hispanic, and 4% multiracial. The patients were an average 16 years old and had been diagnosed for a median 5 years. Three of the intervention’s six modules involved the caregivers or, for older patients, their partners if desired. The communication strategies taught in the program were also tailored to patients’ ages.
“All of these strategies are educational, cognitive, behavioral, mindfulness strategies that target fatigue [and] pain, and they also developed web content for participants to use on their own,” Dr. Levy told attendees.
The researchers had complete postassessment data from 88% of participants, but they also reported some of the statements made during qualitative interviews about the program’s feasibility.
“I think it makes people more aware of themselves to become a better version of themselves, whether that’s in their normal life or in handling a lupus kind of life,” one participant said about the program’s benefits. Another appreciated the “alternative ways of thinking,” including “being more mindful of my thoughts and how those kind of aggravate my stress.”
The quantitative findings revealed a statistically significant reduction in depressive symptoms and fatigue for TEACH participants, compared with treatment as usual. Mood scores fell by an average 13.7 points in the TEACH group, compared with a drop of 2.4 points in the treatment as usual group (P < .001). Scores for fatigue fell 9.16 points in the TEACH group and 2.93 in the control group (P = .003). No statistically significant difference showed up in pain scores between the groups, although pain, medication adherence, and disease activity did improve slightly more in the TEACH group.
In addition to the significant improvements in mood and fatigue, therefore, “completion of TEACH may be associated with improved medication adherence and disease activity versus treatment as usual,” Dr. Levy said.
A much smaller study authored by some of the same researchers also assessed TEACH’s impact not in remote form but in terms of its value specifically for adolescent patients with SLE and elevated depression and fatigue scores. Comparison of 6 high-risk patients with 10 low-risk patients who underwent TEACH suggested that the program was especially effective for improving depression in high-risk patients since these patients had a statistically significantly greater improvement in mood. Fatigue, pain, anxiety, quality of life, and disease activity scores did not statistically differ between the groups.
Authors of the automated depression screening study reported no disclosures or outside funding. The study assessing psychological distress was funded by a CARRA–Arthritis Foundation grant, and the authors reported no disclosures. The remote TEACH study was funded by a CARRA–Arthritis Foundation grant, and all but one author reported no disclosures. One author had disclosures with Janssen, Roche, and Sobi. The high-risk TEACH study was also funded by a CARRA grant, and the authors had no disclosures.
NEW ORLEANS – Pediatric patients with rheumatologic diseases experience a particularly high prevalence of psychological distress and depression and anxiety symptoms, according to research presented at the Pediatric Rheumatology Symposium. Although this finding is not necessarily surprising, the extent to which depression and psychological distress impacts these young patients’ quality of life has led to greater research and innovation in seeking ways to identify, address, and treat depression and anxiety in children and adolescents with diseases such as juvenile idiopathic arthritis (JIA) or systemic lupus erythematosus (SLE).
Accordingly, other studies presented at the conference examined more efficient ways to screen adolescent patients for depression and assessed programs designed to improve symptoms. In fact, the American College of Rheumatology award for the top Quality, Health Services, and Education Research abstract at this year’s symposium went to Lauren Harper, MD, a pediatric rheumatology fellow at Nationwide Children’s Hospital, Columbus, whose research examined the effects of automating depression screening during check-in for adolescent patients with SLE. Her findings revealed that automation of screening increased detection of depression and suicidality, thereby increasing interventions and ultimately resulting in a reduction in depression prevalence.
“The key clinical takeaway is that mental health screening is really important – it affects our patients in so many different ways – and it’s very doable in your rheumatology clinic,” Dr. Harper said in an interview. “It’s also important because they’re coming to us very frequently, but they don’t see their PCP [primary care provider] very often, so we can’t leave screening to the PCPs.”
Two other studies assessed the effectiveness of a 6-week cognitive-behavioral intervention for youth called Treatment and Education Approach for Childhood-Onset Lupus (TEACH). One study found that remote delivery of TEACH resulted in improved mood symptoms and reduced fatigue, and another found the program particularly effective in improving mood for patients deemed “high risk” because of greater depression and fatigue symptoms.
The impact of growing mental health problems has been enormous both in the pediatric rheumatology population and society at large, Daria Sosna, MSc, a research coordinator at the University of Calgary (Alta.), said in an interview as she visited the research posters related to psychological stress and depression.
“We need to do something,” said Ms. Sosna, whose department is currently applying for funding to develop a research project to improve mental health outcomes in adolescents with lupus. “This population, specifically, has higher numbers than anyone else does because they have chronic illness” – and those issues need to be addressed.
High psychological stress levels
The study looking at psychological stress in pediatric rheumatology patients, led by Natalie Rosenwasser, MD, of Seattle Children’s Hospital, relied on cross-sectional data from patients enrolled in two Childhood Arthritis and Rheumatology Research Alliance sites, one in Utah and one in Seattle. The average age of the 71 patients who completed the surveys was 13, and the researchers reported the findings in two separate age groups: those aged 13-17, who completed the surveys themselves, and those aged 8-12, whose parents completed the surveys. Nearly all the patients (94.4%) had JIA, but one had lupus and three had juvenile dermatomyositis.
The participants completed the Patient-Reported Outcomes Measurement Information System (PROMIS) for psychological stress, physical stress, and depressive symptoms. They also filled out the National Institutes of Health–Toolbox Perceived Stress survey, the 9-item Patient Health Questionnaire (PHQ-9), the Screen for Child Anxiety Related Disorders (SCARED), a visual analog scale for COVID-related distress, and a questionnaire asking about how receptive they were to mental health screening. The researchers determined that a score 1 standard deviation above the mean on the PROMIS and NIH-Toolbox assessments qualified as a high level of psychological stress.
“There are data that suggest that psychological stress can be a precursor to depression and anxiety, which raises the concern that not every patient who’s experiencing mental health symptoms is going to be picked up on traditional measures that meet that clinical threshold, but they may really need interventions to protect their mental health,” presenter Erin Treemarcki, DO, an assistant professor of pediatric rheumatology at the University of Utah, Salt Lake City, said in an interview. “Not every patient may necessarily need referral to a mental health specialist, but there are still potential interventions that we can do in the clinical setting to address mental health, which in turn can improve outcomes, including medication compliance and knowing how patients are feeling.”
More than one-third of the patients (39%) reported a high level of psychological stress, and 43% had elevated physical stress. Broken down by age, 26% of the teens and 15% of the younger patients reported high levels of perceived stress. The PROMIS only identified increased depressive symptoms in 26% of the participants, whereas more than half (54%) had a positive PHQ-9 depression screen. Furthermore, half the patients had SCARED scores (50%) that likely indicated anxiety disorder. Only 6% of patients reported severe stress specifically related to the pandemic, but most reported mild distress from the pandemic.
“Psychological stress was highly correlated with physical stress, perceived stress, depressive symptoms [PROMIS and PHQ-9], and anxiety,” the authors reported (P < .05). The authors next plan to expand their assessment to a third CARRA site and then explore the interaction between psychological distress and sociodemographic factors.
“There’s such an increase in mental health disorders right now, and we’re overwhelmed in general,” Ms. Sosna said in an interview. “There have to be interventions that approach this. We can use pharmacological approaches, we can use CBT, we can use a lot of these things that are very well established, and they’re absolutely fantastic, but we don’t necessarily have the resources or capabilities to do that all the time.”
Benefits of automated depression screening
To reduce the likelihood of depression screenings falling through the cracks during visits, Dr. Harper’s study assessed the impact of automating screens in an adolescent population. In her presentation, she noted previous research finding that nearly half of youth with lupus (47%) had depression, compared with 24% of adults with lupus. Pediatric patients have nearly three times the odds of depression and more than five times the odds of suicidal ideation, she told attendees. These mood disorders are correlated with greater physical disability, higher cardiovascular risk, more disease activity, higher risk of premature death, and decreased educational attainment, medication compliance, and quality of life.
Despite recommendations for depression screening from the U.S. Preventive Services Task Force and the American Academy of Pediatrics, only 2% of pediatric rheumatology patients are routinely screened for depression with a validated instrument, and only 7% of those with depressive symptoms are screened, according to a 2016 study that Dr. Harper cited. Yet the same study found that nearly all pediatric rheumatologists (95%) supported routine depression screening every 6-12 months. Hence her team’s decision to test whether automating screening improved their screening rates.
Their population included lupus patients aged 12 and older seen at Nationwide Children’s Hospital between 2014 and 2022. Initially, patients completed the PHQ-9 on paper, which was then transcribed into the electronic health record. The process became automated and administered on an iPad at every visit in 2022. Positive screens – those endorsing suicidality or with a score of at least 10 – caused an alert to pop up for clinicians during their workflow so that they would talk to the mental health team about the patient’s needs.
A total of 149 patients completed 529 screenings during the study’s 8 years. Only 1 patient completed a PHQ-9 in 2014, which increased to just 17 patients in 2017. Automation resulted in 225 screens (P < .01). Subsequently, positive screens increased from 0% in 2014 to 25%-30% in 2018-2021, but then fell to 12% in 2022 (P < .01). The median PHQ-9 score was 3; overall scores decreased as screening increased.
The overall incidence of positive screens during the study period was 20% and prevalence was 38%, the authors reported. Of the 10 automated alerts triggered by positive screens, 90% resulted in a meeting with a psychologist or social worker, and 90% completed a suicide risk assessment. The intrusive alert for clinicians requires them to acknowledge the alert, agreeing to initiate a risk assessment, before they can enter data into the patient’s chart.
The study findings reveal “that you can successfully screen a high-risk population using an automated, seamless process, and you can alert providers without too much disruption to their typical clinic flow,” Dr. Harper told attendees. “And all of these processes have led to sustainability for routine depression screening in our lupus clinic.”
Dr. Harper’s team next plans to expand the automated screenings to populations with other diseases, to add an automated screening for anxiety, and to explore how PHQ-9 scores correlate with disease activity.
Treating patients’ mental health
Another two other abstracts at the symposium looked at another option, the 6-week cognitive-behavioral TEACH program. Deborah Levy, MD, MS, an associate professor of pediatrics at the University of Toronto and the clinical director of rheumatology at The Hospital for Sick Children, and colleagues assessed the program’s success when delivered remotely to adolescent patients with lupus. Pilot testing with TEACH had already shown improvements in fatigue and mood, Dr. Levy told attendees, but barriers to in-person delivery limited its utility even before the pandemic, so this study aimed to determine a remote version’s feasibility and effects, compared with treatment as usual.
The randomized, controlled trial, led by Natoshia Cunningham, PhD, from Michigan State University, Grand Rapids, included 57 participants, aged 12-22, from seven U.S. and Canadian rheumatology sites. All had been diagnosed with childhood-onset SLE by age 18 and had elevated symptoms in fatigue, pain, or depression. A PROMIS Fatigue T score of 60 or greater indicated elevated fatigue scores, whereas a high pain score was at least a 3/10 on a visual analog scale, and a high depression T score was at least a 60 but not higher than 80 on the Children’s Depression Inventory–2 or the Beck Depression Inventory–II (depending on the patient’s age).
Patients with other chronic medical conditions, developmental delays, or untreated major psychiatric illness were excluded from the study, as were patients who were receiving overlapping treatment, such as cognitive-behavioral therapy for pain or mood. Thirty patients were randomly assigned to receive treatment as usual while 27 patients were assigned to participate in the remote TEACH program.
Nearly all the patients (94%) were female, but they were racially diverse, with 42% White, 28% Asian, 19% Black, 19% Hispanic, and 4% multiracial. The patients were an average 16 years old and had been diagnosed for a median 5 years. Three of the intervention’s six modules involved the caregivers or, for older patients, their partners if desired. The communication strategies taught in the program were also tailored to patients’ ages.
“All of these strategies are educational, cognitive, behavioral, mindfulness strategies that target fatigue [and] pain, and they also developed web content for participants to use on their own,” Dr. Levy told attendees.
The researchers had complete postassessment data from 88% of participants, but they also reported some of the statements made during qualitative interviews about the program’s feasibility.
“I think it makes people more aware of themselves to become a better version of themselves, whether that’s in their normal life or in handling a lupus kind of life,” one participant said about the program’s benefits. Another appreciated the “alternative ways of thinking,” including “being more mindful of my thoughts and how those kind of aggravate my stress.”
The quantitative findings revealed a statistically significant reduction in depressive symptoms and fatigue for TEACH participants, compared with treatment as usual. Mood scores fell by an average 13.7 points in the TEACH group, compared with a drop of 2.4 points in the treatment as usual group (P < .001). Scores for fatigue fell 9.16 points in the TEACH group and 2.93 in the control group (P = .003). No statistically significant difference showed up in pain scores between the groups, although pain, medication adherence, and disease activity did improve slightly more in the TEACH group.
In addition to the significant improvements in mood and fatigue, therefore, “completion of TEACH may be associated with improved medication adherence and disease activity versus treatment as usual,” Dr. Levy said.
A much smaller study authored by some of the same researchers also assessed TEACH’s impact not in remote form but in terms of its value specifically for adolescent patients with SLE and elevated depression and fatigue scores. Comparison of 6 high-risk patients with 10 low-risk patients who underwent TEACH suggested that the program was especially effective for improving depression in high-risk patients since these patients had a statistically significantly greater improvement in mood. Fatigue, pain, anxiety, quality of life, and disease activity scores did not statistically differ between the groups.
Authors of the automated depression screening study reported no disclosures or outside funding. The study assessing psychological distress was funded by a CARRA–Arthritis Foundation grant, and the authors reported no disclosures. The remote TEACH study was funded by a CARRA–Arthritis Foundation grant, and all but one author reported no disclosures. One author had disclosures with Janssen, Roche, and Sobi. The high-risk TEACH study was also funded by a CARRA grant, and the authors had no disclosures.
NEW ORLEANS – Pediatric patients with rheumatologic diseases experience a particularly high prevalence of psychological distress and depression and anxiety symptoms, according to research presented at the Pediatric Rheumatology Symposium. Although this finding is not necessarily surprising, the extent to which depression and psychological distress impacts these young patients’ quality of life has led to greater research and innovation in seeking ways to identify, address, and treat depression and anxiety in children and adolescents with diseases such as juvenile idiopathic arthritis (JIA) or systemic lupus erythematosus (SLE).
Accordingly, other studies presented at the conference examined more efficient ways to screen adolescent patients for depression and assessed programs designed to improve symptoms. In fact, the American College of Rheumatology award for the top Quality, Health Services, and Education Research abstract at this year’s symposium went to Lauren Harper, MD, a pediatric rheumatology fellow at Nationwide Children’s Hospital, Columbus, whose research examined the effects of automating depression screening during check-in for adolescent patients with SLE. Her findings revealed that automation of screening increased detection of depression and suicidality, thereby increasing interventions and ultimately resulting in a reduction in depression prevalence.
“The key clinical takeaway is that mental health screening is really important – it affects our patients in so many different ways – and it’s very doable in your rheumatology clinic,” Dr. Harper said in an interview. “It’s also important because they’re coming to us very frequently, but they don’t see their PCP [primary care provider] very often, so we can’t leave screening to the PCPs.”
Two other studies assessed the effectiveness of a 6-week cognitive-behavioral intervention for youth called Treatment and Education Approach for Childhood-Onset Lupus (TEACH). One study found that remote delivery of TEACH resulted in improved mood symptoms and reduced fatigue, and another found the program particularly effective in improving mood for patients deemed “high risk” because of greater depression and fatigue symptoms.
The impact of growing mental health problems has been enormous both in the pediatric rheumatology population and society at large, Daria Sosna, MSc, a research coordinator at the University of Calgary (Alta.), said in an interview as she visited the research posters related to psychological stress and depression.
“We need to do something,” said Ms. Sosna, whose department is currently applying for funding to develop a research project to improve mental health outcomes in adolescents with lupus. “This population, specifically, has higher numbers than anyone else does because they have chronic illness” – and those issues need to be addressed.
High psychological stress levels
The study looking at psychological stress in pediatric rheumatology patients, led by Natalie Rosenwasser, MD, of Seattle Children’s Hospital, relied on cross-sectional data from patients enrolled in two Childhood Arthritis and Rheumatology Research Alliance sites, one in Utah and one in Seattle. The average age of the 71 patients who completed the surveys was 13, and the researchers reported the findings in two separate age groups: those aged 13-17, who completed the surveys themselves, and those aged 8-12, whose parents completed the surveys. Nearly all the patients (94.4%) had JIA, but one had lupus and three had juvenile dermatomyositis.
The participants completed the Patient-Reported Outcomes Measurement Information System (PROMIS) for psychological stress, physical stress, and depressive symptoms. They also filled out the National Institutes of Health–Toolbox Perceived Stress survey, the 9-item Patient Health Questionnaire (PHQ-9), the Screen for Child Anxiety Related Disorders (SCARED), a visual analog scale for COVID-related distress, and a questionnaire asking about how receptive they were to mental health screening. The researchers determined that a score 1 standard deviation above the mean on the PROMIS and NIH-Toolbox assessments qualified as a high level of psychological stress.
“There are data that suggest that psychological stress can be a precursor to depression and anxiety, which raises the concern that not every patient who’s experiencing mental health symptoms is going to be picked up on traditional measures that meet that clinical threshold, but they may really need interventions to protect their mental health,” presenter Erin Treemarcki, DO, an assistant professor of pediatric rheumatology at the University of Utah, Salt Lake City, said in an interview. “Not every patient may necessarily need referral to a mental health specialist, but there are still potential interventions that we can do in the clinical setting to address mental health, which in turn can improve outcomes, including medication compliance and knowing how patients are feeling.”
More than one-third of the patients (39%) reported a high level of psychological stress, and 43% had elevated physical stress. Broken down by age, 26% of the teens and 15% of the younger patients reported high levels of perceived stress. The PROMIS only identified increased depressive symptoms in 26% of the participants, whereas more than half (54%) had a positive PHQ-9 depression screen. Furthermore, half the patients had SCARED scores (50%) that likely indicated anxiety disorder. Only 6% of patients reported severe stress specifically related to the pandemic, but most reported mild distress from the pandemic.
“Psychological stress was highly correlated with physical stress, perceived stress, depressive symptoms [PROMIS and PHQ-9], and anxiety,” the authors reported (P < .05). The authors next plan to expand their assessment to a third CARRA site and then explore the interaction between psychological distress and sociodemographic factors.
“There’s such an increase in mental health disorders right now, and we’re overwhelmed in general,” Ms. Sosna said in an interview. “There have to be interventions that approach this. We can use pharmacological approaches, we can use CBT, we can use a lot of these things that are very well established, and they’re absolutely fantastic, but we don’t necessarily have the resources or capabilities to do that all the time.”
Benefits of automated depression screening
To reduce the likelihood of depression screenings falling through the cracks during visits, Dr. Harper’s study assessed the impact of automating screens in an adolescent population. In her presentation, she noted previous research finding that nearly half of youth with lupus (47%) had depression, compared with 24% of adults with lupus. Pediatric patients have nearly three times the odds of depression and more than five times the odds of suicidal ideation, she told attendees. These mood disorders are correlated with greater physical disability, higher cardiovascular risk, more disease activity, higher risk of premature death, and decreased educational attainment, medication compliance, and quality of life.
Despite recommendations for depression screening from the U.S. Preventive Services Task Force and the American Academy of Pediatrics, only 2% of pediatric rheumatology patients are routinely screened for depression with a validated instrument, and only 7% of those with depressive symptoms are screened, according to a 2016 study that Dr. Harper cited. Yet the same study found that nearly all pediatric rheumatologists (95%) supported routine depression screening every 6-12 months. Hence her team’s decision to test whether automating screening improved their screening rates.
Their population included lupus patients aged 12 and older seen at Nationwide Children’s Hospital between 2014 and 2022. Initially, patients completed the PHQ-9 on paper, which was then transcribed into the electronic health record. The process became automated and administered on an iPad at every visit in 2022. Positive screens – those endorsing suicidality or with a score of at least 10 – caused an alert to pop up for clinicians during their workflow so that they would talk to the mental health team about the patient’s needs.
A total of 149 patients completed 529 screenings during the study’s 8 years. Only 1 patient completed a PHQ-9 in 2014, which increased to just 17 patients in 2017. Automation resulted in 225 screens (P < .01). Subsequently, positive screens increased from 0% in 2014 to 25%-30% in 2018-2021, but then fell to 12% in 2022 (P < .01). The median PHQ-9 score was 3; overall scores decreased as screening increased.
The overall incidence of positive screens during the study period was 20% and prevalence was 38%, the authors reported. Of the 10 automated alerts triggered by positive screens, 90% resulted in a meeting with a psychologist or social worker, and 90% completed a suicide risk assessment. The intrusive alert for clinicians requires them to acknowledge the alert, agreeing to initiate a risk assessment, before they can enter data into the patient’s chart.
The study findings reveal “that you can successfully screen a high-risk population using an automated, seamless process, and you can alert providers without too much disruption to their typical clinic flow,” Dr. Harper told attendees. “And all of these processes have led to sustainability for routine depression screening in our lupus clinic.”
Dr. Harper’s team next plans to expand the automated screenings to populations with other diseases, to add an automated screening for anxiety, and to explore how PHQ-9 scores correlate with disease activity.
Treating patients’ mental health
Another two other abstracts at the symposium looked at another option, the 6-week cognitive-behavioral TEACH program. Deborah Levy, MD, MS, an associate professor of pediatrics at the University of Toronto and the clinical director of rheumatology at The Hospital for Sick Children, and colleagues assessed the program’s success when delivered remotely to adolescent patients with lupus. Pilot testing with TEACH had already shown improvements in fatigue and mood, Dr. Levy told attendees, but barriers to in-person delivery limited its utility even before the pandemic, so this study aimed to determine a remote version’s feasibility and effects, compared with treatment as usual.
The randomized, controlled trial, led by Natoshia Cunningham, PhD, from Michigan State University, Grand Rapids, included 57 participants, aged 12-22, from seven U.S. and Canadian rheumatology sites. All had been diagnosed with childhood-onset SLE by age 18 and had elevated symptoms in fatigue, pain, or depression. A PROMIS Fatigue T score of 60 or greater indicated elevated fatigue scores, whereas a high pain score was at least a 3/10 on a visual analog scale, and a high depression T score was at least a 60 but not higher than 80 on the Children’s Depression Inventory–2 or the Beck Depression Inventory–II (depending on the patient’s age).
Patients with other chronic medical conditions, developmental delays, or untreated major psychiatric illness were excluded from the study, as were patients who were receiving overlapping treatment, such as cognitive-behavioral therapy for pain or mood. Thirty patients were randomly assigned to receive treatment as usual while 27 patients were assigned to participate in the remote TEACH program.
Nearly all the patients (94%) were female, but they were racially diverse, with 42% White, 28% Asian, 19% Black, 19% Hispanic, and 4% multiracial. The patients were an average 16 years old and had been diagnosed for a median 5 years. Three of the intervention’s six modules involved the caregivers or, for older patients, their partners if desired. The communication strategies taught in the program were also tailored to patients’ ages.
“All of these strategies are educational, cognitive, behavioral, mindfulness strategies that target fatigue [and] pain, and they also developed web content for participants to use on their own,” Dr. Levy told attendees.
The researchers had complete postassessment data from 88% of participants, but they also reported some of the statements made during qualitative interviews about the program’s feasibility.
“I think it makes people more aware of themselves to become a better version of themselves, whether that’s in their normal life or in handling a lupus kind of life,” one participant said about the program’s benefits. Another appreciated the “alternative ways of thinking,” including “being more mindful of my thoughts and how those kind of aggravate my stress.”
The quantitative findings revealed a statistically significant reduction in depressive symptoms and fatigue for TEACH participants, compared with treatment as usual. Mood scores fell by an average 13.7 points in the TEACH group, compared with a drop of 2.4 points in the treatment as usual group (P < .001). Scores for fatigue fell 9.16 points in the TEACH group and 2.93 in the control group (P = .003). No statistically significant difference showed up in pain scores between the groups, although pain, medication adherence, and disease activity did improve slightly more in the TEACH group.
In addition to the significant improvements in mood and fatigue, therefore, “completion of TEACH may be associated with improved medication adherence and disease activity versus treatment as usual,” Dr. Levy said.
A much smaller study authored by some of the same researchers also assessed TEACH’s impact not in remote form but in terms of its value specifically for adolescent patients with SLE and elevated depression and fatigue scores. Comparison of 6 high-risk patients with 10 low-risk patients who underwent TEACH suggested that the program was especially effective for improving depression in high-risk patients since these patients had a statistically significantly greater improvement in mood. Fatigue, pain, anxiety, quality of life, and disease activity scores did not statistically differ between the groups.
Authors of the automated depression screening study reported no disclosures or outside funding. The study assessing psychological distress was funded by a CARRA–Arthritis Foundation grant, and the authors reported no disclosures. The remote TEACH study was funded by a CARRA–Arthritis Foundation grant, and all but one author reported no disclosures. One author had disclosures with Janssen, Roche, and Sobi. The high-risk TEACH study was also funded by a CARRA grant, and the authors had no disclosures.
AT PRSYM 2023
Cancer, heart disease vaccines may be ready by 2030, Moderna says
The announcement is yet another sign of what many are calling “the golden age” of vaccine development, which is largely credited to the pandemic’s use of mRNA technology to create COVID-19 vaccines.
“I think what we have learned in recent months is that if you ever thought that mRNA was just for infectious diseases, or just for COVID, the evidence now is that that’s absolutely not the case,” Moderna Chief Medical Officer Paul Burton, MD, PhD, told The Guardian. “It can be applied to all sorts of disease areas; we are in cancer, infectious disease, cardiovascular disease, autoimmune diseases, rare disease. We have studies in all of those areas, and they have all shown tremendous promise.”
The U.S. Food and Drug Administration recently designated two new Moderna vaccines as breakthrough therapies: a shot that prevents respiratory syncytial virus (RSV) in older people and a shot that helps prevent the recurrence of melanoma. The FDA’s breakthrough designation is given when a new treatment’s early trial results are substantially better than an existing therapy.
The mRNA vaccine technology that made headlines for its role in COVID-19 vaccines works by teaching the body how to make a specific protein to help the immune system prevent or target a certain disease.
Dr. Burton anticipates that mRNA technology will result in breakthroughs such as a cancer vaccine that can be personalized based on the features of a specific tumor.
“I think we will have mRNA-based therapies for rare diseases that were previously undruggable, and I think that 10 years from now, we will be approaching a world where you truly can identify the genetic cause of a disease and, with relative simplicity, go and edit that out and repair it using mRNA-based technology,” he said.
The Moderna executive made the statements before its annual update on its vaccine pipeline projects, which the company calls “Vaccines Day.” The Massachusetts-based drugmaker said it has given someone the first dose of a “next-generation” COVID-19 vaccine in a phase III trial, has made progress on a Lyme disease shot, and is developing a vaccine for the highly contagious norovirus.
In all, Moderna expects “six major vaccine product launches in the next few years,” the company said in a statement, adding that it expects the COVID-19 booster market alone to be valued at $15 billion.
A version of this article first appeared on WebMD.com.
The announcement is yet another sign of what many are calling “the golden age” of vaccine development, which is largely credited to the pandemic’s use of mRNA technology to create COVID-19 vaccines.
“I think what we have learned in recent months is that if you ever thought that mRNA was just for infectious diseases, or just for COVID, the evidence now is that that’s absolutely not the case,” Moderna Chief Medical Officer Paul Burton, MD, PhD, told The Guardian. “It can be applied to all sorts of disease areas; we are in cancer, infectious disease, cardiovascular disease, autoimmune diseases, rare disease. We have studies in all of those areas, and they have all shown tremendous promise.”
The U.S. Food and Drug Administration recently designated two new Moderna vaccines as breakthrough therapies: a shot that prevents respiratory syncytial virus (RSV) in older people and a shot that helps prevent the recurrence of melanoma. The FDA’s breakthrough designation is given when a new treatment’s early trial results are substantially better than an existing therapy.
The mRNA vaccine technology that made headlines for its role in COVID-19 vaccines works by teaching the body how to make a specific protein to help the immune system prevent or target a certain disease.
Dr. Burton anticipates that mRNA technology will result in breakthroughs such as a cancer vaccine that can be personalized based on the features of a specific tumor.
“I think we will have mRNA-based therapies for rare diseases that were previously undruggable, and I think that 10 years from now, we will be approaching a world where you truly can identify the genetic cause of a disease and, with relative simplicity, go and edit that out and repair it using mRNA-based technology,” he said.
The Moderna executive made the statements before its annual update on its vaccine pipeline projects, which the company calls “Vaccines Day.” The Massachusetts-based drugmaker said it has given someone the first dose of a “next-generation” COVID-19 vaccine in a phase III trial, has made progress on a Lyme disease shot, and is developing a vaccine for the highly contagious norovirus.
In all, Moderna expects “six major vaccine product launches in the next few years,” the company said in a statement, adding that it expects the COVID-19 booster market alone to be valued at $15 billion.
A version of this article first appeared on WebMD.com.
The announcement is yet another sign of what many are calling “the golden age” of vaccine development, which is largely credited to the pandemic’s use of mRNA technology to create COVID-19 vaccines.
“I think what we have learned in recent months is that if you ever thought that mRNA was just for infectious diseases, or just for COVID, the evidence now is that that’s absolutely not the case,” Moderna Chief Medical Officer Paul Burton, MD, PhD, told The Guardian. “It can be applied to all sorts of disease areas; we are in cancer, infectious disease, cardiovascular disease, autoimmune diseases, rare disease. We have studies in all of those areas, and they have all shown tremendous promise.”
The U.S. Food and Drug Administration recently designated two new Moderna vaccines as breakthrough therapies: a shot that prevents respiratory syncytial virus (RSV) in older people and a shot that helps prevent the recurrence of melanoma. The FDA’s breakthrough designation is given when a new treatment’s early trial results are substantially better than an existing therapy.
The mRNA vaccine technology that made headlines for its role in COVID-19 vaccines works by teaching the body how to make a specific protein to help the immune system prevent or target a certain disease.
Dr. Burton anticipates that mRNA technology will result in breakthroughs such as a cancer vaccine that can be personalized based on the features of a specific tumor.
“I think we will have mRNA-based therapies for rare diseases that were previously undruggable, and I think that 10 years from now, we will be approaching a world where you truly can identify the genetic cause of a disease and, with relative simplicity, go and edit that out and repair it using mRNA-based technology,” he said.
The Moderna executive made the statements before its annual update on its vaccine pipeline projects, which the company calls “Vaccines Day.” The Massachusetts-based drugmaker said it has given someone the first dose of a “next-generation” COVID-19 vaccine in a phase III trial, has made progress on a Lyme disease shot, and is developing a vaccine for the highly contagious norovirus.
In all, Moderna expects “six major vaccine product launches in the next few years,” the company said in a statement, adding that it expects the COVID-19 booster market alone to be valued at $15 billion.
A version of this article first appeared on WebMD.com.
Pretransfer visits with pediatric and adult rheumatologists smooth adolescent transition
NEW ORLEANS – Implementing a pediatric transition program in which a patient meets with both their pediatric and soon-to-be adult rheumatologist during a visit before formal transition resulted in less time setting up the first adult visit, according to research presented at the Pediatric Rheumatology Symposium.
The presentation was one of two that focused on ways to improve the transition from pediatric to adult care for rheumatology patients. The other, a poster from researchers at Baylor College of Medicine, Houston, took the first steps toward learning what factors can help predict a successful transition.
“This period of transitioning from pediatric to adult care, both rheumatology specific and otherwise, is a high-risk time,” John M. Bridges, MD, a fourth-year pediatric rheumatology fellow at the University of Alabama at Birmingham, told attendees. “There are changes in insurance coverage, employment, geographic mobility, and shifting responsibilities between parents and children in the setting of a still-developing frontal lobe that contribute to the risk of this period. Risks include disease flare, and then organ damage, as well as issues with decreasing medication and therapy, adherence, unscheduled care utilization, and increasing loss to follow-up.”
Dr. Bridges developed a structured transition program called the Bridge to Adult Care from Childhood for Young Adults with Rheumatic Disease (BACC YARD) aimed at improving the pediatric transition period. The analysis he presented focused specifically on reducing loss to follow-up by introducing a pretransfer visit with both rheumatologists. The patient first meets with their pediatric rheumatologist.
During that visit, the adult rheumatologist attends and discusses the patient’s history and current therapy with the pediatric rheumatologist before entering the patient’s room and having “a brief introductory conversation, a sort of verbal handoff and handshake, in front of the patient,” Dr. Bridges explained. “Then I assume responsibility for this patient and their next visit is to see me, both proverbially and literally down the street at the adulthood rheumatology clinic, where this patient becomes a part of my continuity cohort.”
Bridges entered patients from this BACC YARD cohort into an observational registry that included their dual provider pretransfer visit and a posttransfer visit, occurring between July 2020 and May 2022. He compared these patients with a historical control cohort of 45 patients from March 2018 to March 2020, who had at least two pediatric rheumatology visits prior to their transfer to adult care and no documentation of outside rheumatology visits during the study period. Specifically, he examined at the requested and actual interval between patients’ final pediatric rheumatology visit and their first adult rheumatology visit.
The intervention cohort included 86 patients, mostly female (73%), with a median age of 20. About two-thirds were White (65%) and one-third (34%) were Black. One patient was Asian, and 7% were Hispanic. Just over half the patients had juvenile idiopathic arthritis (58%), and 30% had lupus and related connective tissue diseases. The other patients had vasculitis, uveitis, inflammatory myopathy, relapsing polychondritis, morphea, or syndrome of undifferentiated recurrent fever.
A total of 8% of these patients had previously been lost to follow-up at Children’s of Alabama before they re-established rheumatology care at UAB, and 3.5% came from a pediatric rheumatologist from somewhere other than Children’s of Alabama but established adult care at UAB through the BACC YARD program. Among the remaining patients, 65% (n = 56) had both a dual provider pretransfer visit and a posttransfer visit.
The BACC YARD patients requested their next rheumatology visit (the first adult one) a median 119 days after their last pediatric visit, and the actual time until that visit was a median 141 days (P < .05). By comparison, the 45 patients in the historical control group had a median 261 days between their last pediatric visit and their first adult visit (P < .001). The median days between visits was shorter for those with JIA (129 days) and lupus (119 days) than for patients with other conditions (149 days).
Bridges acknowledged that the study was limited by the small size of the cohort and potential contextual factors related to individual patients’ circumstances.
“We’re continuing to make iterative changes to this process to try to continue to improve the transition and its outcomes in this cohort,” Dr. Bridges said.
Aimee Hersh, MD, an associate professor of pediatric rheumatology and division chief of pediatric rheumatology at the University of Utah and Primary Children’s Hospital, both in Salt Lake City, attended the presentation and noted that the University of Utah has a very similar transfer program.
“I think one of the challenges of that model, and our model, is that you have to have a very specific type of physician who is both [medical-pediatrics] trained and has a specific interest in transition,” Dr. Hersh said in an interview. She noted that the adult rheumatologist at her institution didn’t train in pediatric rheumatology but did complete a meds-peds residency. “So if you can find an adult rheumatologist who can do something similar, can see older adolescent patients and serve as that transition bridge, then I think it is feasible.”
For practices that don’t have the resources for this kind of program, Dr. Hersh recommended the Got Transition program, which provides transition guidance that can be applied to any adolescent population with chronic illness.
The other study, led by Kristiana Nasto, BS, a third-year medical student at Baylor College of Medicine, reported on the findings from one aspect of a program also developed to improve the transition from pediatric to adult care for rheumatology patients. It included periodic self-reported evaluation using the validated Adolescent Assessment of Preparation for Transition (ADAPT) survey. As the first step to better understanding the factors that can predict successful transition, the researchers surveyed returning patients with any rheumatologic diagnosis, aged 14 years and older, between July 2021 and November 2022.
Since the survey was automated through the electronic medical record, patients and their caregivers could respond during in-person or virtual visit check-in. The researchers calculated three composite scores out of 100 for self-management, prescription management, and transfer planning, using responses from the ADAPT survey. Among 462 patients who returned 670 surveys, 87% provided surveys that could be scored for at least one composite score. Most respondents were female (75%), White (69%), non-Hispanic (64%), English speaking (90%), and aged 14-17 years (83%).
The overall average score for self-management from 401 respondents was 35. For prescription management, the average score was 59 from 288 respondents, and the average transfer planning score was 17 from 367 respondents. Self-management and transfer planning scores both improved with age (P = .0001). Self-management scores rose from an average of 20 at age 14 to an average of 64 at age 18 and older. Transfer planning scores increased from an average of 1 at age 14 to an average of 49 at age 18 and older. Prescription management scores remained high across all ages, from an average of 59 at age 14 to an average score of 66 at age 18 and older (P = .044). Although the scores did not statistically vary by age or race, Hispanic patients did score higher in self-management with an average of 44.5, compared with 31 among other patients (P = .0001).
Only 21% of patients completed two surveys, and 8.4% completed all three surveys. The average time between the first and second surveys was 4 months, during which there was no statistically significant change in self-management or prescription management scores, but transfer planning scores did increase from 14 to 21 (P = .008) among the 90 patients who completed those surveys.
The researchers concluded from their analysis that “participation in the transition pathway can rapidly improve transfer planning scores, [but] opportunities remain to improve readiness in all domains.” The researchers are in the process of developing Spanish-language surveys.
No external funding was noted for either study. Dr. Bridges, Dr. Hersh, and Ms. Nasto reported no relevant financial relationships.
A version of this article first appeared on Medscape.com.
NEW ORLEANS – Implementing a pediatric transition program in which a patient meets with both their pediatric and soon-to-be adult rheumatologist during a visit before formal transition resulted in less time setting up the first adult visit, according to research presented at the Pediatric Rheumatology Symposium.
The presentation was one of two that focused on ways to improve the transition from pediatric to adult care for rheumatology patients. The other, a poster from researchers at Baylor College of Medicine, Houston, took the first steps toward learning what factors can help predict a successful transition.
“This period of transitioning from pediatric to adult care, both rheumatology specific and otherwise, is a high-risk time,” John M. Bridges, MD, a fourth-year pediatric rheumatology fellow at the University of Alabama at Birmingham, told attendees. “There are changes in insurance coverage, employment, geographic mobility, and shifting responsibilities between parents and children in the setting of a still-developing frontal lobe that contribute to the risk of this period. Risks include disease flare, and then organ damage, as well as issues with decreasing medication and therapy, adherence, unscheduled care utilization, and increasing loss to follow-up.”
Dr. Bridges developed a structured transition program called the Bridge to Adult Care from Childhood for Young Adults with Rheumatic Disease (BACC YARD) aimed at improving the pediatric transition period. The analysis he presented focused specifically on reducing loss to follow-up by introducing a pretransfer visit with both rheumatologists. The patient first meets with their pediatric rheumatologist.
During that visit, the adult rheumatologist attends and discusses the patient’s history and current therapy with the pediatric rheumatologist before entering the patient’s room and having “a brief introductory conversation, a sort of verbal handoff and handshake, in front of the patient,” Dr. Bridges explained. “Then I assume responsibility for this patient and their next visit is to see me, both proverbially and literally down the street at the adulthood rheumatology clinic, where this patient becomes a part of my continuity cohort.”
Bridges entered patients from this BACC YARD cohort into an observational registry that included their dual provider pretransfer visit and a posttransfer visit, occurring between July 2020 and May 2022. He compared these patients with a historical control cohort of 45 patients from March 2018 to March 2020, who had at least two pediatric rheumatology visits prior to their transfer to adult care and no documentation of outside rheumatology visits during the study period. Specifically, he examined at the requested and actual interval between patients’ final pediatric rheumatology visit and their first adult rheumatology visit.
The intervention cohort included 86 patients, mostly female (73%), with a median age of 20. About two-thirds were White (65%) and one-third (34%) were Black. One patient was Asian, and 7% were Hispanic. Just over half the patients had juvenile idiopathic arthritis (58%), and 30% had lupus and related connective tissue diseases. The other patients had vasculitis, uveitis, inflammatory myopathy, relapsing polychondritis, morphea, or syndrome of undifferentiated recurrent fever.
A total of 8% of these patients had previously been lost to follow-up at Children’s of Alabama before they re-established rheumatology care at UAB, and 3.5% came from a pediatric rheumatologist from somewhere other than Children’s of Alabama but established adult care at UAB through the BACC YARD program. Among the remaining patients, 65% (n = 56) had both a dual provider pretransfer visit and a posttransfer visit.
The BACC YARD patients requested their next rheumatology visit (the first adult one) a median 119 days after their last pediatric visit, and the actual time until that visit was a median 141 days (P < .05). By comparison, the 45 patients in the historical control group had a median 261 days between their last pediatric visit and their first adult visit (P < .001). The median days between visits was shorter for those with JIA (129 days) and lupus (119 days) than for patients with other conditions (149 days).
Bridges acknowledged that the study was limited by the small size of the cohort and potential contextual factors related to individual patients’ circumstances.
“We’re continuing to make iterative changes to this process to try to continue to improve the transition and its outcomes in this cohort,” Dr. Bridges said.
Aimee Hersh, MD, an associate professor of pediatric rheumatology and division chief of pediatric rheumatology at the University of Utah and Primary Children’s Hospital, both in Salt Lake City, attended the presentation and noted that the University of Utah has a very similar transfer program.
“I think one of the challenges of that model, and our model, is that you have to have a very specific type of physician who is both [medical-pediatrics] trained and has a specific interest in transition,” Dr. Hersh said in an interview. She noted that the adult rheumatologist at her institution didn’t train in pediatric rheumatology but did complete a meds-peds residency. “So if you can find an adult rheumatologist who can do something similar, can see older adolescent patients and serve as that transition bridge, then I think it is feasible.”
For practices that don’t have the resources for this kind of program, Dr. Hersh recommended the Got Transition program, which provides transition guidance that can be applied to any adolescent population with chronic illness.
The other study, led by Kristiana Nasto, BS, a third-year medical student at Baylor College of Medicine, reported on the findings from one aspect of a program also developed to improve the transition from pediatric to adult care for rheumatology patients. It included periodic self-reported evaluation using the validated Adolescent Assessment of Preparation for Transition (ADAPT) survey. As the first step to better understanding the factors that can predict successful transition, the researchers surveyed returning patients with any rheumatologic diagnosis, aged 14 years and older, between July 2021 and November 2022.
Since the survey was automated through the electronic medical record, patients and their caregivers could respond during in-person or virtual visit check-in. The researchers calculated three composite scores out of 100 for self-management, prescription management, and transfer planning, using responses from the ADAPT survey. Among 462 patients who returned 670 surveys, 87% provided surveys that could be scored for at least one composite score. Most respondents were female (75%), White (69%), non-Hispanic (64%), English speaking (90%), and aged 14-17 years (83%).
The overall average score for self-management from 401 respondents was 35. For prescription management, the average score was 59 from 288 respondents, and the average transfer planning score was 17 from 367 respondents. Self-management and transfer planning scores both improved with age (P = .0001). Self-management scores rose from an average of 20 at age 14 to an average of 64 at age 18 and older. Transfer planning scores increased from an average of 1 at age 14 to an average of 49 at age 18 and older. Prescription management scores remained high across all ages, from an average of 59 at age 14 to an average score of 66 at age 18 and older (P = .044). Although the scores did not statistically vary by age or race, Hispanic patients did score higher in self-management with an average of 44.5, compared with 31 among other patients (P = .0001).
Only 21% of patients completed two surveys, and 8.4% completed all three surveys. The average time between the first and second surveys was 4 months, during which there was no statistically significant change in self-management or prescription management scores, but transfer planning scores did increase from 14 to 21 (P = .008) among the 90 patients who completed those surveys.
The researchers concluded from their analysis that “participation in the transition pathway can rapidly improve transfer planning scores, [but] opportunities remain to improve readiness in all domains.” The researchers are in the process of developing Spanish-language surveys.
No external funding was noted for either study. Dr. Bridges, Dr. Hersh, and Ms. Nasto reported no relevant financial relationships.
A version of this article first appeared on Medscape.com.
NEW ORLEANS – Implementing a pediatric transition program in which a patient meets with both their pediatric and soon-to-be adult rheumatologist during a visit before formal transition resulted in less time setting up the first adult visit, according to research presented at the Pediatric Rheumatology Symposium.
The presentation was one of two that focused on ways to improve the transition from pediatric to adult care for rheumatology patients. The other, a poster from researchers at Baylor College of Medicine, Houston, took the first steps toward learning what factors can help predict a successful transition.
“This period of transitioning from pediatric to adult care, both rheumatology specific and otherwise, is a high-risk time,” John M. Bridges, MD, a fourth-year pediatric rheumatology fellow at the University of Alabama at Birmingham, told attendees. “There are changes in insurance coverage, employment, geographic mobility, and shifting responsibilities between parents and children in the setting of a still-developing frontal lobe that contribute to the risk of this period. Risks include disease flare, and then organ damage, as well as issues with decreasing medication and therapy, adherence, unscheduled care utilization, and increasing loss to follow-up.”
Dr. Bridges developed a structured transition program called the Bridge to Adult Care from Childhood for Young Adults with Rheumatic Disease (BACC YARD) aimed at improving the pediatric transition period. The analysis he presented focused specifically on reducing loss to follow-up by introducing a pretransfer visit with both rheumatologists. The patient first meets with their pediatric rheumatologist.
During that visit, the adult rheumatologist attends and discusses the patient’s history and current therapy with the pediatric rheumatologist before entering the patient’s room and having “a brief introductory conversation, a sort of verbal handoff and handshake, in front of the patient,” Dr. Bridges explained. “Then I assume responsibility for this patient and their next visit is to see me, both proverbially and literally down the street at the adulthood rheumatology clinic, where this patient becomes a part of my continuity cohort.”
Bridges entered patients from this BACC YARD cohort into an observational registry that included their dual provider pretransfer visit and a posttransfer visit, occurring between July 2020 and May 2022. He compared these patients with a historical control cohort of 45 patients from March 2018 to March 2020, who had at least two pediatric rheumatology visits prior to their transfer to adult care and no documentation of outside rheumatology visits during the study period. Specifically, he examined at the requested and actual interval between patients’ final pediatric rheumatology visit and their first adult rheumatology visit.
The intervention cohort included 86 patients, mostly female (73%), with a median age of 20. About two-thirds were White (65%) and one-third (34%) were Black. One patient was Asian, and 7% were Hispanic. Just over half the patients had juvenile idiopathic arthritis (58%), and 30% had lupus and related connective tissue diseases. The other patients had vasculitis, uveitis, inflammatory myopathy, relapsing polychondritis, morphea, or syndrome of undifferentiated recurrent fever.
A total of 8% of these patients had previously been lost to follow-up at Children’s of Alabama before they re-established rheumatology care at UAB, and 3.5% came from a pediatric rheumatologist from somewhere other than Children’s of Alabama but established adult care at UAB through the BACC YARD program. Among the remaining patients, 65% (n = 56) had both a dual provider pretransfer visit and a posttransfer visit.
The BACC YARD patients requested their next rheumatology visit (the first adult one) a median 119 days after their last pediatric visit, and the actual time until that visit was a median 141 days (P < .05). By comparison, the 45 patients in the historical control group had a median 261 days between their last pediatric visit and their first adult visit (P < .001). The median days between visits was shorter for those with JIA (129 days) and lupus (119 days) than for patients with other conditions (149 days).
Bridges acknowledged that the study was limited by the small size of the cohort and potential contextual factors related to individual patients’ circumstances.
“We’re continuing to make iterative changes to this process to try to continue to improve the transition and its outcomes in this cohort,” Dr. Bridges said.
Aimee Hersh, MD, an associate professor of pediatric rheumatology and division chief of pediatric rheumatology at the University of Utah and Primary Children’s Hospital, both in Salt Lake City, attended the presentation and noted that the University of Utah has a very similar transfer program.
“I think one of the challenges of that model, and our model, is that you have to have a very specific type of physician who is both [medical-pediatrics] trained and has a specific interest in transition,” Dr. Hersh said in an interview. She noted that the adult rheumatologist at her institution didn’t train in pediatric rheumatology but did complete a meds-peds residency. “So if you can find an adult rheumatologist who can do something similar, can see older adolescent patients and serve as that transition bridge, then I think it is feasible.”
For practices that don’t have the resources for this kind of program, Dr. Hersh recommended the Got Transition program, which provides transition guidance that can be applied to any adolescent population with chronic illness.
The other study, led by Kristiana Nasto, BS, a third-year medical student at Baylor College of Medicine, reported on the findings from one aspect of a program also developed to improve the transition from pediatric to adult care for rheumatology patients. It included periodic self-reported evaluation using the validated Adolescent Assessment of Preparation for Transition (ADAPT) survey. As the first step to better understanding the factors that can predict successful transition, the researchers surveyed returning patients with any rheumatologic diagnosis, aged 14 years and older, between July 2021 and November 2022.
Since the survey was automated through the electronic medical record, patients and their caregivers could respond during in-person or virtual visit check-in. The researchers calculated three composite scores out of 100 for self-management, prescription management, and transfer planning, using responses from the ADAPT survey. Among 462 patients who returned 670 surveys, 87% provided surveys that could be scored for at least one composite score. Most respondents were female (75%), White (69%), non-Hispanic (64%), English speaking (90%), and aged 14-17 years (83%).
The overall average score for self-management from 401 respondents was 35. For prescription management, the average score was 59 from 288 respondents, and the average transfer planning score was 17 from 367 respondents. Self-management and transfer planning scores both improved with age (P = .0001). Self-management scores rose from an average of 20 at age 14 to an average of 64 at age 18 and older. Transfer planning scores increased from an average of 1 at age 14 to an average of 49 at age 18 and older. Prescription management scores remained high across all ages, from an average of 59 at age 14 to an average score of 66 at age 18 and older (P = .044). Although the scores did not statistically vary by age or race, Hispanic patients did score higher in self-management with an average of 44.5, compared with 31 among other patients (P = .0001).
Only 21% of patients completed two surveys, and 8.4% completed all three surveys. The average time between the first and second surveys was 4 months, during which there was no statistically significant change in self-management or prescription management scores, but transfer planning scores did increase from 14 to 21 (P = .008) among the 90 patients who completed those surveys.
The researchers concluded from their analysis that “participation in the transition pathway can rapidly improve transfer planning scores, [but] opportunities remain to improve readiness in all domains.” The researchers are in the process of developing Spanish-language surveys.
No external funding was noted for either study. Dr. Bridges, Dr. Hersh, and Ms. Nasto reported no relevant financial relationships.
A version of this article first appeared on Medscape.com.
AT PRSYM 2023
Childhood lupus severity linked to social determinants of health
NEW ORLEANS – The sociodemographic characteristics of Black and Hispanic children with systemic lupus erythematosus (SLE) appear to play a strong role in influencing the severity of disease in these patients, according to two studies presented at the Pediatric Rheumatology Symposium.
One study showed an association between multiple determinants of health and disease severity among children seen in a large Texas city, and a separate descriptive cross-sectional cohort study of predominantly Black children at two centers in Mississippi and Alabama reinforced the finding of greater severity of disease and social hardships among this racial group.
The findings from both studies supplement existing evidence that the prevalence of childhood-onset SLE is greater among Black and Hispanic children.
“Several demographic and social determinants of health parameters influenced disease severity at levels that reached statistical significance, including insurance status, race/ethnicity, referral source, PCP [primary care provider] availability, primary language, and transportation needs,” Emily Beil, MD, a pediatric rheumatologist at Texas Children’s Hospital in Houston, told attendees at the conference, which was sponsored by the American College of Rheumatology. Her team’s goal, she said, was to “better understand our patient population and social disparities that contribute to disease severity.”
Dr. Beil and her colleagues conducted a retrospective review of 136 children who had been diagnosed with childhood-onset SLE between January 2018 and May 2022 at Texas Children’s Hospital. Only children who were younger than 18 years at the time of diagnosis at Texas Children’s were included. The analysis considered demographics, clinical characteristics, insurance status, social work consultation, access to a primary care provider, transportation needs, primary language, and other parameters related to social determinants of health.
The average age of the patients was 13 years, and most were girls (82%). Just over half were Hispanic (53%), and just over a quarter were Black (26%). Half had Medicaid or participated in the Children’s Health Insurance Program (CHIP), and 1 in 10 were uninsured (10%). Half the diagnoses were made during an inpatient admission; 36% were made on the floor, and 14% were made in the intensive care unit (ICU).
The average Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score was 12.5, and 48.5% of patients had severe disease, indicated by a score of at least 12. Only two in three children were documented as having a primary care physician (66%), and 32% preferred a language other than English. Most of the children (80%) had a social work consult.
Black and biracial children had higher SLEDAI scores at presentation. Non-Hispanic White children were less likely to have a social work consult, compared with other racial/ethnic groups (P = .01 for both). Central nervous system involvement was most prevalent among Black patients (P = .004). Cyclophosphamide was used most often for Black and biracial patients.
Uninsured patients were most likely to be diagnosed on an inpatient floor. The highest proportion of ICU admissions was among patients insured by Medicaid (P = .034). Average SLEDAI scores were highest among uninsured patients, followed by Medicaid patients. More than half of the patients who did not have insurance lacked access to a regular primary care provider, compared with 12% of Medicaid patients and 7% of privately insured patients (P = .001). All the uninsured patients had transportation needs, which was a significantly higher rate than among those with Medicaid (13%) or private insurance (15%) (P = .001). The highest percentage of social work consults was among patients who were insured by Medicaid or were without insurance (P = .001).
Salient demographics and clinical features
In the second presentation, Anita Dhanrajani, MD, assistant professor of pediatrics at the University of Mississippi Medical Center in Jackson, began by noting that Alabama and Mississippi are ranked in the top 10 states for the highest poverty rate: Mississippi is No. 1, and Alabama is No. 7. Further, 40% of children in Mississippi and 29% of children in Alabama are of African American ancestry, she said.
“So, we know that this population that we’re dealing with has several high-risk factors that can lead them to have poor outcomes, and yet, we haven’t really ever characterized their clinical features or their social demographic features,” Dr. Dhanrajani told attendees. “My hope is that with this very miniscule first step, we’re able to move towards solutions to decrease health care disparities in this population.”
She presented findings regarding the first of three aims in the study, which was to describe the baseline clinical, demographic, and socioeconomic profiles of childhood lupus patients at the two centers. The two other aims were to examine genetic factors potentially linked to poor outcomes in the cohort and to assess the mental health status of the population.
The study relied on a retrospective chart review for the 17 patients at the University of Mississippi Medical Center and on Childhood Arthritis and Rheumatology Research Alliance registry data for the 19 patients at the University of Alabama at Birmingham. Most of the patients (86%) were female, Black (78%), and insured by Medicaid (64%). The average age at diagnosis was 13 years. Most (83%) also lived in a ZIP code that met the criteria for a medium-high or high Social Vulnerability Index. The children had to travel an average 75 miles to see a rheumatologist, compared with the national average of 43 miles.
At diagnosis, their average Systemic Lupus International Collaborating Clinics (SLICC) score was 8.8, their average American College of Rheumatology score was 5.2, and their average SLEDAI score was 12.1 – the latter was substantially higher than the average 3.1 score in a multiethnic Canadian cohort (the 1000 Canadian Faces of Lupus Study) with 10% Black children (P < .00001). The SLEDAI score dropped to 6.8 at 6 months and to 4 at 1 year. Nearly half (47%) had a SLICC Damage Index (SDI) greater than 0, and one-third had an SDI of 2 or greater, compared with 16% and 7%, respectively, reported in other recent studies (P < .0001 for both).
“These disparities are very difficult to investigate in terms of causal relationships and [are] likely to be very modifiable,” Coziana Ciurtin, MD, PhD, associate professor of rheumatology at University College London, told this news organization. “I think the socioeconomic status, the level of education, poverty, [type of] medical insurance, and probably genetic variants are all underpinning the presentation, damage, or disease activity being very high, and also organ involvement,” such as the greater CNS involvement seen in non-White patients.
Being mindful of these risk profiles can help doctors in asking about patients’ support at home and their families’ education, beliefs, and cultural practices, Dr. Ciurtin added. “Helping them to engage and be involved in decision-making is probably the most important” aspect of learning this information about families, she said.
Collecting this information should not be the sole responsibility of the physician, added Eve Smith, PhD, MBCHB, an academic clinical lecturer at the University of Liverpool, England, who attended the presentations. Dr. Smith noted a discussion in a work group during the previous day of the conference concerning questionnaires for screening patients regarding the need for social services and for identifying areas in which patients and their families were having difficulties.
“Obviously, if you’re going to do that, you have to have access to someone who can actually help to deal with that. Some hospitals have patient navigators that can help, for example, with a food security issue to highlight resources within the community, so it’s not all on the doctor,” Dr. Smith said. “To really make a difference in this area, it can’t just be down to the doctor. There needs to be social care, there needs to be community-based interventions and things to do about it. Doctors can help identify these patients, or maybe somebody in the [medical] team can help with that, but there needs to be an intervention. Otherwise, you’re left with this problem without a solution that you can’t do anything about.”
The researchers did not note any external funding for either study. Dr. Beil, Dr. Dhanrajani, Dr. Smith, and Dr. Ciurtin reported no relevant financial relationships.
A version of this article originally appeared on Medscape.com.
NEW ORLEANS – The sociodemographic characteristics of Black and Hispanic children with systemic lupus erythematosus (SLE) appear to play a strong role in influencing the severity of disease in these patients, according to two studies presented at the Pediatric Rheumatology Symposium.
One study showed an association between multiple determinants of health and disease severity among children seen in a large Texas city, and a separate descriptive cross-sectional cohort study of predominantly Black children at two centers in Mississippi and Alabama reinforced the finding of greater severity of disease and social hardships among this racial group.
The findings from both studies supplement existing evidence that the prevalence of childhood-onset SLE is greater among Black and Hispanic children.
“Several demographic and social determinants of health parameters influenced disease severity at levels that reached statistical significance, including insurance status, race/ethnicity, referral source, PCP [primary care provider] availability, primary language, and transportation needs,” Emily Beil, MD, a pediatric rheumatologist at Texas Children’s Hospital in Houston, told attendees at the conference, which was sponsored by the American College of Rheumatology. Her team’s goal, she said, was to “better understand our patient population and social disparities that contribute to disease severity.”
Dr. Beil and her colleagues conducted a retrospective review of 136 children who had been diagnosed with childhood-onset SLE between January 2018 and May 2022 at Texas Children’s Hospital. Only children who were younger than 18 years at the time of diagnosis at Texas Children’s were included. The analysis considered demographics, clinical characteristics, insurance status, social work consultation, access to a primary care provider, transportation needs, primary language, and other parameters related to social determinants of health.
The average age of the patients was 13 years, and most were girls (82%). Just over half were Hispanic (53%), and just over a quarter were Black (26%). Half had Medicaid or participated in the Children’s Health Insurance Program (CHIP), and 1 in 10 were uninsured (10%). Half the diagnoses were made during an inpatient admission; 36% were made on the floor, and 14% were made in the intensive care unit (ICU).
The average Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score was 12.5, and 48.5% of patients had severe disease, indicated by a score of at least 12. Only two in three children were documented as having a primary care physician (66%), and 32% preferred a language other than English. Most of the children (80%) had a social work consult.
Black and biracial children had higher SLEDAI scores at presentation. Non-Hispanic White children were less likely to have a social work consult, compared with other racial/ethnic groups (P = .01 for both). Central nervous system involvement was most prevalent among Black patients (P = .004). Cyclophosphamide was used most often for Black and biracial patients.
Uninsured patients were most likely to be diagnosed on an inpatient floor. The highest proportion of ICU admissions was among patients insured by Medicaid (P = .034). Average SLEDAI scores were highest among uninsured patients, followed by Medicaid patients. More than half of the patients who did not have insurance lacked access to a regular primary care provider, compared with 12% of Medicaid patients and 7% of privately insured patients (P = .001). All the uninsured patients had transportation needs, which was a significantly higher rate than among those with Medicaid (13%) or private insurance (15%) (P = .001). The highest percentage of social work consults was among patients who were insured by Medicaid or were without insurance (P = .001).
Salient demographics and clinical features
In the second presentation, Anita Dhanrajani, MD, assistant professor of pediatrics at the University of Mississippi Medical Center in Jackson, began by noting that Alabama and Mississippi are ranked in the top 10 states for the highest poverty rate: Mississippi is No. 1, and Alabama is No. 7. Further, 40% of children in Mississippi and 29% of children in Alabama are of African American ancestry, she said.
“So, we know that this population that we’re dealing with has several high-risk factors that can lead them to have poor outcomes, and yet, we haven’t really ever characterized their clinical features or their social demographic features,” Dr. Dhanrajani told attendees. “My hope is that with this very miniscule first step, we’re able to move towards solutions to decrease health care disparities in this population.”
She presented findings regarding the first of three aims in the study, which was to describe the baseline clinical, demographic, and socioeconomic profiles of childhood lupus patients at the two centers. The two other aims were to examine genetic factors potentially linked to poor outcomes in the cohort and to assess the mental health status of the population.
The study relied on a retrospective chart review for the 17 patients at the University of Mississippi Medical Center and on Childhood Arthritis and Rheumatology Research Alliance registry data for the 19 patients at the University of Alabama at Birmingham. Most of the patients (86%) were female, Black (78%), and insured by Medicaid (64%). The average age at diagnosis was 13 years. Most (83%) also lived in a ZIP code that met the criteria for a medium-high or high Social Vulnerability Index. The children had to travel an average 75 miles to see a rheumatologist, compared with the national average of 43 miles.
At diagnosis, their average Systemic Lupus International Collaborating Clinics (SLICC) score was 8.8, their average American College of Rheumatology score was 5.2, and their average SLEDAI score was 12.1 – the latter was substantially higher than the average 3.1 score in a multiethnic Canadian cohort (the 1000 Canadian Faces of Lupus Study) with 10% Black children (P < .00001). The SLEDAI score dropped to 6.8 at 6 months and to 4 at 1 year. Nearly half (47%) had a SLICC Damage Index (SDI) greater than 0, and one-third had an SDI of 2 or greater, compared with 16% and 7%, respectively, reported in other recent studies (P < .0001 for both).
“These disparities are very difficult to investigate in terms of causal relationships and [are] likely to be very modifiable,” Coziana Ciurtin, MD, PhD, associate professor of rheumatology at University College London, told this news organization. “I think the socioeconomic status, the level of education, poverty, [type of] medical insurance, and probably genetic variants are all underpinning the presentation, damage, or disease activity being very high, and also organ involvement,” such as the greater CNS involvement seen in non-White patients.
Being mindful of these risk profiles can help doctors in asking about patients’ support at home and their families’ education, beliefs, and cultural practices, Dr. Ciurtin added. “Helping them to engage and be involved in decision-making is probably the most important” aspect of learning this information about families, she said.
Collecting this information should not be the sole responsibility of the physician, added Eve Smith, PhD, MBCHB, an academic clinical lecturer at the University of Liverpool, England, who attended the presentations. Dr. Smith noted a discussion in a work group during the previous day of the conference concerning questionnaires for screening patients regarding the need for social services and for identifying areas in which patients and their families were having difficulties.
“Obviously, if you’re going to do that, you have to have access to someone who can actually help to deal with that. Some hospitals have patient navigators that can help, for example, with a food security issue to highlight resources within the community, so it’s not all on the doctor,” Dr. Smith said. “To really make a difference in this area, it can’t just be down to the doctor. There needs to be social care, there needs to be community-based interventions and things to do about it. Doctors can help identify these patients, or maybe somebody in the [medical] team can help with that, but there needs to be an intervention. Otherwise, you’re left with this problem without a solution that you can’t do anything about.”
The researchers did not note any external funding for either study. Dr. Beil, Dr. Dhanrajani, Dr. Smith, and Dr. Ciurtin reported no relevant financial relationships.
A version of this article originally appeared on Medscape.com.
NEW ORLEANS – The sociodemographic characteristics of Black and Hispanic children with systemic lupus erythematosus (SLE) appear to play a strong role in influencing the severity of disease in these patients, according to two studies presented at the Pediatric Rheumatology Symposium.
One study showed an association between multiple determinants of health and disease severity among children seen in a large Texas city, and a separate descriptive cross-sectional cohort study of predominantly Black children at two centers in Mississippi and Alabama reinforced the finding of greater severity of disease and social hardships among this racial group.
The findings from both studies supplement existing evidence that the prevalence of childhood-onset SLE is greater among Black and Hispanic children.
“Several demographic and social determinants of health parameters influenced disease severity at levels that reached statistical significance, including insurance status, race/ethnicity, referral source, PCP [primary care provider] availability, primary language, and transportation needs,” Emily Beil, MD, a pediatric rheumatologist at Texas Children’s Hospital in Houston, told attendees at the conference, which was sponsored by the American College of Rheumatology. Her team’s goal, she said, was to “better understand our patient population and social disparities that contribute to disease severity.”
Dr. Beil and her colleagues conducted a retrospective review of 136 children who had been diagnosed with childhood-onset SLE between January 2018 and May 2022 at Texas Children’s Hospital. Only children who were younger than 18 years at the time of diagnosis at Texas Children’s were included. The analysis considered demographics, clinical characteristics, insurance status, social work consultation, access to a primary care provider, transportation needs, primary language, and other parameters related to social determinants of health.
The average age of the patients was 13 years, and most were girls (82%). Just over half were Hispanic (53%), and just over a quarter were Black (26%). Half had Medicaid or participated in the Children’s Health Insurance Program (CHIP), and 1 in 10 were uninsured (10%). Half the diagnoses were made during an inpatient admission; 36% were made on the floor, and 14% were made in the intensive care unit (ICU).
The average Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score was 12.5, and 48.5% of patients had severe disease, indicated by a score of at least 12. Only two in three children were documented as having a primary care physician (66%), and 32% preferred a language other than English. Most of the children (80%) had a social work consult.
Black and biracial children had higher SLEDAI scores at presentation. Non-Hispanic White children were less likely to have a social work consult, compared with other racial/ethnic groups (P = .01 for both). Central nervous system involvement was most prevalent among Black patients (P = .004). Cyclophosphamide was used most often for Black and biracial patients.
Uninsured patients were most likely to be diagnosed on an inpatient floor. The highest proportion of ICU admissions was among patients insured by Medicaid (P = .034). Average SLEDAI scores were highest among uninsured patients, followed by Medicaid patients. More than half of the patients who did not have insurance lacked access to a regular primary care provider, compared with 12% of Medicaid patients and 7% of privately insured patients (P = .001). All the uninsured patients had transportation needs, which was a significantly higher rate than among those with Medicaid (13%) or private insurance (15%) (P = .001). The highest percentage of social work consults was among patients who were insured by Medicaid or were without insurance (P = .001).
Salient demographics and clinical features
In the second presentation, Anita Dhanrajani, MD, assistant professor of pediatrics at the University of Mississippi Medical Center in Jackson, began by noting that Alabama and Mississippi are ranked in the top 10 states for the highest poverty rate: Mississippi is No. 1, and Alabama is No. 7. Further, 40% of children in Mississippi and 29% of children in Alabama are of African American ancestry, she said.
“So, we know that this population that we’re dealing with has several high-risk factors that can lead them to have poor outcomes, and yet, we haven’t really ever characterized their clinical features or their social demographic features,” Dr. Dhanrajani told attendees. “My hope is that with this very miniscule first step, we’re able to move towards solutions to decrease health care disparities in this population.”
She presented findings regarding the first of three aims in the study, which was to describe the baseline clinical, demographic, and socioeconomic profiles of childhood lupus patients at the two centers. The two other aims were to examine genetic factors potentially linked to poor outcomes in the cohort and to assess the mental health status of the population.
The study relied on a retrospective chart review for the 17 patients at the University of Mississippi Medical Center and on Childhood Arthritis and Rheumatology Research Alliance registry data for the 19 patients at the University of Alabama at Birmingham. Most of the patients (86%) were female, Black (78%), and insured by Medicaid (64%). The average age at diagnosis was 13 years. Most (83%) also lived in a ZIP code that met the criteria for a medium-high or high Social Vulnerability Index. The children had to travel an average 75 miles to see a rheumatologist, compared with the national average of 43 miles.
At diagnosis, their average Systemic Lupus International Collaborating Clinics (SLICC) score was 8.8, their average American College of Rheumatology score was 5.2, and their average SLEDAI score was 12.1 – the latter was substantially higher than the average 3.1 score in a multiethnic Canadian cohort (the 1000 Canadian Faces of Lupus Study) with 10% Black children (P < .00001). The SLEDAI score dropped to 6.8 at 6 months and to 4 at 1 year. Nearly half (47%) had a SLICC Damage Index (SDI) greater than 0, and one-third had an SDI of 2 or greater, compared with 16% and 7%, respectively, reported in other recent studies (P < .0001 for both).
“These disparities are very difficult to investigate in terms of causal relationships and [are] likely to be very modifiable,” Coziana Ciurtin, MD, PhD, associate professor of rheumatology at University College London, told this news organization. “I think the socioeconomic status, the level of education, poverty, [type of] medical insurance, and probably genetic variants are all underpinning the presentation, damage, or disease activity being very high, and also organ involvement,” such as the greater CNS involvement seen in non-White patients.
Being mindful of these risk profiles can help doctors in asking about patients’ support at home and their families’ education, beliefs, and cultural practices, Dr. Ciurtin added. “Helping them to engage and be involved in decision-making is probably the most important” aspect of learning this information about families, she said.
Collecting this information should not be the sole responsibility of the physician, added Eve Smith, PhD, MBCHB, an academic clinical lecturer at the University of Liverpool, England, who attended the presentations. Dr. Smith noted a discussion in a work group during the previous day of the conference concerning questionnaires for screening patients regarding the need for social services and for identifying areas in which patients and their families were having difficulties.
“Obviously, if you’re going to do that, you have to have access to someone who can actually help to deal with that. Some hospitals have patient navigators that can help, for example, with a food security issue to highlight resources within the community, so it’s not all on the doctor,” Dr. Smith said. “To really make a difference in this area, it can’t just be down to the doctor. There needs to be social care, there needs to be community-based interventions and things to do about it. Doctors can help identify these patients, or maybe somebody in the [medical] team can help with that, but there needs to be an intervention. Otherwise, you’re left with this problem without a solution that you can’t do anything about.”
The researchers did not note any external funding for either study. Dr. Beil, Dr. Dhanrajani, Dr. Smith, and Dr. Ciurtin reported no relevant financial relationships.
A version of this article originally appeared on Medscape.com.
AT PRSYM 2023
Little change in rheumatology faculty coverage in pediatric residency programs in nearly 20 years
NEW ORLEANS – More than one-third of pediatric residency programs do not have a pediatric rheumatologist on faculty, a situation that has changed little since 2004, according to a poster presented at the Pediatric Rheumatology Symposium 2023 conference.
“This shortage has significant downstream effects,” according to author Miriah Gillispie-Taylor, MD, an assistant professor of pediatric rheumatology at Baylor College of Medicine and Texas Children’s Hospital in Houston. Without adequate education, it’s unreasonable to expect that a pediatrician will recognize the great diversity of presentations among rheumatic diseases, for example. “Without recognition, patients are not referred in a timely manner, and earlier identification and treatment of rheumatic diseases leads to improved outcomes,” Dr. Gillispie-Taylor said.
Currently, eight U.S. states do not have a board-certified pediatric rheumatologist, including Alaska. Dr. Gillispie-Taylor cited a 2006 study that found that one-third of medical schools (33%) and 40% of U.S. pediatric residency programs did not have an on-site pediatric rheumatologist in 2004.
As the long-standing workforce shortage in pediatric rheumatology continues, Dr. Gillispie-Taylor and her colleagues investigated whether increasing awareness of this problem has influenced the number of United States and Puerto Rico residency training programs with pediatric rheumatology faculty from 2004 to present.
The researchers identified 212 pediatric residency programs accredited by the Accreditation Council for Graduate Medical Education for 2022-2023 and reviewed their program website to see which ones had affiliated pediatric rheumatology faculty. After determining the faculty from the website for 85% of the programs, the researchers emailed the other programs to find out whether a pediatric rheumatologist was on faculty, filling out another 6% of the programs. Most of the remaining uncategorized programs (7%) were categorized at a meeting of the Childhood Arthritis and Rheumatology Research Alliance medical education workgroup. Only 2% of programs could not be ultimately categorized.
The region with the greatest proportion of pediatric residency programs that had a pediatric rheumatologist was the Southeast, where 95% (36 of 38 programs) of programs had one on faculty. The Southwest, comprising Texas, Oklahoma, New Mexico, and Arizona, had the lowest proportion: 43% (9 of 21 programs). For the other regions, 69% of the West/Pacific Northwest (18 of 26), 62% of the Midwest (28 of 45), and 61% of the Northeast (39 of 64) programs had a pediatric rheumatologist on faculty. Three of Puerto Rico’s four programs had one as well.
Overall, 63% of programs had a pediatric rheumatologist on faculty, and 36% did not; the state of three programs was unknown.
The large proportion of programs without a pediatric rheumatologist “limits exposure to rheumatologic conditions and learning opportunities during residency and contribute to declining fellow match rates,” the authors concluded. They noted that only 62.8% of pediatric rheumatology fellowship positions were filled in 2022, down slightly from the 69.2% filled in 2021, according to report data from the National Matching Resident Program.
The researchers acknowledged that their results could be skewed if website information was outdated for any programs, and it’s difficult to determine which programs might lack resources on the basis of only publicly available information. Though programs without pediatric rheumatologists might benefit from visiting professorships, it can be difficult to identify which ones, they added.
The authors recommend two next steps: one, establishing areas of essential knowledge in pediatric rheumatology to enable the creation of learning objectives so programs can focus their educational efforts; and two, continuing efforts to understand residents’ motivation to pursue fellowships in pediatric rheumatology for the purpose of improving recruitment.
Two medical students at Dr. Gillispie-Taylor’s institution spoke with this news organization about their thoughts on the findings and how they were approaching their own career goals in medicine in light of these findings.
Kyla Fergason, a second-year medical student at Baylor College of Medicine, said that she thinks she wants to pursue pediatrics or meds-peds. Though she’s not sure whether she specifically wants to pursue pediatric rheumatology, she is very interested in the area and said that she has learned much from the Pediatric Rheumatology Symposium conference. She found the dearth of pediatric rheumatology faculty at residency programs worrisome, particularly in states like Alaska and Hawaii because they aren’t contiguous with the rest of the United States. Only three pediatric rheumatologists are practicing in Hawaii.
“It’s really concerning that sometimes there is not any rheumatologist there to see the patient,” Ms. Fergason told this news organization. “These are diseases that affect people chronically throughout their entire lives, so it’s definitely concerning to think that, at a time when they could be helped and there could be interventions made, none are made because there’s just no one available.”
Kristiana Nasto, a third-year medical student at Baylor College of Medicine, is similarly interested in pediatrics but leaning more toward meds-peds and has an interest in rheumatology as well. She was surprised at how many programs had no pediatric rheumatologist on faculty because Baylor has a robust program.
“I was not aware of the fact that other states or other parts of Texas do not have the luxury of the great rheumatologists that we have at Baylor College of Medicine,” Ms. Nasto said. “That can definitely impact care for many patients because some of these rheumatologic diseases are so unique and challenging to treat that they require specialized care, so it makes me a bit sad that this is the case.”
Dr. Gillispie-Taylor has received an educational grant from Pfizer. Ms. Fergason and Ms. Nasto had no disclosures. No external funding was noted for the study.
A version of this article first appeared on Medscape.com.
NEW ORLEANS – More than one-third of pediatric residency programs do not have a pediatric rheumatologist on faculty, a situation that has changed little since 2004, according to a poster presented at the Pediatric Rheumatology Symposium 2023 conference.
“This shortage has significant downstream effects,” according to author Miriah Gillispie-Taylor, MD, an assistant professor of pediatric rheumatology at Baylor College of Medicine and Texas Children’s Hospital in Houston. Without adequate education, it’s unreasonable to expect that a pediatrician will recognize the great diversity of presentations among rheumatic diseases, for example. “Without recognition, patients are not referred in a timely manner, and earlier identification and treatment of rheumatic diseases leads to improved outcomes,” Dr. Gillispie-Taylor said.
Currently, eight U.S. states do not have a board-certified pediatric rheumatologist, including Alaska. Dr. Gillispie-Taylor cited a 2006 study that found that one-third of medical schools (33%) and 40% of U.S. pediatric residency programs did not have an on-site pediatric rheumatologist in 2004.
As the long-standing workforce shortage in pediatric rheumatology continues, Dr. Gillispie-Taylor and her colleagues investigated whether increasing awareness of this problem has influenced the number of United States and Puerto Rico residency training programs with pediatric rheumatology faculty from 2004 to present.
The researchers identified 212 pediatric residency programs accredited by the Accreditation Council for Graduate Medical Education for 2022-2023 and reviewed their program website to see which ones had affiliated pediatric rheumatology faculty. After determining the faculty from the website for 85% of the programs, the researchers emailed the other programs to find out whether a pediatric rheumatologist was on faculty, filling out another 6% of the programs. Most of the remaining uncategorized programs (7%) were categorized at a meeting of the Childhood Arthritis and Rheumatology Research Alliance medical education workgroup. Only 2% of programs could not be ultimately categorized.
The region with the greatest proportion of pediatric residency programs that had a pediatric rheumatologist was the Southeast, where 95% (36 of 38 programs) of programs had one on faculty. The Southwest, comprising Texas, Oklahoma, New Mexico, and Arizona, had the lowest proportion: 43% (9 of 21 programs). For the other regions, 69% of the West/Pacific Northwest (18 of 26), 62% of the Midwest (28 of 45), and 61% of the Northeast (39 of 64) programs had a pediatric rheumatologist on faculty. Three of Puerto Rico’s four programs had one as well.
Overall, 63% of programs had a pediatric rheumatologist on faculty, and 36% did not; the state of three programs was unknown.
The large proportion of programs without a pediatric rheumatologist “limits exposure to rheumatologic conditions and learning opportunities during residency and contribute to declining fellow match rates,” the authors concluded. They noted that only 62.8% of pediatric rheumatology fellowship positions were filled in 2022, down slightly from the 69.2% filled in 2021, according to report data from the National Matching Resident Program.
The researchers acknowledged that their results could be skewed if website information was outdated for any programs, and it’s difficult to determine which programs might lack resources on the basis of only publicly available information. Though programs without pediatric rheumatologists might benefit from visiting professorships, it can be difficult to identify which ones, they added.
The authors recommend two next steps: one, establishing areas of essential knowledge in pediatric rheumatology to enable the creation of learning objectives so programs can focus their educational efforts; and two, continuing efforts to understand residents’ motivation to pursue fellowships in pediatric rheumatology for the purpose of improving recruitment.
Two medical students at Dr. Gillispie-Taylor’s institution spoke with this news organization about their thoughts on the findings and how they were approaching their own career goals in medicine in light of these findings.
Kyla Fergason, a second-year medical student at Baylor College of Medicine, said that she thinks she wants to pursue pediatrics or meds-peds. Though she’s not sure whether she specifically wants to pursue pediatric rheumatology, she is very interested in the area and said that she has learned much from the Pediatric Rheumatology Symposium conference. She found the dearth of pediatric rheumatology faculty at residency programs worrisome, particularly in states like Alaska and Hawaii because they aren’t contiguous with the rest of the United States. Only three pediatric rheumatologists are practicing in Hawaii.
“It’s really concerning that sometimes there is not any rheumatologist there to see the patient,” Ms. Fergason told this news organization. “These are diseases that affect people chronically throughout their entire lives, so it’s definitely concerning to think that, at a time when they could be helped and there could be interventions made, none are made because there’s just no one available.”
Kristiana Nasto, a third-year medical student at Baylor College of Medicine, is similarly interested in pediatrics but leaning more toward meds-peds and has an interest in rheumatology as well. She was surprised at how many programs had no pediatric rheumatologist on faculty because Baylor has a robust program.
“I was not aware of the fact that other states or other parts of Texas do not have the luxury of the great rheumatologists that we have at Baylor College of Medicine,” Ms. Nasto said. “That can definitely impact care for many patients because some of these rheumatologic diseases are so unique and challenging to treat that they require specialized care, so it makes me a bit sad that this is the case.”
Dr. Gillispie-Taylor has received an educational grant from Pfizer. Ms. Fergason and Ms. Nasto had no disclosures. No external funding was noted for the study.
A version of this article first appeared on Medscape.com.
NEW ORLEANS – More than one-third of pediatric residency programs do not have a pediatric rheumatologist on faculty, a situation that has changed little since 2004, according to a poster presented at the Pediatric Rheumatology Symposium 2023 conference.
“This shortage has significant downstream effects,” according to author Miriah Gillispie-Taylor, MD, an assistant professor of pediatric rheumatology at Baylor College of Medicine and Texas Children’s Hospital in Houston. Without adequate education, it’s unreasonable to expect that a pediatrician will recognize the great diversity of presentations among rheumatic diseases, for example. “Without recognition, patients are not referred in a timely manner, and earlier identification and treatment of rheumatic diseases leads to improved outcomes,” Dr. Gillispie-Taylor said.
Currently, eight U.S. states do not have a board-certified pediatric rheumatologist, including Alaska. Dr. Gillispie-Taylor cited a 2006 study that found that one-third of medical schools (33%) and 40% of U.S. pediatric residency programs did not have an on-site pediatric rheumatologist in 2004.
As the long-standing workforce shortage in pediatric rheumatology continues, Dr. Gillispie-Taylor and her colleagues investigated whether increasing awareness of this problem has influenced the number of United States and Puerto Rico residency training programs with pediatric rheumatology faculty from 2004 to present.
The researchers identified 212 pediatric residency programs accredited by the Accreditation Council for Graduate Medical Education for 2022-2023 and reviewed their program website to see which ones had affiliated pediatric rheumatology faculty. After determining the faculty from the website for 85% of the programs, the researchers emailed the other programs to find out whether a pediatric rheumatologist was on faculty, filling out another 6% of the programs. Most of the remaining uncategorized programs (7%) were categorized at a meeting of the Childhood Arthritis and Rheumatology Research Alliance medical education workgroup. Only 2% of programs could not be ultimately categorized.
The region with the greatest proportion of pediatric residency programs that had a pediatric rheumatologist was the Southeast, where 95% (36 of 38 programs) of programs had one on faculty. The Southwest, comprising Texas, Oklahoma, New Mexico, and Arizona, had the lowest proportion: 43% (9 of 21 programs). For the other regions, 69% of the West/Pacific Northwest (18 of 26), 62% of the Midwest (28 of 45), and 61% of the Northeast (39 of 64) programs had a pediatric rheumatologist on faculty. Three of Puerto Rico’s four programs had one as well.
Overall, 63% of programs had a pediatric rheumatologist on faculty, and 36% did not; the state of three programs was unknown.
The large proportion of programs without a pediatric rheumatologist “limits exposure to rheumatologic conditions and learning opportunities during residency and contribute to declining fellow match rates,” the authors concluded. They noted that only 62.8% of pediatric rheumatology fellowship positions were filled in 2022, down slightly from the 69.2% filled in 2021, according to report data from the National Matching Resident Program.
The researchers acknowledged that their results could be skewed if website information was outdated for any programs, and it’s difficult to determine which programs might lack resources on the basis of only publicly available information. Though programs without pediatric rheumatologists might benefit from visiting professorships, it can be difficult to identify which ones, they added.
The authors recommend two next steps: one, establishing areas of essential knowledge in pediatric rheumatology to enable the creation of learning objectives so programs can focus their educational efforts; and two, continuing efforts to understand residents’ motivation to pursue fellowships in pediatric rheumatology for the purpose of improving recruitment.
Two medical students at Dr. Gillispie-Taylor’s institution spoke with this news organization about their thoughts on the findings and how they were approaching their own career goals in medicine in light of these findings.
Kyla Fergason, a second-year medical student at Baylor College of Medicine, said that she thinks she wants to pursue pediatrics or meds-peds. Though she’s not sure whether she specifically wants to pursue pediatric rheumatology, she is very interested in the area and said that she has learned much from the Pediatric Rheumatology Symposium conference. She found the dearth of pediatric rheumatology faculty at residency programs worrisome, particularly in states like Alaska and Hawaii because they aren’t contiguous with the rest of the United States. Only three pediatric rheumatologists are practicing in Hawaii.
“It’s really concerning that sometimes there is not any rheumatologist there to see the patient,” Ms. Fergason told this news organization. “These are diseases that affect people chronically throughout their entire lives, so it’s definitely concerning to think that, at a time when they could be helped and there could be interventions made, none are made because there’s just no one available.”
Kristiana Nasto, a third-year medical student at Baylor College of Medicine, is similarly interested in pediatrics but leaning more toward meds-peds and has an interest in rheumatology as well. She was surprised at how many programs had no pediatric rheumatologist on faculty because Baylor has a robust program.
“I was not aware of the fact that other states or other parts of Texas do not have the luxury of the great rheumatologists that we have at Baylor College of Medicine,” Ms. Nasto said. “That can definitely impact care for many patients because some of these rheumatologic diseases are so unique and challenging to treat that they require specialized care, so it makes me a bit sad that this is the case.”
Dr. Gillispie-Taylor has received an educational grant from Pfizer. Ms. Fergason and Ms. Nasto had no disclosures. No external funding was noted for the study.
A version of this article first appeared on Medscape.com.
AT PRSYM 2023
Biosimilars and patients: Discussions should address safety, cost, and anxiety about change
Rheumatologist Marcus Snow, MD, is comfortable with prescribing biosimilars as a first-line, first-time biologic, and discussing them with patients.
“If a biosimilar is on the market, it has gone through rigorous study proving its effectiveness and equivalence to a bio-originator,” said Dr. Snow, a rheumatologist with the University of Nebraska Medical Center, Omaha, and chair of the American College of Rheumatology’s Committee on Rheumatologic Care.
The formulary makes a big difference in the conversation about options, he said. “The formularies dictate what we can prescribe. It may not be appropriate, but it is reality. The cost of biologics for a patient without insurance coverage makes it impossible to afford.”
He will often tell patients that he’ll fight any changes or formulary restrictions he does not agree with. “However, when I see patients in follow-up, even if there is no known change on the horizon, I may bring up biosimilars when we have a moment to chat about them to familiarize them with what may happen in the future.”
The need for patient education on biosimilars presents a barrier to realizing their potential to save money and expand choice, noted Cardinal Health in its 2023 biosimilars report. Of 103 rheumatologists who responded to a Cardinal Health survey, 85% agreed that patient education was important. But those conversations can take an uncomfortable turn if the patient pushes back against taking a biosimilar owing to cost or safety concerns.
It’s not uncommon for a patient to express some anxiety about biosimilars, especially if they’re doing well on a current treatment plan. Most patients do not want any changes that may lead to worsening disease control, Dr. Snow said.
Patients and physicians alike often don’t understand the mechanics of biosimilars. “There’s a lot of misinformation about this,” said Sameer Awsare, MD, an associate executive director for The Permanente Medical Group in Campbell, Calif. Patients should know that a biosimilar will be as clinically efficacious as the medicine they’ve been on, with the same safety profiles, said Dr. Awsare, who works with Kaiser Permanente’s pharmacy partners on biosimilars.
Insurance often drives the conversation
The global anti-inflammatory biologics market is anticipated to reach $150 billion by 2027, according to a recent CVS report. As of March 2023, the Food and Drug Administration had approved 40 biosimilars to 11 different reference products. There are 28 on the U.S. market and 100 more in development. Projected to save more than $180 billion over the next 5 years, they are anticipated to expand choice and drive competition.
Rheumatologists, dermatologists, and gastroenterologists are frequent prescribers, although their choices for immune-mediated inflammatory diseases are limited to tumor necrosis factor inhibitors (infliximab [Remicade] originator and adalimumab [Humira] originator) and anti-CD20 agents, such as rituximab (Rituxan) originator.
Benefit design or formulary usually dictates what medicine a patient receives. “Because of significantly higher out-of-pocket cost or formulary positioning, patients may end up with a generic or a biosimilar instead of a brand-name medicine or branded biologic,” said Robert Popovian, PharmD, MS, chief science policy officer of the Global Healthy Living Foundation.
Insurers rarely offer both Remicade and biosimilar infliximab, allowing the doctor to choose, said Miguel Regueiro, MD, chair of the Cleveland Clinic’s Digestive Disease & Surgery Institute, who prescribes infliximab biosimilars. Most often, the payer will choose the lower-cost biosimilar. “I am fine with the biosimilar, either as a new start or a switch from the reference product.”
However, the patient might feel differently. They can form an attachment to the reference medication if it has prevented severe illness. “They do not want to change, as they feel they are going on a ‘new’ medication that will not work as well,” Dr. Regueiro said.
This is where the education comes in: to reassure patients that a biosimilar will work just as well as the reference product. “For patients who have done well for years on a biologic, more time needs to be spent reassuring them and answering questions,” compared with a patient just starting on a biosimilar, he advised.
But not all physicians are quick to prescribe biosimilars.
Especially with psoriasis, which has so many strong options for reference drugs, a switch may be hard to justify, said dermatologist Stephanie K. Fabbro, MD, assistant professor at Northeast Ohio Medical University, Rootstown. “If I have a preference, I would rather switch a patient to a drug from a different class without a biosimilar option to reduce the possibility of pushback.”
Dr. Fabbro, part of the core faculty in the Riverside Methodist Hospital Dermatology Residency Program in Columbus, will share data from clinical trials and postmarket surveillance with patients to support her decision.
Conversations about cost
Patients may also push back if they don’t save money when switching to a biosimilar. “This dilemma raises the question of who is profiting when a biosimilar is dispensed,” Dr. Popovian said. Insurers and pharmacy benefit managers (PBMs) that take additional concessions from biopharmaceutical manufacturers in the form of rebates and fees will often pocket this money as profit instead of passing savings back to the patient to help reduce their out-of-pocket requirement, he added.
If an originator biologic and a biosimilar are available, “as a pharmacist, I will choose the medicine that will incur the lowest out-of-pocket cost for the patient,” Dr. Popovian said.
Discussing cost – and who dictates which biosimilar is on the formulary – is an important conversation to have with patients, said Vivek Kaul, MD, Segal-Watson Professor of Medicine at the University of Rochester (N.Y.) Medical Center.
Providing equivalent clinical efficacy while saving costs is the economic reality of biosimilars, Dr. Kaul said. Third-party payers regularly evaluate how to provide the same quality of care while saving money. Physicians and patients alike “must be mindful that as time goes on, if the science on biosimilars stays robust, if the adoption is more widespread and the cost-saving proposition turns out to be true, more formularies will be attracted to replacing the reference product with the biosimilar counterpart.”
Providers and patients can weigh the options if a formulary suddenly switches to a biosimilar, Dr. Kaul continued. “You can accept the novel product on the formulary or may have to face out-of-pocket expenses as a patient.” If providers and patients have concerns about the biosimilar, they can always appeal if there’s solid scientific evidence that supports reverting back to the reference product.
“If you think the biosimilar is equally efficacious, comes at a lower cost, and is right for the patient, then the providers should tell the patient that,” he added.
Some studies have questioned whether the biosimilars will save money, compared with the reference drug, Dr. Fabbro noted. Medicare, for example, may pay only for a certain percentage of an approved biosimilar, saddling the patient with a monthly copay costing thousands of dollars. “It is unclear whether biosimilar manufacturers will have the same level of patient support programs as the reference drug companies.”
For that reason, physicians should also inform patients about the robust patient assistance and copay assistance programs many reference drug manufacturers offer, she said.
Biosimilars 101: Familiarizing patients
Safety and ease of use are other common concerns about biosimilars. Patients may ask if the application is different, or why it’s advantageous to switch to a biosimilar, Dr. Awsare said.
Sometimes the syringe or injector for a biosimilar might look different from that of the originator drug, he said.
Anecdotally, Dr. Fabbro has heard stories of patients having injection reactions that they did not experience with the reference drug or having a disease flare-up after starting a biosimilar. 
As is the case with reference products, in their conversations with patients, clinicians should address the adverse event profile of biosimilars, offering data points from published studies and clinical guidelines that support the use of these products. “There should be an emphasis on patient education around efficacy and any side effects, and how the profile of the reference product compares with a proposed biosimilar,” Dr. Kaul suggested.
When Dr. Snow discusses biosimilars and generics, “I make sure to share this in an understandable way based on the patient’s scientific background, or lack thereof,” he said. If there is enough time, he also discusses how European- and U.S.-sourced biologics are slightly different.
Pharmacists should tell patients to expect the same clinical outcomes from a biosimilar, Dr. Popovian said. However, if they have any reduction in efficacy or potential safety concerns, they should communicate with their physician or pharmacist immediately.
In Dr. Regueiro’s practice, a pharmacist specializing in inflammatory bowel disease often has a one-on-one meeting with patients to educate and answer questions. “Additionally, we provide them the Crohn’s and Colitis Foundation web link on biosimilars,” said Dr. Regueiro.
A village approach to education
When biosimilars first came out, there were no formal education materials, Dr. Awsare said. Kaiser Permanente decided to create its own educational materials, not just for patients but also to help educate its primary care doctors; the rheumatologists, dermatologists, and gastroenterologists using the biosimilars; the nurses infusing patients; and the pharmacists preparing the biosimilars.
The health system also has a different approach to choosing medication. Instead of having an insurance company or PBM decide what’s in the formulary, clinicians work with the pharmacists at Kaiser to look at clinical evidence and decide which biosimilar to use. Most of its plans also provide lower copays to patients when they use the biosimilar.
This was the approach for Humira biosimilars, Dr. Awsare said. Eight will be on the market in 2023. “Our rheumatologists, dermatologists, and gastroenterologists looked at the data from Europe, looked at some real-world evidence, and then said: ‘We think this one’s going to be the best one for our patients.’ ”
Having clinicians choose the biosimilar instead of a health plan makes it a lot easier to have conversations with patients, he said. “Once we’ve moved that market share to that particular biosimilar, we give our physicians the time to have those discussions.”
Clinical pharmacists also provide educational support, offering guidance on issues such as side effects, as patients transition to the biosimilar. “We like to use the word ‘transition’ because it’s essentially the same biologic. So, you’re not actually switching,” Dr. Awsare said.
No consensus on interchangeability
Whether the conversation on interchangeability will affect patient conversations with physicians depends on who you ask.
If a biosimilar has an interchangeability designation, it means that the pharmacist can substitute it without the intervention of the clinician who prescribed the reference product. It does not relate to the quality, safety, or effectiveness of biosimilars or interchangeable biosimilar products, Dr. Popovian said.
The United States is the only country that has this designation. Even though it’s not identical to the originator drug, a biosimilar has the same clinical efficacy and safety profile. “So clinically, interchangeability is meaningless,” Dr. Awsare said.
In its report on biosimilars in the autoimmune category, CVS acknowledged that interchangeability was important but would not be a significant factor in driving adoption of biosimilars. However, in a Cardinal Health survey of 72 gastroenterologists, 38% cited the interchangeability of biosimilars as a top concern for adalimumab biosimilars, along with transitioning patients from Humira to a biosimilar (44%).
“Patient education regarding biosimilar safety, efficacy, and interchangeability appears paramount to the acceptance of these products, particularly for patients who are switched from a reference product,” Dr. Kaul noted in the Cardinal Health report.
Wherever supported by data, Dr. Kaul recommends incorporating biosimilar use and interchangeability into best practice guidelines going forward. “That will go a long way in disseminating the latest information on this topic and position this paradigm for increased adoption among providers.”
Some physicians like Dr. Snow aren’t that concerned with interchangeability. This hasn’t affected conversations with patients, he said. Multiple studies demonstrating the lack of antibody formation with multiple switches from different biosimilar drugs has eased his concern about multiple switches causing problems.
“Initially, there was a gap in demonstrating the long-term effect of multiple switches on antibody production and drug effectiveness. That gap has started to close as more data from Europe’s experience with biosimilars becomes available,” Dr. Snow said.
Resources for physicians, patients
The federal government has taken steps to advance biosimilars education and adoption. In 2021, President Biden signed the Advancing Education on Biosimilars Act into law, which directs the FDA to develop or improve continuing education programs that address prescribing of biosimilars and biological products.
The FDA provides educational materials on its website, including a comprehensive curriculum toolkit. The Accreditation Council for Medical Affairs has also created an online 40-hour curriculum for health care professionals called the Board-Certified Biologics and Biosimilars Specialist Program.
Dr. Fabbro recommended patients use the FDA page Biosimilar Basics for Patients to educate themselves on biosimilars. The Global Healthy Living Foundation’s podcast, Breaking Down Biosimilars, is another free resource for patients.
“While much has changed, the continued need for multistakeholder education, awareness, and dedicated research remains even more important as we expand into newer therapeutic areas and classes,” wrote the authors of the Cardinal Health report.
Help patients understand biologics and biosimilars by using AGA resources for providers and patients available at gastro.org/biosimilars.
Dr. Regueiro is on advisory boards and consults for AbbVie, Janssen, UCB, Takeda, Pfizer, Bristol-Myers Squibb, Organon, Amgen, Genentech, Gilead, Salix, Prometheus, Lilly, Celgene, TARGET PharmaSolutions, Trellis, and Boehringer Ingelheim. Dr. Fabbro is a principal investigator for Castle Biosciences, on the speakers bureau for Valchlor, and on the advisory boards of Janssen and Bristol-Myers Squibb. Dr. Popovian, Dr. Snow, Dr. Awsare, and Dr. Kaul had no disclosures.
A version of this article originally appeared on Medscape.com.
Rheumatologist Marcus Snow, MD, is comfortable with prescribing biosimilars as a first-line, first-time biologic, and discussing them with patients.
“If a biosimilar is on the market, it has gone through rigorous study proving its effectiveness and equivalence to a bio-originator,” said Dr. Snow, a rheumatologist with the University of Nebraska Medical Center, Omaha, and chair of the American College of Rheumatology’s Committee on Rheumatologic Care.
The formulary makes a big difference in the conversation about options, he said. “The formularies dictate what we can prescribe. It may not be appropriate, but it is reality. The cost of biologics for a patient without insurance coverage makes it impossible to afford.”
He will often tell patients that he’ll fight any changes or formulary restrictions he does not agree with. “However, when I see patients in follow-up, even if there is no known change on the horizon, I may bring up biosimilars when we have a moment to chat about them to familiarize them with what may happen in the future.”
The need for patient education on biosimilars presents a barrier to realizing their potential to save money and expand choice, noted Cardinal Health in its 2023 biosimilars report. Of 103 rheumatologists who responded to a Cardinal Health survey, 85% agreed that patient education was important. But those conversations can take an uncomfortable turn if the patient pushes back against taking a biosimilar owing to cost or safety concerns.
It’s not uncommon for a patient to express some anxiety about biosimilars, especially if they’re doing well on a current treatment plan. Most patients do not want any changes that may lead to worsening disease control, Dr. Snow said.
Patients and physicians alike often don’t understand the mechanics of biosimilars. “There’s a lot of misinformation about this,” said Sameer Awsare, MD, an associate executive director for The Permanente Medical Group in Campbell, Calif. Patients should know that a biosimilar will be as clinically efficacious as the medicine they’ve been on, with the same safety profiles, said Dr. Awsare, who works with Kaiser Permanente’s pharmacy partners on biosimilars.
Insurance often drives the conversation
The global anti-inflammatory biologics market is anticipated to reach $150 billion by 2027, according to a recent CVS report. As of March 2023, the Food and Drug Administration had approved 40 biosimilars to 11 different reference products. There are 28 on the U.S. market and 100 more in development. Projected to save more than $180 billion over the next 5 years, they are anticipated to expand choice and drive competition.
Rheumatologists, dermatologists, and gastroenterologists are frequent prescribers, although their choices for immune-mediated inflammatory diseases are limited to tumor necrosis factor inhibitors (infliximab [Remicade] originator and adalimumab [Humira] originator) and anti-CD20 agents, such as rituximab (Rituxan) originator.
Benefit design or formulary usually dictates what medicine a patient receives. “Because of significantly higher out-of-pocket cost or formulary positioning, patients may end up with a generic or a biosimilar instead of a brand-name medicine or branded biologic,” said Robert Popovian, PharmD, MS, chief science policy officer of the Global Healthy Living Foundation.
Insurers rarely offer both Remicade and biosimilar infliximab, allowing the doctor to choose, said Miguel Regueiro, MD, chair of the Cleveland Clinic’s Digestive Disease & Surgery Institute, who prescribes infliximab biosimilars. Most often, the payer will choose the lower-cost biosimilar. “I am fine with the biosimilar, either as a new start or a switch from the reference product.”
However, the patient might feel differently. They can form an attachment to the reference medication if it has prevented severe illness. “They do not want to change, as they feel they are going on a ‘new’ medication that will not work as well,” Dr. Regueiro said.
This is where the education comes in: to reassure patients that a biosimilar will work just as well as the reference product. “For patients who have done well for years on a biologic, more time needs to be spent reassuring them and answering questions,” compared with a patient just starting on a biosimilar, he advised.
But not all physicians are quick to prescribe biosimilars.
Especially with psoriasis, which has so many strong options for reference drugs, a switch may be hard to justify, said dermatologist Stephanie K. Fabbro, MD, assistant professor at Northeast Ohio Medical University, Rootstown. “If I have a preference, I would rather switch a patient to a drug from a different class without a biosimilar option to reduce the possibility of pushback.”
Dr. Fabbro, part of the core faculty in the Riverside Methodist Hospital Dermatology Residency Program in Columbus, will share data from clinical trials and postmarket surveillance with patients to support her decision.
Conversations about cost
Patients may also push back if they don’t save money when switching to a biosimilar. “This dilemma raises the question of who is profiting when a biosimilar is dispensed,” Dr. Popovian said. Insurers and pharmacy benefit managers (PBMs) that take additional concessions from biopharmaceutical manufacturers in the form of rebates and fees will often pocket this money as profit instead of passing savings back to the patient to help reduce their out-of-pocket requirement, he added.
If an originator biologic and a biosimilar are available, “as a pharmacist, I will choose the medicine that will incur the lowest out-of-pocket cost for the patient,” Dr. Popovian said.
Discussing cost – and who dictates which biosimilar is on the formulary – is an important conversation to have with patients, said Vivek Kaul, MD, Segal-Watson Professor of Medicine at the University of Rochester (N.Y.) Medical Center.
Providing equivalent clinical efficacy while saving costs is the economic reality of biosimilars, Dr. Kaul said. Third-party payers regularly evaluate how to provide the same quality of care while saving money. Physicians and patients alike “must be mindful that as time goes on, if the science on biosimilars stays robust, if the adoption is more widespread and the cost-saving proposition turns out to be true, more formularies will be attracted to replacing the reference product with the biosimilar counterpart.”
Providers and patients can weigh the options if a formulary suddenly switches to a biosimilar, Dr. Kaul continued. “You can accept the novel product on the formulary or may have to face out-of-pocket expenses as a patient.” If providers and patients have concerns about the biosimilar, they can always appeal if there’s solid scientific evidence that supports reverting back to the reference product.
“If you think the biosimilar is equally efficacious, comes at a lower cost, and is right for the patient, then the providers should tell the patient that,” he added.
Some studies have questioned whether the biosimilars will save money, compared with the reference drug, Dr. Fabbro noted. Medicare, for example, may pay only for a certain percentage of an approved biosimilar, saddling the patient with a monthly copay costing thousands of dollars. “It is unclear whether biosimilar manufacturers will have the same level of patient support programs as the reference drug companies.”
For that reason, physicians should also inform patients about the robust patient assistance and copay assistance programs many reference drug manufacturers offer, she said.
Biosimilars 101: Familiarizing patients
Safety and ease of use are other common concerns about biosimilars. Patients may ask if the application is different, or why it’s advantageous to switch to a biosimilar, Dr. Awsare said.
Sometimes the syringe or injector for a biosimilar might look different from that of the originator drug, he said.
Anecdotally, Dr. Fabbro has heard stories of patients having injection reactions that they did not experience with the reference drug or having a disease flare-up after starting a biosimilar.
As is the case with reference products, in their conversations with patients, clinicians should address the adverse event profile of biosimilars, offering data points from published studies and clinical guidelines that support the use of these products. “There should be an emphasis on patient education around efficacy and any side effects, and how the profile of the reference product compares with a proposed biosimilar,” Dr. Kaul suggested.
When Dr. Snow discusses biosimilars and generics, “I make sure to share this in an understandable way based on the patient’s scientific background, or lack thereof,” he said. If there is enough time, he also discusses how European- and U.S.-sourced biologics are slightly different.
Pharmacists should tell patients to expect the same clinical outcomes from a biosimilar, Dr. Popovian said. However, if they have any reduction in efficacy or potential safety concerns, they should communicate with their physician or pharmacist immediately.
In Dr. Regueiro’s practice, a pharmacist specializing in inflammatory bowel disease often has a one-on-one meeting with patients to educate and answer questions. “Additionally, we provide them the Crohn’s and Colitis Foundation web link on biosimilars,” said Dr. Regueiro.
A village approach to education
When biosimilars first came out, there were no formal education materials, Dr. Awsare said. Kaiser Permanente decided to create its own educational materials, not just for patients but also to help educate its primary care doctors; the rheumatologists, dermatologists, and gastroenterologists using the biosimilars; the nurses infusing patients; and the pharmacists preparing the biosimilars.
The health system also has a different approach to choosing medication. Instead of having an insurance company or PBM decide what’s in the formulary, clinicians work with the pharmacists at Kaiser to look at clinical evidence and decide which biosimilar to use. Most of its plans also provide lower copays to patients when they use the biosimilar.
This was the approach for Humira biosimilars, Dr. Awsare said. Eight will be on the market in 2023. “Our rheumatologists, dermatologists, and gastroenterologists looked at the data from Europe, looked at some real-world evidence, and then said: ‘We think this one’s going to be the best one for our patients.’ ”
Having clinicians choose the biosimilar instead of a health plan makes it a lot easier to have conversations with patients, he said. “Once we’ve moved that market share to that particular biosimilar, we give our physicians the time to have those discussions.”
Clinical pharmacists also provide educational support, offering guidance on issues such as side effects, as patients transition to the biosimilar. “We like to use the word ‘transition’ because it’s essentially the same biologic. So, you’re not actually switching,” Dr. Awsare said.
No consensus on interchangeability
Whether the conversation on interchangeability will affect patient conversations with physicians depends on who you ask.
If a biosimilar has an interchangeability designation, it means that the pharmacist can substitute it without the intervention of the clinician who prescribed the reference product. It does not relate to the quality, safety, or effectiveness of biosimilars or interchangeable biosimilar products, Dr. Popovian said.
The United States is the only country that has this designation. Even though it’s not identical to the originator drug, a biosimilar has the same clinical efficacy and safety profile. “So clinically, interchangeability is meaningless,” Dr. Awsare said.
In its report on biosimilars in the autoimmune category, CVS acknowledged that interchangeability was important but would not be a significant factor in driving adoption of biosimilars. However, in a Cardinal Health survey of 72 gastroenterologists, 38% cited the interchangeability of biosimilars as a top concern for adalimumab biosimilars, along with transitioning patients from Humira to a biosimilar (44%).
“Patient education regarding biosimilar safety, efficacy, and interchangeability appears paramount to the acceptance of these products, particularly for patients who are switched from a reference product,” Dr. Kaul noted in the Cardinal Health report.
Wherever supported by data, Dr. Kaul recommends incorporating biosimilar use and interchangeability into best practice guidelines going forward. “That will go a long way in disseminating the latest information on this topic and position this paradigm for increased adoption among providers.”
Some physicians like Dr. Snow aren’t that concerned with interchangeability. This hasn’t affected conversations with patients, he said. Multiple studies demonstrating the lack of antibody formation with multiple switches from different biosimilar drugs has eased his concern about multiple switches causing problems.
“Initially, there was a gap in demonstrating the long-term effect of multiple switches on antibody production and drug effectiveness. That gap has started to close as more data from Europe’s experience with biosimilars becomes available,” Dr. Snow said.
Resources for physicians, patients
The federal government has taken steps to advance biosimilars education and adoption. In 2021, President Biden signed the Advancing Education on Biosimilars Act into law, which directs the FDA to develop or improve continuing education programs that address prescribing of biosimilars and biological products.
The FDA provides educational materials on its website, including a comprehensive curriculum toolkit. The Accreditation Council for Medical Affairs has also created an online 40-hour curriculum for health care professionals called the Board-Certified Biologics and Biosimilars Specialist Program.
Dr. Fabbro recommended patients use the FDA page Biosimilar Basics for Patients to educate themselves on biosimilars. The Global Healthy Living Foundation’s podcast, Breaking Down Biosimilars, is another free resource for patients.
“While much has changed, the continued need for multistakeholder education, awareness, and dedicated research remains even more important as we expand into newer therapeutic areas and classes,” wrote the authors of the Cardinal Health report.
Help patients understand biologics and biosimilars by using AGA resources for providers and patients available at gastro.org/biosimilars.
Dr. Regueiro is on advisory boards and consults for AbbVie, Janssen, UCB, Takeda, Pfizer, Bristol-Myers Squibb, Organon, Amgen, Genentech, Gilead, Salix, Prometheus, Lilly, Celgene, TARGET PharmaSolutions, Trellis, and Boehringer Ingelheim. Dr. Fabbro is a principal investigator for Castle Biosciences, on the speakers bureau for Valchlor, and on the advisory boards of Janssen and Bristol-Myers Squibb. Dr. Popovian, Dr. Snow, Dr. Awsare, and Dr. Kaul had no disclosures.
A version of this article originally appeared on Medscape.com.
Rheumatologist Marcus Snow, MD, is comfortable with prescribing biosimilars as a first-line, first-time biologic, and discussing them with patients.
“If a biosimilar is on the market, it has gone through rigorous study proving its effectiveness and equivalence to a bio-originator,” said Dr. Snow, a rheumatologist with the University of Nebraska Medical Center, Omaha, and chair of the American College of Rheumatology’s Committee on Rheumatologic Care.
The formulary makes a big difference in the conversation about options, he said. “The formularies dictate what we can prescribe. It may not be appropriate, but it is reality. The cost of biologics for a patient without insurance coverage makes it impossible to afford.”
He will often tell patients that he’ll fight any changes or formulary restrictions he does not agree with. “However, when I see patients in follow-up, even if there is no known change on the horizon, I may bring up biosimilars when we have a moment to chat about them to familiarize them with what may happen in the future.”
The need for patient education on biosimilars presents a barrier to realizing their potential to save money and expand choice, noted Cardinal Health in its 2023 biosimilars report. Of 103 rheumatologists who responded to a Cardinal Health survey, 85% agreed that patient education was important. But those conversations can take an uncomfortable turn if the patient pushes back against taking a biosimilar owing to cost or safety concerns.
It’s not uncommon for a patient to express some anxiety about biosimilars, especially if they’re doing well on a current treatment plan. Most patients do not want any changes that may lead to worsening disease control, Dr. Snow said.
Patients and physicians alike often don’t understand the mechanics of biosimilars. “There’s a lot of misinformation about this,” said Sameer Awsare, MD, an associate executive director for The Permanente Medical Group in Campbell, Calif. Patients should know that a biosimilar will be as clinically efficacious as the medicine they’ve been on, with the same safety profiles, said Dr. Awsare, who works with Kaiser Permanente’s pharmacy partners on biosimilars.
Insurance often drives the conversation
The global anti-inflammatory biologics market is anticipated to reach $150 billion by 2027, according to a recent CVS report. As of March 2023, the Food and Drug Administration had approved 40 biosimilars to 11 different reference products. There are 28 on the U.S. market and 100 more in development. Projected to save more than $180 billion over the next 5 years, they are anticipated to expand choice and drive competition.
Rheumatologists, dermatologists, and gastroenterologists are frequent prescribers, although their choices for immune-mediated inflammatory diseases are limited to tumor necrosis factor inhibitors (infliximab [Remicade] originator and adalimumab [Humira] originator) and anti-CD20 agents, such as rituximab (Rituxan) originator.
Benefit design or formulary usually dictates what medicine a patient receives. “Because of significantly higher out-of-pocket cost or formulary positioning, patients may end up with a generic or a biosimilar instead of a brand-name medicine or branded biologic,” said Robert Popovian, PharmD, MS, chief science policy officer of the Global Healthy Living Foundation.
Insurers rarely offer both Remicade and biosimilar infliximab, allowing the doctor to choose, said Miguel Regueiro, MD, chair of the Cleveland Clinic’s Digestive Disease & Surgery Institute, who prescribes infliximab biosimilars. Most often, the payer will choose the lower-cost biosimilar. “I am fine with the biosimilar, either as a new start or a switch from the reference product.”
However, the patient might feel differently. They can form an attachment to the reference medication if it has prevented severe illness. “They do not want to change, as they feel they are going on a ‘new’ medication that will not work as well,” Dr. Regueiro said.
This is where the education comes in: to reassure patients that a biosimilar will work just as well as the reference product. “For patients who have done well for years on a biologic, more time needs to be spent reassuring them and answering questions,” compared with a patient just starting on a biosimilar, he advised.
But not all physicians are quick to prescribe biosimilars.
Especially with psoriasis, which has so many strong options for reference drugs, a switch may be hard to justify, said dermatologist Stephanie K. Fabbro, MD, assistant professor at Northeast Ohio Medical University, Rootstown. “If I have a preference, I would rather switch a patient to a drug from a different class without a biosimilar option to reduce the possibility of pushback.”
Dr. Fabbro, part of the core faculty in the Riverside Methodist Hospital Dermatology Residency Program in Columbus, will share data from clinical trials and postmarket surveillance with patients to support her decision.
Conversations about cost
Patients may also push back if they don’t save money when switching to a biosimilar. “This dilemma raises the question of who is profiting when a biosimilar is dispensed,” Dr. Popovian said. Insurers and pharmacy benefit managers (PBMs) that take additional concessions from biopharmaceutical manufacturers in the form of rebates and fees will often pocket this money as profit instead of passing savings back to the patient to help reduce their out-of-pocket requirement, he added.
If an originator biologic and a biosimilar are available, “as a pharmacist, I will choose the medicine that will incur the lowest out-of-pocket cost for the patient,” Dr. Popovian said.
Discussing cost – and who dictates which biosimilar is on the formulary – is an important conversation to have with patients, said Vivek Kaul, MD, Segal-Watson Professor of Medicine at the University of Rochester (N.Y.) Medical Center.
Providing equivalent clinical efficacy while saving costs is the economic reality of biosimilars, Dr. Kaul said. Third-party payers regularly evaluate how to provide the same quality of care while saving money. Physicians and patients alike “must be mindful that as time goes on, if the science on biosimilars stays robust, if the adoption is more widespread and the cost-saving proposition turns out to be true, more formularies will be attracted to replacing the reference product with the biosimilar counterpart.”
Providers and patients can weigh the options if a formulary suddenly switches to a biosimilar, Dr. Kaul continued. “You can accept the novel product on the formulary or may have to face out-of-pocket expenses as a patient.” If providers and patients have concerns about the biosimilar, they can always appeal if there’s solid scientific evidence that supports reverting back to the reference product.
“If you think the biosimilar is equally efficacious, comes at a lower cost, and is right for the patient, then the providers should tell the patient that,” he added.
Some studies have questioned whether the biosimilars will save money, compared with the reference drug, Dr. Fabbro noted. Medicare, for example, may pay only for a certain percentage of an approved biosimilar, saddling the patient with a monthly copay costing thousands of dollars. “It is unclear whether biosimilar manufacturers will have the same level of patient support programs as the reference drug companies.”
For that reason, physicians should also inform patients about the robust patient assistance and copay assistance programs many reference drug manufacturers offer, she said.
Biosimilars 101: Familiarizing patients
Safety and ease of use are other common concerns about biosimilars. Patients may ask if the application is different, or why it’s advantageous to switch to a biosimilar, Dr. Awsare said.
Sometimes the syringe or injector for a biosimilar might look different from that of the originator drug, he said.
Anecdotally, Dr. Fabbro has heard stories of patients having injection reactions that they did not experience with the reference drug or having a disease flare-up after starting a biosimilar.
As is the case with reference products, in their conversations with patients, clinicians should address the adverse event profile of biosimilars, offering data points from published studies and clinical guidelines that support the use of these products. “There should be an emphasis on patient education around efficacy and any side effects, and how the profile of the reference product compares with a proposed biosimilar,” Dr. Kaul suggested.
When Dr. Snow discusses biosimilars and generics, “I make sure to share this in an understandable way based on the patient’s scientific background, or lack thereof,” he said. If there is enough time, he also discusses how European- and U.S.-sourced biologics are slightly different.
Pharmacists should tell patients to expect the same clinical outcomes from a biosimilar, Dr. Popovian said. However, if they have any reduction in efficacy or potential safety concerns, they should communicate with their physician or pharmacist immediately.
In Dr. Regueiro’s practice, a pharmacist specializing in inflammatory bowel disease often has a one-on-one meeting with patients to educate and answer questions. “Additionally, we provide them the Crohn’s and Colitis Foundation web link on biosimilars,” said Dr. Regueiro.
A village approach to education
When biosimilars first came out, there were no formal education materials, Dr. Awsare said. Kaiser Permanente decided to create its own educational materials, not just for patients but also to help educate its primary care doctors; the rheumatologists, dermatologists, and gastroenterologists using the biosimilars; the nurses infusing patients; and the pharmacists preparing the biosimilars.
The health system also has a different approach to choosing medication. Instead of having an insurance company or PBM decide what’s in the formulary, clinicians work with the pharmacists at Kaiser to look at clinical evidence and decide which biosimilar to use. Most of its plans also provide lower copays to patients when they use the biosimilar.
This was the approach for Humira biosimilars, Dr. Awsare said. Eight will be on the market in 2023. “Our rheumatologists, dermatologists, and gastroenterologists looked at the data from Europe, looked at some real-world evidence, and then said: ‘We think this one’s going to be the best one for our patients.’ ”
Having clinicians choose the biosimilar instead of a health plan makes it a lot easier to have conversations with patients, he said. “Once we’ve moved that market share to that particular biosimilar, we give our physicians the time to have those discussions.”
Clinical pharmacists also provide educational support, offering guidance on issues such as side effects, as patients transition to the biosimilar. “We like to use the word ‘transition’ because it’s essentially the same biologic. So, you’re not actually switching,” Dr. Awsare said.
No consensus on interchangeability
Whether the conversation on interchangeability will affect patient conversations with physicians depends on who you ask.
If a biosimilar has an interchangeability designation, it means that the pharmacist can substitute it without the intervention of the clinician who prescribed the reference product. It does not relate to the quality, safety, or effectiveness of biosimilars or interchangeable biosimilar products, Dr. Popovian said.
The United States is the only country that has this designation. Even though it’s not identical to the originator drug, a biosimilar has the same clinical efficacy and safety profile. “So clinically, interchangeability is meaningless,” Dr. Awsare said.
In its report on biosimilars in the autoimmune category, CVS acknowledged that interchangeability was important but would not be a significant factor in driving adoption of biosimilars. However, in a Cardinal Health survey of 72 gastroenterologists, 38% cited the interchangeability of biosimilars as a top concern for adalimumab biosimilars, along with transitioning patients from Humira to a biosimilar (44%).
“Patient education regarding biosimilar safety, efficacy, and interchangeability appears paramount to the acceptance of these products, particularly for patients who are switched from a reference product,” Dr. Kaul noted in the Cardinal Health report.
Wherever supported by data, Dr. Kaul recommends incorporating biosimilar use and interchangeability into best practice guidelines going forward. “That will go a long way in disseminating the latest information on this topic and position this paradigm for increased adoption among providers.”
Some physicians like Dr. Snow aren’t that concerned with interchangeability. This hasn’t affected conversations with patients, he said. Multiple studies demonstrating the lack of antibody formation with multiple switches from different biosimilar drugs has eased his concern about multiple switches causing problems.
“Initially, there was a gap in demonstrating the long-term effect of multiple switches on antibody production and drug effectiveness. That gap has started to close as more data from Europe’s experience with biosimilars becomes available,” Dr. Snow said.
Resources for physicians, patients
The federal government has taken steps to advance biosimilars education and adoption. In 2021, President Biden signed the Advancing Education on Biosimilars Act into law, which directs the FDA to develop or improve continuing education programs that address prescribing of biosimilars and biological products.
The FDA provides educational materials on its website, including a comprehensive curriculum toolkit. The Accreditation Council for Medical Affairs has also created an online 40-hour curriculum for health care professionals called the Board-Certified Biologics and Biosimilars Specialist Program.
Dr. Fabbro recommended patients use the FDA page Biosimilar Basics for Patients to educate themselves on biosimilars. The Global Healthy Living Foundation’s podcast, Breaking Down Biosimilars, is another free resource for patients.
“While much has changed, the continued need for multistakeholder education, awareness, and dedicated research remains even more important as we expand into newer therapeutic areas and classes,” wrote the authors of the Cardinal Health report.
Help patients understand biologics and biosimilars by using AGA resources for providers and patients available at gastro.org/biosimilars.
Dr. Regueiro is on advisory boards and consults for AbbVie, Janssen, UCB, Takeda, Pfizer, Bristol-Myers Squibb, Organon, Amgen, Genentech, Gilead, Salix, Prometheus, Lilly, Celgene, TARGET PharmaSolutions, Trellis, and Boehringer Ingelheim. Dr. Fabbro is a principal investigator for Castle Biosciences, on the speakers bureau for Valchlor, and on the advisory boards of Janssen and Bristol-Myers Squibb. Dr. Popovian, Dr. Snow, Dr. Awsare, and Dr. Kaul had no disclosures.
A version of this article originally appeared on Medscape.com.
CT results in myositis inform cancer screening strategies
Targeting cancer screenings based on idiopathic inflammatory myositis (IIM) subtype, autoantibodies, and age may help to maximize cancer detection while limiting false positives.
In a retrospective, single-center study conducted at Johns Hopkins University in Baltimore, researchers found that when screening patients with IIM for cancer via CT imaging, the diagnostic yield (number of cancers detected/tests performed) was highest in patients with dermatomyositis and the autoantibody anti–TIF1-gamma. Screening patients below age 40 years was associated with lower diagnostic yields and higher false positives, regardless of subtype.
Because of the well-known association between IIM and contemporaneous cancer, newly diagnosed patients with IIM often undergo screening. Yet, there is little research on the most efficient assessment approaches, Christopher Mecoli, MD, an assistant professor of medicine at John Hopkins University School of Medicine and lead author of the study, told this news organization. “There has been a lot written about how these patients should be evaluated for cancer. Unfortunately, the majority of literature is based on eminence,” he said. This study is “one of the first pieces of real data to inform that conversation,” he added.
The research was published online in Arthritis Care & Research.
In the study, Dr. Mecoli and colleagues looked at 1,086 patients enrolled in the center’s Myositis Research Registry from 2003 through 2020. The analysis included patients with a diagnosis of dermatomyositis, polymyositis, immune-mediated necrotizing myopathy (IMNM), and antisynthetase syndrome (ASyS). The researchers also looked at myositis-specific autoantibodies, including anti–TIF1-gamma, –Jo1, and –HMGCR. Patients were excluded from the analysis if they had a cancer diagnosis prior to their IIM onset.
Among patients included in the analysis, the average age of IIM onset was 49 years, and median follow-up duration was 5.3 years. Most patients were female (71%), 68% were white, 21% were Black, 3.6% were Asian, and 7.4% had a listed race of other or unknown. About 66% of all patients received a chest CT scan within 3 years of IIM onset, and 51% received an abdomen/pelvis CT in that same time frame. False positives were defined as the percentage of scans that led to a noncancerous biopsy.
During the study period, 62 patients had a cancer diagnosis within the first 3 years of IIM onset, with the most common cancers being breast (19%), melanoma (13%), and cervical/uterine (10%). Of 1,011 chest scans performed, 9 led to a cancer diagnosis (0.9%), compared with 12 of the 657 abdomen/pelvis (a/p) CT scans (1.8%). Patients with the dermatomyositis-specific autoantibody anti–TIF1-gamma had the highest diagnostic yield (2.9% in chest CT and 2.4% in a/p CT). Regardless of autoantibodies, dermatomyositis patients above 40 years of age had a diagnostic yield of 1.4% in chest CT and 2.7% in a/p CT. For patients under the age of 40 with polymyositis, IMNM, and ASyS, the diagnostic yield for all CT scans was 0.0%. The diagnostic yield in patients under 40 with dermatomyositis was also low (0.0% in chest CT, 0.8% in a/p CT).
The false-positive rate for all chest CT scans was 2.8%, with patients with IMNM and ASyS having the highest frequency of false positivity (both 4.4%). “Based on our data, CT chest imaging in ASyS and IMNM patients are associated with the most harm from a cancer screening perspective,” the authors write. In a/p CT, patients with dermatomyositis under 40 and patients with ASyS had the highest false-positive rates (4.9% and 3.8%, respectively).
“Age was a really big deal in terms of predicting diagnostic yield and false-positivity rate,” Dr. Mecoli said, particularly in patients with dermatomyositis. “This subgroup has historically been thought to have the biggest dissociation with cancer,” he said, but in patients under 40, “it doesn’t look like CT scans were that helpful. They were not picking up a lot of cancers, and they were leading to a lot of false-positive results.”
Still, Rohit Aggarwal, MD, of the division of rheumatology and clinical immunology at the University of Pittsburgh, Pennsylvania, noted that the diagnostic yields of 1%-2% and even 2%-4% in higher-risk populations were high. By comparison, lung cancer screening trials had a diagnostic yield of about 1%, and trials examining CT screening for colorectal cancers had diagnostic yields of 0.5%, the authors write.
“The key message for me is that we should definitely perform CT scans of the chest, abdomen, and pelvis within 3 years of diagnosis – typically at presentation – if the patient has any risk factor for increased risk of cancer, which include dermatomyositis and age above 40,” Dr. Aggarwal toldthis news organization. He was not involved with the research. There are also other clinical factors to consider that were not included in the study, he added, such as severe dysphagia, patients with refractory treatment, and male sex.
Both Dr. Aggarwal and Dr. Mecoli agreed that there are limitations to this single-center, retrospective study that make it difficult to generalize the results. Similar studies should be conducted at other institutions to see if these associations hold true, Dr. Mecoli said. A prospective study could also help control for factors such as selection bias, Dr. Aggarwal added. “I don’t think these are definitive data, but I think these data were needed at retrospective levels” to plan future research, he said.
The study was supported in part by grants from the National Institutes of Health, the Jerome L. Greene Foundation, the Donald B. and Dorothy L. Stabler Foundation, the Huayi and Siuling Zhang Discovery Fund, and Dr. Peter Buck. Dr. Mecoli and Dr. Aggarwal have disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Targeting cancer screenings based on idiopathic inflammatory myositis (IIM) subtype, autoantibodies, and age may help to maximize cancer detection while limiting false positives.
In a retrospective, single-center study conducted at Johns Hopkins University in Baltimore, researchers found that when screening patients with IIM for cancer via CT imaging, the diagnostic yield (number of cancers detected/tests performed) was highest in patients with dermatomyositis and the autoantibody anti–TIF1-gamma. Screening patients below age 40 years was associated with lower diagnostic yields and higher false positives, regardless of subtype.
Because of the well-known association between IIM and contemporaneous cancer, newly diagnosed patients with IIM often undergo screening. Yet, there is little research on the most efficient assessment approaches, Christopher Mecoli, MD, an assistant professor of medicine at John Hopkins University School of Medicine and lead author of the study, told this news organization. “There has been a lot written about how these patients should be evaluated for cancer. Unfortunately, the majority of literature is based on eminence,” he said. This study is “one of the first pieces of real data to inform that conversation,” he added.
The research was published online in Arthritis Care & Research.
In the study, Dr. Mecoli and colleagues looked at 1,086 patients enrolled in the center’s Myositis Research Registry from 2003 through 2020. The analysis included patients with a diagnosis of dermatomyositis, polymyositis, immune-mediated necrotizing myopathy (IMNM), and antisynthetase syndrome (ASyS). The researchers also looked at myositis-specific autoantibodies, including anti–TIF1-gamma, –Jo1, and –HMGCR. Patients were excluded from the analysis if they had a cancer diagnosis prior to their IIM onset.
Among patients included in the analysis, the average age of IIM onset was 49 years, and median follow-up duration was 5.3 years. Most patients were female (71%), 68% were white, 21% were Black, 3.6% were Asian, and 7.4% had a listed race of other or unknown. About 66% of all patients received a chest CT scan within 3 years of IIM onset, and 51% received an abdomen/pelvis CT in that same time frame. False positives were defined as the percentage of scans that led to a noncancerous biopsy.
During the study period, 62 patients had a cancer diagnosis within the first 3 years of IIM onset, with the most common cancers being breast (19%), melanoma (13%), and cervical/uterine (10%). Of 1,011 chest scans performed, 9 led to a cancer diagnosis (0.9%), compared with 12 of the 657 abdomen/pelvis (a/p) CT scans (1.8%). Patients with the dermatomyositis-specific autoantibody anti–TIF1-gamma had the highest diagnostic yield (2.9% in chest CT and 2.4% in a/p CT). Regardless of autoantibodies, dermatomyositis patients above 40 years of age had a diagnostic yield of 1.4% in chest CT and 2.7% in a/p CT. For patients under the age of 40 with polymyositis, IMNM, and ASyS, the diagnostic yield for all CT scans was 0.0%. The diagnostic yield in patients under 40 with dermatomyositis was also low (0.0% in chest CT, 0.8% in a/p CT).
The false-positive rate for all chest CT scans was 2.8%, with patients with IMNM and ASyS having the highest frequency of false positivity (both 4.4%). “Based on our data, CT chest imaging in ASyS and IMNM patients are associated with the most harm from a cancer screening perspective,” the authors write. In a/p CT, patients with dermatomyositis under 40 and patients with ASyS had the highest false-positive rates (4.9% and 3.8%, respectively).
“Age was a really big deal in terms of predicting diagnostic yield and false-positivity rate,” Dr. Mecoli said, particularly in patients with dermatomyositis. “This subgroup has historically been thought to have the biggest dissociation with cancer,” he said, but in patients under 40, “it doesn’t look like CT scans were that helpful. They were not picking up a lot of cancers, and they were leading to a lot of false-positive results.”
Still, Rohit Aggarwal, MD, of the division of rheumatology and clinical immunology at the University of Pittsburgh, Pennsylvania, noted that the diagnostic yields of 1%-2% and even 2%-4% in higher-risk populations were high. By comparison, lung cancer screening trials had a diagnostic yield of about 1%, and trials examining CT screening for colorectal cancers had diagnostic yields of 0.5%, the authors write.
“The key message for me is that we should definitely perform CT scans of the chest, abdomen, and pelvis within 3 years of diagnosis – typically at presentation – if the patient has any risk factor for increased risk of cancer, which include dermatomyositis and age above 40,” Dr. Aggarwal toldthis news organization. He was not involved with the research. There are also other clinical factors to consider that were not included in the study, he added, such as severe dysphagia, patients with refractory treatment, and male sex.
Both Dr. Aggarwal and Dr. Mecoli agreed that there are limitations to this single-center, retrospective study that make it difficult to generalize the results. Similar studies should be conducted at other institutions to see if these associations hold true, Dr. Mecoli said. A prospective study could also help control for factors such as selection bias, Dr. Aggarwal added. “I don’t think these are definitive data, but I think these data were needed at retrospective levels” to plan future research, he said.
The study was supported in part by grants from the National Institutes of Health, the Jerome L. Greene Foundation, the Donald B. and Dorothy L. Stabler Foundation, the Huayi and Siuling Zhang Discovery Fund, and Dr. Peter Buck. Dr. Mecoli and Dr. Aggarwal have disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Targeting cancer screenings based on idiopathic inflammatory myositis (IIM) subtype, autoantibodies, and age may help to maximize cancer detection while limiting false positives.
In a retrospective, single-center study conducted at Johns Hopkins University in Baltimore, researchers found that when screening patients with IIM for cancer via CT imaging, the diagnostic yield (number of cancers detected/tests performed) was highest in patients with dermatomyositis and the autoantibody anti–TIF1-gamma. Screening patients below age 40 years was associated with lower diagnostic yields and higher false positives, regardless of subtype.
Because of the well-known association between IIM and contemporaneous cancer, newly diagnosed patients with IIM often undergo screening. Yet, there is little research on the most efficient assessment approaches, Christopher Mecoli, MD, an assistant professor of medicine at John Hopkins University School of Medicine and lead author of the study, told this news organization. “There has been a lot written about how these patients should be evaluated for cancer. Unfortunately, the majority of literature is based on eminence,” he said. This study is “one of the first pieces of real data to inform that conversation,” he added.
The research was published online in Arthritis Care & Research.
In the study, Dr. Mecoli and colleagues looked at 1,086 patients enrolled in the center’s Myositis Research Registry from 2003 through 2020. The analysis included patients with a diagnosis of dermatomyositis, polymyositis, immune-mediated necrotizing myopathy (IMNM), and antisynthetase syndrome (ASyS). The researchers also looked at myositis-specific autoantibodies, including anti–TIF1-gamma, –Jo1, and –HMGCR. Patients were excluded from the analysis if they had a cancer diagnosis prior to their IIM onset.
Among patients included in the analysis, the average age of IIM onset was 49 years, and median follow-up duration was 5.3 years. Most patients were female (71%), 68% were white, 21% were Black, 3.6% were Asian, and 7.4% had a listed race of other or unknown. About 66% of all patients received a chest CT scan within 3 years of IIM onset, and 51% received an abdomen/pelvis CT in that same time frame. False positives were defined as the percentage of scans that led to a noncancerous biopsy.
During the study period, 62 patients had a cancer diagnosis within the first 3 years of IIM onset, with the most common cancers being breast (19%), melanoma (13%), and cervical/uterine (10%). Of 1,011 chest scans performed, 9 led to a cancer diagnosis (0.9%), compared with 12 of the 657 abdomen/pelvis (a/p) CT scans (1.8%). Patients with the dermatomyositis-specific autoantibody anti–TIF1-gamma had the highest diagnostic yield (2.9% in chest CT and 2.4% in a/p CT). Regardless of autoantibodies, dermatomyositis patients above 40 years of age had a diagnostic yield of 1.4% in chest CT and 2.7% in a/p CT. For patients under the age of 40 with polymyositis, IMNM, and ASyS, the diagnostic yield for all CT scans was 0.0%. The diagnostic yield in patients under 40 with dermatomyositis was also low (0.0% in chest CT, 0.8% in a/p CT).
The false-positive rate for all chest CT scans was 2.8%, with patients with IMNM and ASyS having the highest frequency of false positivity (both 4.4%). “Based on our data, CT chest imaging in ASyS and IMNM patients are associated with the most harm from a cancer screening perspective,” the authors write. In a/p CT, patients with dermatomyositis under 40 and patients with ASyS had the highest false-positive rates (4.9% and 3.8%, respectively).
“Age was a really big deal in terms of predicting diagnostic yield and false-positivity rate,” Dr. Mecoli said, particularly in patients with dermatomyositis. “This subgroup has historically been thought to have the biggest dissociation with cancer,” he said, but in patients under 40, “it doesn’t look like CT scans were that helpful. They were not picking up a lot of cancers, and they were leading to a lot of false-positive results.”
Still, Rohit Aggarwal, MD, of the division of rheumatology and clinical immunology at the University of Pittsburgh, Pennsylvania, noted that the diagnostic yields of 1%-2% and even 2%-4% in higher-risk populations were high. By comparison, lung cancer screening trials had a diagnostic yield of about 1%, and trials examining CT screening for colorectal cancers had diagnostic yields of 0.5%, the authors write.
“The key message for me is that we should definitely perform CT scans of the chest, abdomen, and pelvis within 3 years of diagnosis – typically at presentation – if the patient has any risk factor for increased risk of cancer, which include dermatomyositis and age above 40,” Dr. Aggarwal toldthis news organization. He was not involved with the research. There are also other clinical factors to consider that were not included in the study, he added, such as severe dysphagia, patients with refractory treatment, and male sex.
Both Dr. Aggarwal and Dr. Mecoli agreed that there are limitations to this single-center, retrospective study that make it difficult to generalize the results. Similar studies should be conducted at other institutions to see if these associations hold true, Dr. Mecoli said. A prospective study could also help control for factors such as selection bias, Dr. Aggarwal added. “I don’t think these are definitive data, but I think these data were needed at retrospective levels” to plan future research, he said.
The study was supported in part by grants from the National Institutes of Health, the Jerome L. Greene Foundation, the Donald B. and Dorothy L. Stabler Foundation, the Huayi and Siuling Zhang Discovery Fund, and Dr. Peter Buck. Dr. Mecoli and Dr. Aggarwal have disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
FROM ARTHRITIS CARE & RESEARCH