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Neurofilament Light Chain Detects Early Chemotherapy-Related Neurotoxicity
Investigators found Nfl levels increased in cancer patients following a first infusion of the medication paclitaxel and corresponded to neuropathy severity 6-12 months post-treatment, suggesting the blood protein may provide an early CIPN biomarker.
“Nfl after a single cycle could detect axonal degeneration,” said lead investigator Masarra Joda, a researcher and PhD candidate at the University of Sydney in Australia. She added that “quantification of Nfl may provide a clinically useful marker of emerging neurotoxicity in patients vulnerable to CIPN.”
The findings were presented at the Peripheral Nerve Society (PNS) 2024 annual meeting.
Common, Burdensome Side Effect
A common side effect of chemotherapy, CIPN manifests as sensory neuropathy and causes degeneration of the peripheral axons. A protein biomarker of axonal degeneration, Nfl has previously been investigated as a way of identifying patients at risk of CIPN.
The goal of the current study was to identify the potential link between Nfl with neurophysiological markers of axon degeneration in patients receiving the neurotoxin chemotherapy paclitaxel.
The study included 93 cancer patients. All were assessed at the beginning, middle, and end of treatment. CIPN was assessed using blood samples of Nfl and the Total Neuropathy Score (TNS), the Common Terminology Criteria for Adverse Events (CTCAE) neuropathy scale, and patient-reported measures using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire–Chemotherapy-Induced Peripheral Neuropathy Module (EORTC-CIPN20).
Axonal degeneration was measured with neurophysiological tests including sural nerve compound sensory action potential (CSAP) for the lower limbs, and sensory median nerve CSAP, as well as stimulus threshold testing, for the upper limbs.
Almost all of study participants (97%) were female. The majority (66%) had breast cancer and 30% had gynecological cancer. Most (73%) were receiving a weekly regimen of paclitaxel, and the remainder were treated with taxanes plus platinum once every 3 weeks. By the end of treatment, 82% of the patients had developed CIPN, which was mild in 44% and moderate/severe in 38%.
Nfl levels increased significantly from baseline to after the first dose of chemotherapy (P < .001), “highlighting that nerve damage occurs from the very beginning of treatment,” senior investigator Susanna Park, PhD, told this news organization.
In addition, “patients with higher Nfl levels after a single paclitaxel treatment had greater neuropathy at the end of treatment (higher EORTC scores [P ≤ .026], and higher TNS scores [P ≤ .00]),” added Dr. Park, who is associate professor at the University of Sydney.
“Importantly, we also looked at long-term outcomes beyond the end of chemotherapy, because chronic neuropathy produces a significant burden in cancer survivors,” said Dr. Park.
“Among a total of 44 patients who completed the 6- to 12-month post-treatment follow-up, NfL levels after a single treatment were linked to severity of nerve damage quantified with neurophysiological tests, and greater Nfl levels at mid-treatment were correlated with worse patient and neurologically graded neuropathy at 6-12 months.”
Dr. Park said the results suggest that NfL may provide a biomarker of long-term axon damage and that Nfl assays “may enable clinicians to evaluate the risk of long-term toxicity early during paclitaxel treatment to hopefully provide clinically significant information to guide better treatment titration.”
Currently, she said, CIPN is a prominent cause of dose reduction and early chemotherapy cessation.
“For example, in early breast cancer around 25% of patients experience a dose reduction due to the severity of neuropathy symptoms.” But, she said, “there is no standardized way of identifying which patients are at risk of long-term neuropathy and therefore, may benefit more from dose reduction. In this setting, a biomarker such as Nfl could provide oncologists with more information about the risk of long-term toxicity and take that into account in dose decision-making.”
For some cancers, she added, there are multiple potential therapy options.
“A biomarker such as NfL could assist in determining risk-benefit profile in terms of switching to alternate therapies. However, further studies will be needed to fully define the utility of NfL as a biomarker of paclitaxel neuropathy.”
Promising Research
Commenting on the research for this news organization, Maryam Lustberg, MD, associate professor, director of the Center for Breast Cancer at Smilow Cancer Hospital and Yale Cancer Center, and chief of Breast Medical Oncology at Yale Cancer Center, in New Haven, Connecticut, said the study “builds on a body of work previously reported by others showing that neurofilament light chains as detected in the blood can be associated with early signs of neurotoxic injury.”
She added that the research “is promising, since existing clinical and patient-reported measures tend to under-detect chemotherapy-induced neuropathy until more permanent injury might have occurred.”
Dr. Lustberg, who is immediate past president of the Multinational Association of Supportive Care in Cancer, said future studies are needed before Nfl testing can be implemented in routine practice, but that “early detection will allow earlier initiation of supportive care strategies such as physical therapy and exercise, as well as dose modifications, which may be helpful for preventing permanent damage and improving quality of life.”
The investigators and Dr. Lustberg report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Investigators found Nfl levels increased in cancer patients following a first infusion of the medication paclitaxel and corresponded to neuropathy severity 6-12 months post-treatment, suggesting the blood protein may provide an early CIPN biomarker.
“Nfl after a single cycle could detect axonal degeneration,” said lead investigator Masarra Joda, a researcher and PhD candidate at the University of Sydney in Australia. She added that “quantification of Nfl may provide a clinically useful marker of emerging neurotoxicity in patients vulnerable to CIPN.”
The findings were presented at the Peripheral Nerve Society (PNS) 2024 annual meeting.
Common, Burdensome Side Effect
A common side effect of chemotherapy, CIPN manifests as sensory neuropathy and causes degeneration of the peripheral axons. A protein biomarker of axonal degeneration, Nfl has previously been investigated as a way of identifying patients at risk of CIPN.
The goal of the current study was to identify the potential link between Nfl with neurophysiological markers of axon degeneration in patients receiving the neurotoxin chemotherapy paclitaxel.
The study included 93 cancer patients. All were assessed at the beginning, middle, and end of treatment. CIPN was assessed using blood samples of Nfl and the Total Neuropathy Score (TNS), the Common Terminology Criteria for Adverse Events (CTCAE) neuropathy scale, and patient-reported measures using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire–Chemotherapy-Induced Peripheral Neuropathy Module (EORTC-CIPN20).
Axonal degeneration was measured with neurophysiological tests including sural nerve compound sensory action potential (CSAP) for the lower limbs, and sensory median nerve CSAP, as well as stimulus threshold testing, for the upper limbs.
Almost all of study participants (97%) were female. The majority (66%) had breast cancer and 30% had gynecological cancer. Most (73%) were receiving a weekly regimen of paclitaxel, and the remainder were treated with taxanes plus platinum once every 3 weeks. By the end of treatment, 82% of the patients had developed CIPN, which was mild in 44% and moderate/severe in 38%.
Nfl levels increased significantly from baseline to after the first dose of chemotherapy (P < .001), “highlighting that nerve damage occurs from the very beginning of treatment,” senior investigator Susanna Park, PhD, told this news organization.
In addition, “patients with higher Nfl levels after a single paclitaxel treatment had greater neuropathy at the end of treatment (higher EORTC scores [P ≤ .026], and higher TNS scores [P ≤ .00]),” added Dr. Park, who is associate professor at the University of Sydney.
“Importantly, we also looked at long-term outcomes beyond the end of chemotherapy, because chronic neuropathy produces a significant burden in cancer survivors,” said Dr. Park.
“Among a total of 44 patients who completed the 6- to 12-month post-treatment follow-up, NfL levels after a single treatment were linked to severity of nerve damage quantified with neurophysiological tests, and greater Nfl levels at mid-treatment were correlated with worse patient and neurologically graded neuropathy at 6-12 months.”
Dr. Park said the results suggest that NfL may provide a biomarker of long-term axon damage and that Nfl assays “may enable clinicians to evaluate the risk of long-term toxicity early during paclitaxel treatment to hopefully provide clinically significant information to guide better treatment titration.”
Currently, she said, CIPN is a prominent cause of dose reduction and early chemotherapy cessation.
“For example, in early breast cancer around 25% of patients experience a dose reduction due to the severity of neuropathy symptoms.” But, she said, “there is no standardized way of identifying which patients are at risk of long-term neuropathy and therefore, may benefit more from dose reduction. In this setting, a biomarker such as Nfl could provide oncologists with more information about the risk of long-term toxicity and take that into account in dose decision-making.”
For some cancers, she added, there are multiple potential therapy options.
“A biomarker such as NfL could assist in determining risk-benefit profile in terms of switching to alternate therapies. However, further studies will be needed to fully define the utility of NfL as a biomarker of paclitaxel neuropathy.”
Promising Research
Commenting on the research for this news organization, Maryam Lustberg, MD, associate professor, director of the Center for Breast Cancer at Smilow Cancer Hospital and Yale Cancer Center, and chief of Breast Medical Oncology at Yale Cancer Center, in New Haven, Connecticut, said the study “builds on a body of work previously reported by others showing that neurofilament light chains as detected in the blood can be associated with early signs of neurotoxic injury.”
She added that the research “is promising, since existing clinical and patient-reported measures tend to under-detect chemotherapy-induced neuropathy until more permanent injury might have occurred.”
Dr. Lustberg, who is immediate past president of the Multinational Association of Supportive Care in Cancer, said future studies are needed before Nfl testing can be implemented in routine practice, but that “early detection will allow earlier initiation of supportive care strategies such as physical therapy and exercise, as well as dose modifications, which may be helpful for preventing permanent damage and improving quality of life.”
The investigators and Dr. Lustberg report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Investigators found Nfl levels increased in cancer patients following a first infusion of the medication paclitaxel and corresponded to neuropathy severity 6-12 months post-treatment, suggesting the blood protein may provide an early CIPN biomarker.
“Nfl after a single cycle could detect axonal degeneration,” said lead investigator Masarra Joda, a researcher and PhD candidate at the University of Sydney in Australia. She added that “quantification of Nfl may provide a clinically useful marker of emerging neurotoxicity in patients vulnerable to CIPN.”
The findings were presented at the Peripheral Nerve Society (PNS) 2024 annual meeting.
Common, Burdensome Side Effect
A common side effect of chemotherapy, CIPN manifests as sensory neuropathy and causes degeneration of the peripheral axons. A protein biomarker of axonal degeneration, Nfl has previously been investigated as a way of identifying patients at risk of CIPN.
The goal of the current study was to identify the potential link between Nfl with neurophysiological markers of axon degeneration in patients receiving the neurotoxin chemotherapy paclitaxel.
The study included 93 cancer patients. All were assessed at the beginning, middle, and end of treatment. CIPN was assessed using blood samples of Nfl and the Total Neuropathy Score (TNS), the Common Terminology Criteria for Adverse Events (CTCAE) neuropathy scale, and patient-reported measures using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire–Chemotherapy-Induced Peripheral Neuropathy Module (EORTC-CIPN20).
Axonal degeneration was measured with neurophysiological tests including sural nerve compound sensory action potential (CSAP) for the lower limbs, and sensory median nerve CSAP, as well as stimulus threshold testing, for the upper limbs.
Almost all of study participants (97%) were female. The majority (66%) had breast cancer and 30% had gynecological cancer. Most (73%) were receiving a weekly regimen of paclitaxel, and the remainder were treated with taxanes plus platinum once every 3 weeks. By the end of treatment, 82% of the patients had developed CIPN, which was mild in 44% and moderate/severe in 38%.
Nfl levels increased significantly from baseline to after the first dose of chemotherapy (P < .001), “highlighting that nerve damage occurs from the very beginning of treatment,” senior investigator Susanna Park, PhD, told this news organization.
In addition, “patients with higher Nfl levels after a single paclitaxel treatment had greater neuropathy at the end of treatment (higher EORTC scores [P ≤ .026], and higher TNS scores [P ≤ .00]),” added Dr. Park, who is associate professor at the University of Sydney.
“Importantly, we also looked at long-term outcomes beyond the end of chemotherapy, because chronic neuropathy produces a significant burden in cancer survivors,” said Dr. Park.
“Among a total of 44 patients who completed the 6- to 12-month post-treatment follow-up, NfL levels after a single treatment were linked to severity of nerve damage quantified with neurophysiological tests, and greater Nfl levels at mid-treatment were correlated with worse patient and neurologically graded neuropathy at 6-12 months.”
Dr. Park said the results suggest that NfL may provide a biomarker of long-term axon damage and that Nfl assays “may enable clinicians to evaluate the risk of long-term toxicity early during paclitaxel treatment to hopefully provide clinically significant information to guide better treatment titration.”
Currently, she said, CIPN is a prominent cause of dose reduction and early chemotherapy cessation.
“For example, in early breast cancer around 25% of patients experience a dose reduction due to the severity of neuropathy symptoms.” But, she said, “there is no standardized way of identifying which patients are at risk of long-term neuropathy and therefore, may benefit more from dose reduction. In this setting, a biomarker such as Nfl could provide oncologists with more information about the risk of long-term toxicity and take that into account in dose decision-making.”
For some cancers, she added, there are multiple potential therapy options.
“A biomarker such as NfL could assist in determining risk-benefit profile in terms of switching to alternate therapies. However, further studies will be needed to fully define the utility of NfL as a biomarker of paclitaxel neuropathy.”
Promising Research
Commenting on the research for this news organization, Maryam Lustberg, MD, associate professor, director of the Center for Breast Cancer at Smilow Cancer Hospital and Yale Cancer Center, and chief of Breast Medical Oncology at Yale Cancer Center, in New Haven, Connecticut, said the study “builds on a body of work previously reported by others showing that neurofilament light chains as detected in the blood can be associated with early signs of neurotoxic injury.”
She added that the research “is promising, since existing clinical and patient-reported measures tend to under-detect chemotherapy-induced neuropathy until more permanent injury might have occurred.”
Dr. Lustberg, who is immediate past president of the Multinational Association of Supportive Care in Cancer, said future studies are needed before Nfl testing can be implemented in routine practice, but that “early detection will allow earlier initiation of supportive care strategies such as physical therapy and exercise, as well as dose modifications, which may be helpful for preventing permanent damage and improving quality of life.”
The investigators and Dr. Lustberg report no relevant financial relationships.
A version of this article appeared on Medscape.com.
AT PNS 2024
First-line Canakinumab Without Steroids Shows Effectiveness for Systemic Juvenile Idiopathic Arthritis
VIENNA — The interleukin-1 receptor antagonist (IL-1RA) canakinumab provided control of systemic juvenile idiopathic arthritis (sJIA) without the use of glucocorticoids for up to a year in most study participants after three monthly injections.
In this study of 20 patients with newly diagnosed sJIA treated off glucocorticoids, fever was controlled after a single injection in all patients, and 16 patients reached the primary outcome of remission after three injections, said Gerd Horneff, MD, PhD, Asklepios Children’s Hospital, Sankt Augustin, Germany.
Results of this open-label study, called CANAKINUMAB FIRST, were presented as late-breaking findings at the European Alliance of Associations for Rheumatology (EULAR) 2024 Annual Meeting.
“Steroid-free, first-line treatment with canakinumab led to sustained responses in most patients, with a considerable number achieving remission,” said Dr. Horneff, adding that the observation in this group is ongoing.
Building on Earlier Data
The efficacy of canakinumab was previously reported in anecdotal experiences and one small patient series published 10 years ago. Dr. Horneff noted that he has offered this drug off label to patients with challenging cases.
The objective was to evaluate canakinumab as a first-line monotherapy administered in the absence of glucocorticoids. The study was open to children aged 2-18 years with active sJIA/juvenile Still disease confirmed with published criteria. All were naive to biologic or nonbiologic disease-modifying antirheumatic drugs as well as steroids.
The median age of the children was 8.4 years. A total of 60% were men. The median disease duration at the time of entry was 1.2 months. Most had fever (95%) and rash (80%) with high levels of inflammatory markers at baseline. The mean number of painful joints was 3.1, and the mean number of systemic manifestations was 2.8. No patient was without any systemic involvement, but four of the patients did not have any painful joints.
At enrollment, patients were scheduled to receive three injections of canakinumab at monthly intervals during an active treatment phase, after which they entered an observation phase lasting 40 weeks. In the event of nonresponse or flares in either phase, they were transitioned to usual care.
Symptoms Resolve After Single Injection
After the first injection, active joint disease and all systemic manifestations resolved in 16 (80%) of the 20 patients. Joint activity and systemic manifestations also remained controlled after the second and third injections in 16 of the 20 patients.
One patient in this series achieved inactive disease after a single injection but developed what appeared to be a treatment-related allergic reaction. He received no further treatment and was excluded from the study, although he is being followed separately.
“According to sJADAS [systemic JIA Disease Activity Score] criteria at month 3, 14 had inactive disease, three had minimal disease activity, and one patient had moderate disease activity,” Dr. Horneff said.
At week 24, or 3 months after the last injection, there was still no joint activity in 16 patients. Systemic manifestations remained controlled in 13 patients, but 1 patient by this point had a flare. Another flare occurred after this point, and other patients have not yet completed the 52-week observation period.
“Of the 10 patients who remained in the study and have completed the 52-week observation period, eight have had a drug-free remission,” Dr. Horneff said.
MAS Event Observed in One Patient
In addition to the allergic skin reaction, which was considered probably related to the study drug, there were three flares, one of which was a macrophage activation syndrome (MAS) event. The MAS occurred 8 weeks after the last injection, but it was managed successfully.
Of 30 infections that developed during the observation period, 18 involved the upper airway. All were treated successfully. There were also two injection-site reactions and one case of cytopenia.
Among the studies planned for follow-up, investigators will examine genomic and gene activation in relation to disease activity and the effect of canakinumab.
Comoderator of the abstract session and chair of the EULAR 2024 Abstract Selection Committee, Christian Dejaco, MD, PhD, a consultant rheumatologist and associate professor at the Medical University of Graz in Graz, Austria, suggested that these are highly encouraging data for a disease that does not currently have any approved therapies. Clearly, larger studies with a longer follow-up period are needed, but he pointed out that phase 3 trials in a rare disease like sJIA are challenging.
Because of the limited number of cases, “it will be difficult to conduct a placebo-controlled trial,” he pointed out. However, he hopes this study will provide the basis for larger studies and sufficient data to lead to an indication for this therapy.
In the meantime, he also believes that these data are likely to support empirical use in a difficult disease, even in advance of formal regulatory approval.
“We heard that canakinumab is already being used off label in JIA, and these data might encourage more of that,” he said.
Dr. Horneff reported financial relationships with AbbVie, Boehringer Ingelheim, Celgene, Chugai, GlaxoSmithKline, Janssen, Merck Sharpe & Dohme, Novartis, Pfizer, Roche, Sanofi, and Sobe. Dr. Dejaco reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
VIENNA — The interleukin-1 receptor antagonist (IL-1RA) canakinumab provided control of systemic juvenile idiopathic arthritis (sJIA) without the use of glucocorticoids for up to a year in most study participants after three monthly injections.
In this study of 20 patients with newly diagnosed sJIA treated off glucocorticoids, fever was controlled after a single injection in all patients, and 16 patients reached the primary outcome of remission after three injections, said Gerd Horneff, MD, PhD, Asklepios Children’s Hospital, Sankt Augustin, Germany.
Results of this open-label study, called CANAKINUMAB FIRST, were presented as late-breaking findings at the European Alliance of Associations for Rheumatology (EULAR) 2024 Annual Meeting.
“Steroid-free, first-line treatment with canakinumab led to sustained responses in most patients, with a considerable number achieving remission,” said Dr. Horneff, adding that the observation in this group is ongoing.
Building on Earlier Data
The efficacy of canakinumab was previously reported in anecdotal experiences and one small patient series published 10 years ago. Dr. Horneff noted that he has offered this drug off label to patients with challenging cases.
The objective was to evaluate canakinumab as a first-line monotherapy administered in the absence of glucocorticoids. The study was open to children aged 2-18 years with active sJIA/juvenile Still disease confirmed with published criteria. All were naive to biologic or nonbiologic disease-modifying antirheumatic drugs as well as steroids.
The median age of the children was 8.4 years. A total of 60% were men. The median disease duration at the time of entry was 1.2 months. Most had fever (95%) and rash (80%) with high levels of inflammatory markers at baseline. The mean number of painful joints was 3.1, and the mean number of systemic manifestations was 2.8. No patient was without any systemic involvement, but four of the patients did not have any painful joints.
At enrollment, patients were scheduled to receive three injections of canakinumab at monthly intervals during an active treatment phase, after which they entered an observation phase lasting 40 weeks. In the event of nonresponse or flares in either phase, they were transitioned to usual care.
Symptoms Resolve After Single Injection
After the first injection, active joint disease and all systemic manifestations resolved in 16 (80%) of the 20 patients. Joint activity and systemic manifestations also remained controlled after the second and third injections in 16 of the 20 patients.
One patient in this series achieved inactive disease after a single injection but developed what appeared to be a treatment-related allergic reaction. He received no further treatment and was excluded from the study, although he is being followed separately.
“According to sJADAS [systemic JIA Disease Activity Score] criteria at month 3, 14 had inactive disease, three had minimal disease activity, and one patient had moderate disease activity,” Dr. Horneff said.
At week 24, or 3 months after the last injection, there was still no joint activity in 16 patients. Systemic manifestations remained controlled in 13 patients, but 1 patient by this point had a flare. Another flare occurred after this point, and other patients have not yet completed the 52-week observation period.
“Of the 10 patients who remained in the study and have completed the 52-week observation period, eight have had a drug-free remission,” Dr. Horneff said.
MAS Event Observed in One Patient
In addition to the allergic skin reaction, which was considered probably related to the study drug, there were three flares, one of which was a macrophage activation syndrome (MAS) event. The MAS occurred 8 weeks after the last injection, but it was managed successfully.
Of 30 infections that developed during the observation period, 18 involved the upper airway. All were treated successfully. There were also two injection-site reactions and one case of cytopenia.
Among the studies planned for follow-up, investigators will examine genomic and gene activation in relation to disease activity and the effect of canakinumab.
Comoderator of the abstract session and chair of the EULAR 2024 Abstract Selection Committee, Christian Dejaco, MD, PhD, a consultant rheumatologist and associate professor at the Medical University of Graz in Graz, Austria, suggested that these are highly encouraging data for a disease that does not currently have any approved therapies. Clearly, larger studies with a longer follow-up period are needed, but he pointed out that phase 3 trials in a rare disease like sJIA are challenging.
Because of the limited number of cases, “it will be difficult to conduct a placebo-controlled trial,” he pointed out. However, he hopes this study will provide the basis for larger studies and sufficient data to lead to an indication for this therapy.
In the meantime, he also believes that these data are likely to support empirical use in a difficult disease, even in advance of formal regulatory approval.
“We heard that canakinumab is already being used off label in JIA, and these data might encourage more of that,” he said.
Dr. Horneff reported financial relationships with AbbVie, Boehringer Ingelheim, Celgene, Chugai, GlaxoSmithKline, Janssen, Merck Sharpe & Dohme, Novartis, Pfizer, Roche, Sanofi, and Sobe. Dr. Dejaco reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
VIENNA — The interleukin-1 receptor antagonist (IL-1RA) canakinumab provided control of systemic juvenile idiopathic arthritis (sJIA) without the use of glucocorticoids for up to a year in most study participants after three monthly injections.
In this study of 20 patients with newly diagnosed sJIA treated off glucocorticoids, fever was controlled after a single injection in all patients, and 16 patients reached the primary outcome of remission after three injections, said Gerd Horneff, MD, PhD, Asklepios Children’s Hospital, Sankt Augustin, Germany.
Results of this open-label study, called CANAKINUMAB FIRST, were presented as late-breaking findings at the European Alliance of Associations for Rheumatology (EULAR) 2024 Annual Meeting.
“Steroid-free, first-line treatment with canakinumab led to sustained responses in most patients, with a considerable number achieving remission,” said Dr. Horneff, adding that the observation in this group is ongoing.
Building on Earlier Data
The efficacy of canakinumab was previously reported in anecdotal experiences and one small patient series published 10 years ago. Dr. Horneff noted that he has offered this drug off label to patients with challenging cases.
The objective was to evaluate canakinumab as a first-line monotherapy administered in the absence of glucocorticoids. The study was open to children aged 2-18 years with active sJIA/juvenile Still disease confirmed with published criteria. All were naive to biologic or nonbiologic disease-modifying antirheumatic drugs as well as steroids.
The median age of the children was 8.4 years. A total of 60% were men. The median disease duration at the time of entry was 1.2 months. Most had fever (95%) and rash (80%) with high levels of inflammatory markers at baseline. The mean number of painful joints was 3.1, and the mean number of systemic manifestations was 2.8. No patient was without any systemic involvement, but four of the patients did not have any painful joints.
At enrollment, patients were scheduled to receive three injections of canakinumab at monthly intervals during an active treatment phase, after which they entered an observation phase lasting 40 weeks. In the event of nonresponse or flares in either phase, they were transitioned to usual care.
Symptoms Resolve After Single Injection
After the first injection, active joint disease and all systemic manifestations resolved in 16 (80%) of the 20 patients. Joint activity and systemic manifestations also remained controlled after the second and third injections in 16 of the 20 patients.
One patient in this series achieved inactive disease after a single injection but developed what appeared to be a treatment-related allergic reaction. He received no further treatment and was excluded from the study, although he is being followed separately.
“According to sJADAS [systemic JIA Disease Activity Score] criteria at month 3, 14 had inactive disease, three had minimal disease activity, and one patient had moderate disease activity,” Dr. Horneff said.
At week 24, or 3 months after the last injection, there was still no joint activity in 16 patients. Systemic manifestations remained controlled in 13 patients, but 1 patient by this point had a flare. Another flare occurred after this point, and other patients have not yet completed the 52-week observation period.
“Of the 10 patients who remained in the study and have completed the 52-week observation period, eight have had a drug-free remission,” Dr. Horneff said.
MAS Event Observed in One Patient
In addition to the allergic skin reaction, which was considered probably related to the study drug, there were three flares, one of which was a macrophage activation syndrome (MAS) event. The MAS occurred 8 weeks after the last injection, but it was managed successfully.
Of 30 infections that developed during the observation period, 18 involved the upper airway. All were treated successfully. There were also two injection-site reactions and one case of cytopenia.
Among the studies planned for follow-up, investigators will examine genomic and gene activation in relation to disease activity and the effect of canakinumab.
Comoderator of the abstract session and chair of the EULAR 2024 Abstract Selection Committee, Christian Dejaco, MD, PhD, a consultant rheumatologist and associate professor at the Medical University of Graz in Graz, Austria, suggested that these are highly encouraging data for a disease that does not currently have any approved therapies. Clearly, larger studies with a longer follow-up period are needed, but he pointed out that phase 3 trials in a rare disease like sJIA are challenging.
Because of the limited number of cases, “it will be difficult to conduct a placebo-controlled trial,” he pointed out. However, he hopes this study will provide the basis for larger studies and sufficient data to lead to an indication for this therapy.
In the meantime, he also believes that these data are likely to support empirical use in a difficult disease, even in advance of formal regulatory approval.
“We heard that canakinumab is already being used off label in JIA, and these data might encourage more of that,” he said.
Dr. Horneff reported financial relationships with AbbVie, Boehringer Ingelheim, Celgene, Chugai, GlaxoSmithKline, Janssen, Merck Sharpe & Dohme, Novartis, Pfizer, Roche, Sanofi, and Sobe. Dr. Dejaco reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
FROM EULAR 2024
CMS Announces End to Cyberattack Relief Program
The Centers for Medicare & Medicaid Services (CMS) has announced the conclusion of a program that provided billions in early Medicare payments to those affected by the Change Healthcare/UnitedHealth Group cyberattack last winter.
CMS reported that the program advanced more than $2.55 billion in Medicare payments to > 4200 Part A providers, including hospitals, and more than $717.18 million in payments to Part B suppliers such as physicians, nonphysician practitioners, and durable medical equipment suppliers.
According to CMS, the Medicare billing system is now functioning properly, and 96% of the early payments have been recovered. The advances were to represent ≤ 30 days of typical claims payments in a 3-month period of 2023, with full repayment expected within 90 days through “automatic recoupment from Medicare claims” — no extensions allowed.
The agency took a victory lap regarding its response. “In the face of one of the most widespread cyberattacks on the US health care industry, CMS promptly took action to get providers and suppliers access to the funds they needed to continue providing patients with vital care,” CMS Administrator Chiquita Brooks-LaSure said in a statement. “Our efforts helped minimize the disruptive fallout from this incident, and we will remain vigilant to be ready to address future events.”
Ongoing Concerns from Health Care Organizations
Ben Teicher, an American Hospital Association spokesman, said that the organization hopes that CMS will be responsive if there’s more need for action after the advance payment program expires. The organization represents about 5000 hospitals, health care systems, and other providers.
“Our members report that the aftereffects of this event will likely be felt throughout the remainder of the year,” he said. According to Teicher, hospitals remain concerned about their ability to process claims and appeal denials, the safety of reconnecting to cyber services, and access to information needed to bill patients and reconcile payments.
In addition, hospitals are concerned about “financial support to mitigate the considerable costs incurred as a result of the cyberattack,” he said.
Charlene MacDonald, executive vice-president of public affairs at the Federation of American Hospitals, which represents more than 1000 for-profit hospitals, sent a statement to this news organization that said some providers “are still feeling the effects of care denials and delays caused by insurer inaction.
“We appreciate that the Administration acted within its authority to support providers during this unprecedented crisis and blunt these devastating impacts, especially because a vast majority of managed care companies failed to step up to the plate,” she said. “It is now time to shift our focus to holding plans accountable for using tactics to delay and deny needed patient care.”
Cyberattack Impact and Response
The ransom-based cyberattack against Change Healthcare/UnitedHealth Group targeted an electronic data interchange clearing house processing payer reimbursement systems, disrupting cash flows at hospitals and medical practices, and affecting patient access to prescriptions and life-saving therapy.
Change Healthcare — part of the UnitedHealth Group subsidiary Optum — processes half of all medical claims, according to a Department of Justice lawsuit. The American Hospital Association described the cyberattack as “the most significant and consequential incident of its kind” in US history.
By late March, UnitedHealth Group said nearly all medical and pharmacy claims were processing properly, while a deputy secretary of the US Department of Health & Human Services told clinicians that officials were focusing on the last group of clinicians who were facing cash-flow problems.
Still, a senior advisor with CMS told providers at that time that “we have heard from so many providers over the last several weeks who are really struggling to make ends meet right now or who are worried that they will not be able to make payroll in the weeks to come.”
Randy Dotinga is a freelance health/medical reporter and board member of the Association of Health Care Journalists.
A version of this article appeared on Medscape.com.
The Centers for Medicare & Medicaid Services (CMS) has announced the conclusion of a program that provided billions in early Medicare payments to those affected by the Change Healthcare/UnitedHealth Group cyberattack last winter.
CMS reported that the program advanced more than $2.55 billion in Medicare payments to > 4200 Part A providers, including hospitals, and more than $717.18 million in payments to Part B suppliers such as physicians, nonphysician practitioners, and durable medical equipment suppliers.
According to CMS, the Medicare billing system is now functioning properly, and 96% of the early payments have been recovered. The advances were to represent ≤ 30 days of typical claims payments in a 3-month period of 2023, with full repayment expected within 90 days through “automatic recoupment from Medicare claims” — no extensions allowed.
The agency took a victory lap regarding its response. “In the face of one of the most widespread cyberattacks on the US health care industry, CMS promptly took action to get providers and suppliers access to the funds they needed to continue providing patients with vital care,” CMS Administrator Chiquita Brooks-LaSure said in a statement. “Our efforts helped minimize the disruptive fallout from this incident, and we will remain vigilant to be ready to address future events.”
Ongoing Concerns from Health Care Organizations
Ben Teicher, an American Hospital Association spokesman, said that the organization hopes that CMS will be responsive if there’s more need for action after the advance payment program expires. The organization represents about 5000 hospitals, health care systems, and other providers.
“Our members report that the aftereffects of this event will likely be felt throughout the remainder of the year,” he said. According to Teicher, hospitals remain concerned about their ability to process claims and appeal denials, the safety of reconnecting to cyber services, and access to information needed to bill patients and reconcile payments.
In addition, hospitals are concerned about “financial support to mitigate the considerable costs incurred as a result of the cyberattack,” he said.
Charlene MacDonald, executive vice-president of public affairs at the Federation of American Hospitals, which represents more than 1000 for-profit hospitals, sent a statement to this news organization that said some providers “are still feeling the effects of care denials and delays caused by insurer inaction.
“We appreciate that the Administration acted within its authority to support providers during this unprecedented crisis and blunt these devastating impacts, especially because a vast majority of managed care companies failed to step up to the plate,” she said. “It is now time to shift our focus to holding plans accountable for using tactics to delay and deny needed patient care.”
Cyberattack Impact and Response
The ransom-based cyberattack against Change Healthcare/UnitedHealth Group targeted an electronic data interchange clearing house processing payer reimbursement systems, disrupting cash flows at hospitals and medical practices, and affecting patient access to prescriptions and life-saving therapy.
Change Healthcare — part of the UnitedHealth Group subsidiary Optum — processes half of all medical claims, according to a Department of Justice lawsuit. The American Hospital Association described the cyberattack as “the most significant and consequential incident of its kind” in US history.
By late March, UnitedHealth Group said nearly all medical and pharmacy claims were processing properly, while a deputy secretary of the US Department of Health & Human Services told clinicians that officials were focusing on the last group of clinicians who were facing cash-flow problems.
Still, a senior advisor with CMS told providers at that time that “we have heard from so many providers over the last several weeks who are really struggling to make ends meet right now or who are worried that they will not be able to make payroll in the weeks to come.”
Randy Dotinga is a freelance health/medical reporter and board member of the Association of Health Care Journalists.
A version of this article appeared on Medscape.com.
The Centers for Medicare & Medicaid Services (CMS) has announced the conclusion of a program that provided billions in early Medicare payments to those affected by the Change Healthcare/UnitedHealth Group cyberattack last winter.
CMS reported that the program advanced more than $2.55 billion in Medicare payments to > 4200 Part A providers, including hospitals, and more than $717.18 million in payments to Part B suppliers such as physicians, nonphysician practitioners, and durable medical equipment suppliers.
According to CMS, the Medicare billing system is now functioning properly, and 96% of the early payments have been recovered. The advances were to represent ≤ 30 days of typical claims payments in a 3-month period of 2023, with full repayment expected within 90 days through “automatic recoupment from Medicare claims” — no extensions allowed.
The agency took a victory lap regarding its response. “In the face of one of the most widespread cyberattacks on the US health care industry, CMS promptly took action to get providers and suppliers access to the funds they needed to continue providing patients with vital care,” CMS Administrator Chiquita Brooks-LaSure said in a statement. “Our efforts helped minimize the disruptive fallout from this incident, and we will remain vigilant to be ready to address future events.”
Ongoing Concerns from Health Care Organizations
Ben Teicher, an American Hospital Association spokesman, said that the organization hopes that CMS will be responsive if there’s more need for action after the advance payment program expires. The organization represents about 5000 hospitals, health care systems, and other providers.
“Our members report that the aftereffects of this event will likely be felt throughout the remainder of the year,” he said. According to Teicher, hospitals remain concerned about their ability to process claims and appeal denials, the safety of reconnecting to cyber services, and access to information needed to bill patients and reconcile payments.
In addition, hospitals are concerned about “financial support to mitigate the considerable costs incurred as a result of the cyberattack,” he said.
Charlene MacDonald, executive vice-president of public affairs at the Federation of American Hospitals, which represents more than 1000 for-profit hospitals, sent a statement to this news organization that said some providers “are still feeling the effects of care denials and delays caused by insurer inaction.
“We appreciate that the Administration acted within its authority to support providers during this unprecedented crisis and blunt these devastating impacts, especially because a vast majority of managed care companies failed to step up to the plate,” she said. “It is now time to shift our focus to holding plans accountable for using tactics to delay and deny needed patient care.”
Cyberattack Impact and Response
The ransom-based cyberattack against Change Healthcare/UnitedHealth Group targeted an electronic data interchange clearing house processing payer reimbursement systems, disrupting cash flows at hospitals and medical practices, and affecting patient access to prescriptions and life-saving therapy.
Change Healthcare — part of the UnitedHealth Group subsidiary Optum — processes half of all medical claims, according to a Department of Justice lawsuit. The American Hospital Association described the cyberattack as “the most significant and consequential incident of its kind” in US history.
By late March, UnitedHealth Group said nearly all medical and pharmacy claims were processing properly, while a deputy secretary of the US Department of Health & Human Services told clinicians that officials were focusing on the last group of clinicians who were facing cash-flow problems.
Still, a senior advisor with CMS told providers at that time that “we have heard from so many providers over the last several weeks who are really struggling to make ends meet right now or who are worried that they will not be able to make payroll in the weeks to come.”
Randy Dotinga is a freelance health/medical reporter and board member of the Association of Health Care Journalists.
A version of this article appeared on Medscape.com.
Is This Journal Legit? Predatory Publishers
This transcript has been edited for clarity.
Andrew N. Wilner, MD: My guest today is Dr. Jose Merino, editor in chief of the Neurology family of journals and professor of neurology and co-vice chair of education at Georgetown University in Washington, DC.
Our program today is a follow-up of Dr. Merino’s presentation at the recent American Academy of Neurology meeting in Denver, Colorado. Along with two other panelists, Dr. Merino discussed the role of open-access publication and the dangers of predatory journals.
Jose G. Merino, MD, MPhil: Thank you for having me here. It’s a pleasure.
Open Access Defined
Dr. Wilner: I remember when publication in neurology was pretty straightforward. It was either the green journal or the blue journal, but things have certainly changed. I think one topic that is not clear to everyone is this concept of open access. Could you define that for us?
Dr. Merino: Sure. Open access is a mode of publication that fosters more open or accessible science. The idea of open access is that it combines two main elements. One is that the papers that are published become immediately available to anybody with an internet connection anywhere in the world without any restrictions.
The second important element from open access, which makes it different from other models we can talk about, is the fact that the authors retain the copyright of their work, but they give the journal and readers a license to use, reproduce, and modify the content.
This is different, for example, from instances where we have funder mandates. For example, NIH papers have to become available 6 months after publication, so they’re available to everybody but not immediately.
Dr. Wilner: I remember that when a journal article was published, say, in Neurology, if you didn’t have a subscription to Neurology, you went to the library that hopefully had a subscription.
If they didn’t have it, you would write to the author and say, “Hey, I heard you have this great paper because the abstract was out there. Could you send me a reprint?” Has that whole universe evaporated?
Dr. Merino: It depends on how the paper is published. For example, in Neurology, some of the research we publish is open access. Basically, if you have an internet connection, you can access the paper.
That’s the case for papers published in our wholly open-access journals in the Neurology family like Neurology Neuroimmunology & Neuroinflammation, Neurology Genetics, or Neurology Education.
For other papers that are published in Neurology, not under open access, there is a paywall. For some of them, the paywall comes down after a few months based on funder mandates and so on. As I was mentioning, the NIH-funded papers are available 6 months later.
In the first 6 months, you may have to go to your library, and if your library has a subscription, you can download it directly. [This is also true for] those that always stay behind the paywall, where you have to have a subscription or your library has to have a subscription.
Is Pay to Publish a Red Flag?
Dr. Wilner: I’m a professional writer. With any luck, when I write something, I get paid to write it. There’s been a long tradition in academic medicine that when you submit an article to, say, Neurology, you don’t get paid as an author for the publication. Your reward is the honor of it being published.
Neurology supports itself in various ways, including advertising and so on. That’s been the contract: free publication for work that merits it, and the journal survives on its own.
With open access, one of the things that’s happened is that — and I’ve published open access myself — is that I get a notification that I need to pay to have my article that I’ve slaved over published. Explain that, please.
Dr. Merino: This is the issue with open access. As I mentioned, the paper gets published. You’re giving the journal a license to publish it. You’re retaining the copyright of your work. That means that the journal cannot make money or support itself by just publishing open access because they belong to you.
Typically, open-access journals are not in print and don’t have much in terms of advertising. The contract is you’re giving me a license to publish it, but it’s your journal, so you’re paying a fee for the journal expenses to basically produce your paper. That’s what’s happening with open access.
That’s been recognized with many funders, for example, with NIH funding or many of the European funders, they’re including open-access fees as part of their funding for research. Now, of course, this doesn’t help if you’re not a funded researcher or if you’re a fellow who’s doing work and so on.
Typically, most journals will have waived fees or lower fees for these situations. The reason for the open-access fee is the fact that you’re retaining the copyright. You’re not giving it to the journal who can then use it to generate its revenue for supporting itself, the editorial staff, and so on.
Dr. Wilner: This idea of charging for publication has created a satellite business of what are called predatory journals. How does one know if the open-access journal that I’m submitting to is really just in the business of wanting my $300 or my $900 to get published? How do I know if that’s a reasonable place to publish?
Predatory Journals
Dr. Merino: That’s a big challenge that has come with this whole idea of open access and the fact that now, many journals are online only, so you’re no longer seeing a physical copy. That has given rise to the predatory journals.
The predatory journal, by definition, is a journal that claims to be open access. They’ll take your paper and publish it, but they don’t provide all the other services that you would typically expect from the fact that you’re paying an open-access fee. This includes getting appropriate peer review, production of the manuscript, and long-term curation and storage of the manuscript.
Many predatory journals will take your open-access fee, accept any paper that you submit, regardless of the quality, because they’re charging the fees for that. They don’t send it to real peer review, and then in a few months, the journal disappears so there’s no way for anybody to actually find your paper anymore.
There are certain checklists. Dr. David Moher at the University of Toronto has produced some work trying to help us identify predatory journals.
One thing I typically suggest to people who ask me this question is: Have you ever heard of this journal before? Does the journal have a track record? How far back does the story of the journal go? Is it supported by a publisher that you know? Do you know anybody who has published there? Is it something you can easily access?
If in doubt, always ask your friendly medical librarian. There used to be lists that were kept in terms of predatory journals that were being constantly updated, but those had to be shut down. As far as I understand, there were legal issues in terms of how things got on that list.
I think that overall, if you’ve heard of it, if it’s relevant, if it’s known in your field, and if your librarian knows it, it’s probably a good legitimate open-access journal. There are many very good legitimate open-access journals.
I mentioned the two that we have in our family, but all the other major journals have their own open-access journal within their family. There are some, like BMC or PLOS, that are completely open-access and legitimate journals.
Impact Factor
Dr. Wilner: What about impact factor? Many journals boast about their impact factor. I’m not sure how to interpret that number.
Dr. Merino: Impact factor is very interesting. The impact factor was developed by medical librarians to try to identify the journals they should be subscribing to. It’s a measure of the average citations to an average paper in the journal.
It doesn’t tell you about specific papers. It tells you, on average, how many of the papers in this journal get cited so many times. It’s calculated by the number of articles that were cited divided by the number of articles that were published. Journals that publish many papers, like Neurology, have a hard time bringing up their impact factor beyond a certain level.
Similarly, very small journals with one or two very highly cited papers have a very high impact factor. It’s being used as a measure, perhaps inappropriately, of how good or how reputable a journal is. We all say we don’t care about journal impact factors, but we all know our journal impact factor and we used to know it to three decimals. Now, they changed the system, and there’s only one decimal point, which makes more sense.
This is more important, for example, for authors when deciding where to submit papers. I know that in some countries, particularly in Europe, the impact factor of the journal where you publish has an impact on your promotion decisions.
I would say what’s even more important than the impact factor, is to say, “Well, is this the journal that fits the scope of my paper? Is this the journal that reaches the audience that I want to reach when I write my paper?”
There are some papers, for example, that are very influential. The impact factor just captures citations. There are some papers that are very influential that may not get cited very often. There may be papers that change clinical practice.
If you read a paper that tells you that you should be changing how you treat your patients with myasthenia based on this paper, that may not get cited. It’s a very clinically focused paper, but it’s probably more impactful than one that gets cited very much in some respect, or they make it to public policy decisions, and so on.
I think it’s important to look more at the audience and the journal scope when you submit your papers.
Dr. Wilner: One other technical question. The journals also say they’re indexed in PubMed or Google Scholar. If I want to publish my paper and I want it indexed where the right people are going to find it, where does it need to be indexed?
Dr. Merino: I grew up using Index Medicus, MedlinePlus, and the Library of Science. I still do. If I need to find something, I go to PubMed. Ideally, papers are listed in MedlinePlus or can be found in PubMed. They’re not the same thing, but you can find them through them.
That would be an important thing. Nowadays, a lot more people are using Google Scholar or Google just to identify papers. It may be a little bit less relevant, but it’s still a measure of the quality of the journal before they get indexed in some of these. For example, if you get listed in MedlinePlus, it has gone through certain quality checks by the index itself to see whether they would accept the journal or not. That’s something you want to check.
Typically, most of the large journals or the journals you and I know about are listed in more than one place, right? They’re listed in Scopus and Web of Science. They’re listed in MedlinePlus and so on. Again, if you’re submitting your paper, go somewhere where you know the journal and you’ve heard about it.
Dr. Wilner: I’m not going to ask you about artificial intelligence. We can do that another time. I want to ask something closer to me, which is this question of publish or perish.
There seems to be, in academics, more emphasis on the number of papers that one has published rather than their quality. How does a younger academician or one who really needs to publish cope with that?
Dr. Merino: Many people are writing up research that may not be relevant or that may not be high quality just because you need to have a long list of papers to get promoted, for example, if you’re an academician.
Doug Altman, who was a very influential person in the field quality of not only medical statistics but also medical publishing, had the idea that we need less research, but we need better research.
We often receive papers where you say, well, what’s the rationale behind the question in this paper? It’s like they had a large amount of data and were trying to squeeze as much as they could out of that. I think, as a young academician, the important thing to think about is whether it is an important question that matters to you and to the field, from whatever perspective, whether it’s going to advance research, advance clinical care, or have public policy implications.
Is this one where the answer will be important no matter what the answer is? If you’re thinking of that, your work will be well recognized, people will know you, and you’ll get invited to collaborate. I think that’s the most important thing rather than just churning out a large number of papers.
The productivity will come from the fact that you start by saying, let me ask something that’s really meaningful to me and to the field, with a good question and using strong research methodology.
Dr. Wilner: Thanks for that, Dr. Merino. I think that’s very valuable for all of us. This has been a great discussion. Do you have any final comments before we wrap up?
Dr. Merino: I want to encourage people to continue reading medical journals all the time and submitting to us, again, good research and important questions with robust methodology. That’s what we’re looking for in Neurology and most serious medical journals.
Dr. Wilner is an associate professor of neurology at the University of Tennessee Health Science Center, Memphis. Dr. Merino is a professor in the department of neurology at Georgetown University Medical Center, Washington, DC. Dr. Wilner reported conflicts of interest with Accordant Health Services and Lulu Publishing. Dr. Merino reported no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
This transcript has been edited for clarity.
Andrew N. Wilner, MD: My guest today is Dr. Jose Merino, editor in chief of the Neurology family of journals and professor of neurology and co-vice chair of education at Georgetown University in Washington, DC.
Our program today is a follow-up of Dr. Merino’s presentation at the recent American Academy of Neurology meeting in Denver, Colorado. Along with two other panelists, Dr. Merino discussed the role of open-access publication and the dangers of predatory journals.
Jose G. Merino, MD, MPhil: Thank you for having me here. It’s a pleasure.
Open Access Defined
Dr. Wilner: I remember when publication in neurology was pretty straightforward. It was either the green journal or the blue journal, but things have certainly changed. I think one topic that is not clear to everyone is this concept of open access. Could you define that for us?
Dr. Merino: Sure. Open access is a mode of publication that fosters more open or accessible science. The idea of open access is that it combines two main elements. One is that the papers that are published become immediately available to anybody with an internet connection anywhere in the world without any restrictions.
The second important element from open access, which makes it different from other models we can talk about, is the fact that the authors retain the copyright of their work, but they give the journal and readers a license to use, reproduce, and modify the content.
This is different, for example, from instances where we have funder mandates. For example, NIH papers have to become available 6 months after publication, so they’re available to everybody but not immediately.
Dr. Wilner: I remember that when a journal article was published, say, in Neurology, if you didn’t have a subscription to Neurology, you went to the library that hopefully had a subscription.
If they didn’t have it, you would write to the author and say, “Hey, I heard you have this great paper because the abstract was out there. Could you send me a reprint?” Has that whole universe evaporated?
Dr. Merino: It depends on how the paper is published. For example, in Neurology, some of the research we publish is open access. Basically, if you have an internet connection, you can access the paper.
That’s the case for papers published in our wholly open-access journals in the Neurology family like Neurology Neuroimmunology & Neuroinflammation, Neurology Genetics, or Neurology Education.
For other papers that are published in Neurology, not under open access, there is a paywall. For some of them, the paywall comes down after a few months based on funder mandates and so on. As I was mentioning, the NIH-funded papers are available 6 months later.
In the first 6 months, you may have to go to your library, and if your library has a subscription, you can download it directly. [This is also true for] those that always stay behind the paywall, where you have to have a subscription or your library has to have a subscription.
Is Pay to Publish a Red Flag?
Dr. Wilner: I’m a professional writer. With any luck, when I write something, I get paid to write it. There’s been a long tradition in academic medicine that when you submit an article to, say, Neurology, you don’t get paid as an author for the publication. Your reward is the honor of it being published.
Neurology supports itself in various ways, including advertising and so on. That’s been the contract: free publication for work that merits it, and the journal survives on its own.
With open access, one of the things that’s happened is that — and I’ve published open access myself — is that I get a notification that I need to pay to have my article that I’ve slaved over published. Explain that, please.
Dr. Merino: This is the issue with open access. As I mentioned, the paper gets published. You’re giving the journal a license to publish it. You’re retaining the copyright of your work. That means that the journal cannot make money or support itself by just publishing open access because they belong to you.
Typically, open-access journals are not in print and don’t have much in terms of advertising. The contract is you’re giving me a license to publish it, but it’s your journal, so you’re paying a fee for the journal expenses to basically produce your paper. That’s what’s happening with open access.
That’s been recognized with many funders, for example, with NIH funding or many of the European funders, they’re including open-access fees as part of their funding for research. Now, of course, this doesn’t help if you’re not a funded researcher or if you’re a fellow who’s doing work and so on.
Typically, most journals will have waived fees or lower fees for these situations. The reason for the open-access fee is the fact that you’re retaining the copyright. You’re not giving it to the journal who can then use it to generate its revenue for supporting itself, the editorial staff, and so on.
Dr. Wilner: This idea of charging for publication has created a satellite business of what are called predatory journals. How does one know if the open-access journal that I’m submitting to is really just in the business of wanting my $300 or my $900 to get published? How do I know if that’s a reasonable place to publish?
Predatory Journals
Dr. Merino: That’s a big challenge that has come with this whole idea of open access and the fact that now, many journals are online only, so you’re no longer seeing a physical copy. That has given rise to the predatory journals.
The predatory journal, by definition, is a journal that claims to be open access. They’ll take your paper and publish it, but they don’t provide all the other services that you would typically expect from the fact that you’re paying an open-access fee. This includes getting appropriate peer review, production of the manuscript, and long-term curation and storage of the manuscript.
Many predatory journals will take your open-access fee, accept any paper that you submit, regardless of the quality, because they’re charging the fees for that. They don’t send it to real peer review, and then in a few months, the journal disappears so there’s no way for anybody to actually find your paper anymore.
There are certain checklists. Dr. David Moher at the University of Toronto has produced some work trying to help us identify predatory journals.
One thing I typically suggest to people who ask me this question is: Have you ever heard of this journal before? Does the journal have a track record? How far back does the story of the journal go? Is it supported by a publisher that you know? Do you know anybody who has published there? Is it something you can easily access?
If in doubt, always ask your friendly medical librarian. There used to be lists that were kept in terms of predatory journals that were being constantly updated, but those had to be shut down. As far as I understand, there were legal issues in terms of how things got on that list.
I think that overall, if you’ve heard of it, if it’s relevant, if it’s known in your field, and if your librarian knows it, it’s probably a good legitimate open-access journal. There are many very good legitimate open-access journals.
I mentioned the two that we have in our family, but all the other major journals have their own open-access journal within their family. There are some, like BMC or PLOS, that are completely open-access and legitimate journals.
Impact Factor
Dr. Wilner: What about impact factor? Many journals boast about their impact factor. I’m not sure how to interpret that number.
Dr. Merino: Impact factor is very interesting. The impact factor was developed by medical librarians to try to identify the journals they should be subscribing to. It’s a measure of the average citations to an average paper in the journal.
It doesn’t tell you about specific papers. It tells you, on average, how many of the papers in this journal get cited so many times. It’s calculated by the number of articles that were cited divided by the number of articles that were published. Journals that publish many papers, like Neurology, have a hard time bringing up their impact factor beyond a certain level.
Similarly, very small journals with one or two very highly cited papers have a very high impact factor. It’s being used as a measure, perhaps inappropriately, of how good or how reputable a journal is. We all say we don’t care about journal impact factors, but we all know our journal impact factor and we used to know it to three decimals. Now, they changed the system, and there’s only one decimal point, which makes more sense.
This is more important, for example, for authors when deciding where to submit papers. I know that in some countries, particularly in Europe, the impact factor of the journal where you publish has an impact on your promotion decisions.
I would say what’s even more important than the impact factor, is to say, “Well, is this the journal that fits the scope of my paper? Is this the journal that reaches the audience that I want to reach when I write my paper?”
There are some papers, for example, that are very influential. The impact factor just captures citations. There are some papers that are very influential that may not get cited very often. There may be papers that change clinical practice.
If you read a paper that tells you that you should be changing how you treat your patients with myasthenia based on this paper, that may not get cited. It’s a very clinically focused paper, but it’s probably more impactful than one that gets cited very much in some respect, or they make it to public policy decisions, and so on.
I think it’s important to look more at the audience and the journal scope when you submit your papers.
Dr. Wilner: One other technical question. The journals also say they’re indexed in PubMed or Google Scholar. If I want to publish my paper and I want it indexed where the right people are going to find it, where does it need to be indexed?
Dr. Merino: I grew up using Index Medicus, MedlinePlus, and the Library of Science. I still do. If I need to find something, I go to PubMed. Ideally, papers are listed in MedlinePlus or can be found in PubMed. They’re not the same thing, but you can find them through them.
That would be an important thing. Nowadays, a lot more people are using Google Scholar or Google just to identify papers. It may be a little bit less relevant, but it’s still a measure of the quality of the journal before they get indexed in some of these. For example, if you get listed in MedlinePlus, it has gone through certain quality checks by the index itself to see whether they would accept the journal or not. That’s something you want to check.
Typically, most of the large journals or the journals you and I know about are listed in more than one place, right? They’re listed in Scopus and Web of Science. They’re listed in MedlinePlus and so on. Again, if you’re submitting your paper, go somewhere where you know the journal and you’ve heard about it.
Dr. Wilner: I’m not going to ask you about artificial intelligence. We can do that another time. I want to ask something closer to me, which is this question of publish or perish.
There seems to be, in academics, more emphasis on the number of papers that one has published rather than their quality. How does a younger academician or one who really needs to publish cope with that?
Dr. Merino: Many people are writing up research that may not be relevant or that may not be high quality just because you need to have a long list of papers to get promoted, for example, if you’re an academician.
Doug Altman, who was a very influential person in the field quality of not only medical statistics but also medical publishing, had the idea that we need less research, but we need better research.
We often receive papers where you say, well, what’s the rationale behind the question in this paper? It’s like they had a large amount of data and were trying to squeeze as much as they could out of that. I think, as a young academician, the important thing to think about is whether it is an important question that matters to you and to the field, from whatever perspective, whether it’s going to advance research, advance clinical care, or have public policy implications.
Is this one where the answer will be important no matter what the answer is? If you’re thinking of that, your work will be well recognized, people will know you, and you’ll get invited to collaborate. I think that’s the most important thing rather than just churning out a large number of papers.
The productivity will come from the fact that you start by saying, let me ask something that’s really meaningful to me and to the field, with a good question and using strong research methodology.
Dr. Wilner: Thanks for that, Dr. Merino. I think that’s very valuable for all of us. This has been a great discussion. Do you have any final comments before we wrap up?
Dr. Merino: I want to encourage people to continue reading medical journals all the time and submitting to us, again, good research and important questions with robust methodology. That’s what we’re looking for in Neurology and most serious medical journals.
Dr. Wilner is an associate professor of neurology at the University of Tennessee Health Science Center, Memphis. Dr. Merino is a professor in the department of neurology at Georgetown University Medical Center, Washington, DC. Dr. Wilner reported conflicts of interest with Accordant Health Services and Lulu Publishing. Dr. Merino reported no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
This transcript has been edited for clarity.
Andrew N. Wilner, MD: My guest today is Dr. Jose Merino, editor in chief of the Neurology family of journals and professor of neurology and co-vice chair of education at Georgetown University in Washington, DC.
Our program today is a follow-up of Dr. Merino’s presentation at the recent American Academy of Neurology meeting in Denver, Colorado. Along with two other panelists, Dr. Merino discussed the role of open-access publication and the dangers of predatory journals.
Jose G. Merino, MD, MPhil: Thank you for having me here. It’s a pleasure.
Open Access Defined
Dr. Wilner: I remember when publication in neurology was pretty straightforward. It was either the green journal or the blue journal, but things have certainly changed. I think one topic that is not clear to everyone is this concept of open access. Could you define that for us?
Dr. Merino: Sure. Open access is a mode of publication that fosters more open or accessible science. The idea of open access is that it combines two main elements. One is that the papers that are published become immediately available to anybody with an internet connection anywhere in the world without any restrictions.
The second important element from open access, which makes it different from other models we can talk about, is the fact that the authors retain the copyright of their work, but they give the journal and readers a license to use, reproduce, and modify the content.
This is different, for example, from instances where we have funder mandates. For example, NIH papers have to become available 6 months after publication, so they’re available to everybody but not immediately.
Dr. Wilner: I remember that when a journal article was published, say, in Neurology, if you didn’t have a subscription to Neurology, you went to the library that hopefully had a subscription.
If they didn’t have it, you would write to the author and say, “Hey, I heard you have this great paper because the abstract was out there. Could you send me a reprint?” Has that whole universe evaporated?
Dr. Merino: It depends on how the paper is published. For example, in Neurology, some of the research we publish is open access. Basically, if you have an internet connection, you can access the paper.
That’s the case for papers published in our wholly open-access journals in the Neurology family like Neurology Neuroimmunology & Neuroinflammation, Neurology Genetics, or Neurology Education.
For other papers that are published in Neurology, not under open access, there is a paywall. For some of them, the paywall comes down after a few months based on funder mandates and so on. As I was mentioning, the NIH-funded papers are available 6 months later.
In the first 6 months, you may have to go to your library, and if your library has a subscription, you can download it directly. [This is also true for] those that always stay behind the paywall, where you have to have a subscription or your library has to have a subscription.
Is Pay to Publish a Red Flag?
Dr. Wilner: I’m a professional writer. With any luck, when I write something, I get paid to write it. There’s been a long tradition in academic medicine that when you submit an article to, say, Neurology, you don’t get paid as an author for the publication. Your reward is the honor of it being published.
Neurology supports itself in various ways, including advertising and so on. That’s been the contract: free publication for work that merits it, and the journal survives on its own.
With open access, one of the things that’s happened is that — and I’ve published open access myself — is that I get a notification that I need to pay to have my article that I’ve slaved over published. Explain that, please.
Dr. Merino: This is the issue with open access. As I mentioned, the paper gets published. You’re giving the journal a license to publish it. You’re retaining the copyright of your work. That means that the journal cannot make money or support itself by just publishing open access because they belong to you.
Typically, open-access journals are not in print and don’t have much in terms of advertising. The contract is you’re giving me a license to publish it, but it’s your journal, so you’re paying a fee for the journal expenses to basically produce your paper. That’s what’s happening with open access.
That’s been recognized with many funders, for example, with NIH funding or many of the European funders, they’re including open-access fees as part of their funding for research. Now, of course, this doesn’t help if you’re not a funded researcher or if you’re a fellow who’s doing work and so on.
Typically, most journals will have waived fees or lower fees for these situations. The reason for the open-access fee is the fact that you’re retaining the copyright. You’re not giving it to the journal who can then use it to generate its revenue for supporting itself, the editorial staff, and so on.
Dr. Wilner: This idea of charging for publication has created a satellite business of what are called predatory journals. How does one know if the open-access journal that I’m submitting to is really just in the business of wanting my $300 or my $900 to get published? How do I know if that’s a reasonable place to publish?
Predatory Journals
Dr. Merino: That’s a big challenge that has come with this whole idea of open access and the fact that now, many journals are online only, so you’re no longer seeing a physical copy. That has given rise to the predatory journals.
The predatory journal, by definition, is a journal that claims to be open access. They’ll take your paper and publish it, but they don’t provide all the other services that you would typically expect from the fact that you’re paying an open-access fee. This includes getting appropriate peer review, production of the manuscript, and long-term curation and storage of the manuscript.
Many predatory journals will take your open-access fee, accept any paper that you submit, regardless of the quality, because they’re charging the fees for that. They don’t send it to real peer review, and then in a few months, the journal disappears so there’s no way for anybody to actually find your paper anymore.
There are certain checklists. Dr. David Moher at the University of Toronto has produced some work trying to help us identify predatory journals.
One thing I typically suggest to people who ask me this question is: Have you ever heard of this journal before? Does the journal have a track record? How far back does the story of the journal go? Is it supported by a publisher that you know? Do you know anybody who has published there? Is it something you can easily access?
If in doubt, always ask your friendly medical librarian. There used to be lists that were kept in terms of predatory journals that were being constantly updated, but those had to be shut down. As far as I understand, there were legal issues in terms of how things got on that list.
I think that overall, if you’ve heard of it, if it’s relevant, if it’s known in your field, and if your librarian knows it, it’s probably a good legitimate open-access journal. There are many very good legitimate open-access journals.
I mentioned the two that we have in our family, but all the other major journals have their own open-access journal within their family. There are some, like BMC or PLOS, that are completely open-access and legitimate journals.
Impact Factor
Dr. Wilner: What about impact factor? Many journals boast about their impact factor. I’m not sure how to interpret that number.
Dr. Merino: Impact factor is very interesting. The impact factor was developed by medical librarians to try to identify the journals they should be subscribing to. It’s a measure of the average citations to an average paper in the journal.
It doesn’t tell you about specific papers. It tells you, on average, how many of the papers in this journal get cited so many times. It’s calculated by the number of articles that were cited divided by the number of articles that were published. Journals that publish many papers, like Neurology, have a hard time bringing up their impact factor beyond a certain level.
Similarly, very small journals with one or two very highly cited papers have a very high impact factor. It’s being used as a measure, perhaps inappropriately, of how good or how reputable a journal is. We all say we don’t care about journal impact factors, but we all know our journal impact factor and we used to know it to three decimals. Now, they changed the system, and there’s only one decimal point, which makes more sense.
This is more important, for example, for authors when deciding where to submit papers. I know that in some countries, particularly in Europe, the impact factor of the journal where you publish has an impact on your promotion decisions.
I would say what’s even more important than the impact factor, is to say, “Well, is this the journal that fits the scope of my paper? Is this the journal that reaches the audience that I want to reach when I write my paper?”
There are some papers, for example, that are very influential. The impact factor just captures citations. There are some papers that are very influential that may not get cited very often. There may be papers that change clinical practice.
If you read a paper that tells you that you should be changing how you treat your patients with myasthenia based on this paper, that may not get cited. It’s a very clinically focused paper, but it’s probably more impactful than one that gets cited very much in some respect, or they make it to public policy decisions, and so on.
I think it’s important to look more at the audience and the journal scope when you submit your papers.
Dr. Wilner: One other technical question. The journals also say they’re indexed in PubMed or Google Scholar. If I want to publish my paper and I want it indexed where the right people are going to find it, where does it need to be indexed?
Dr. Merino: I grew up using Index Medicus, MedlinePlus, and the Library of Science. I still do. If I need to find something, I go to PubMed. Ideally, papers are listed in MedlinePlus or can be found in PubMed. They’re not the same thing, but you can find them through them.
That would be an important thing. Nowadays, a lot more people are using Google Scholar or Google just to identify papers. It may be a little bit less relevant, but it’s still a measure of the quality of the journal before they get indexed in some of these. For example, if you get listed in MedlinePlus, it has gone through certain quality checks by the index itself to see whether they would accept the journal or not. That’s something you want to check.
Typically, most of the large journals or the journals you and I know about are listed in more than one place, right? They’re listed in Scopus and Web of Science. They’re listed in MedlinePlus and so on. Again, if you’re submitting your paper, go somewhere where you know the journal and you’ve heard about it.
Dr. Wilner: I’m not going to ask you about artificial intelligence. We can do that another time. I want to ask something closer to me, which is this question of publish or perish.
There seems to be, in academics, more emphasis on the number of papers that one has published rather than their quality. How does a younger academician or one who really needs to publish cope with that?
Dr. Merino: Many people are writing up research that may not be relevant or that may not be high quality just because you need to have a long list of papers to get promoted, for example, if you’re an academician.
Doug Altman, who was a very influential person in the field quality of not only medical statistics but also medical publishing, had the idea that we need less research, but we need better research.
We often receive papers where you say, well, what’s the rationale behind the question in this paper? It’s like they had a large amount of data and were trying to squeeze as much as they could out of that. I think, as a young academician, the important thing to think about is whether it is an important question that matters to you and to the field, from whatever perspective, whether it’s going to advance research, advance clinical care, or have public policy implications.
Is this one where the answer will be important no matter what the answer is? If you’re thinking of that, your work will be well recognized, people will know you, and you’ll get invited to collaborate. I think that’s the most important thing rather than just churning out a large number of papers.
The productivity will come from the fact that you start by saying, let me ask something that’s really meaningful to me and to the field, with a good question and using strong research methodology.
Dr. Wilner: Thanks for that, Dr. Merino. I think that’s very valuable for all of us. This has been a great discussion. Do you have any final comments before we wrap up?
Dr. Merino: I want to encourage people to continue reading medical journals all the time and submitting to us, again, good research and important questions with robust methodology. That’s what we’re looking for in Neurology and most serious medical journals.
Dr. Wilner is an associate professor of neurology at the University of Tennessee Health Science Center, Memphis. Dr. Merino is a professor in the department of neurology at Georgetown University Medical Center, Washington, DC. Dr. Wilner reported conflicts of interest with Accordant Health Services and Lulu Publishing. Dr. Merino reported no relevant conflicts of interest.
A version of this article first appeared on Medscape.com.
Oncology Mergers Are on the Rise. How Can Independent Practices Survive?
When he completed his fellowship at Fox Chase Cancer Center in Philadelphia, Moshe Chasky, MD, joined a small five-person practice that rented space from the city’s Jefferson Hospital in Philadelphia. The arrangement seemed to work well for the hospital and the small practice, which remained independent.
Within 10 years, the hospital sought to buy the practice, Alliance Cancer Specialists.
But the oncologists at Alliance did not want to join Jefferson.
The hospital eventually entered into an exclusive agreement with its own medical group to provide inpatient oncology/hematology services at three Jefferson Health–Northeast hospitals and stripped Dr. Chasky and his colleagues of their privileges at those facilities, Medscape Medical News reported last year.
said Jeff Patton, MD, CEO of OneOncology, a management services organization.
A 2020 report from the Community Oncology Alliance (COA), for instance, tracked mergers, acquisitions, and closures in the community oncology setting and found the number of practices acquired by hospitals, known as vertical integration, nearly tripled from 2010 to 2020.
“Some hospitals are pretty predatory in their approach,” Dr. Patton said. If hospitals have their own oncology program, “they’ll employ the referring doctors and then discourage them or prevent them from referring patients to our independent practices that are not owned by the hospital.”
Still, in the face of growing pressure to join hospitals, some community oncology practices are finding ways to survive and maintain their independence.
A Growing Trend
The latest data continue to show a clear trend: Consolidation in oncology is on the rise.
A 2024 study revealed that the pace of consolidation seems to be increasing.
The analysis found that, between 2015 and 2022, the number of medical oncologists increased by 14% and the number of medical oncologists per practice increased by 40%, while the number of practices decreased by 18%.
While about 44% of practices remain independent, the percentage of medical oncologists working in practices with more than 25 clinicians has increased from 34% in 2015 to 44% in 2022. By 2022, the largest 102 practices in the United States employed more than 40% of all medical oncologists.
“The rate of consolidation seems to be rapid,” study coauthor Parsa Erfani, MD, an internal medicine resident at Brigham & Women’s Hospital, Boston, explained.
Consolidation appears to breed more consolidation. The researchers found, for instance, that markets with greater hospital consolidation and more hospital beds per capita were more likely to undergo consolidation in oncology.
Consolidation may be higher in these markets “because hospitals or health systems are buying up oncology practices or conversely because oncology practices are merging to compete more effectively with larger hospitals in the area,” Dr. Erfani told this news organization.
Mergers among independent practices, known as horizontal integration, have also been on the rise, according to the 2020 COA report. These mergers can help counter pressures from hospitals seeking to acquire community practices as well as prevent practices and their clinics from closing.
Although Dr. Erfani’s research wasn’t designed to determine the factors behind consolidation, he and his colleagues point to the Affordable Care Act (ACA) and the federal 340B Drug Pricing Program as potential drivers of this trend.
The ACA encouraged consolidation as a way to improve efficiency and created the need for ever-larger information systems to collect and report quality data. But these data collection and reporting requirements have become increasingly difficult for smaller practices to take on.
The 340B Program, however, may be a bigger contributing factor to consolidation. Created in 1992, the 340B Program allows qualifying hospitals and clinics that treat low-income and uninsured patients to buy outpatient prescription drugs at a 25%-50% discount.
Hospitals seeking to capitalize on the margins possible under the 340B Program will “buy all the referring physicians in a market so that the medical oncology group is left with little choice but to sell to the hospital,” said Dr. Patton.
“Those 340B dollars are worth a lot to hospitals,” said David A. Eagle, MD, a hematologist/oncologist with New York Cancer & Blood Specialists and past president of COA. The program “creates an appetite for nonprofit hospitals to want to grow their medical oncology programs,” he told this news organization.
Declining Medicare reimbursement has also hit independent practices hard.
Over the past 15 years, compared with inflation, physicians have gotten “a pay rate decrease from Medicare,” said Dr. Patton. Payers have followed that lead and tried to cut pay for clinicians, especially those who do not have market share, he said. Paying them less is “disingenuous knowing that our costs of providing care are going up,” he said.
Less Access, Higher Costs, Worse Care?
Many studies have demonstrated that, when hospitals become behemoths in a given market, healthcare costs go up.
“There are robust data showing that consolidation increases healthcare costs by reducing competition, including in oncology,” wrote Dr. Erfani and colleagues.
Oncology practices that are owned by hospitals bill facility fees for outpatient chemotherapy treatment, adding another layer of cost, the researchers explained, citing a 2019 Health Economics study.
Another analysis, published in 2020, found that hospital prices for the top 37 infused cancer drugs averaged 86% more per unit than the price charged by physician offices. Hospital outpatient departments charged even more, on average, for drugs — 128% more for nivolumab and 428% more for fluorouracil, for instance.
In their 2024 analysis, Dr. Erfani and colleagues also found that increased hospital market concentration was associated with worse quality of care, across all assessed patient satisfaction measures, and may result in worse access to care as well.
Overall, these consolidation “trends have important implications for cancer care cost, quality, and access,” the authors concluded.
Navigating the Consolidation Trend
In the face of mounting pressure to join hospitals, community oncology practices have typically relied on horizontal mergers to maintain their independence. An increasing number of practices, however, are now turning to another strategy: Management services organizations.
According to some oncologists, a core benefit of joining a management services organization is their community practices can maintain autonomy, hold on to referrals, and benefit from access to a wider network of peers and recently approved treatments such as chimeric antigen receptor T-cell therapies.
In these arrangements, the management company also provides business assistance to practices, including help with billing and collection, payer negotiations, supply chain issues, and credentialing, as well as recruiting, hiring, and marketing.
These management organizations, which include American Oncology Network, Integrated Oncology Network, OneOncology, and Verdi Oncology, are, however, backed by private equity. According to a 2022 report, private equity–backed management organizations have ramped up arrangements with community oncology practices over the past few years — a trend that has concerned some experts.
The authors of a recent analysis in JAMA Internal Medicine explained that, although private equity involvement in physician practices may enable operational efficiencies, “critics point to potential conflicts of interest” and highlight concerns that patients “may face additional barriers to both accessibility and affordability of care.”
The difference, according to some oncologists, is their practices are not owned by the management services organization; instead, the practices enter contracts that outline the boundaries of the relationship and stipulate fees to the management organizations.
In 2020, Dr. Chasky’s practice, Alliance Cancer Specialists, joined The US Oncology Network, a management services organization wholly owned by McKesson. The organization provides the practice with capital and other resources, as well as access to the Sarah Cannon Research Institute, so patients can participate in clinical trials.
“We totally function as an independent practice,” said Dr. Chasky. “We make our own management decisions,” he said. For instance, if Alliance wants to hire a new clinician, US Oncology helps with the recruitment. “But at the end of the day, it’s our practice,” he said.
Davey Daniel, MD — whose community practice joined the management services organization OneOncology — has seen the benefits of being part of a larger network. For instance, bispecific therapies for leukemias, lymphomas, and multiple myeloma are typically administered at academic centers because of the risk for cytokine release syndrome.
However, physician leaders in the OneOncology network “came up with a playbook on how to do it safely” in the community setting, said Dr. Daniel. “It meant that we were adopting FDA newly approved therapies in a very short course.”
Being able to draw from a wider pool of expertise has had other advantages. Dr. Daniel can lean on pathologists and research scientists in the network for advice on targeted therapy use. “We’re actually bringing precision medicine expertise to the community,” Dr. Daniel said.
Dr. Chasky and Dr. Eagle, whose practice is also part of OneOncology, said that continuing to work in the community setting has allowed them greater flexibility.
Dr. Eagle explained that New York Cancer & Blood Specialists tries to offer patients an appointment within 2 days of a referral, and it allows walk-in visits.
Dr. Chasky leans into the flexibility by having staff stay late, when needed, to ensure that all patients are seen. “We’re there for our patients at all hours,” Dr. Chasky said, adding that often “you don’t have that flexibility when you work for a big hospital system.”
The bottom line is community oncology can still thrive, said Nick Ferreyros, managing director of COA, “as long as we have a healthy competitive ecosystem where [we] are valued and seen as an important part of our cancer care system.”
A version of this article first appeared on Medscape.com.
When he completed his fellowship at Fox Chase Cancer Center in Philadelphia, Moshe Chasky, MD, joined a small five-person practice that rented space from the city’s Jefferson Hospital in Philadelphia. The arrangement seemed to work well for the hospital and the small practice, which remained independent.
Within 10 years, the hospital sought to buy the practice, Alliance Cancer Specialists.
But the oncologists at Alliance did not want to join Jefferson.
The hospital eventually entered into an exclusive agreement with its own medical group to provide inpatient oncology/hematology services at three Jefferson Health–Northeast hospitals and stripped Dr. Chasky and his colleagues of their privileges at those facilities, Medscape Medical News reported last year.
said Jeff Patton, MD, CEO of OneOncology, a management services organization.
A 2020 report from the Community Oncology Alliance (COA), for instance, tracked mergers, acquisitions, and closures in the community oncology setting and found the number of practices acquired by hospitals, known as vertical integration, nearly tripled from 2010 to 2020.
“Some hospitals are pretty predatory in their approach,” Dr. Patton said. If hospitals have their own oncology program, “they’ll employ the referring doctors and then discourage them or prevent them from referring patients to our independent practices that are not owned by the hospital.”
Still, in the face of growing pressure to join hospitals, some community oncology practices are finding ways to survive and maintain their independence.
A Growing Trend
The latest data continue to show a clear trend: Consolidation in oncology is on the rise.
A 2024 study revealed that the pace of consolidation seems to be increasing.
The analysis found that, between 2015 and 2022, the number of medical oncologists increased by 14% and the number of medical oncologists per practice increased by 40%, while the number of practices decreased by 18%.
While about 44% of practices remain independent, the percentage of medical oncologists working in practices with more than 25 clinicians has increased from 34% in 2015 to 44% in 2022. By 2022, the largest 102 practices in the United States employed more than 40% of all medical oncologists.
“The rate of consolidation seems to be rapid,” study coauthor Parsa Erfani, MD, an internal medicine resident at Brigham & Women’s Hospital, Boston, explained.
Consolidation appears to breed more consolidation. The researchers found, for instance, that markets with greater hospital consolidation and more hospital beds per capita were more likely to undergo consolidation in oncology.
Consolidation may be higher in these markets “because hospitals or health systems are buying up oncology practices or conversely because oncology practices are merging to compete more effectively with larger hospitals in the area,” Dr. Erfani told this news organization.
Mergers among independent practices, known as horizontal integration, have also been on the rise, according to the 2020 COA report. These mergers can help counter pressures from hospitals seeking to acquire community practices as well as prevent practices and their clinics from closing.
Although Dr. Erfani’s research wasn’t designed to determine the factors behind consolidation, he and his colleagues point to the Affordable Care Act (ACA) and the federal 340B Drug Pricing Program as potential drivers of this trend.
The ACA encouraged consolidation as a way to improve efficiency and created the need for ever-larger information systems to collect and report quality data. But these data collection and reporting requirements have become increasingly difficult for smaller practices to take on.
The 340B Program, however, may be a bigger contributing factor to consolidation. Created in 1992, the 340B Program allows qualifying hospitals and clinics that treat low-income and uninsured patients to buy outpatient prescription drugs at a 25%-50% discount.
Hospitals seeking to capitalize on the margins possible under the 340B Program will “buy all the referring physicians in a market so that the medical oncology group is left with little choice but to sell to the hospital,” said Dr. Patton.
“Those 340B dollars are worth a lot to hospitals,” said David A. Eagle, MD, a hematologist/oncologist with New York Cancer & Blood Specialists and past president of COA. The program “creates an appetite for nonprofit hospitals to want to grow their medical oncology programs,” he told this news organization.
Declining Medicare reimbursement has also hit independent practices hard.
Over the past 15 years, compared with inflation, physicians have gotten “a pay rate decrease from Medicare,” said Dr. Patton. Payers have followed that lead and tried to cut pay for clinicians, especially those who do not have market share, he said. Paying them less is “disingenuous knowing that our costs of providing care are going up,” he said.
Less Access, Higher Costs, Worse Care?
Many studies have demonstrated that, when hospitals become behemoths in a given market, healthcare costs go up.
“There are robust data showing that consolidation increases healthcare costs by reducing competition, including in oncology,” wrote Dr. Erfani and colleagues.
Oncology practices that are owned by hospitals bill facility fees for outpatient chemotherapy treatment, adding another layer of cost, the researchers explained, citing a 2019 Health Economics study.
Another analysis, published in 2020, found that hospital prices for the top 37 infused cancer drugs averaged 86% more per unit than the price charged by physician offices. Hospital outpatient departments charged even more, on average, for drugs — 128% more for nivolumab and 428% more for fluorouracil, for instance.
In their 2024 analysis, Dr. Erfani and colleagues also found that increased hospital market concentration was associated with worse quality of care, across all assessed patient satisfaction measures, and may result in worse access to care as well.
Overall, these consolidation “trends have important implications for cancer care cost, quality, and access,” the authors concluded.
Navigating the Consolidation Trend
In the face of mounting pressure to join hospitals, community oncology practices have typically relied on horizontal mergers to maintain their independence. An increasing number of practices, however, are now turning to another strategy: Management services organizations.
According to some oncologists, a core benefit of joining a management services organization is their community practices can maintain autonomy, hold on to referrals, and benefit from access to a wider network of peers and recently approved treatments such as chimeric antigen receptor T-cell therapies.
In these arrangements, the management company also provides business assistance to practices, including help with billing and collection, payer negotiations, supply chain issues, and credentialing, as well as recruiting, hiring, and marketing.
These management organizations, which include American Oncology Network, Integrated Oncology Network, OneOncology, and Verdi Oncology, are, however, backed by private equity. According to a 2022 report, private equity–backed management organizations have ramped up arrangements with community oncology practices over the past few years — a trend that has concerned some experts.
The authors of a recent analysis in JAMA Internal Medicine explained that, although private equity involvement in physician practices may enable operational efficiencies, “critics point to potential conflicts of interest” and highlight concerns that patients “may face additional barriers to both accessibility and affordability of care.”
The difference, according to some oncologists, is their practices are not owned by the management services organization; instead, the practices enter contracts that outline the boundaries of the relationship and stipulate fees to the management organizations.
In 2020, Dr. Chasky’s practice, Alliance Cancer Specialists, joined The US Oncology Network, a management services organization wholly owned by McKesson. The organization provides the practice with capital and other resources, as well as access to the Sarah Cannon Research Institute, so patients can participate in clinical trials.
“We totally function as an independent practice,” said Dr. Chasky. “We make our own management decisions,” he said. For instance, if Alliance wants to hire a new clinician, US Oncology helps with the recruitment. “But at the end of the day, it’s our practice,” he said.
Davey Daniel, MD — whose community practice joined the management services organization OneOncology — has seen the benefits of being part of a larger network. For instance, bispecific therapies for leukemias, lymphomas, and multiple myeloma are typically administered at academic centers because of the risk for cytokine release syndrome.
However, physician leaders in the OneOncology network “came up with a playbook on how to do it safely” in the community setting, said Dr. Daniel. “It meant that we were adopting FDA newly approved therapies in a very short course.”
Being able to draw from a wider pool of expertise has had other advantages. Dr. Daniel can lean on pathologists and research scientists in the network for advice on targeted therapy use. “We’re actually bringing precision medicine expertise to the community,” Dr. Daniel said.
Dr. Chasky and Dr. Eagle, whose practice is also part of OneOncology, said that continuing to work in the community setting has allowed them greater flexibility.
Dr. Eagle explained that New York Cancer & Blood Specialists tries to offer patients an appointment within 2 days of a referral, and it allows walk-in visits.
Dr. Chasky leans into the flexibility by having staff stay late, when needed, to ensure that all patients are seen. “We’re there for our patients at all hours,” Dr. Chasky said, adding that often “you don’t have that flexibility when you work for a big hospital system.”
The bottom line is community oncology can still thrive, said Nick Ferreyros, managing director of COA, “as long as we have a healthy competitive ecosystem where [we] are valued and seen as an important part of our cancer care system.”
A version of this article first appeared on Medscape.com.
When he completed his fellowship at Fox Chase Cancer Center in Philadelphia, Moshe Chasky, MD, joined a small five-person practice that rented space from the city’s Jefferson Hospital in Philadelphia. The arrangement seemed to work well for the hospital and the small practice, which remained independent.
Within 10 years, the hospital sought to buy the practice, Alliance Cancer Specialists.
But the oncologists at Alliance did not want to join Jefferson.
The hospital eventually entered into an exclusive agreement with its own medical group to provide inpatient oncology/hematology services at three Jefferson Health–Northeast hospitals and stripped Dr. Chasky and his colleagues of their privileges at those facilities, Medscape Medical News reported last year.
said Jeff Patton, MD, CEO of OneOncology, a management services organization.
A 2020 report from the Community Oncology Alliance (COA), for instance, tracked mergers, acquisitions, and closures in the community oncology setting and found the number of practices acquired by hospitals, known as vertical integration, nearly tripled from 2010 to 2020.
“Some hospitals are pretty predatory in their approach,” Dr. Patton said. If hospitals have their own oncology program, “they’ll employ the referring doctors and then discourage them or prevent them from referring patients to our independent practices that are not owned by the hospital.”
Still, in the face of growing pressure to join hospitals, some community oncology practices are finding ways to survive and maintain their independence.
A Growing Trend
The latest data continue to show a clear trend: Consolidation in oncology is on the rise.
A 2024 study revealed that the pace of consolidation seems to be increasing.
The analysis found that, between 2015 and 2022, the number of medical oncologists increased by 14% and the number of medical oncologists per practice increased by 40%, while the number of practices decreased by 18%.
While about 44% of practices remain independent, the percentage of medical oncologists working in practices with more than 25 clinicians has increased from 34% in 2015 to 44% in 2022. By 2022, the largest 102 practices in the United States employed more than 40% of all medical oncologists.
“The rate of consolidation seems to be rapid,” study coauthor Parsa Erfani, MD, an internal medicine resident at Brigham & Women’s Hospital, Boston, explained.
Consolidation appears to breed more consolidation. The researchers found, for instance, that markets with greater hospital consolidation and more hospital beds per capita were more likely to undergo consolidation in oncology.
Consolidation may be higher in these markets “because hospitals or health systems are buying up oncology practices or conversely because oncology practices are merging to compete more effectively with larger hospitals in the area,” Dr. Erfani told this news organization.
Mergers among independent practices, known as horizontal integration, have also been on the rise, according to the 2020 COA report. These mergers can help counter pressures from hospitals seeking to acquire community practices as well as prevent practices and their clinics from closing.
Although Dr. Erfani’s research wasn’t designed to determine the factors behind consolidation, he and his colleagues point to the Affordable Care Act (ACA) and the federal 340B Drug Pricing Program as potential drivers of this trend.
The ACA encouraged consolidation as a way to improve efficiency and created the need for ever-larger information systems to collect and report quality data. But these data collection and reporting requirements have become increasingly difficult for smaller practices to take on.
The 340B Program, however, may be a bigger contributing factor to consolidation. Created in 1992, the 340B Program allows qualifying hospitals and clinics that treat low-income and uninsured patients to buy outpatient prescription drugs at a 25%-50% discount.
Hospitals seeking to capitalize on the margins possible under the 340B Program will “buy all the referring physicians in a market so that the medical oncology group is left with little choice but to sell to the hospital,” said Dr. Patton.
“Those 340B dollars are worth a lot to hospitals,” said David A. Eagle, MD, a hematologist/oncologist with New York Cancer & Blood Specialists and past president of COA. The program “creates an appetite for nonprofit hospitals to want to grow their medical oncology programs,” he told this news organization.
Declining Medicare reimbursement has also hit independent practices hard.
Over the past 15 years, compared with inflation, physicians have gotten “a pay rate decrease from Medicare,” said Dr. Patton. Payers have followed that lead and tried to cut pay for clinicians, especially those who do not have market share, he said. Paying them less is “disingenuous knowing that our costs of providing care are going up,” he said.
Less Access, Higher Costs, Worse Care?
Many studies have demonstrated that, when hospitals become behemoths in a given market, healthcare costs go up.
“There are robust data showing that consolidation increases healthcare costs by reducing competition, including in oncology,” wrote Dr. Erfani and colleagues.
Oncology practices that are owned by hospitals bill facility fees for outpatient chemotherapy treatment, adding another layer of cost, the researchers explained, citing a 2019 Health Economics study.
Another analysis, published in 2020, found that hospital prices for the top 37 infused cancer drugs averaged 86% more per unit than the price charged by physician offices. Hospital outpatient departments charged even more, on average, for drugs — 128% more for nivolumab and 428% more for fluorouracil, for instance.
In their 2024 analysis, Dr. Erfani and colleagues also found that increased hospital market concentration was associated with worse quality of care, across all assessed patient satisfaction measures, and may result in worse access to care as well.
Overall, these consolidation “trends have important implications for cancer care cost, quality, and access,” the authors concluded.
Navigating the Consolidation Trend
In the face of mounting pressure to join hospitals, community oncology practices have typically relied on horizontal mergers to maintain their independence. An increasing number of practices, however, are now turning to another strategy: Management services organizations.
According to some oncologists, a core benefit of joining a management services organization is their community practices can maintain autonomy, hold on to referrals, and benefit from access to a wider network of peers and recently approved treatments such as chimeric antigen receptor T-cell therapies.
In these arrangements, the management company also provides business assistance to practices, including help with billing and collection, payer negotiations, supply chain issues, and credentialing, as well as recruiting, hiring, and marketing.
These management organizations, which include American Oncology Network, Integrated Oncology Network, OneOncology, and Verdi Oncology, are, however, backed by private equity. According to a 2022 report, private equity–backed management organizations have ramped up arrangements with community oncology practices over the past few years — a trend that has concerned some experts.
The authors of a recent analysis in JAMA Internal Medicine explained that, although private equity involvement in physician practices may enable operational efficiencies, “critics point to potential conflicts of interest” and highlight concerns that patients “may face additional barriers to both accessibility and affordability of care.”
The difference, according to some oncologists, is their practices are not owned by the management services organization; instead, the practices enter contracts that outline the boundaries of the relationship and stipulate fees to the management organizations.
In 2020, Dr. Chasky’s practice, Alliance Cancer Specialists, joined The US Oncology Network, a management services organization wholly owned by McKesson. The organization provides the practice with capital and other resources, as well as access to the Sarah Cannon Research Institute, so patients can participate in clinical trials.
“We totally function as an independent practice,” said Dr. Chasky. “We make our own management decisions,” he said. For instance, if Alliance wants to hire a new clinician, US Oncology helps with the recruitment. “But at the end of the day, it’s our practice,” he said.
Davey Daniel, MD — whose community practice joined the management services organization OneOncology — has seen the benefits of being part of a larger network. For instance, bispecific therapies for leukemias, lymphomas, and multiple myeloma are typically administered at academic centers because of the risk for cytokine release syndrome.
However, physician leaders in the OneOncology network “came up with a playbook on how to do it safely” in the community setting, said Dr. Daniel. “It meant that we were adopting FDA newly approved therapies in a very short course.”
Being able to draw from a wider pool of expertise has had other advantages. Dr. Daniel can lean on pathologists and research scientists in the network for advice on targeted therapy use. “We’re actually bringing precision medicine expertise to the community,” Dr. Daniel said.
Dr. Chasky and Dr. Eagle, whose practice is also part of OneOncology, said that continuing to work in the community setting has allowed them greater flexibility.
Dr. Eagle explained that New York Cancer & Blood Specialists tries to offer patients an appointment within 2 days of a referral, and it allows walk-in visits.
Dr. Chasky leans into the flexibility by having staff stay late, when needed, to ensure that all patients are seen. “We’re there for our patients at all hours,” Dr. Chasky said, adding that often “you don’t have that flexibility when you work for a big hospital system.”
The bottom line is community oncology can still thrive, said Nick Ferreyros, managing director of COA, “as long as we have a healthy competitive ecosystem where [we] are valued and seen as an important part of our cancer care system.”
A version of this article first appeared on Medscape.com.
FDA Approves Topical Anticholinergic for Axillary Hyperhidrosis
The .
According to a press release from Botanix Pharmaceuticals, which developed the product and will market it under the brand name Sofdra, approval was based on results from two phase 3 studies that enrolled 710 patients with primary axillary hyperhidrosis. In the trials, patients treated with sofpironium topical gel, 12.45%, experienced “clinically and statistically meaningful changes” from baseline in the Gravimetric Sweat Production and the Hyperhidrosis Disease Severity Measure–Axillary seven-item score, according to the company.
Botanix plans to enable qualified patients to gain early access to the product in the third quarter of 2024, with commercial sales expected in the fourth quarter of 2024.
A version of this article first appeared on Medscape.com.
The .
According to a press release from Botanix Pharmaceuticals, which developed the product and will market it under the brand name Sofdra, approval was based on results from two phase 3 studies that enrolled 710 patients with primary axillary hyperhidrosis. In the trials, patients treated with sofpironium topical gel, 12.45%, experienced “clinically and statistically meaningful changes” from baseline in the Gravimetric Sweat Production and the Hyperhidrosis Disease Severity Measure–Axillary seven-item score, according to the company.
Botanix plans to enable qualified patients to gain early access to the product in the third quarter of 2024, with commercial sales expected in the fourth quarter of 2024.
A version of this article first appeared on Medscape.com.
The .
According to a press release from Botanix Pharmaceuticals, which developed the product and will market it under the brand name Sofdra, approval was based on results from two phase 3 studies that enrolled 710 patients with primary axillary hyperhidrosis. In the trials, patients treated with sofpironium topical gel, 12.45%, experienced “clinically and statistically meaningful changes” from baseline in the Gravimetric Sweat Production and the Hyperhidrosis Disease Severity Measure–Axillary seven-item score, according to the company.
Botanix plans to enable qualified patients to gain early access to the product in the third quarter of 2024, with commercial sales expected in the fourth quarter of 2024.
A version of this article first appeared on Medscape.com.
Ixekizumab Met Phase 3 Trial Endpoint in Juvenile PsA, Enthesitis-Related Arthritis
VIENNA — Ixekizumab (Taltz), an interleukin-17A inhibitor that’s already approved for the treatment of psoriatic arthritis and axial spondyloarthritis in adults appears likely to be granted the same corresponding indications for children, based on initial results from an open-label, phase 3 trial that employed adalimumab as a reference.
With a safety profile comparable with that seen in adult patients, ixekizumab “met the prespecified criterion for success at 16 weeks,” reported Athimalaipet V. Ramanan, MD, PhD, of Bristol Royal Hospital for Children and Translational Health Sciences, Bristol, England.
In this multicenter, randomized, open-label trial called COSPIRIT-JIA, which is still ongoing, investigators enrolled 101 children with active juvenile PsA (JPsA) or enthesitis-related arthritis (ERA), which is akin to spondyloarthritis in adults.
The efficacy and safety data at 16 weeks were presented as a late-breaking abstract at the annual European Congress of Rheumatology. Dr. Ramanan said that the open-label extension to 104 weeks is underway and further follow-up out to 264 weeks is planned.
Nearly 90% Achieve ACR30
The trial had an adaptive design in which the first 40 patients without biologics experience were randomized to ixekizumab or adalimumab, stratified by JPsA or ERA diagnosis, and the following 61 patients with either no biologic experience or an inadequate response or intolerance to biologics all received ixekizumab. The drugs were dosed according to weight. Dr. Ramanan explained that a placebo-controlled trial was considered unethical because of the strong evidence of benefit from biologics for JPsA and ERA.
The trial easily met its predefined threshold for success, which required ≥ 80% probability, based on Bayesian analysis, that ≥ 50% of patients would have 30% improvement in American College of Rheumatology response criteria (ACR30) at week 16. ACR30 was achieved in 88.9% of those treated with ixekizumab overall vs 95.0% of those treated with adalimumab, but the trial was not designed as a head-to-head comparison. Rather, adalimumab served as a reference.
When compared for the distinct diseases, the ACR30 rates were also numerically lower for ixekizumab relative to adalimumab for both ERA (88.9% vs 93.8%) and JPsA (88.9% vs 100%), but all of the adalimumab patients were naive to biologics. In comparison, about 75% of patients receiving ixekizumab were biologic-therapy naive.
Response rates to ixekizumab overall were numerically higher for patients without previous biologic experience than for those with experience (90.0% vs 85.7%), and this was also the case for patients with ERA (92.5% vs 78.6%). However, in the JPsA group, biologic-experienced patients had higher numerical response rates to ixekizumab (100% vs 85.0%).
An ACR30 is not a clinical goal that satisfies most patients and clinicians, Dr. Ramanan conceded, but he noted that ACR50 was reached with ixekizumab by 81.5% with ERA and 74.1% with JPsA, and ACR70 was reached by 68.5% and 55.6%, respectively. The highest responses of ACR90 (27.8% and 33.3%) and ACR100 (14.8% and 25.9%) were lower but still substantial in the ERA and JPsA groups, respectively.
Through week 16, 58.0% of those treated with ixekizumab had an adverse event considered treatment-related. Nearly half were of mild severity, and the remainder were moderate. Only 3.7% were considered serious. No patient discontinued study treatment because of an adverse event.
In this study, the presence of at least three active peripheral joints was an inclusion criterion. The median age was about 13 years in the biologic-naive adalimumab and ixekizumab groups and 14 years in the ixekizumab biologic-experienced group. The youngest patient in the study was aged 5 years, and the oldest was aged 18 years. Although about 40% of patients were women in the two biologic-naive subgroups, it was 60% in the biologic-experienced group.
On average, patients in the biologic-naive group were entered about 1 year after diagnosis. In the experienced patients, the average duration of disease at entry was nearly 4 years. About 45% of patients remained on conventional synthetic disease-modifying antirheumatic drugs while receiving ixekizumab. The proportion was 35% in the adalimumab reference arm.
Ixekizumab Might Fulfill Need for More Options
There are several biologics that have received regulatory approval or are already widely used for the treatment of JPsA or ERA, but more options are needed, according to Dr. Ramanan and the chair of the abstract session in which these data were reported. According to Caroline Ospelt, MD, PhD, a researcher at the Center for Experimental Rheumatology, University Hospital Zurich, Switzerland, regulatory approval of ixekizumab will depend on sustained efficacy and safety in longer follow-up from the COSPIRIT-JIA trial, but this trial supports continued development.
Despite a novel mechanism of action, “the data so far suggest a level of efficacy similar to that of anti-TNF [anti-tumor necrosis factor] biologics,” said Dr. Ospelt, who, in addition to moderating the late-breaking session, served as Scientific Program Chair of EULAR 2024.
While Dr. Ospelt emphasized that she is a researcher involved in translational rheumatology studies and not a clinician, she said there was consensus within the program committee to select this abstract from other high-quality latebreaker submissions on the basis of its potential clinical significance.
Dr. Ramanan reported financial relationships with AbbVie, AstraZeneca, Novartis, Pfizer, Roche, SOBI, UCB, and Eli Lilly, which provided funding for this study. Dr. Ospelt reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
VIENNA — Ixekizumab (Taltz), an interleukin-17A inhibitor that’s already approved for the treatment of psoriatic arthritis and axial spondyloarthritis in adults appears likely to be granted the same corresponding indications for children, based on initial results from an open-label, phase 3 trial that employed adalimumab as a reference.
With a safety profile comparable with that seen in adult patients, ixekizumab “met the prespecified criterion for success at 16 weeks,” reported Athimalaipet V. Ramanan, MD, PhD, of Bristol Royal Hospital for Children and Translational Health Sciences, Bristol, England.
In this multicenter, randomized, open-label trial called COSPIRIT-JIA, which is still ongoing, investigators enrolled 101 children with active juvenile PsA (JPsA) or enthesitis-related arthritis (ERA), which is akin to spondyloarthritis in adults.
The efficacy and safety data at 16 weeks were presented as a late-breaking abstract at the annual European Congress of Rheumatology. Dr. Ramanan said that the open-label extension to 104 weeks is underway and further follow-up out to 264 weeks is planned.
Nearly 90% Achieve ACR30
The trial had an adaptive design in which the first 40 patients without biologics experience were randomized to ixekizumab or adalimumab, stratified by JPsA or ERA diagnosis, and the following 61 patients with either no biologic experience or an inadequate response or intolerance to biologics all received ixekizumab. The drugs were dosed according to weight. Dr. Ramanan explained that a placebo-controlled trial was considered unethical because of the strong evidence of benefit from biologics for JPsA and ERA.
The trial easily met its predefined threshold for success, which required ≥ 80% probability, based on Bayesian analysis, that ≥ 50% of patients would have 30% improvement in American College of Rheumatology response criteria (ACR30) at week 16. ACR30 was achieved in 88.9% of those treated with ixekizumab overall vs 95.0% of those treated with adalimumab, but the trial was not designed as a head-to-head comparison. Rather, adalimumab served as a reference.
When compared for the distinct diseases, the ACR30 rates were also numerically lower for ixekizumab relative to adalimumab for both ERA (88.9% vs 93.8%) and JPsA (88.9% vs 100%), but all of the adalimumab patients were naive to biologics. In comparison, about 75% of patients receiving ixekizumab were biologic-therapy naive.
Response rates to ixekizumab overall were numerically higher for patients without previous biologic experience than for those with experience (90.0% vs 85.7%), and this was also the case for patients with ERA (92.5% vs 78.6%). However, in the JPsA group, biologic-experienced patients had higher numerical response rates to ixekizumab (100% vs 85.0%).
An ACR30 is not a clinical goal that satisfies most patients and clinicians, Dr. Ramanan conceded, but he noted that ACR50 was reached with ixekizumab by 81.5% with ERA and 74.1% with JPsA, and ACR70 was reached by 68.5% and 55.6%, respectively. The highest responses of ACR90 (27.8% and 33.3%) and ACR100 (14.8% and 25.9%) were lower but still substantial in the ERA and JPsA groups, respectively.
Through week 16, 58.0% of those treated with ixekizumab had an adverse event considered treatment-related. Nearly half were of mild severity, and the remainder were moderate. Only 3.7% were considered serious. No patient discontinued study treatment because of an adverse event.
In this study, the presence of at least three active peripheral joints was an inclusion criterion. The median age was about 13 years in the biologic-naive adalimumab and ixekizumab groups and 14 years in the ixekizumab biologic-experienced group. The youngest patient in the study was aged 5 years, and the oldest was aged 18 years. Although about 40% of patients were women in the two biologic-naive subgroups, it was 60% in the biologic-experienced group.
On average, patients in the biologic-naive group were entered about 1 year after diagnosis. In the experienced patients, the average duration of disease at entry was nearly 4 years. About 45% of patients remained on conventional synthetic disease-modifying antirheumatic drugs while receiving ixekizumab. The proportion was 35% in the adalimumab reference arm.
Ixekizumab Might Fulfill Need for More Options
There are several biologics that have received regulatory approval or are already widely used for the treatment of JPsA or ERA, but more options are needed, according to Dr. Ramanan and the chair of the abstract session in which these data were reported. According to Caroline Ospelt, MD, PhD, a researcher at the Center for Experimental Rheumatology, University Hospital Zurich, Switzerland, regulatory approval of ixekizumab will depend on sustained efficacy and safety in longer follow-up from the COSPIRIT-JIA trial, but this trial supports continued development.
Despite a novel mechanism of action, “the data so far suggest a level of efficacy similar to that of anti-TNF [anti-tumor necrosis factor] biologics,” said Dr. Ospelt, who, in addition to moderating the late-breaking session, served as Scientific Program Chair of EULAR 2024.
While Dr. Ospelt emphasized that she is a researcher involved in translational rheumatology studies and not a clinician, she said there was consensus within the program committee to select this abstract from other high-quality latebreaker submissions on the basis of its potential clinical significance.
Dr. Ramanan reported financial relationships with AbbVie, AstraZeneca, Novartis, Pfizer, Roche, SOBI, UCB, and Eli Lilly, which provided funding for this study. Dr. Ospelt reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
VIENNA — Ixekizumab (Taltz), an interleukin-17A inhibitor that’s already approved for the treatment of psoriatic arthritis and axial spondyloarthritis in adults appears likely to be granted the same corresponding indications for children, based on initial results from an open-label, phase 3 trial that employed adalimumab as a reference.
With a safety profile comparable with that seen in adult patients, ixekizumab “met the prespecified criterion for success at 16 weeks,” reported Athimalaipet V. Ramanan, MD, PhD, of Bristol Royal Hospital for Children and Translational Health Sciences, Bristol, England.
In this multicenter, randomized, open-label trial called COSPIRIT-JIA, which is still ongoing, investigators enrolled 101 children with active juvenile PsA (JPsA) or enthesitis-related arthritis (ERA), which is akin to spondyloarthritis in adults.
The efficacy and safety data at 16 weeks were presented as a late-breaking abstract at the annual European Congress of Rheumatology. Dr. Ramanan said that the open-label extension to 104 weeks is underway and further follow-up out to 264 weeks is planned.
Nearly 90% Achieve ACR30
The trial had an adaptive design in which the first 40 patients without biologics experience were randomized to ixekizumab or adalimumab, stratified by JPsA or ERA diagnosis, and the following 61 patients with either no biologic experience or an inadequate response or intolerance to biologics all received ixekizumab. The drugs were dosed according to weight. Dr. Ramanan explained that a placebo-controlled trial was considered unethical because of the strong evidence of benefit from biologics for JPsA and ERA.
The trial easily met its predefined threshold for success, which required ≥ 80% probability, based on Bayesian analysis, that ≥ 50% of patients would have 30% improvement in American College of Rheumatology response criteria (ACR30) at week 16. ACR30 was achieved in 88.9% of those treated with ixekizumab overall vs 95.0% of those treated with adalimumab, but the trial was not designed as a head-to-head comparison. Rather, adalimumab served as a reference.
When compared for the distinct diseases, the ACR30 rates were also numerically lower for ixekizumab relative to adalimumab for both ERA (88.9% vs 93.8%) and JPsA (88.9% vs 100%), but all of the adalimumab patients were naive to biologics. In comparison, about 75% of patients receiving ixekizumab were biologic-therapy naive.
Response rates to ixekizumab overall were numerically higher for patients without previous biologic experience than for those with experience (90.0% vs 85.7%), and this was also the case for patients with ERA (92.5% vs 78.6%). However, in the JPsA group, biologic-experienced patients had higher numerical response rates to ixekizumab (100% vs 85.0%).
An ACR30 is not a clinical goal that satisfies most patients and clinicians, Dr. Ramanan conceded, but he noted that ACR50 was reached with ixekizumab by 81.5% with ERA and 74.1% with JPsA, and ACR70 was reached by 68.5% and 55.6%, respectively. The highest responses of ACR90 (27.8% and 33.3%) and ACR100 (14.8% and 25.9%) were lower but still substantial in the ERA and JPsA groups, respectively.
Through week 16, 58.0% of those treated with ixekizumab had an adverse event considered treatment-related. Nearly half were of mild severity, and the remainder were moderate. Only 3.7% were considered serious. No patient discontinued study treatment because of an adverse event.
In this study, the presence of at least three active peripheral joints was an inclusion criterion. The median age was about 13 years in the biologic-naive adalimumab and ixekizumab groups and 14 years in the ixekizumab biologic-experienced group. The youngest patient in the study was aged 5 years, and the oldest was aged 18 years. Although about 40% of patients were women in the two biologic-naive subgroups, it was 60% in the biologic-experienced group.
On average, patients in the biologic-naive group were entered about 1 year after diagnosis. In the experienced patients, the average duration of disease at entry was nearly 4 years. About 45% of patients remained on conventional synthetic disease-modifying antirheumatic drugs while receiving ixekizumab. The proportion was 35% in the adalimumab reference arm.
Ixekizumab Might Fulfill Need for More Options
There are several biologics that have received regulatory approval or are already widely used for the treatment of JPsA or ERA, but more options are needed, according to Dr. Ramanan and the chair of the abstract session in which these data were reported. According to Caroline Ospelt, MD, PhD, a researcher at the Center for Experimental Rheumatology, University Hospital Zurich, Switzerland, regulatory approval of ixekizumab will depend on sustained efficacy and safety in longer follow-up from the COSPIRIT-JIA trial, but this trial supports continued development.
Despite a novel mechanism of action, “the data so far suggest a level of efficacy similar to that of anti-TNF [anti-tumor necrosis factor] biologics,” said Dr. Ospelt, who, in addition to moderating the late-breaking session, served as Scientific Program Chair of EULAR 2024.
While Dr. Ospelt emphasized that she is a researcher involved in translational rheumatology studies and not a clinician, she said there was consensus within the program committee to select this abstract from other high-quality latebreaker submissions on the basis of its potential clinical significance.
Dr. Ramanan reported financial relationships with AbbVie, AstraZeneca, Novartis, Pfizer, Roche, SOBI, UCB, and Eli Lilly, which provided funding for this study. Dr. Ospelt reported no potential conflicts of interest.
A version of this article first appeared on Medscape.com.
FROM EULAR 2024
See the Medical World Through Neurodivergent Doctors’ Eyes
Some 15%-20% of the world’s population are neurodivergent, with conditions such as autism, dyslexia, Tourette syndrome, attention-deficit/hyperactivity disorder (ADHD), and others. With different strengths and challenges around learning, engaging socially, or completing certain tasks, neurodivergent people can face barriers in the workforce.
Meanwhile, studies suggest that neurodivergent people may be overrepresented in STEM fields such as medicine. The medical field may self-select for traits associated with neurodivergent conditions, researchers say, including a hyperfocus on intense interests, pattern recognition, increased curiosity and empathy, and thinking quickly under pressure.
But . They struggle with stigma, a culture of nondisclosure, and lack of accommodations, which can lead to burnout and poor mental health.
“The medical system and the mental health system are some of the spaces that are holding on tightly to some of the outdated understandings of things like autism and ADHD,” says Megan Anna Neff, PsyD, a psychologist with autism and ADHD based in Portland, Oregon.
Situations can get dire: A 2023 survey of more than 200 autistic doctors from several countries found that 77% had considered suicide and 24% had attempted it.
But here’s the crux of it: Many neurodivergent doctors believe their unique ways of thinking and outside-the-box creativity are skills and strengths that can benefit the field. And they say making medicine more inclusive — and better understanding how a neurodivergent physician’s brain works — would allow them to thrive.
Blending In and Breaking Down
The exact number of neurodivergent physicians in the workforce remains unknown. Existing studies are small and focus mainly on autism. But researchers believe the percentage could be higher than we think, because neurodiversity can be underidentified.
Although autism can sometimes be diagnosed as early as 18 months, it’s not uncommon to receive a diagnosis well into adulthood. “Like many late-identified autistic adults, I got my autism diagnosis in the context of autistic burnout,” says Melissa Houser, MD, a primary care physician who received a diagnosis in 2021. Dr. Houser, who uses the pronouns she/they, explains that her experience is common, “a consequence of chronically having your life’s demands exceed your capacity.”
Dr. Houser, who also has ADHD and dyslexia, among other neurodivergent conditions, says that before her diagnosis, she worked in a traditional practice setting. Eventually, she began to notice intense dysregulation and fatigue. “I began to have a lot more difficulties with communication and my motor planning and sequencing,” Dr. Houser says. “I was sleep-deprived, and my needs were not being met. I was in a situation where I had a complete lack of autonomy over my practice.”
Deep in burnout, Dr. Houser says she lost her ability to “mask,” a term used to describe how some neurodivergent people work to “blend in” with societal expectations. This led to further communication breakdowns with her supervisor. Finally, Dr. Houser saw a psychiatrist.
Shortly after her diagnosis, Dr. Houser quit her job and founded All Brains Belong, a nonprofit that provides neurodiversity-affirming medical care, education, and advocacy. Research has found that people with autism are at increased risk for physical health conditions, including immune conditions, gastrointestinal disorders, metabolic conditions, and increased mortality in hospital settings. Understanding these connections can “mean the difference between life and death” for neurodivergent patients, Dr. Houser says.
Yet, in a 2015 study that assessed providers’ ability to recognize autism, a high proportion were not aware that they had patients with autism spectrum disorder, and most reported lacking both the skills and the tools to care for them.
Different as a Doctor and a Patient
Bernadette Grosjean, MD, a retired associate professor of psychiatry at David Geffen School of Medicine at UCLA and a distinguished Fellow of the American Psychiatric Association, also found insight into lifelong experiences as both a doctor and a patient with her autism diagnosis, which came when she was 61.
“Looking back, I was a smart kid but kind of clumsy and different in other ways,” Dr. Grosjean says. According to a 2021 survey by Cambridge University, autistic individuals are significantly more likely to identify as LGBTQ+, and Dr. Grosjean, who is gay, says that not being fully accepted by family or friends played a role in her struggles with mental health issues.
Throughout her mental health treatment, Dr. Grosjean felt as though her providers “were expecting from me things that I didn’t know how to do or fix. I didn’t know how to be a ‘good’ patient,” she recalls.
As a psychiatrist, Dr. Grosjean started to notice that many of the women she treated for borderline personality disorder, which is categorized by unstable relationships and emotions, were autistic. “I then started asking lots of questions about myself — the fact that I’ve always been very sensitive or that I’ve been accused of being both hypersensitive and not having emotions, and I understood a lot.”
When Dr. Grosjean came across Autistic Doctors International, a group of over 800 autistic doctors worldwide, she says, “I found my tribe.” She now serves as the US lead for psychiatry for the group, which is focused on support, advocacy, research, and education around neurodiversity.
Psychiatric comorbidities can accompany neurodivergent conditions. But a growing body of research, including a 2022 study published in the European Archives of Psychiatry and Clinical Neuroscience, indicates that autism and ADHD are frequently misdiagnosed as depression or anxiety.
Dr. Neff was unaware of her conditions until one of her children was diagnosed with autism in 2021. She started to research it. “As I was learning about autism and girls, I was like, ‘Oh, my gosh, this is me,’ ” Dr. Neff recalls. Within a few weeks, she had her own diagnosis.
In hindsight, Dr. Neff has more clarity regarding her struggles in the traditional medical space. She had found it difficult to fit patients into short appointment windows and keep their notes concise. Although she loved hospital work, the environment had been overwhelming and led to burnout.
‘A Deficit-Based Lens’
Dr. Houser believes that too often, autism is viewed through a “deficit-based lens.” Stressors like sensory overload, changes in routine, or unexpected events can exacerbate behavioral challenges for neurodivergent people in the workplace. The DSM-5 criteria for autism, she points out, are largely based on autistic “stress behaviors.”
The result, Dr. Houser says, is that neurodivergent doctors are judged by their response to stressors that put them at a disadvantage rather than their capabilities under more positive circumstances. “The more dysregulated someone is,” she says, “the more likely they are to manifest those observable behaviors.”
Dr. Neff notes that medicine is a very “sensory overwhelming work environment.” Working in ob.gyn. and primary care clinics, she remembers often coming home with a headache and a low-grade fever. “I had no idea why, but I now realize it’s because I was so sensory sick.”
Fearing for her job, Dr. Neff intentionally waited until she was in private practice to disclose her neurodiversity. “I don’t think it would have been received well if I was in a hospital system,” she says. “There’s a lot of invalidation that can come when someone chooses to self-disclose, and their colleagues don’t have a framework in mind to understand.” In one instance, after revealing her diagnosis, she remembers a well-known researcher telling her she wasn’t autistic.
Dr. Grosjean has also had former colleagues invalidate her diagnosis, something she says “keeps people quiet.”
Understanding the Neurodivergent Brain
The general lack of education on how neurodivergent brains work, physicians with these conditions say, means they are not often recognized for how they can function with certain accommodations and how they could contribute in unique ways if their workplace challenges were reduced.
“What we know about autistic brains is that we are systems-thinking pattern matchers,” says Dr. Houser, who formed an interdisciplinary task force to explore medical conditions that are more common in autistic people. Through that comprehensive approach, she has worked to find best practices to treat the constellation of conditions that can arise among these patients. “My autistic brain allowed me to do that,” Dr. Houser says.
Catriona McVey, a medical student in the United Kingdom and creator of the blog Attention Deficit Doctor, points out that “ADHD brains are interest-driven; they can be very focused when you’re doing something enjoyable or new due to increased dopaminergic stimulation.” Ms. McVey speaks from personal experience. “I’ve hyperfocused before on an essay that interested me for over 10 hours,” she recalls, “so I imagine if I was interested in surgery, I could easily hyperfocus on a long operation.”
Empathy is another key part of medical practice. Contrary to stereotypes of neurodivergent people lacking empathy, current research suggests this isn’t true. A concept known as the “double empathy problem,” a term coined by British researcher Damian Milton in 2012, challenges the misconception that autistic people do not have empathy, explains Dr. Grosjean.
Mr. Milton theorized that there are two types of empathy: emotional, when you feel someone else’s pain, and cognitive, which involves critical thinking to understand someone’s emotions or thoughts. “Autistic people have, in general, a lot of emotional empathy,” Dr. Grosjean says, “but the cognitive empathy they don’t have as well.”
Dr. Neff has experienced this in her practice. “I will often feel what my clients are feeling as they’re feeling it,” she says, adding that she has always had an innate ability to analyze and connect with clients. She’s good at observing the interplay of health conditions, incorporating biology, psychology, and social conceptualizations of issues, with nuance. She feels that recognizing behavioral patterns or psychological triggers in her patients helps her see them holistically and provide better care. “That was a skill even before I realized I was autistic, but I always thought it was just intuitive to everyone,” she says.
Support Can Lead to Success
The Americans with Disabilities Act requires employers to provide reasonable accommodations to neurodivergent employees. However, getting those accommodations involves disclosure, which many physicians have reasons to avoid.
It also means more work. Requesting and putting adjustments in place can take a lot of time and energy to organize. Ms. McVey says they can be “long-winded, multistep tasks” that are not very compatible with ADHD. “Some doctors report that service pressures and funding are used as excuses to refuse adjustments,” she adds.
Ms. McVey lists several workplace accommodations that could be helpful, including flexible working hours, a quiet space to complete paperwork, dictation software, and extra time for medical students to complete written exams.
Neurodivergent physicians have also called for increased diversity of senior leadership and utilizing “cognitive apprenticeship models,” where employees explain their thought processes and receive timely feedback.
But far too often, there is little intervention until a doctor reaches a crisis point. “I look forward to the day when we don’t have to wait until people are profoundly depleted to discover how their brains work,” says Dr. Houser.
Beyond logistical and structural changes in the medical field, Dr. Grosjean speaks of the simple need to listen to colleagues with an open mind and believe them when they express their feelings and experiences. “Everyone has a role to play in challenging stigma, misconceptions, and stereotypes,” Ms. McVey agrees. Ask yourself the old question, she suggests: “If not me, then who? If not now, then when?”
A version of this article first appeared on Medscape.com.
Some 15%-20% of the world’s population are neurodivergent, with conditions such as autism, dyslexia, Tourette syndrome, attention-deficit/hyperactivity disorder (ADHD), and others. With different strengths and challenges around learning, engaging socially, or completing certain tasks, neurodivergent people can face barriers in the workforce.
Meanwhile, studies suggest that neurodivergent people may be overrepresented in STEM fields such as medicine. The medical field may self-select for traits associated with neurodivergent conditions, researchers say, including a hyperfocus on intense interests, pattern recognition, increased curiosity and empathy, and thinking quickly under pressure.
But . They struggle with stigma, a culture of nondisclosure, and lack of accommodations, which can lead to burnout and poor mental health.
“The medical system and the mental health system are some of the spaces that are holding on tightly to some of the outdated understandings of things like autism and ADHD,” says Megan Anna Neff, PsyD, a psychologist with autism and ADHD based in Portland, Oregon.
Situations can get dire: A 2023 survey of more than 200 autistic doctors from several countries found that 77% had considered suicide and 24% had attempted it.
But here’s the crux of it: Many neurodivergent doctors believe their unique ways of thinking and outside-the-box creativity are skills and strengths that can benefit the field. And they say making medicine more inclusive — and better understanding how a neurodivergent physician’s brain works — would allow them to thrive.
Blending In and Breaking Down
The exact number of neurodivergent physicians in the workforce remains unknown. Existing studies are small and focus mainly on autism. But researchers believe the percentage could be higher than we think, because neurodiversity can be underidentified.
Although autism can sometimes be diagnosed as early as 18 months, it’s not uncommon to receive a diagnosis well into adulthood. “Like many late-identified autistic adults, I got my autism diagnosis in the context of autistic burnout,” says Melissa Houser, MD, a primary care physician who received a diagnosis in 2021. Dr. Houser, who uses the pronouns she/they, explains that her experience is common, “a consequence of chronically having your life’s demands exceed your capacity.”
Dr. Houser, who also has ADHD and dyslexia, among other neurodivergent conditions, says that before her diagnosis, she worked in a traditional practice setting. Eventually, she began to notice intense dysregulation and fatigue. “I began to have a lot more difficulties with communication and my motor planning and sequencing,” Dr. Houser says. “I was sleep-deprived, and my needs were not being met. I was in a situation where I had a complete lack of autonomy over my practice.”
Deep in burnout, Dr. Houser says she lost her ability to “mask,” a term used to describe how some neurodivergent people work to “blend in” with societal expectations. This led to further communication breakdowns with her supervisor. Finally, Dr. Houser saw a psychiatrist.
Shortly after her diagnosis, Dr. Houser quit her job and founded All Brains Belong, a nonprofit that provides neurodiversity-affirming medical care, education, and advocacy. Research has found that people with autism are at increased risk for physical health conditions, including immune conditions, gastrointestinal disorders, metabolic conditions, and increased mortality in hospital settings. Understanding these connections can “mean the difference between life and death” for neurodivergent patients, Dr. Houser says.
Yet, in a 2015 study that assessed providers’ ability to recognize autism, a high proportion were not aware that they had patients with autism spectrum disorder, and most reported lacking both the skills and the tools to care for them.
Different as a Doctor and a Patient
Bernadette Grosjean, MD, a retired associate professor of psychiatry at David Geffen School of Medicine at UCLA and a distinguished Fellow of the American Psychiatric Association, also found insight into lifelong experiences as both a doctor and a patient with her autism diagnosis, which came when she was 61.
“Looking back, I was a smart kid but kind of clumsy and different in other ways,” Dr. Grosjean says. According to a 2021 survey by Cambridge University, autistic individuals are significantly more likely to identify as LGBTQ+, and Dr. Grosjean, who is gay, says that not being fully accepted by family or friends played a role in her struggles with mental health issues.
Throughout her mental health treatment, Dr. Grosjean felt as though her providers “were expecting from me things that I didn’t know how to do or fix. I didn’t know how to be a ‘good’ patient,” she recalls.
As a psychiatrist, Dr. Grosjean started to notice that many of the women she treated for borderline personality disorder, which is categorized by unstable relationships and emotions, were autistic. “I then started asking lots of questions about myself — the fact that I’ve always been very sensitive or that I’ve been accused of being both hypersensitive and not having emotions, and I understood a lot.”
When Dr. Grosjean came across Autistic Doctors International, a group of over 800 autistic doctors worldwide, she says, “I found my tribe.” She now serves as the US lead for psychiatry for the group, which is focused on support, advocacy, research, and education around neurodiversity.
Psychiatric comorbidities can accompany neurodivergent conditions. But a growing body of research, including a 2022 study published in the European Archives of Psychiatry and Clinical Neuroscience, indicates that autism and ADHD are frequently misdiagnosed as depression or anxiety.
Dr. Neff was unaware of her conditions until one of her children was diagnosed with autism in 2021. She started to research it. “As I was learning about autism and girls, I was like, ‘Oh, my gosh, this is me,’ ” Dr. Neff recalls. Within a few weeks, she had her own diagnosis.
In hindsight, Dr. Neff has more clarity regarding her struggles in the traditional medical space. She had found it difficult to fit patients into short appointment windows and keep their notes concise. Although she loved hospital work, the environment had been overwhelming and led to burnout.
‘A Deficit-Based Lens’
Dr. Houser believes that too often, autism is viewed through a “deficit-based lens.” Stressors like sensory overload, changes in routine, or unexpected events can exacerbate behavioral challenges for neurodivergent people in the workplace. The DSM-5 criteria for autism, she points out, are largely based on autistic “stress behaviors.”
The result, Dr. Houser says, is that neurodivergent doctors are judged by their response to stressors that put them at a disadvantage rather than their capabilities under more positive circumstances. “The more dysregulated someone is,” she says, “the more likely they are to manifest those observable behaviors.”
Dr. Neff notes that medicine is a very “sensory overwhelming work environment.” Working in ob.gyn. and primary care clinics, she remembers often coming home with a headache and a low-grade fever. “I had no idea why, but I now realize it’s because I was so sensory sick.”
Fearing for her job, Dr. Neff intentionally waited until she was in private practice to disclose her neurodiversity. “I don’t think it would have been received well if I was in a hospital system,” she says. “There’s a lot of invalidation that can come when someone chooses to self-disclose, and their colleagues don’t have a framework in mind to understand.” In one instance, after revealing her diagnosis, she remembers a well-known researcher telling her she wasn’t autistic.
Dr. Grosjean has also had former colleagues invalidate her diagnosis, something she says “keeps people quiet.”
Understanding the Neurodivergent Brain
The general lack of education on how neurodivergent brains work, physicians with these conditions say, means they are not often recognized for how they can function with certain accommodations and how they could contribute in unique ways if their workplace challenges were reduced.
“What we know about autistic brains is that we are systems-thinking pattern matchers,” says Dr. Houser, who formed an interdisciplinary task force to explore medical conditions that are more common in autistic people. Through that comprehensive approach, she has worked to find best practices to treat the constellation of conditions that can arise among these patients. “My autistic brain allowed me to do that,” Dr. Houser says.
Catriona McVey, a medical student in the United Kingdom and creator of the blog Attention Deficit Doctor, points out that “ADHD brains are interest-driven; they can be very focused when you’re doing something enjoyable or new due to increased dopaminergic stimulation.” Ms. McVey speaks from personal experience. “I’ve hyperfocused before on an essay that interested me for over 10 hours,” she recalls, “so I imagine if I was interested in surgery, I could easily hyperfocus on a long operation.”
Empathy is another key part of medical practice. Contrary to stereotypes of neurodivergent people lacking empathy, current research suggests this isn’t true. A concept known as the “double empathy problem,” a term coined by British researcher Damian Milton in 2012, challenges the misconception that autistic people do not have empathy, explains Dr. Grosjean.
Mr. Milton theorized that there are two types of empathy: emotional, when you feel someone else’s pain, and cognitive, which involves critical thinking to understand someone’s emotions or thoughts. “Autistic people have, in general, a lot of emotional empathy,” Dr. Grosjean says, “but the cognitive empathy they don’t have as well.”
Dr. Neff has experienced this in her practice. “I will often feel what my clients are feeling as they’re feeling it,” she says, adding that she has always had an innate ability to analyze and connect with clients. She’s good at observing the interplay of health conditions, incorporating biology, psychology, and social conceptualizations of issues, with nuance. She feels that recognizing behavioral patterns or psychological triggers in her patients helps her see them holistically and provide better care. “That was a skill even before I realized I was autistic, but I always thought it was just intuitive to everyone,” she says.
Support Can Lead to Success
The Americans with Disabilities Act requires employers to provide reasonable accommodations to neurodivergent employees. However, getting those accommodations involves disclosure, which many physicians have reasons to avoid.
It also means more work. Requesting and putting adjustments in place can take a lot of time and energy to organize. Ms. McVey says they can be “long-winded, multistep tasks” that are not very compatible with ADHD. “Some doctors report that service pressures and funding are used as excuses to refuse adjustments,” she adds.
Ms. McVey lists several workplace accommodations that could be helpful, including flexible working hours, a quiet space to complete paperwork, dictation software, and extra time for medical students to complete written exams.
Neurodivergent physicians have also called for increased diversity of senior leadership and utilizing “cognitive apprenticeship models,” where employees explain their thought processes and receive timely feedback.
But far too often, there is little intervention until a doctor reaches a crisis point. “I look forward to the day when we don’t have to wait until people are profoundly depleted to discover how their brains work,” says Dr. Houser.
Beyond logistical and structural changes in the medical field, Dr. Grosjean speaks of the simple need to listen to colleagues with an open mind and believe them when they express their feelings and experiences. “Everyone has a role to play in challenging stigma, misconceptions, and stereotypes,” Ms. McVey agrees. Ask yourself the old question, she suggests: “If not me, then who? If not now, then when?”
A version of this article first appeared on Medscape.com.
Some 15%-20% of the world’s population are neurodivergent, with conditions such as autism, dyslexia, Tourette syndrome, attention-deficit/hyperactivity disorder (ADHD), and others. With different strengths and challenges around learning, engaging socially, or completing certain tasks, neurodivergent people can face barriers in the workforce.
Meanwhile, studies suggest that neurodivergent people may be overrepresented in STEM fields such as medicine. The medical field may self-select for traits associated with neurodivergent conditions, researchers say, including a hyperfocus on intense interests, pattern recognition, increased curiosity and empathy, and thinking quickly under pressure.
But . They struggle with stigma, a culture of nondisclosure, and lack of accommodations, which can lead to burnout and poor mental health.
“The medical system and the mental health system are some of the spaces that are holding on tightly to some of the outdated understandings of things like autism and ADHD,” says Megan Anna Neff, PsyD, a psychologist with autism and ADHD based in Portland, Oregon.
Situations can get dire: A 2023 survey of more than 200 autistic doctors from several countries found that 77% had considered suicide and 24% had attempted it.
But here’s the crux of it: Many neurodivergent doctors believe their unique ways of thinking and outside-the-box creativity are skills and strengths that can benefit the field. And they say making medicine more inclusive — and better understanding how a neurodivergent physician’s brain works — would allow them to thrive.
Blending In and Breaking Down
The exact number of neurodivergent physicians in the workforce remains unknown. Existing studies are small and focus mainly on autism. But researchers believe the percentage could be higher than we think, because neurodiversity can be underidentified.
Although autism can sometimes be diagnosed as early as 18 months, it’s not uncommon to receive a diagnosis well into adulthood. “Like many late-identified autistic adults, I got my autism diagnosis in the context of autistic burnout,” says Melissa Houser, MD, a primary care physician who received a diagnosis in 2021. Dr. Houser, who uses the pronouns she/they, explains that her experience is common, “a consequence of chronically having your life’s demands exceed your capacity.”
Dr. Houser, who also has ADHD and dyslexia, among other neurodivergent conditions, says that before her diagnosis, she worked in a traditional practice setting. Eventually, she began to notice intense dysregulation and fatigue. “I began to have a lot more difficulties with communication and my motor planning and sequencing,” Dr. Houser says. “I was sleep-deprived, and my needs were not being met. I was in a situation where I had a complete lack of autonomy over my practice.”
Deep in burnout, Dr. Houser says she lost her ability to “mask,” a term used to describe how some neurodivergent people work to “blend in” with societal expectations. This led to further communication breakdowns with her supervisor. Finally, Dr. Houser saw a psychiatrist.
Shortly after her diagnosis, Dr. Houser quit her job and founded All Brains Belong, a nonprofit that provides neurodiversity-affirming medical care, education, and advocacy. Research has found that people with autism are at increased risk for physical health conditions, including immune conditions, gastrointestinal disorders, metabolic conditions, and increased mortality in hospital settings. Understanding these connections can “mean the difference between life and death” for neurodivergent patients, Dr. Houser says.
Yet, in a 2015 study that assessed providers’ ability to recognize autism, a high proportion were not aware that they had patients with autism spectrum disorder, and most reported lacking both the skills and the tools to care for them.
Different as a Doctor and a Patient
Bernadette Grosjean, MD, a retired associate professor of psychiatry at David Geffen School of Medicine at UCLA and a distinguished Fellow of the American Psychiatric Association, also found insight into lifelong experiences as both a doctor and a patient with her autism diagnosis, which came when she was 61.
“Looking back, I was a smart kid but kind of clumsy and different in other ways,” Dr. Grosjean says. According to a 2021 survey by Cambridge University, autistic individuals are significantly more likely to identify as LGBTQ+, and Dr. Grosjean, who is gay, says that not being fully accepted by family or friends played a role in her struggles with mental health issues.
Throughout her mental health treatment, Dr. Grosjean felt as though her providers “were expecting from me things that I didn’t know how to do or fix. I didn’t know how to be a ‘good’ patient,” she recalls.
As a psychiatrist, Dr. Grosjean started to notice that many of the women she treated for borderline personality disorder, which is categorized by unstable relationships and emotions, were autistic. “I then started asking lots of questions about myself — the fact that I’ve always been very sensitive or that I’ve been accused of being both hypersensitive and not having emotions, and I understood a lot.”
When Dr. Grosjean came across Autistic Doctors International, a group of over 800 autistic doctors worldwide, she says, “I found my tribe.” She now serves as the US lead for psychiatry for the group, which is focused on support, advocacy, research, and education around neurodiversity.
Psychiatric comorbidities can accompany neurodivergent conditions. But a growing body of research, including a 2022 study published in the European Archives of Psychiatry and Clinical Neuroscience, indicates that autism and ADHD are frequently misdiagnosed as depression or anxiety.
Dr. Neff was unaware of her conditions until one of her children was diagnosed with autism in 2021. She started to research it. “As I was learning about autism and girls, I was like, ‘Oh, my gosh, this is me,’ ” Dr. Neff recalls. Within a few weeks, she had her own diagnosis.
In hindsight, Dr. Neff has more clarity regarding her struggles in the traditional medical space. She had found it difficult to fit patients into short appointment windows and keep their notes concise. Although she loved hospital work, the environment had been overwhelming and led to burnout.
‘A Deficit-Based Lens’
Dr. Houser believes that too often, autism is viewed through a “deficit-based lens.” Stressors like sensory overload, changes in routine, or unexpected events can exacerbate behavioral challenges for neurodivergent people in the workplace. The DSM-5 criteria for autism, she points out, are largely based on autistic “stress behaviors.”
The result, Dr. Houser says, is that neurodivergent doctors are judged by their response to stressors that put them at a disadvantage rather than their capabilities under more positive circumstances. “The more dysregulated someone is,” she says, “the more likely they are to manifest those observable behaviors.”
Dr. Neff notes that medicine is a very “sensory overwhelming work environment.” Working in ob.gyn. and primary care clinics, she remembers often coming home with a headache and a low-grade fever. “I had no idea why, but I now realize it’s because I was so sensory sick.”
Fearing for her job, Dr. Neff intentionally waited until she was in private practice to disclose her neurodiversity. “I don’t think it would have been received well if I was in a hospital system,” she says. “There’s a lot of invalidation that can come when someone chooses to self-disclose, and their colleagues don’t have a framework in mind to understand.” In one instance, after revealing her diagnosis, she remembers a well-known researcher telling her she wasn’t autistic.
Dr. Grosjean has also had former colleagues invalidate her diagnosis, something she says “keeps people quiet.”
Understanding the Neurodivergent Brain
The general lack of education on how neurodivergent brains work, physicians with these conditions say, means they are not often recognized for how they can function with certain accommodations and how they could contribute in unique ways if their workplace challenges were reduced.
“What we know about autistic brains is that we are systems-thinking pattern matchers,” says Dr. Houser, who formed an interdisciplinary task force to explore medical conditions that are more common in autistic people. Through that comprehensive approach, she has worked to find best practices to treat the constellation of conditions that can arise among these patients. “My autistic brain allowed me to do that,” Dr. Houser says.
Catriona McVey, a medical student in the United Kingdom and creator of the blog Attention Deficit Doctor, points out that “ADHD brains are interest-driven; they can be very focused when you’re doing something enjoyable or new due to increased dopaminergic stimulation.” Ms. McVey speaks from personal experience. “I’ve hyperfocused before on an essay that interested me for over 10 hours,” she recalls, “so I imagine if I was interested in surgery, I could easily hyperfocus on a long operation.”
Empathy is another key part of medical practice. Contrary to stereotypes of neurodivergent people lacking empathy, current research suggests this isn’t true. A concept known as the “double empathy problem,” a term coined by British researcher Damian Milton in 2012, challenges the misconception that autistic people do not have empathy, explains Dr. Grosjean.
Mr. Milton theorized that there are two types of empathy: emotional, when you feel someone else’s pain, and cognitive, which involves critical thinking to understand someone’s emotions or thoughts. “Autistic people have, in general, a lot of emotional empathy,” Dr. Grosjean says, “but the cognitive empathy they don’t have as well.”
Dr. Neff has experienced this in her practice. “I will often feel what my clients are feeling as they’re feeling it,” she says, adding that she has always had an innate ability to analyze and connect with clients. She’s good at observing the interplay of health conditions, incorporating biology, psychology, and social conceptualizations of issues, with nuance. She feels that recognizing behavioral patterns or psychological triggers in her patients helps her see them holistically and provide better care. “That was a skill even before I realized I was autistic, but I always thought it was just intuitive to everyone,” she says.
Support Can Lead to Success
The Americans with Disabilities Act requires employers to provide reasonable accommodations to neurodivergent employees. However, getting those accommodations involves disclosure, which many physicians have reasons to avoid.
It also means more work. Requesting and putting adjustments in place can take a lot of time and energy to organize. Ms. McVey says they can be “long-winded, multistep tasks” that are not very compatible with ADHD. “Some doctors report that service pressures and funding are used as excuses to refuse adjustments,” she adds.
Ms. McVey lists several workplace accommodations that could be helpful, including flexible working hours, a quiet space to complete paperwork, dictation software, and extra time for medical students to complete written exams.
Neurodivergent physicians have also called for increased diversity of senior leadership and utilizing “cognitive apprenticeship models,” where employees explain their thought processes and receive timely feedback.
But far too often, there is little intervention until a doctor reaches a crisis point. “I look forward to the day when we don’t have to wait until people are profoundly depleted to discover how their brains work,” says Dr. Houser.
Beyond logistical and structural changes in the medical field, Dr. Grosjean speaks of the simple need to listen to colleagues with an open mind and believe them when they express their feelings and experiences. “Everyone has a role to play in challenging stigma, misconceptions, and stereotypes,” Ms. McVey agrees. Ask yourself the old question, she suggests: “If not me, then who? If not now, then when?”
A version of this article first appeared on Medscape.com.
Atopic Dermatitis: Study Compares Prevalence by Gender, Age, and Ethnic Background
than adults from other ethnic backgrounds.
Those are among the key findings from an analysis of nationally representative cross-sectional data that were presented during a late-breaking abstract session at the Revolutionizing Atopic Dermatitis conference in Chicago.
“In the past few years, there has been a much-needed focus on better understanding disparities in atopic dermatitis,” one of the study authors, Raj Chovatiya, MD, PhD, clinical associate professor at Chicago Medical School, Rosalind Franklin University, North Chicago, told this news organization after the conference.
“Epidemiology is one of the key ways in which we can query differences in AD at a population level.”
Drawing from the 2021 National Health Interview Survey, the researchers identified 3103 respondents who reported being diagnosed with AD or eczema. They estimated the prevalence rates of AD for the overall population and each subgroup by dividing US frequency estimates by their corresponding US population totals and used multivariable logistic regression to assess the odds of having AD.
More than half of the respondents (1643) were aged between 18 and 64 years, 522 were aged 65 years and older, and 922 were children younger than 18 years. Overall, the prevalence of AD was 7.6% in adults aged 18-64 years and 6.1% in adults aged 65 years and older, for a weighted US estimate of 15.3 and 3.2 million, respectively. The prevalence of AD varied by race/ethnicity and was highest for those from “other single and multiple races” group (12.4%), followed by Black/African American (8.5%), White (7.7%), Asian (6.5%), American Indian/Alaskan Native (4.9%), and Hispanic (4.8%) populations.
In children, race/ethnicity prevalence were highest for those from other single and multiple races (15.2.%), followed by Black/African American (14.2%), American Indian/Alaskan Native (12%), White (10.2%), Hispanic (9.5%), and Asian (9%) populations.
When the researchers combined all age groups, they observed higher prevalence rates of AD among females than among males. However, in an analysis limited to children, the prevalence rates were similar between girls and boys (10.8% vs 10.7%, respectively), for a weighted US estimate of 7.8 million children with AD.
On multiple regression, the odds of having AD were greater among women than among men (odds ratio [OR], 1.4), among adults aged 18-64 years than among those aged 65 years and older (OR, 1.4), among those younger than 18 years than among those aged 65 years and older (OR, 2.0), and among Black/African American individuals than among White individuals (OR, 1.2). Hispanic adults had a lower risk for AD than non-Hispanic White adults (OR, 0.69) as did Asian adults than White adults (OR, 0.82).
“We found AD prevalence rates were higher in children and adult females, Hispanic adults had a lower prevalence of AD than all other adult groups, and there were numerical differences in AD prevalence across racial groups,” Dr. Chovatiya said in the interview. “While there are of course limitations to the use of any nationally representative cross-sectional dataset that requires weighting to project results from a smaller sample to reflect a larger more heterogeneous group, these results are important for us to consider targeted strategies to address AD burden.”
Jonathan I. Silverberg, MD, PhD, professor of dermatology at The George Washington University, Washington, who was asked to comment on the study, said that while the prevalence of AD in children has been well documented in prior research, “this study fills an important gap by showing us that the prevalence does remain high in adults.”
In addition, “it has not shown any evidence of AD decreasing over time; if anything, it might be slightly increasing,” he said. “We’re also seeing differences [in AD] by race and ethnicity. We have seen that demonstrated in children but [has been] less clearly demonstrated in adults.”
Eli Lilly and Company funded the analysis. Dr. Chovatiya and Dr. Silverberg disclosed ties to several pharmaceutical companies, including Eli Lilly.
A version of this article appeared on Medscape.com .
than adults from other ethnic backgrounds.
Those are among the key findings from an analysis of nationally representative cross-sectional data that were presented during a late-breaking abstract session at the Revolutionizing Atopic Dermatitis conference in Chicago.
“In the past few years, there has been a much-needed focus on better understanding disparities in atopic dermatitis,” one of the study authors, Raj Chovatiya, MD, PhD, clinical associate professor at Chicago Medical School, Rosalind Franklin University, North Chicago, told this news organization after the conference.
“Epidemiology is one of the key ways in which we can query differences in AD at a population level.”
Drawing from the 2021 National Health Interview Survey, the researchers identified 3103 respondents who reported being diagnosed with AD or eczema. They estimated the prevalence rates of AD for the overall population and each subgroup by dividing US frequency estimates by their corresponding US population totals and used multivariable logistic regression to assess the odds of having AD.
More than half of the respondents (1643) were aged between 18 and 64 years, 522 were aged 65 years and older, and 922 were children younger than 18 years. Overall, the prevalence of AD was 7.6% in adults aged 18-64 years and 6.1% in adults aged 65 years and older, for a weighted US estimate of 15.3 and 3.2 million, respectively. The prevalence of AD varied by race/ethnicity and was highest for those from “other single and multiple races” group (12.4%), followed by Black/African American (8.5%), White (7.7%), Asian (6.5%), American Indian/Alaskan Native (4.9%), and Hispanic (4.8%) populations.
In children, race/ethnicity prevalence were highest for those from other single and multiple races (15.2.%), followed by Black/African American (14.2%), American Indian/Alaskan Native (12%), White (10.2%), Hispanic (9.5%), and Asian (9%) populations.
When the researchers combined all age groups, they observed higher prevalence rates of AD among females than among males. However, in an analysis limited to children, the prevalence rates were similar between girls and boys (10.8% vs 10.7%, respectively), for a weighted US estimate of 7.8 million children with AD.
On multiple regression, the odds of having AD were greater among women than among men (odds ratio [OR], 1.4), among adults aged 18-64 years than among those aged 65 years and older (OR, 1.4), among those younger than 18 years than among those aged 65 years and older (OR, 2.0), and among Black/African American individuals than among White individuals (OR, 1.2). Hispanic adults had a lower risk for AD than non-Hispanic White adults (OR, 0.69) as did Asian adults than White adults (OR, 0.82).
“We found AD prevalence rates were higher in children and adult females, Hispanic adults had a lower prevalence of AD than all other adult groups, and there were numerical differences in AD prevalence across racial groups,” Dr. Chovatiya said in the interview. “While there are of course limitations to the use of any nationally representative cross-sectional dataset that requires weighting to project results from a smaller sample to reflect a larger more heterogeneous group, these results are important for us to consider targeted strategies to address AD burden.”
Jonathan I. Silverberg, MD, PhD, professor of dermatology at The George Washington University, Washington, who was asked to comment on the study, said that while the prevalence of AD in children has been well documented in prior research, “this study fills an important gap by showing us that the prevalence does remain high in adults.”
In addition, “it has not shown any evidence of AD decreasing over time; if anything, it might be slightly increasing,” he said. “We’re also seeing differences [in AD] by race and ethnicity. We have seen that demonstrated in children but [has been] less clearly demonstrated in adults.”
Eli Lilly and Company funded the analysis. Dr. Chovatiya and Dr. Silverberg disclosed ties to several pharmaceutical companies, including Eli Lilly.
A version of this article appeared on Medscape.com .
than adults from other ethnic backgrounds.
Those are among the key findings from an analysis of nationally representative cross-sectional data that were presented during a late-breaking abstract session at the Revolutionizing Atopic Dermatitis conference in Chicago.
“In the past few years, there has been a much-needed focus on better understanding disparities in atopic dermatitis,” one of the study authors, Raj Chovatiya, MD, PhD, clinical associate professor at Chicago Medical School, Rosalind Franklin University, North Chicago, told this news organization after the conference.
“Epidemiology is one of the key ways in which we can query differences in AD at a population level.”
Drawing from the 2021 National Health Interview Survey, the researchers identified 3103 respondents who reported being diagnosed with AD or eczema. They estimated the prevalence rates of AD for the overall population and each subgroup by dividing US frequency estimates by their corresponding US population totals and used multivariable logistic regression to assess the odds of having AD.
More than half of the respondents (1643) were aged between 18 and 64 years, 522 were aged 65 years and older, and 922 were children younger than 18 years. Overall, the prevalence of AD was 7.6% in adults aged 18-64 years and 6.1% in adults aged 65 years and older, for a weighted US estimate of 15.3 and 3.2 million, respectively. The prevalence of AD varied by race/ethnicity and was highest for those from “other single and multiple races” group (12.4%), followed by Black/African American (8.5%), White (7.7%), Asian (6.5%), American Indian/Alaskan Native (4.9%), and Hispanic (4.8%) populations.
In children, race/ethnicity prevalence were highest for those from other single and multiple races (15.2.%), followed by Black/African American (14.2%), American Indian/Alaskan Native (12%), White (10.2%), Hispanic (9.5%), and Asian (9%) populations.
When the researchers combined all age groups, they observed higher prevalence rates of AD among females than among males. However, in an analysis limited to children, the prevalence rates were similar between girls and boys (10.8% vs 10.7%, respectively), for a weighted US estimate of 7.8 million children with AD.
On multiple regression, the odds of having AD were greater among women than among men (odds ratio [OR], 1.4), among adults aged 18-64 years than among those aged 65 years and older (OR, 1.4), among those younger than 18 years than among those aged 65 years and older (OR, 2.0), and among Black/African American individuals than among White individuals (OR, 1.2). Hispanic adults had a lower risk for AD than non-Hispanic White adults (OR, 0.69) as did Asian adults than White adults (OR, 0.82).
“We found AD prevalence rates were higher in children and adult females, Hispanic adults had a lower prevalence of AD than all other adult groups, and there were numerical differences in AD prevalence across racial groups,” Dr. Chovatiya said in the interview. “While there are of course limitations to the use of any nationally representative cross-sectional dataset that requires weighting to project results from a smaller sample to reflect a larger more heterogeneous group, these results are important for us to consider targeted strategies to address AD burden.”
Jonathan I. Silverberg, MD, PhD, professor of dermatology at The George Washington University, Washington, who was asked to comment on the study, said that while the prevalence of AD in children has been well documented in prior research, “this study fills an important gap by showing us that the prevalence does remain high in adults.”
In addition, “it has not shown any evidence of AD decreasing over time; if anything, it might be slightly increasing,” he said. “We’re also seeing differences [in AD] by race and ethnicity. We have seen that demonstrated in children but [has been] less clearly demonstrated in adults.”
Eli Lilly and Company funded the analysis. Dr. Chovatiya and Dr. Silverberg disclosed ties to several pharmaceutical companies, including Eli Lilly.
A version of this article appeared on Medscape.com .
Topical Ruxolitinib Effective for AD in Study of Children Ages 2-11 years
) affecting ≥ 35% or more of their body surface area (BSA), results from a small open-label maximum-use trial showed.
When approved for this age group, ruxolitinib cream will provide a topical nonsteroidal option for patients aged 2-11, which will “simplify the treatment regimen,” one of the study investigators, Linda Stein Gold, MD, director of clinical research and division head of dermatology at the Henry Ford Health System, Detroit, said in an interview after the Revolutionizing Atopic Dermatitis conference, where the study was presented during a late-breaking abstract session.
A topical formulation of the selective Janus kinase (JAK) 1/JAK2 inhibitor, ruxolitinib cream 1.5% is currently approved by the Food and Drug Administration for the short-term and noncontinuous chronic treatment of mild to moderate AD in non-immunocompromised adult and pediatric patients aged 12 years and older, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.
In previous reports of this trial in children aged 2-11 years with ≥ 35% affected BSA, ruxolitinib cream 1.5% was generally well tolerated, with rapid anti-inflammatory and antipruritic effects and improvements in patient-reported outcomes observed with ≤ 4 weeks of continuous treatment and maintained with as-needed treatment from 4 to 8 weeks.
For the current trial, investigators evaluated data on tolerability, safety, systemic exposure, and clinical and patient-reported outcomes through 52 weeks to determine whether clinical benefits and tolerability observed through 8 weeks were sustained.
Dr. Stein Gold and colleagues reported results from 29 children who received ruxolitinib cream 1.5% from baseline through week 8. Of these, 22 continued into the long-term safety period from week 8 through 52. From baseline through week 8, patients applied a mean of 6.5 g per day of ruxolitinib cream; this dropped to a mean of 3.2 g per day from weeks 8 through 52. The mean steady-state plasma concentration of ruxolitinib throughout the study was 98.2 nM, which is “well below half-maximal concentration of JAK-mediated myelosuppression in adults (281 nM),” the researchers stated in their abstract.
No treatment-related interruptions, discontinuations, or serious adverse events were observed between baseline and week 52. One patient (3.4%) had two treatment-related application site reactions (paresthesia and folliculitis). At weeks 4 and 52, 53.8% of patients achieved treatment success, which was defined as an Investigator Global Assessment of 0/1 with a ≥ 2-grade improvement from baseline. The mean affected BSA decreased from 58.0% at baseline to 11.4% at week 4 and continued to decrease to 2.2% through week 52. “I was surprised that patients could maintain control over the long-term using the medication as needed,” Dr. Stein Gold told this news organization. “I was also pleased to see that there was low systemic exposure even when used on large body surface areas.”
In other findings, the mean total Patient Oriented Eczema Measure score dropped from a baseline of 19.4 to a mean of 4.5 at week 8 and 3.6 at week 52 and the mean total Children’s Dermatology Life Quality Index score fell from a baseline of 15.4 to a mean of 5.3 at week 8 and a mean of 2.1 at week 52. Meanwhile, the mean total Infants’ Dermatology Quality of Life Index score fell from a mean of 12.3 at baseline to a mean of 2.8 at week 8 and a mean of 0.7 at week 52.
Dr. Stein Gold noted certain limitations of the study, including the fact that it did not study children aged younger than 2 years.
The study was funded by Incyte, which markets ruxolitinib cream 1.5% as Opzelura. Dr. Stein Gold disclosed that she has served as an investigator, advisor, and/or speaker for several pharmaceutical companies, including Incyte.
A version of this article appeared on Medscape.com.
) affecting ≥ 35% or more of their body surface area (BSA), results from a small open-label maximum-use trial showed.
When approved for this age group, ruxolitinib cream will provide a topical nonsteroidal option for patients aged 2-11, which will “simplify the treatment regimen,” one of the study investigators, Linda Stein Gold, MD, director of clinical research and division head of dermatology at the Henry Ford Health System, Detroit, said in an interview after the Revolutionizing Atopic Dermatitis conference, where the study was presented during a late-breaking abstract session.
A topical formulation of the selective Janus kinase (JAK) 1/JAK2 inhibitor, ruxolitinib cream 1.5% is currently approved by the Food and Drug Administration for the short-term and noncontinuous chronic treatment of mild to moderate AD in non-immunocompromised adult and pediatric patients aged 12 years and older, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.
In previous reports of this trial in children aged 2-11 years with ≥ 35% affected BSA, ruxolitinib cream 1.5% was generally well tolerated, with rapid anti-inflammatory and antipruritic effects and improvements in patient-reported outcomes observed with ≤ 4 weeks of continuous treatment and maintained with as-needed treatment from 4 to 8 weeks.
For the current trial, investigators evaluated data on tolerability, safety, systemic exposure, and clinical and patient-reported outcomes through 52 weeks to determine whether clinical benefits and tolerability observed through 8 weeks were sustained.
Dr. Stein Gold and colleagues reported results from 29 children who received ruxolitinib cream 1.5% from baseline through week 8. Of these, 22 continued into the long-term safety period from week 8 through 52. From baseline through week 8, patients applied a mean of 6.5 g per day of ruxolitinib cream; this dropped to a mean of 3.2 g per day from weeks 8 through 52. The mean steady-state plasma concentration of ruxolitinib throughout the study was 98.2 nM, which is “well below half-maximal concentration of JAK-mediated myelosuppression in adults (281 nM),” the researchers stated in their abstract.
No treatment-related interruptions, discontinuations, or serious adverse events were observed between baseline and week 52. One patient (3.4%) had two treatment-related application site reactions (paresthesia and folliculitis). At weeks 4 and 52, 53.8% of patients achieved treatment success, which was defined as an Investigator Global Assessment of 0/1 with a ≥ 2-grade improvement from baseline. The mean affected BSA decreased from 58.0% at baseline to 11.4% at week 4 and continued to decrease to 2.2% through week 52. “I was surprised that patients could maintain control over the long-term using the medication as needed,” Dr. Stein Gold told this news organization. “I was also pleased to see that there was low systemic exposure even when used on large body surface areas.”
In other findings, the mean total Patient Oriented Eczema Measure score dropped from a baseline of 19.4 to a mean of 4.5 at week 8 and 3.6 at week 52 and the mean total Children’s Dermatology Life Quality Index score fell from a baseline of 15.4 to a mean of 5.3 at week 8 and a mean of 2.1 at week 52. Meanwhile, the mean total Infants’ Dermatology Quality of Life Index score fell from a mean of 12.3 at baseline to a mean of 2.8 at week 8 and a mean of 0.7 at week 52.
Dr. Stein Gold noted certain limitations of the study, including the fact that it did not study children aged younger than 2 years.
The study was funded by Incyte, which markets ruxolitinib cream 1.5% as Opzelura. Dr. Stein Gold disclosed that she has served as an investigator, advisor, and/or speaker for several pharmaceutical companies, including Incyte.
A version of this article appeared on Medscape.com.
) affecting ≥ 35% or more of their body surface area (BSA), results from a small open-label maximum-use trial showed.
When approved for this age group, ruxolitinib cream will provide a topical nonsteroidal option for patients aged 2-11, which will “simplify the treatment regimen,” one of the study investigators, Linda Stein Gold, MD, director of clinical research and division head of dermatology at the Henry Ford Health System, Detroit, said in an interview after the Revolutionizing Atopic Dermatitis conference, where the study was presented during a late-breaking abstract session.
A topical formulation of the selective Janus kinase (JAK) 1/JAK2 inhibitor, ruxolitinib cream 1.5% is currently approved by the Food and Drug Administration for the short-term and noncontinuous chronic treatment of mild to moderate AD in non-immunocompromised adult and pediatric patients aged 12 years and older, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.
In previous reports of this trial in children aged 2-11 years with ≥ 35% affected BSA, ruxolitinib cream 1.5% was generally well tolerated, with rapid anti-inflammatory and antipruritic effects and improvements in patient-reported outcomes observed with ≤ 4 weeks of continuous treatment and maintained with as-needed treatment from 4 to 8 weeks.
For the current trial, investigators evaluated data on tolerability, safety, systemic exposure, and clinical and patient-reported outcomes through 52 weeks to determine whether clinical benefits and tolerability observed through 8 weeks were sustained.
Dr. Stein Gold and colleagues reported results from 29 children who received ruxolitinib cream 1.5% from baseline through week 8. Of these, 22 continued into the long-term safety period from week 8 through 52. From baseline through week 8, patients applied a mean of 6.5 g per day of ruxolitinib cream; this dropped to a mean of 3.2 g per day from weeks 8 through 52. The mean steady-state plasma concentration of ruxolitinib throughout the study was 98.2 nM, which is “well below half-maximal concentration of JAK-mediated myelosuppression in adults (281 nM),” the researchers stated in their abstract.
No treatment-related interruptions, discontinuations, or serious adverse events were observed between baseline and week 52. One patient (3.4%) had two treatment-related application site reactions (paresthesia and folliculitis). At weeks 4 and 52, 53.8% of patients achieved treatment success, which was defined as an Investigator Global Assessment of 0/1 with a ≥ 2-grade improvement from baseline. The mean affected BSA decreased from 58.0% at baseline to 11.4% at week 4 and continued to decrease to 2.2% through week 52. “I was surprised that patients could maintain control over the long-term using the medication as needed,” Dr. Stein Gold told this news organization. “I was also pleased to see that there was low systemic exposure even when used on large body surface areas.”
In other findings, the mean total Patient Oriented Eczema Measure score dropped from a baseline of 19.4 to a mean of 4.5 at week 8 and 3.6 at week 52 and the mean total Children’s Dermatology Life Quality Index score fell from a baseline of 15.4 to a mean of 5.3 at week 8 and a mean of 2.1 at week 52. Meanwhile, the mean total Infants’ Dermatology Quality of Life Index score fell from a mean of 12.3 at baseline to a mean of 2.8 at week 8 and a mean of 0.7 at week 52.
Dr. Stein Gold noted certain limitations of the study, including the fact that it did not study children aged younger than 2 years.
The study was funded by Incyte, which markets ruxolitinib cream 1.5% as Opzelura. Dr. Stein Gold disclosed that she has served as an investigator, advisor, and/or speaker for several pharmaceutical companies, including Incyte.
A version of this article appeared on Medscape.com.