Dermatology News

Patients with treatment-refractory cancers who did not meet eligibility criteria for a pan-cancer clinical trial but received waivers allowing them to participate had similar outcomes to patients who participated without waivers, a new analysis revealed.

The study, published online in Clinical Cancer Research, highlighted the potential benefits of broadening eligibility criteria for clinical trials.

“It is well known that results in an ‘ideal’ population do not always translate to the real-world population,” senior author Hans Gelderblom, MD, chair of the Department of Medical Oncology at the Leiden University Medical Center, Leiden, the Netherlands, said in a press release. “Eligibility criteria are often too strict, and educated exemptions by experienced investigators can help individual patients, especially in a last-resort trial.”

Although experts have expressed interest in improving trial inclusivity, it’s unclear how doing so might impact treatment safety and efficacy.

In the Drug Rediscovery Protocol (DRUP), Dr. Gelderblom and colleagues examined the impact of broadening trial eligibility on patient outcomes. DRUP is an ongoing Dutch national, multicenter, pan-cancer, nonrandomized clinical trial in which patients are treated off-label with approved molecularly targeted or immunotherapies.

In the trial, 1019 patients with treatment-refractory disease were matched to one of the available study drugs based on their tumor molecular profile and enrolled in parallel cohorts. Cohorts were defined by tumor type, molecular profile, and study drug.

Among these patients, 82 patients — 8% of the cohort — were granted waivers to participate. Most waivers (45%) were granted as exceptions to general- or drug-related eligibility criteria, often because of out-of-range lab results. Other categories included treatment and testing exceptions, as well as out-of-window testing. 

The researchers then compared safety and efficacy outcomes between the 82 participants granted waivers and the 937 who did not receive waivers. 

Overall, Dr. Gelderblom’s team found that the rate of serious adverse events was similar between patients who received a waiver and those who did not: 39% vs 41%, respectively.

A relationship between waivers and serious adverse events was deemed “unlikely” for 86% of patients and “possible” for 14%. In two cases concerning a direct relationship, for instance, patients who received waivers for decreased hemoglobin levels developed anemia.

The rate of clinical benefit — defined as an objective response or stable disease for at least 16 weeks — was similar between the groups. Overall, 40% of patients who received a waiver (33 of 82) had a clinical benefit vs 33% of patients without a waiver (P = .43). Median overall survival for patients that received a waiver was also similar — 11 months in the waiver group and 8 months in the nonwaiver group (hazard ratio, 0.87; P = .33).

“Safety and clinical benefit were preserved in patients for whom a waiver was granted,” the authors concluded.

The study had several limitations. The diversity of cancer types, treatments, and reasons for protocol exemptions precluded subgroup analyses. In addition, because the decision to grant waivers depended in large part on the likelihood of clinical benefit, “it is possible that patients who received waivers were positively selected for clinical benefit compared with the general study population,” the authors wrote.

So, “although the clinical benefit rate of the patient group for whom a waiver was granted appears to be slightly higher, this difference might be explained by the selection process of the central study team, in which each waiver request was carefully considered, weighing the risks and potential benefits for the patient in question,” the authors explained.

Overall, “these findings advocate for a broader and more inclusive design when establishing novel trials, paving the way for a more effective and tailored application of cancer therapies in patients with advanced or refractory disease,” Dr. Gelderblom said.

Commenting on the study, Bishal Gyawali, MD, PhD, said that “relaxing eligibility criteria is important, and I support this. Trials should include patients that are more representative of the real-world, so that results are generalizable.”

However, “the paper overemphasized efficacy,” said Dr. Gyawali, from Queen’s University, Kingston, Ontario, Canada. The sample size of waiver-granted patients was small, plus “the clinical benefit rate is not a marker of efficacy.

“The response rate is somewhat better, but for a heterogeneous study with multiple targets and drugs, it is difficult to say much about treatment effects here,” Dr. Gyawali added. Overall, “we shouldn’t read too much into treatment benefits based on these numbers.”

Funding for the study was provided by the Stelvio for Life Foundation, the Dutch Cancer Society, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, pharma&, Eisai Co., Ipsen, Merck Sharp & Dohme, Novartis, Pfizer, and Roche. Dr. Gelderblom declared no conflicts of interest, and Dr. Gyawali declared no conflicts of interest related to his comment.
 

A version of this article appeared on Medscape.com.

Patients with treatment-refractory cancers who did not meet eligibility criteria for a pan-cancer clinical trial but received waivers allowing them to participate had similar outcomes to patients who participated without waivers, a new analysis revealed.

The study, published online in Clinical Cancer Research, highlighted the potential benefits of broadening eligibility criteria for clinical trials.

“It is well known that results in an ‘ideal’ population do not always translate to the real-world population,” senior author Hans Gelderblom, MD, chair of the Department of Medical Oncology at the Leiden University Medical Center, Leiden, the Netherlands, said in a press release. “Eligibility criteria are often too strict, and educated exemptions by experienced investigators can help individual patients, especially in a last-resort trial.”

Although experts have expressed interest in improving trial inclusivity, it’s unclear how doing so might impact treatment safety and efficacy.

In the Drug Rediscovery Protocol (DRUP), Dr. Gelderblom and colleagues examined the impact of broadening trial eligibility on patient outcomes. DRUP is an ongoing Dutch national, multicenter, pan-cancer, nonrandomized clinical trial in which patients are treated off-label with approved molecularly targeted or immunotherapies.

In the trial, 1019 patients with treatment-refractory disease were matched to one of the available study drugs based on their tumor molecular profile and enrolled in parallel cohorts. Cohorts were defined by tumor type, molecular profile, and study drug.

Among these patients, 82 patients — 8% of the cohort — were granted waivers to participate. Most waivers (45%) were granted as exceptions to general- or drug-related eligibility criteria, often because of out-of-range lab results. Other categories included treatment and testing exceptions, as well as out-of-window testing. 

The researchers then compared safety and efficacy outcomes between the 82 participants granted waivers and the 937 who did not receive waivers. 

Overall, Dr. Gelderblom’s team found that the rate of serious adverse events was similar between patients who received a waiver and those who did not: 39% vs 41%, respectively.

A relationship between waivers and serious adverse events was deemed “unlikely” for 86% of patients and “possible” for 14%. In two cases concerning a direct relationship, for instance, patients who received waivers for decreased hemoglobin levels developed anemia.

The rate of clinical benefit — defined as an objective response or stable disease for at least 16 weeks — was similar between the groups. Overall, 40% of patients who received a waiver (33 of 82) had a clinical benefit vs 33% of patients without a waiver (P = .43). Median overall survival for patients that received a waiver was also similar — 11 months in the waiver group and 8 months in the nonwaiver group (hazard ratio, 0.87; P = .33).

“Safety and clinical benefit were preserved in patients for whom a waiver was granted,” the authors concluded.

The study had several limitations. The diversity of cancer types, treatments, and reasons for protocol exemptions precluded subgroup analyses. In addition, because the decision to grant waivers depended in large part on the likelihood of clinical benefit, “it is possible that patients who received waivers were positively selected for clinical benefit compared with the general study population,” the authors wrote.

So, “although the clinical benefit rate of the patient group for whom a waiver was granted appears to be slightly higher, this difference might be explained by the selection process of the central study team, in which each waiver request was carefully considered, weighing the risks and potential benefits for the patient in question,” the authors explained.

Overall, “these findings advocate for a broader and more inclusive design when establishing novel trials, paving the way for a more effective and tailored application of cancer therapies in patients with advanced or refractory disease,” Dr. Gelderblom said.

Commenting on the study, Bishal Gyawali, MD, PhD, said that “relaxing eligibility criteria is important, and I support this. Trials should include patients that are more representative of the real-world, so that results are generalizable.”

However, “the paper overemphasized efficacy,” said Dr. Gyawali, from Queen’s University, Kingston, Ontario, Canada. The sample size of waiver-granted patients was small, plus “the clinical benefit rate is not a marker of efficacy.

“The response rate is somewhat better, but for a heterogeneous study with multiple targets and drugs, it is difficult to say much about treatment effects here,” Dr. Gyawali added. Overall, “we shouldn’t read too much into treatment benefits based on these numbers.”

Funding for the study was provided by the Stelvio for Life Foundation, the Dutch Cancer Society, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, pharma&, Eisai Co., Ipsen, Merck Sharp & Dohme, Novartis, Pfizer, and Roche. Dr. Gelderblom declared no conflicts of interest, and Dr. Gyawali declared no conflicts of interest related to his comment.
 

A version of this article appeared on Medscape.com.

Patients with treatment-refractory cancers who did not meet eligibility criteria for a pan-cancer clinical trial but received waivers allowing them to participate had similar outcomes to patients who participated without waivers, a new analysis revealed.

The study, published online in Clinical Cancer Research, highlighted the potential benefits of broadening eligibility criteria for clinical trials.

“It is well known that results in an ‘ideal’ population do not always translate to the real-world population,” senior author Hans Gelderblom, MD, chair of the Department of Medical Oncology at the Leiden University Medical Center, Leiden, the Netherlands, said in a press release. “Eligibility criteria are often too strict, and educated exemptions by experienced investigators can help individual patients, especially in a last-resort trial.”

Although experts have expressed interest in improving trial inclusivity, it’s unclear how doing so might impact treatment safety and efficacy.

In the Drug Rediscovery Protocol (DRUP), Dr. Gelderblom and colleagues examined the impact of broadening trial eligibility on patient outcomes. DRUP is an ongoing Dutch national, multicenter, pan-cancer, nonrandomized clinical trial in which patients are treated off-label with approved molecularly targeted or immunotherapies.

In the trial, 1019 patients with treatment-refractory disease were matched to one of the available study drugs based on their tumor molecular profile and enrolled in parallel cohorts. Cohorts were defined by tumor type, molecular profile, and study drug.

Among these patients, 82 patients — 8% of the cohort — were granted waivers to participate. Most waivers (45%) were granted as exceptions to general- or drug-related eligibility criteria, often because of out-of-range lab results. Other categories included treatment and testing exceptions, as well as out-of-window testing. 

The researchers then compared safety and efficacy outcomes between the 82 participants granted waivers and the 937 who did not receive waivers. 

Overall, Dr. Gelderblom’s team found that the rate of serious adverse events was similar between patients who received a waiver and those who did not: 39% vs 41%, respectively.

A relationship between waivers and serious adverse events was deemed “unlikely” for 86% of patients and “possible” for 14%. In two cases concerning a direct relationship, for instance, patients who received waivers for decreased hemoglobin levels developed anemia.

The rate of clinical benefit — defined as an objective response or stable disease for at least 16 weeks — was similar between the groups. Overall, 40% of patients who received a waiver (33 of 82) had a clinical benefit vs 33% of patients without a waiver (P = .43). Median overall survival for patients that received a waiver was also similar — 11 months in the waiver group and 8 months in the nonwaiver group (hazard ratio, 0.87; P = .33).

“Safety and clinical benefit were preserved in patients for whom a waiver was granted,” the authors concluded.

The study had several limitations. The diversity of cancer types, treatments, and reasons for protocol exemptions precluded subgroup analyses. In addition, because the decision to grant waivers depended in large part on the likelihood of clinical benefit, “it is possible that patients who received waivers were positively selected for clinical benefit compared with the general study population,” the authors wrote.

So, “although the clinical benefit rate of the patient group for whom a waiver was granted appears to be slightly higher, this difference might be explained by the selection process of the central study team, in which each waiver request was carefully considered, weighing the risks and potential benefits for the patient in question,” the authors explained.

Overall, “these findings advocate for a broader and more inclusive design when establishing novel trials, paving the way for a more effective and tailored application of cancer therapies in patients with advanced or refractory disease,” Dr. Gelderblom said.

Commenting on the study, Bishal Gyawali, MD, PhD, said that “relaxing eligibility criteria is important, and I support this. Trials should include patients that are more representative of the real-world, so that results are generalizable.”

However, “the paper overemphasized efficacy,” said Dr. Gyawali, from Queen’s University, Kingston, Ontario, Canada. The sample size of waiver-granted patients was small, plus “the clinical benefit rate is not a marker of efficacy.

“The response rate is somewhat better, but for a heterogeneous study with multiple targets and drugs, it is difficult to say much about treatment effects here,” Dr. Gyawali added. Overall, “we shouldn’t read too much into treatment benefits based on these numbers.”

Funding for the study was provided by the Stelvio for Life Foundation, the Dutch Cancer Society, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, pharma&, Eisai Co., Ipsen, Merck Sharp & Dohme, Novartis, Pfizer, and Roche. Dr. Gelderblom declared no conflicts of interest, and Dr. Gyawali declared no conflicts of interest related to his comment.
 

A version of this article appeared on Medscape.com.

VIENNA — From a dozen or so studies and sessions devoted to the role of chimeric antigen receptor (CAR) T cells in rheumatic diseases at the annual European Congress of Rheumatology, the message was uniformly positive, supporting growing evidence that drugs in this class are heading toward a paradigm shift in refractory rheumatic diseases.

Of the reports, an update from a 15-patient case series with at least 1 year of follow-up provides “the first long-term evidence of safety and efficacy in multiple rheumatic diseases,” according to Georg Schett, MD, PhD, director of rheumatology and immunology, University of Erlangen-Nürnberg, Erlangen, Germany.

Ted Bosworth/Medscape Medical News

The report of high rates of activity and low relative risk of serious adverse events from the same series was published earlier this year in The New England Journal of Medicine when the median follow-up was 15 months. Almost all of the patients have now completed at least 1 year of follow-up and about a third have completed more than 2 years.
 

SLE Is Frequently Targeted in CAR T-Cell Studies

The three rheumatic diseases represented in this series of patients, all of whom had failed multiple previous immune suppressive treatments, were systemic lupus erythematosus (SLE), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). After the autologous T cells were harvested, they were expanded and transfected with the CD19 CAR. The proprietary investigational product, called MB-CART19.1 (Miltenyi Biotec), was administered in a single dose of one million cells per kg bodyweight.

The response rates have been, and continue to be, impressive. For the eight patients with SLE, all achieved the definition of remission in SLE criteria after one dose of treatment. Complete resolution of all major symptom types was achieved after 6 months of follow-up. So far, no patient has relapsed.

For the three patients with IIM, all reached the American College of Rheumatology–EULAR criteria for a major response. All creatine kinase levels had normalized by 3 months. In this group, there was one relapse, which occurred after 18 months of follow-up.

All four patients with SSc achieved a major response on the European Scleroderma Trials and Research (EUSTAR) group activity index. The median reduction from baseline in the EUSTAR score was 4.2 points, and this has been maintained in follow-up to date.
 

Remissions Have Persisted Off All Therapies

These remissions were achieved and maintained after a single dose of CAR T-cell therapy despite discontinuation of all immunosuppressive therapies. With the exception of the single relapse, all remissions have persisted through follow-up to date.

These responses were achieved with manageable side effects, according to Dr. Schett. The most serious adverse event was a grade 4 neutropenia that developed 4 months after receiving CAR T cells. It resolved with granulocyte colony-stimulating factor treatment. Cytokine release syndrome (CRS) has occurred in 10 patients, but it was grade 1 in eight patients and grade 2 in the others. There has been no neurotoxicity.

Almost all patients have experienced an infection during follow-up, but there has been no discernible pattern in relationship to the timing or types of infections. The most common have involved the upper respiratory tract and have been of mild severity, with cases disseminated similarly over early vs late follow-up. There was one case of pneumonia involving antibiotic treatment and a hospital stay, but it resolved.

Dr. Schett acknowledged that safety is a bigger concern in autoimmune diseases, which are often serious but rarely fatal, than in the hematologic malignancies for which CAR T cells were initially tested, but the low rates of serious adverse events in his and other early studies have supported the premise that the risks are not the same.

Asked specifically if CAR T cells can be considered a game changer in autoimmune rheumatic diseases, Dr. Schett was cautious. One reason is the CAR T cells are a complex therapy relative to biologic disease-modifying antirheumatic drugs. He thinks, therefore, that much more data are needed to confirm safety and efficacy. In addition, they are expensive, so it is not yet clear how they will be integrated with other options.

Yet, he thinks the evidence so far suggests a profound effect on the fundamental drivers of autoimmune disease. Their specific mechanism of benefit is still being evaluated, but he considers the clinical responses consistent with a “reset” hypothesis.

After a response, “we are seeing drug-free remissions in some patients as long as they have been followed,” Dr. Schett said. Based on the fact that disease control is being observed off all other therapies, “this only makes sense to me if there is some sort of immunologic reset.”
 

CAR T-Cell Studies in Autoimmune Diseases Are Proliferating

At last count, there were about 40 studies being performed with CAR T cells in various autoimmune diseases, most of which were rheumatologic disorders, according to Dr. Schett. He noted that funding is coming from multinational drug companies, small biotech startups, and investigator-initiated studies at academic centers.

At EULAR, beyond case studies and anecdotal reports, all of the clinical studies were still at the level of phase 1 or 1/2. Consistent with the data presented by Dr. Schett, the drugs have been nearly uniformly effective, with major responses persisting in patients off other therapies. Adverse events have been manageable.

Examples include a phase 1/2 multinational study with the investigational CAR T-cell therapy YTB323 (Novartis), which demonstrated acceptable safety and a strong signal of benefit in six patients with SLE. In this report, CRS was also common, but no case of CRS was more severe than grade 2. There was no neurotoxicity. Infections did occur but were of relatively mild grades and resolved with treatment.

For efficacy in the ongoing follow-up, SLE symptoms as measured with the SLE Disease Activity Index began to abate at about 14 days after the single-infusion treatment. Improvement on the Physician Global Assessment was also observed between 14 and 28 days. C3 and C4 complement levels started to rise at about 28 days. While the responses have correlated with the observed changes in biomarkers of immune function, they have endured through a median follow-up that now exceeds 6 months.
 

Complete B-Cell Depletion Is Followed by Full Recovery

“Pharmacokinetic and pharmacodynamic studies revealed peak expansion of CAR T cells approximately 13-21 days post infusion, which was accompanied by deep B-cell depletion followed by subsequent B-cell recovery,” reported Josefina Cortés-Hernández, MD, PhD, a senior lecturer at Vall d’Hebron Research Institute, Barcelona, Spain.

Dr. Schett had reported the same pattern of expansion followed by a rapid elimination of detectable CAR T cells despite the sustained clinical benefit.

Dr. Cortés-Hernández said that the signal of efficacy in the context of acceptable safety supports an expansion of clinical studies with this CAR T-cell product in SLE and perhaps other autoimmune disorders.

In another early-stage study, patients with SLE who had failed multiple prior lines of therapy have been enrolled in an ongoing study with a compound CAR (cCAR) T cell. This experimental proprietary product (iCAR Bio Therapeutics, Zhongshan, China) targets both the B-cell maturation antigen and CD19, according to Greg Deener, the chief executive officer of iCell Gene Therapeutics, New York City.

Ted Bosworth/Medscape Medical News

cCAR T-Cell Construct Targets Immune Reset

With this construct, the goal is to deplete long-lived plasma cells as well as B cells in order to achieve a more complete humoral reset. While preliminary data from the phase 1 trial were published earlier this year in Annals of the Rheumatic Diseases, Mr. Deener focused his presentation at EULAR 2024 on 12 patients with SLE and lupus nephritis, a severe form of SLE that threatens glomerular structures and can lead to end-stage liver disease.

B cells in the peripheral blood could not be detected within 10 days of the cCAR infusion, and the immunoglobulins IgM and IgA were undetectable by day 42.

However, after B-cell recovery by day 150, “flow cytometry and B-cell receptor sequencing confirmed full humoral reset was achieved,” Mr. Deener said.

The remission has been durable in 11 of the 12 patients after a mean follow-up of 458 days, Mr. Deener reported. He noted that an improvement in renal function has been observed in the majority of patients.

Like others, he reported that treatment has been relatively well tolerated. In this series of patients, there have been no cases of CRS more severe than grade 1.

Overall, the cCAR data in lupus nephritis support the hypothesis that CAR T cells are reprogramming the immune system, according to Mr. Deener.

Combined with a reasonable safety profile, the consistency of benefit from CAR T cells in autoimmune rheumatic diseases is good news, but all of the investigators who spoke at EULAR agreed that there are still many unanswered questions. Not least, it is unclear whether patients can be effectively and safely retreated when and if relapses occur. Even though Dr. Schett did report a response with retreatment following a relapse, he said that there is no conclusion to draw from a single patient.

Yet, the high rates of remissions in patients with disease refractory to other therapeutic options is highly encouraging, particularly with the manageable side effects now reported by multiple investigators using different CAR T-cell products.

“Roughly 100 patients with rheumatic diseases have been treated with CAR T-cells, and we have not seen a high-grade CRS or neurotoxicity,” he said.

Long-term efficacy is less clear. With the first clinical studies in autoimmune diseases initiated in 2021, few patients have been followed for more than 2 years. Even with the high rates of response that will certainly fuel efforts to rapidly bring these treatments forward, long-term data are now the missing piece.
 

Other Case Series Presented at EULAR

Several other abstracts reported on patients with SSc who were treated with CD19-targeting CAR T cells:

Three patients for whom autologous hematopoietic stem cell transplantation was contraindicated or unsuccessful were successfully and safely treated.

Six patients with diffuse and progressive disease achieved stable disease activity without additional immunosuppression for up to 1 year after treatment.

Dr. Schett reported no potential conflicts of interest, and the study he presented was not funded by industry. Dr. Cortés-Hernández reported a financial relationship with Novartis, which funded the study of the CAR T-cell therapy YTB323, as well as with GlaxoSmithKline, which was not involved in the study she presented.  Mr. Deener is an employee of iCell Gene Therapeutics, which provided funding for the trial he presented.

August 7, 2024 — Editor's note: This article was updated with additional disclosure information for Dr. Josefina Cortés-Hernández.

A version of this article appeared on Medscape.com.

VIENNA — From a dozen or so studies and sessions devoted to the role of chimeric antigen receptor (CAR) T cells in rheumatic diseases at the annual European Congress of Rheumatology, the message was uniformly positive, supporting growing evidence that drugs in this class are heading toward a paradigm shift in refractory rheumatic diseases.

Of the reports, an update from a 15-patient case series with at least 1 year of follow-up provides “the first long-term evidence of safety and efficacy in multiple rheumatic diseases,” according to Georg Schett, MD, PhD, director of rheumatology and immunology, University of Erlangen-Nürnberg, Erlangen, Germany.

Ted Bosworth/Medscape Medical News

The report of high rates of activity and low relative risk of serious adverse events from the same series was published earlier this year in The New England Journal of Medicine when the median follow-up was 15 months. Almost all of the patients have now completed at least 1 year of follow-up and about a third have completed more than 2 years.
 

SLE Is Frequently Targeted in CAR T-Cell Studies

The three rheumatic diseases represented in this series of patients, all of whom had failed multiple previous immune suppressive treatments, were systemic lupus erythematosus (SLE), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). After the autologous T cells were harvested, they were expanded and transfected with the CD19 CAR. The proprietary investigational product, called MB-CART19.1 (Miltenyi Biotec), was administered in a single dose of one million cells per kg bodyweight.

The response rates have been, and continue to be, impressive. For the eight patients with SLE, all achieved the definition of remission in SLE criteria after one dose of treatment. Complete resolution of all major symptom types was achieved after 6 months of follow-up. So far, no patient has relapsed.

For the three patients with IIM, all reached the American College of Rheumatology–EULAR criteria for a major response. All creatine kinase levels had normalized by 3 months. In this group, there was one relapse, which occurred after 18 months of follow-up.

All four patients with SSc achieved a major response on the European Scleroderma Trials and Research (EUSTAR) group activity index. The median reduction from baseline in the EUSTAR score was 4.2 points, and this has been maintained in follow-up to date.
 

Remissions Have Persisted Off All Therapies

These remissions were achieved and maintained after a single dose of CAR T-cell therapy despite discontinuation of all immunosuppressive therapies. With the exception of the single relapse, all remissions have persisted through follow-up to date.

These responses were achieved with manageable side effects, according to Dr. Schett. The most serious adverse event was a grade 4 neutropenia that developed 4 months after receiving CAR T cells. It resolved with granulocyte colony-stimulating factor treatment. Cytokine release syndrome (CRS) has occurred in 10 patients, but it was grade 1 in eight patients and grade 2 in the others. There has been no neurotoxicity.

Almost all patients have experienced an infection during follow-up, but there has been no discernible pattern in relationship to the timing or types of infections. The most common have involved the upper respiratory tract and have been of mild severity, with cases disseminated similarly over early vs late follow-up. There was one case of pneumonia involving antibiotic treatment and a hospital stay, but it resolved.

Dr. Schett acknowledged that safety is a bigger concern in autoimmune diseases, which are often serious but rarely fatal, than in the hematologic malignancies for which CAR T cells were initially tested, but the low rates of serious adverse events in his and other early studies have supported the premise that the risks are not the same.

Asked specifically if CAR T cells can be considered a game changer in autoimmune rheumatic diseases, Dr. Schett was cautious. One reason is the CAR T cells are a complex therapy relative to biologic disease-modifying antirheumatic drugs. He thinks, therefore, that much more data are needed to confirm safety and efficacy. In addition, they are expensive, so it is not yet clear how they will be integrated with other options.

Yet, he thinks the evidence so far suggests a profound effect on the fundamental drivers of autoimmune disease. Their specific mechanism of benefit is still being evaluated, but he considers the clinical responses consistent with a “reset” hypothesis.

After a response, “we are seeing drug-free remissions in some patients as long as they have been followed,” Dr. Schett said. Based on the fact that disease control is being observed off all other therapies, “this only makes sense to me if there is some sort of immunologic reset.”
 

CAR T-Cell Studies in Autoimmune Diseases Are Proliferating

At last count, there were about 40 studies being performed with CAR T cells in various autoimmune diseases, most of which were rheumatologic disorders, according to Dr. Schett. He noted that funding is coming from multinational drug companies, small biotech startups, and investigator-initiated studies at academic centers.

At EULAR, beyond case studies and anecdotal reports, all of the clinical studies were still at the level of phase 1 or 1/2. Consistent with the data presented by Dr. Schett, the drugs have been nearly uniformly effective, with major responses persisting in patients off other therapies. Adverse events have been manageable.

Examples include a phase 1/2 multinational study with the investigational CAR T-cell therapy YTB323 (Novartis), which demonstrated acceptable safety and a strong signal of benefit in six patients with SLE. In this report, CRS was also common, but no case of CRS was more severe than grade 2. There was no neurotoxicity. Infections did occur but were of relatively mild grades and resolved with treatment.

For efficacy in the ongoing follow-up, SLE symptoms as measured with the SLE Disease Activity Index began to abate at about 14 days after the single-infusion treatment. Improvement on the Physician Global Assessment was also observed between 14 and 28 days. C3 and C4 complement levels started to rise at about 28 days. While the responses have correlated with the observed changes in biomarkers of immune function, they have endured through a median follow-up that now exceeds 6 months.
 

Complete B-Cell Depletion Is Followed by Full Recovery

“Pharmacokinetic and pharmacodynamic studies revealed peak expansion of CAR T cells approximately 13-21 days post infusion, which was accompanied by deep B-cell depletion followed by subsequent B-cell recovery,” reported Josefina Cortés-Hernández, MD, PhD, a senior lecturer at Vall d’Hebron Research Institute, Barcelona, Spain.

Dr. Schett had reported the same pattern of expansion followed by a rapid elimination of detectable CAR T cells despite the sustained clinical benefit.

Dr. Cortés-Hernández said that the signal of efficacy in the context of acceptable safety supports an expansion of clinical studies with this CAR T-cell product in SLE and perhaps other autoimmune disorders.

In another early-stage study, patients with SLE who had failed multiple prior lines of therapy have been enrolled in an ongoing study with a compound CAR (cCAR) T cell. This experimental proprietary product (iCAR Bio Therapeutics, Zhongshan, China) targets both the B-cell maturation antigen and CD19, according to Greg Deener, the chief executive officer of iCell Gene Therapeutics, New York City.

Ted Bosworth/Medscape Medical News

cCAR T-Cell Construct Targets Immune Reset

With this construct, the goal is to deplete long-lived plasma cells as well as B cells in order to achieve a more complete humoral reset. While preliminary data from the phase 1 trial were published earlier this year in Annals of the Rheumatic Diseases, Mr. Deener focused his presentation at EULAR 2024 on 12 patients with SLE and lupus nephritis, a severe form of SLE that threatens glomerular structures and can lead to end-stage liver disease.

B cells in the peripheral blood could not be detected within 10 days of the cCAR infusion, and the immunoglobulins IgM and IgA were undetectable by day 42.

However, after B-cell recovery by day 150, “flow cytometry and B-cell receptor sequencing confirmed full humoral reset was achieved,” Mr. Deener said.

The remission has been durable in 11 of the 12 patients after a mean follow-up of 458 days, Mr. Deener reported. He noted that an improvement in renal function has been observed in the majority of patients.

Like others, he reported that treatment has been relatively well tolerated. In this series of patients, there have been no cases of CRS more severe than grade 1.

Overall, the cCAR data in lupus nephritis support the hypothesis that CAR T cells are reprogramming the immune system, according to Mr. Deener.

Combined with a reasonable safety profile, the consistency of benefit from CAR T cells in autoimmune rheumatic diseases is good news, but all of the investigators who spoke at EULAR agreed that there are still many unanswered questions. Not least, it is unclear whether patients can be effectively and safely retreated when and if relapses occur. Even though Dr. Schett did report a response with retreatment following a relapse, he said that there is no conclusion to draw from a single patient.

Yet, the high rates of remissions in patients with disease refractory to other therapeutic options is highly encouraging, particularly with the manageable side effects now reported by multiple investigators using different CAR T-cell products.

“Roughly 100 patients with rheumatic diseases have been treated with CAR T-cells, and we have not seen a high-grade CRS or neurotoxicity,” he said.

Long-term efficacy is less clear. With the first clinical studies in autoimmune diseases initiated in 2021, few patients have been followed for more than 2 years. Even with the high rates of response that will certainly fuel efforts to rapidly bring these treatments forward, long-term data are now the missing piece.
 

Other Case Series Presented at EULAR

Several other abstracts reported on patients with SSc who were treated with CD19-targeting CAR T cells:

Three patients for whom autologous hematopoietic stem cell transplantation was contraindicated or unsuccessful were successfully and safely treated.

Six patients with diffuse and progressive disease achieved stable disease activity without additional immunosuppression for up to 1 year after treatment.

Dr. Schett reported no potential conflicts of interest, and the study he presented was not funded by industry. Dr. Cortés-Hernández reported a financial relationship with Novartis, which funded the study of the CAR T-cell therapy YTB323, as well as with GlaxoSmithKline, which was not involved in the study she presented.  Mr. Deener is an employee of iCell Gene Therapeutics, which provided funding for the trial he presented.

August 7, 2024 — Editor's note: This article was updated with additional disclosure information for Dr. Josefina Cortés-Hernández.

A version of this article appeared on Medscape.com.

VIENNA — From a dozen or so studies and sessions devoted to the role of chimeric antigen receptor (CAR) T cells in rheumatic diseases at the annual European Congress of Rheumatology, the message was uniformly positive, supporting growing evidence that drugs in this class are heading toward a paradigm shift in refractory rheumatic diseases.

Of the reports, an update from a 15-patient case series with at least 1 year of follow-up provides “the first long-term evidence of safety and efficacy in multiple rheumatic diseases,” according to Georg Schett, MD, PhD, director of rheumatology and immunology, University of Erlangen-Nürnberg, Erlangen, Germany.

Ted Bosworth/Medscape Medical News

The report of high rates of activity and low relative risk of serious adverse events from the same series was published earlier this year in The New England Journal of Medicine when the median follow-up was 15 months. Almost all of the patients have now completed at least 1 year of follow-up and about a third have completed more than 2 years.
 

SLE Is Frequently Targeted in CAR T-Cell Studies

The three rheumatic diseases represented in this series of patients, all of whom had failed multiple previous immune suppressive treatments, were systemic lupus erythematosus (SLE), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). After the autologous T cells were harvested, they were expanded and transfected with the CD19 CAR. The proprietary investigational product, called MB-CART19.1 (Miltenyi Biotec), was administered in a single dose of one million cells per kg bodyweight.

The response rates have been, and continue to be, impressive. For the eight patients with SLE, all achieved the definition of remission in SLE criteria after one dose of treatment. Complete resolution of all major symptom types was achieved after 6 months of follow-up. So far, no patient has relapsed.

For the three patients with IIM, all reached the American College of Rheumatology–EULAR criteria for a major response. All creatine kinase levels had normalized by 3 months. In this group, there was one relapse, which occurred after 18 months of follow-up.

All four patients with SSc achieved a major response on the European Scleroderma Trials and Research (EUSTAR) group activity index. The median reduction from baseline in the EUSTAR score was 4.2 points, and this has been maintained in follow-up to date.
 

Remissions Have Persisted Off All Therapies

These remissions were achieved and maintained after a single dose of CAR T-cell therapy despite discontinuation of all immunosuppressive therapies. With the exception of the single relapse, all remissions have persisted through follow-up to date.

These responses were achieved with manageable side effects, according to Dr. Schett. The most serious adverse event was a grade 4 neutropenia that developed 4 months after receiving CAR T cells. It resolved with granulocyte colony-stimulating factor treatment. Cytokine release syndrome (CRS) has occurred in 10 patients, but it was grade 1 in eight patients and grade 2 in the others. There has been no neurotoxicity.

Almost all patients have experienced an infection during follow-up, but there has been no discernible pattern in relationship to the timing or types of infections. The most common have involved the upper respiratory tract and have been of mild severity, with cases disseminated similarly over early vs late follow-up. There was one case of pneumonia involving antibiotic treatment and a hospital stay, but it resolved.

Dr. Schett acknowledged that safety is a bigger concern in autoimmune diseases, which are often serious but rarely fatal, than in the hematologic malignancies for which CAR T cells were initially tested, but the low rates of serious adverse events in his and other early studies have supported the premise that the risks are not the same.

Asked specifically if CAR T cells can be considered a game changer in autoimmune rheumatic diseases, Dr. Schett was cautious. One reason is the CAR T cells are a complex therapy relative to biologic disease-modifying antirheumatic drugs. He thinks, therefore, that much more data are needed to confirm safety and efficacy. In addition, they are expensive, so it is not yet clear how they will be integrated with other options.

Yet, he thinks the evidence so far suggests a profound effect on the fundamental drivers of autoimmune disease. Their specific mechanism of benefit is still being evaluated, but he considers the clinical responses consistent with a “reset” hypothesis.

After a response, “we are seeing drug-free remissions in some patients as long as they have been followed,” Dr. Schett said. Based on the fact that disease control is being observed off all other therapies, “this only makes sense to me if there is some sort of immunologic reset.”
 

CAR T-Cell Studies in Autoimmune Diseases Are Proliferating

At last count, there were about 40 studies being performed with CAR T cells in various autoimmune diseases, most of which were rheumatologic disorders, according to Dr. Schett. He noted that funding is coming from multinational drug companies, small biotech startups, and investigator-initiated studies at academic centers.

At EULAR, beyond case studies and anecdotal reports, all of the clinical studies were still at the level of phase 1 or 1/2. Consistent with the data presented by Dr. Schett, the drugs have been nearly uniformly effective, with major responses persisting in patients off other therapies. Adverse events have been manageable.

Examples include a phase 1/2 multinational study with the investigational CAR T-cell therapy YTB323 (Novartis), which demonstrated acceptable safety and a strong signal of benefit in six patients with SLE. In this report, CRS was also common, but no case of CRS was more severe than grade 2. There was no neurotoxicity. Infections did occur but were of relatively mild grades and resolved with treatment.

For efficacy in the ongoing follow-up, SLE symptoms as measured with the SLE Disease Activity Index began to abate at about 14 days after the single-infusion treatment. Improvement on the Physician Global Assessment was also observed between 14 and 28 days. C3 and C4 complement levels started to rise at about 28 days. While the responses have correlated with the observed changes in biomarkers of immune function, they have endured through a median follow-up that now exceeds 6 months.
 

Complete B-Cell Depletion Is Followed by Full Recovery

“Pharmacokinetic and pharmacodynamic studies revealed peak expansion of CAR T cells approximately 13-21 days post infusion, which was accompanied by deep B-cell depletion followed by subsequent B-cell recovery,” reported Josefina Cortés-Hernández, MD, PhD, a senior lecturer at Vall d’Hebron Research Institute, Barcelona, Spain.

Dr. Schett had reported the same pattern of expansion followed by a rapid elimination of detectable CAR T cells despite the sustained clinical benefit.

Dr. Cortés-Hernández said that the signal of efficacy in the context of acceptable safety supports an expansion of clinical studies with this CAR T-cell product in SLE and perhaps other autoimmune disorders.

In another early-stage study, patients with SLE who had failed multiple prior lines of therapy have been enrolled in an ongoing study with a compound CAR (cCAR) T cell. This experimental proprietary product (iCAR Bio Therapeutics, Zhongshan, China) targets both the B-cell maturation antigen and CD19, according to Greg Deener, the chief executive officer of iCell Gene Therapeutics, New York City.

Ted Bosworth/Medscape Medical News

cCAR T-Cell Construct Targets Immune Reset

With this construct, the goal is to deplete long-lived plasma cells as well as B cells in order to achieve a more complete humoral reset. While preliminary data from the phase 1 trial were published earlier this year in Annals of the Rheumatic Diseases, Mr. Deener focused his presentation at EULAR 2024 on 12 patients with SLE and lupus nephritis, a severe form of SLE that threatens glomerular structures and can lead to end-stage liver disease.

B cells in the peripheral blood could not be detected within 10 days of the cCAR infusion, and the immunoglobulins IgM and IgA were undetectable by day 42.

However, after B-cell recovery by day 150, “flow cytometry and B-cell receptor sequencing confirmed full humoral reset was achieved,” Mr. Deener said.

The remission has been durable in 11 of the 12 patients after a mean follow-up of 458 days, Mr. Deener reported. He noted that an improvement in renal function has been observed in the majority of patients.

Like others, he reported that treatment has been relatively well tolerated. In this series of patients, there have been no cases of CRS more severe than grade 1.

Overall, the cCAR data in lupus nephritis support the hypothesis that CAR T cells are reprogramming the immune system, according to Mr. Deener.

Combined with a reasonable safety profile, the consistency of benefit from CAR T cells in autoimmune rheumatic diseases is good news, but all of the investigators who spoke at EULAR agreed that there are still many unanswered questions. Not least, it is unclear whether patients can be effectively and safely retreated when and if relapses occur. Even though Dr. Schett did report a response with retreatment following a relapse, he said that there is no conclusion to draw from a single patient.

Yet, the high rates of remissions in patients with disease refractory to other therapeutic options is highly encouraging, particularly with the manageable side effects now reported by multiple investigators using different CAR T-cell products.

“Roughly 100 patients with rheumatic diseases have been treated with CAR T-cells, and we have not seen a high-grade CRS or neurotoxicity,” he said.

Long-term efficacy is less clear. With the first clinical studies in autoimmune diseases initiated in 2021, few patients have been followed for more than 2 years. Even with the high rates of response that will certainly fuel efforts to rapidly bring these treatments forward, long-term data are now the missing piece.
 

Other Case Series Presented at EULAR

Several other abstracts reported on patients with SSc who were treated with CD19-targeting CAR T cells:

Three patients for whom autologous hematopoietic stem cell transplantation was contraindicated or unsuccessful were successfully and safely treated.

Six patients with diffuse and progressive disease achieved stable disease activity without additional immunosuppression for up to 1 year after treatment.

Dr. Schett reported no potential conflicts of interest, and the study he presented was not funded by industry. Dr. Cortés-Hernández reported a financial relationship with Novartis, which funded the study of the CAR T-cell therapy YTB323, as well as with GlaxoSmithKline, which was not involved in the study she presented.  Mr. Deener is an employee of iCell Gene Therapeutics, which provided funding for the trial he presented.

August 7, 2024 — Editor's note: This article was updated with additional disclosure information for Dr. Josefina Cortés-Hernández.

A version of this article appeared on Medscape.com.

Nonphysician operators (NPOs) using laser and energy-based devices are accounting for an increasing share of malpractice lawsuits in dermatology, and when they use these devices in a dermatologic practice or a dermatologist-owned medical spa, the dermatologist can be on the hook for liability. Dermatologists can protect themselves by understanding malpractice trends and taking preventive steps, such as making sure NPOs have appropriate training and using a rigorous informed consent process, according to a dermatology resident and a dermatologist who have researched recent trends in dermatology lawsuits.

“It’s really important that physicians recognize their responsibility when delegating procedures to nonphysician operators and the physician’s role in supervision of these procedures,” Scott Stratman, MD, MPH, a dermatology resident at the Icahn School of Medicine at Mount Sinai, New York City, told this news organization. He led a  study recently published in the Journal of the American Academy of Dermatology, which found that the majority (52%) of malpractice cases for cutaneous energy-based device procedures in the LexisNexis database from 1985 to September 2023 involved NPOs. The study did not break the data down between different types of NPOs.

Dr. Stratman

Trends in Dermatology Malpractice

This follows a similar trend reported in a 2014 study led by Mathew M. Avram, MD, JD, director of the MGH Dermatology Laser and Cosmetic Center at Massachusetts General Hospital, Boston. The study analyzed liability claims related to cutaneous laser surgery performed by nonphysicians from January 1999 to December 2012.

“With nonphysician litigation data, we saw trend lines beginning in 2008 where the proportion of cases began to increase,” Dr. Avram said at the American Society for Laser Medicine and Surgery (ASLMS) meeting on April 12, 2024. “Over a period of 2008-2012, it went from 36% of cases to about 78%,” he said.

About a quarter (23.4%) of those were in medical offices; 76.6% were in nontraditional settings such as medical spas, he added. The proportion of NPOs was similar in a 2022 study that looked at causes of litigation in cutaneous laser surgery from 2012 to 2020, Dr. Avram said. Again, neither study broke down cases involving NPOs by specific type, but the 2014 study reported that 64% of cases by NPOs occurred outside of a traditional medical setting.

“So it seems that the location and potentially the supervision are issues that are important to patient safety,” Dr. Avram said at the meeting. While state laws regarding laser delegation vary widely, “depending on where you practice, it’s incumbent upon you to know that.”

Dr. Avram and colleagues were also the authors of a study published in June in Dermatologic Surgery that looked at the reasons behind ligations involving dermatologists in a retrospective analysis of 48 state and federal cases between 2011 and 2022. The majority of cases — 54.2% — were for unexpected harm, followed by wrong or delayed diagnoses, which accounted for a third of litigations.

Dr. Stratman’s study found that laser hair removal was the most common procedure for malpractice claims in dermatology among cutaneous energy-based device procedures. Complications from energy-based devices included burns, scarring, and pigmentation changes.

The growth of malpractice suits involving NPOs could be because NPOs are performing a greater proportion of dermatologic procedures, “particularly those practicing without direct supervision, such as in the context of a medical spa,” Dr. Stratman said in the interview. “Again, this highlights a physician’s responsibility in delegating these kinds of procedures to NPOs.”
 

Training Is a Must — But Not Standardized

Comprehensive training for physicians, staff, NPOs, and physicians “is all necessary and paramount in order to diminish adverse outcomes and legal risk, and then, of course, all these practitioners, be it staff or [NPOs], and, of course, physicians, are all held to the same standard of care,” Dr. Stratman said.

However, he added, “There is really no standardized training to operate these devices. That being said, it’s really important to know that both providers and facility owners have a significant obligation to their patients to make sure that their staff in their centers are appropriately trained.”

Training not only involves protocols and procedures but also how to handle patient interactions, Dr. Stratman said.

The legal concept of respondeat superior applies when nonphysicians participate in a patient’s care, Dr. Avram said at the ASLMS meeting. The physician is held liable for a nonphysician’s “negligence provided he or she is an employee receiving a salary [and] benefits and is performing within the scope of his or her duty,” regardless of whether the physician saw the patient or not at that visit, he said. Again, supervision of nonphysician laser procedures varies from state to state, he added.

“So the take-home point is to provide excellent training and appropriate supervision, and if you’re the owner of that practice, you are liable in the event of negligence even though you never were part of the treatment,” Dr. Avram said.

Ins and Outs of Informed Consent

When a patient outcome is less than desirable, or at least less than what the patient expected, a transparent and thorough informed consent process can protect the practice and physician, Dr. Avram said at the meeting.

“Malpractice and consent have nothing to do with each other,” he said. “Consent is getting permission to do a procedure. It’s needed actually for any medical intervention that you perform. What you need to do is to provide information to enable the patient or guardian or to choose knowledgeably among reasonable medical alternatives. This places the patient in control of the course of their medical treatment.”

The information conveyed to the patient should include the diagnosis, the medical causes, the nature and purpose of the treatment, and the risks and alternatives of procedure, “particularly if they’re high risk,” Dr. Avram said.

“Failure to obtain informed consent constitutes a civil battery, and the physician is liable for civil damages,” he said. “The patient need only show that he or she was not informed of the medical nature of the medical touching; physical injury is not necessary.”

A battery could occur if a procedure extends beyond the scope or area of treatment the patient agreed to — for example, extending a liposuction to an area that wasn’t originally targeted, or extending a laser procedure to an area of the body as a presumed favor to the patient. “It does not require a standard of care or an expert witness,” Dr. Avram said. “One only needs to show nonconsensual touching.”

Informed consents should include plain language, he said. “The whole idea is the patient understands what the risks and benefits are,” Dr. Avram said. “You don’t need to use medical jargon.” As an example, he suggested using the term “blisters” instead of “bullae.” If the treatment involves an off-label procedure, include that too, he said.

He also advised avoiding blanket authorizations. “Courts disfavor them,” he noted. “They need more specificity. So those are not valid.”

Dr. Stratman added that providers should think about the setting in which they obtain informed consent. “It’s really important that providers are consenting their patients in private and quiet places, free from distractions, that they accommodate patients who might have disabilities or limitations in English proficiency, using a teach-back method to help patients understand or demonstrate their understanding of the procedure in order to gauge comprehension,” he said.

Both Dr. Avram and Dr. Stratman pointed out that another strategy to prevent malpractice is to build trusting patient-provider relationships. “The patient-provider relationship is paramount not only to the success of the procedure but to the clinical visit as a whole,” Dr. Stratman said.

That’s a two-way street, he added. Patients should be able to trust that their provider provides them with the best treatment based on their own history, and providers should also be able to trust that patients are providing them with an accurate history, asking relevant questions, or expressing any level of apprehension about the procedure or visit. “The patient-provider relationship is everything,” Dr. Stratman said.

Dr. Stratman and Dr. Avram had no relevant disclosures.
 

A version of this article appeared on Medscape.com.

Nonphysician operators (NPOs) using laser and energy-based devices are accounting for an increasing share of malpractice lawsuits in dermatology, and when they use these devices in a dermatologic practice or a dermatologist-owned medical spa, the dermatologist can be on the hook for liability. Dermatologists can protect themselves by understanding malpractice trends and taking preventive steps, such as making sure NPOs have appropriate training and using a rigorous informed consent process, according to a dermatology resident and a dermatologist who have researched recent trends in dermatology lawsuits.

“It’s really important that physicians recognize their responsibility when delegating procedures to nonphysician operators and the physician’s role in supervision of these procedures,” Scott Stratman, MD, MPH, a dermatology resident at the Icahn School of Medicine at Mount Sinai, New York City, told this news organization. He led a  study recently published in the Journal of the American Academy of Dermatology, which found that the majority (52%) of malpractice cases for cutaneous energy-based device procedures in the LexisNexis database from 1985 to September 2023 involved NPOs. The study did not break the data down between different types of NPOs.

Dr. Stratman

Trends in Dermatology Malpractice

This follows a similar trend reported in a 2014 study led by Mathew M. Avram, MD, JD, director of the MGH Dermatology Laser and Cosmetic Center at Massachusetts General Hospital, Boston. The study analyzed liability claims related to cutaneous laser surgery performed by nonphysicians from January 1999 to December 2012.

“With nonphysician litigation data, we saw trend lines beginning in 2008 where the proportion of cases began to increase,” Dr. Avram said at the American Society for Laser Medicine and Surgery (ASLMS) meeting on April 12, 2024. “Over a period of 2008-2012, it went from 36% of cases to about 78%,” he said.

About a quarter (23.4%) of those were in medical offices; 76.6% were in nontraditional settings such as medical spas, he added. The proportion of NPOs was similar in a 2022 study that looked at causes of litigation in cutaneous laser surgery from 2012 to 2020, Dr. Avram said. Again, neither study broke down cases involving NPOs by specific type, but the 2014 study reported that 64% of cases by NPOs occurred outside of a traditional medical setting.

“So it seems that the location and potentially the supervision are issues that are important to patient safety,” Dr. Avram said at the meeting. While state laws regarding laser delegation vary widely, “depending on where you practice, it’s incumbent upon you to know that.”

Dr. Avram and colleagues were also the authors of a study published in June in Dermatologic Surgery that looked at the reasons behind ligations involving dermatologists in a retrospective analysis of 48 state and federal cases between 2011 and 2022. The majority of cases — 54.2% — were for unexpected harm, followed by wrong or delayed diagnoses, which accounted for a third of litigations.

Dr. Stratman’s study found that laser hair removal was the most common procedure for malpractice claims in dermatology among cutaneous energy-based device procedures. Complications from energy-based devices included burns, scarring, and pigmentation changes.

The growth of malpractice suits involving NPOs could be because NPOs are performing a greater proportion of dermatologic procedures, “particularly those practicing without direct supervision, such as in the context of a medical spa,” Dr. Stratman said in the interview. “Again, this highlights a physician’s responsibility in delegating these kinds of procedures to NPOs.”
 

Training Is a Must — But Not Standardized

Comprehensive training for physicians, staff, NPOs, and physicians “is all necessary and paramount in order to diminish adverse outcomes and legal risk, and then, of course, all these practitioners, be it staff or [NPOs], and, of course, physicians, are all held to the same standard of care,” Dr. Stratman said.

However, he added, “There is really no standardized training to operate these devices. That being said, it’s really important to know that both providers and facility owners have a significant obligation to their patients to make sure that their staff in their centers are appropriately trained.”

Training not only involves protocols and procedures but also how to handle patient interactions, Dr. Stratman said.

The legal concept of respondeat superior applies when nonphysicians participate in a patient’s care, Dr. Avram said at the ASLMS meeting. The physician is held liable for a nonphysician’s “negligence provided he or she is an employee receiving a salary [and] benefits and is performing within the scope of his or her duty,” regardless of whether the physician saw the patient or not at that visit, he said. Again, supervision of nonphysician laser procedures varies from state to state, he added.

“So the take-home point is to provide excellent training and appropriate supervision, and if you’re the owner of that practice, you are liable in the event of negligence even though you never were part of the treatment,” Dr. Avram said.

Ins and Outs of Informed Consent

When a patient outcome is less than desirable, or at least less than what the patient expected, a transparent and thorough informed consent process can protect the practice and physician, Dr. Avram said at the meeting.

“Malpractice and consent have nothing to do with each other,” he said. “Consent is getting permission to do a procedure. It’s needed actually for any medical intervention that you perform. What you need to do is to provide information to enable the patient or guardian or to choose knowledgeably among reasonable medical alternatives. This places the patient in control of the course of their medical treatment.”

The information conveyed to the patient should include the diagnosis, the medical causes, the nature and purpose of the treatment, and the risks and alternatives of procedure, “particularly if they’re high risk,” Dr. Avram said.

“Failure to obtain informed consent constitutes a civil battery, and the physician is liable for civil damages,” he said. “The patient need only show that he or she was not informed of the medical nature of the medical touching; physical injury is not necessary.”

A battery could occur if a procedure extends beyond the scope or area of treatment the patient agreed to — for example, extending a liposuction to an area that wasn’t originally targeted, or extending a laser procedure to an area of the body as a presumed favor to the patient. “It does not require a standard of care or an expert witness,” Dr. Avram said. “One only needs to show nonconsensual touching.”

Informed consents should include plain language, he said. “The whole idea is the patient understands what the risks and benefits are,” Dr. Avram said. “You don’t need to use medical jargon.” As an example, he suggested using the term “blisters” instead of “bullae.” If the treatment involves an off-label procedure, include that too, he said.

He also advised avoiding blanket authorizations. “Courts disfavor them,” he noted. “They need more specificity. So those are not valid.”

Dr. Stratman added that providers should think about the setting in which they obtain informed consent. “It’s really important that providers are consenting their patients in private and quiet places, free from distractions, that they accommodate patients who might have disabilities or limitations in English proficiency, using a teach-back method to help patients understand or demonstrate their understanding of the procedure in order to gauge comprehension,” he said.

Both Dr. Avram and Dr. Stratman pointed out that another strategy to prevent malpractice is to build trusting patient-provider relationships. “The patient-provider relationship is paramount not only to the success of the procedure but to the clinical visit as a whole,” Dr. Stratman said.

That’s a two-way street, he added. Patients should be able to trust that their provider provides them with the best treatment based on their own history, and providers should also be able to trust that patients are providing them with an accurate history, asking relevant questions, or expressing any level of apprehension about the procedure or visit. “The patient-provider relationship is everything,” Dr. Stratman said.

Dr. Stratman and Dr. Avram had no relevant disclosures.
 

A version of this article appeared on Medscape.com.

Nonphysician operators (NPOs) using laser and energy-based devices are accounting for an increasing share of malpractice lawsuits in dermatology, and when they use these devices in a dermatologic practice or a dermatologist-owned medical spa, the dermatologist can be on the hook for liability. Dermatologists can protect themselves by understanding malpractice trends and taking preventive steps, such as making sure NPOs have appropriate training and using a rigorous informed consent process, according to a dermatology resident and a dermatologist who have researched recent trends in dermatology lawsuits.

“It’s really important that physicians recognize their responsibility when delegating procedures to nonphysician operators and the physician’s role in supervision of these procedures,” Scott Stratman, MD, MPH, a dermatology resident at the Icahn School of Medicine at Mount Sinai, New York City, told this news organization. He led a  study recently published in the Journal of the American Academy of Dermatology, which found that the majority (52%) of malpractice cases for cutaneous energy-based device procedures in the LexisNexis database from 1985 to September 2023 involved NPOs. The study did not break the data down between different types of NPOs.

Dr. Stratman

Trends in Dermatology Malpractice

This follows a similar trend reported in a 2014 study led by Mathew M. Avram, MD, JD, director of the MGH Dermatology Laser and Cosmetic Center at Massachusetts General Hospital, Boston. The study analyzed liability claims related to cutaneous laser surgery performed by nonphysicians from January 1999 to December 2012.

“With nonphysician litigation data, we saw trend lines beginning in 2008 where the proportion of cases began to increase,” Dr. Avram said at the American Society for Laser Medicine and Surgery (ASLMS) meeting on April 12, 2024. “Over a period of 2008-2012, it went from 36% of cases to about 78%,” he said.

About a quarter (23.4%) of those were in medical offices; 76.6% were in nontraditional settings such as medical spas, he added. The proportion of NPOs was similar in a 2022 study that looked at causes of litigation in cutaneous laser surgery from 2012 to 2020, Dr. Avram said. Again, neither study broke down cases involving NPOs by specific type, but the 2014 study reported that 64% of cases by NPOs occurred outside of a traditional medical setting.

“So it seems that the location and potentially the supervision are issues that are important to patient safety,” Dr. Avram said at the meeting. While state laws regarding laser delegation vary widely, “depending on where you practice, it’s incumbent upon you to know that.”

Dr. Avram and colleagues were also the authors of a study published in June in Dermatologic Surgery that looked at the reasons behind ligations involving dermatologists in a retrospective analysis of 48 state and federal cases between 2011 and 2022. The majority of cases — 54.2% — were for unexpected harm, followed by wrong or delayed diagnoses, which accounted for a third of litigations.

Dr. Stratman’s study found that laser hair removal was the most common procedure for malpractice claims in dermatology among cutaneous energy-based device procedures. Complications from energy-based devices included burns, scarring, and pigmentation changes.

The growth of malpractice suits involving NPOs could be because NPOs are performing a greater proportion of dermatologic procedures, “particularly those practicing without direct supervision, such as in the context of a medical spa,” Dr. Stratman said in the interview. “Again, this highlights a physician’s responsibility in delegating these kinds of procedures to NPOs.”
 

Training Is a Must — But Not Standardized

Comprehensive training for physicians, staff, NPOs, and physicians “is all necessary and paramount in order to diminish adverse outcomes and legal risk, and then, of course, all these practitioners, be it staff or [NPOs], and, of course, physicians, are all held to the same standard of care,” Dr. Stratman said.

However, he added, “There is really no standardized training to operate these devices. That being said, it’s really important to know that both providers and facility owners have a significant obligation to their patients to make sure that their staff in their centers are appropriately trained.”

Training not only involves protocols and procedures but also how to handle patient interactions, Dr. Stratman said.

The legal concept of respondeat superior applies when nonphysicians participate in a patient’s care, Dr. Avram said at the ASLMS meeting. The physician is held liable for a nonphysician’s “negligence provided he or she is an employee receiving a salary [and] benefits and is performing within the scope of his or her duty,” regardless of whether the physician saw the patient or not at that visit, he said. Again, supervision of nonphysician laser procedures varies from state to state, he added.

“So the take-home point is to provide excellent training and appropriate supervision, and if you’re the owner of that practice, you are liable in the event of negligence even though you never were part of the treatment,” Dr. Avram said.

Ins and Outs of Informed Consent

When a patient outcome is less than desirable, or at least less than what the patient expected, a transparent and thorough informed consent process can protect the practice and physician, Dr. Avram said at the meeting.

“Malpractice and consent have nothing to do with each other,” he said. “Consent is getting permission to do a procedure. It’s needed actually for any medical intervention that you perform. What you need to do is to provide information to enable the patient or guardian or to choose knowledgeably among reasonable medical alternatives. This places the patient in control of the course of their medical treatment.”

The information conveyed to the patient should include the diagnosis, the medical causes, the nature and purpose of the treatment, and the risks and alternatives of procedure, “particularly if they’re high risk,” Dr. Avram said.

“Failure to obtain informed consent constitutes a civil battery, and the physician is liable for civil damages,” he said. “The patient need only show that he or she was not informed of the medical nature of the medical touching; physical injury is not necessary.”

A battery could occur if a procedure extends beyond the scope or area of treatment the patient agreed to — for example, extending a liposuction to an area that wasn’t originally targeted, or extending a laser procedure to an area of the body as a presumed favor to the patient. “It does not require a standard of care or an expert witness,” Dr. Avram said. “One only needs to show nonconsensual touching.”

Informed consents should include plain language, he said. “The whole idea is the patient understands what the risks and benefits are,” Dr. Avram said. “You don’t need to use medical jargon.” As an example, he suggested using the term “blisters” instead of “bullae.” If the treatment involves an off-label procedure, include that too, he said.

He also advised avoiding blanket authorizations. “Courts disfavor them,” he noted. “They need more specificity. So those are not valid.”

Dr. Stratman added that providers should think about the setting in which they obtain informed consent. “It’s really important that providers are consenting their patients in private and quiet places, free from distractions, that they accommodate patients who might have disabilities or limitations in English proficiency, using a teach-back method to help patients understand or demonstrate their understanding of the procedure in order to gauge comprehension,” he said.

Both Dr. Avram and Dr. Stratman pointed out that another strategy to prevent malpractice is to build trusting patient-provider relationships. “The patient-provider relationship is paramount not only to the success of the procedure but to the clinical visit as a whole,” Dr. Stratman said.

That’s a two-way street, he added. Patients should be able to trust that their provider provides them with the best treatment based on their own history, and providers should also be able to trust that patients are providing them with an accurate history, asking relevant questions, or expressing any level of apprehension about the procedure or visit. “The patient-provider relationship is everything,” Dr. Stratman said.

Dr. Stratman and Dr. Avram had no relevant disclosures.
 

A version of this article appeared on Medscape.com.

Patients who live in areas with high levels of sun exposure may require higher than normal doses of botulinum toxin A to the glabella to achieve maximal results, findings from a comparative cohort study suggested.

“Botulinum toxin A to the glabella is a popular cosmetic intervention,” researchers led by Kim L. Borsky, MD, MBBS, of the Department of Plastic and Reconstructive Surgery at Stoke Mandeville Hospital, Aylesbury, England, and colleagues wrote in their study, which was published in Plastic and Reconstructive Surgery. “Functional musculature differences may arise from chronic behavioral adjustment to high sun exposure levels, requiring greater doses. This could affect clinical practice globally.”

To investigate the effect of climate on real-world doses of the product, the researchers enrolled 523 women aged 35-60 years who received glabellar botulinum toxin treatment at two centers between 2012 and 2019: one in the United Kingdom and one in Malta. They evaluated data on 292 patients treated during the summer months at the Malta center (classified as the high sun-exposure group), and 231 patients treated during the winter months at the UK center (classified as the low sun-exposure group). The primary outcomes of interest were the required top-up doses and the total dose to achieve full paralysis. Smokers were excluded from the analysis, as were those who did not seek maximal paralysis, those documented as not compliant with posttreatment advice, and those with colds or fevers. They used univariable and multivariable analyses to compare the high vs low sun-exposure groups.

The researchers found that 68.5% of women in the high-sun group required a top-up dose to achieve full paralysis, compared with 61.5% in the low-sun group, a difference that did not reach statistical significance (P = .1032). All patients achieved full paralysis with the treatment protocol used. However, in the high-sun group, the mean top-up dose was significantly higher than that in the low-sun group (a mean of 9.30 vs 7.06 units, respectively; P = .0009), as was the mean total dose (a mean of 29.23 vs 27.25 units; P = .0031).

“Patients subject to less sun exposure require a lower dose than patients with high sun exposure, and this was present and persisted when controlling for potential confounders,” the researchers wrote. “Although robustly demonstrated, the difference in doses seen here was small, and so may not directly impact at a health economic level, as the difference would not necessarily change the number of vials used. However, it may be of relevance to training and protocolization of treatments. Rigid protocols about doses and distributions may lead to undertreatment if applied in sunnier climates.”

They acknowledged certain limitations of their study, including its unblinded design and the fact that they did not evaluate or control for ethnicity. They also characterized the population of Malta as “very homogeneous, mainly made up of Maltese with less than 5% foreigners,” while the demographics of the United Kingdom and especially London, where the injections were performed, “are much more diverse.”

Asked to comment on the results, Pooja Sodha, MD, director of the Center for Laser and Cosmetic Dermatology at George Washington University, Washington, DC, said that the study highlights the importance of tailoring neuromodulator treatment to the individual patient based not just on gender but also on lifestyle and climate. “The conclusion [of the study] is logical, but it’s encouraging that the data supports this,” Dr. Sodha told this news organization. “The potential confounders, such as injection technique (5 point vs 3 point), nonblinding of the evaluator, history of prior treatments, and variation in treatment effect by different botulinum toxin products may be important as well in how we consider this data in practice.”

This study did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors. Neither the researchers nor Dr. Sodha reported having financial disclosures.

A version of this article appeared on Medscape.com.

Patients who live in areas with high levels of sun exposure may require higher than normal doses of botulinum toxin A to the glabella to achieve maximal results, findings from a comparative cohort study suggested.

“Botulinum toxin A to the glabella is a popular cosmetic intervention,” researchers led by Kim L. Borsky, MD, MBBS, of the Department of Plastic and Reconstructive Surgery at Stoke Mandeville Hospital, Aylesbury, England, and colleagues wrote in their study, which was published in Plastic and Reconstructive Surgery. “Functional musculature differences may arise from chronic behavioral adjustment to high sun exposure levels, requiring greater doses. This could affect clinical practice globally.”

To investigate the effect of climate on real-world doses of the product, the researchers enrolled 523 women aged 35-60 years who received glabellar botulinum toxin treatment at two centers between 2012 and 2019: one in the United Kingdom and one in Malta. They evaluated data on 292 patients treated during the summer months at the Malta center (classified as the high sun-exposure group), and 231 patients treated during the winter months at the UK center (classified as the low sun-exposure group). The primary outcomes of interest were the required top-up doses and the total dose to achieve full paralysis. Smokers were excluded from the analysis, as were those who did not seek maximal paralysis, those documented as not compliant with posttreatment advice, and those with colds or fevers. They used univariable and multivariable analyses to compare the high vs low sun-exposure groups.

The researchers found that 68.5% of women in the high-sun group required a top-up dose to achieve full paralysis, compared with 61.5% in the low-sun group, a difference that did not reach statistical significance (P = .1032). All patients achieved full paralysis with the treatment protocol used. However, in the high-sun group, the mean top-up dose was significantly higher than that in the low-sun group (a mean of 9.30 vs 7.06 units, respectively; P = .0009), as was the mean total dose (a mean of 29.23 vs 27.25 units; P = .0031).

“Patients subject to less sun exposure require a lower dose than patients with high sun exposure, and this was present and persisted when controlling for potential confounders,” the researchers wrote. “Although robustly demonstrated, the difference in doses seen here was small, and so may not directly impact at a health economic level, as the difference would not necessarily change the number of vials used. However, it may be of relevance to training and protocolization of treatments. Rigid protocols about doses and distributions may lead to undertreatment if applied in sunnier climates.”

They acknowledged certain limitations of their study, including its unblinded design and the fact that they did not evaluate or control for ethnicity. They also characterized the population of Malta as “very homogeneous, mainly made up of Maltese with less than 5% foreigners,” while the demographics of the United Kingdom and especially London, where the injections were performed, “are much more diverse.”

Asked to comment on the results, Pooja Sodha, MD, director of the Center for Laser and Cosmetic Dermatology at George Washington University, Washington, DC, said that the study highlights the importance of tailoring neuromodulator treatment to the individual patient based not just on gender but also on lifestyle and climate. “The conclusion [of the study] is logical, but it’s encouraging that the data supports this,” Dr. Sodha told this news organization. “The potential confounders, such as injection technique (5 point vs 3 point), nonblinding of the evaluator, history of prior treatments, and variation in treatment effect by different botulinum toxin products may be important as well in how we consider this data in practice.”

This study did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors. Neither the researchers nor Dr. Sodha reported having financial disclosures.

A version of this article appeared on Medscape.com.

Patients who live in areas with high levels of sun exposure may require higher than normal doses of botulinum toxin A to the glabella to achieve maximal results, findings from a comparative cohort study suggested.

“Botulinum toxin A to the glabella is a popular cosmetic intervention,” researchers led by Kim L. Borsky, MD, MBBS, of the Department of Plastic and Reconstructive Surgery at Stoke Mandeville Hospital, Aylesbury, England, and colleagues wrote in their study, which was published in Plastic and Reconstructive Surgery. “Functional musculature differences may arise from chronic behavioral adjustment to high sun exposure levels, requiring greater doses. This could affect clinical practice globally.”

To investigate the effect of climate on real-world doses of the product, the researchers enrolled 523 women aged 35-60 years who received glabellar botulinum toxin treatment at two centers between 2012 and 2019: one in the United Kingdom and one in Malta. They evaluated data on 292 patients treated during the summer months at the Malta center (classified as the high sun-exposure group), and 231 patients treated during the winter months at the UK center (classified as the low sun-exposure group). The primary outcomes of interest were the required top-up doses and the total dose to achieve full paralysis. Smokers were excluded from the analysis, as were those who did not seek maximal paralysis, those documented as not compliant with posttreatment advice, and those with colds or fevers. They used univariable and multivariable analyses to compare the high vs low sun-exposure groups.

The researchers found that 68.5% of women in the high-sun group required a top-up dose to achieve full paralysis, compared with 61.5% in the low-sun group, a difference that did not reach statistical significance (P = .1032). All patients achieved full paralysis with the treatment protocol used. However, in the high-sun group, the mean top-up dose was significantly higher than that in the low-sun group (a mean of 9.30 vs 7.06 units, respectively; P = .0009), as was the mean total dose (a mean of 29.23 vs 27.25 units; P = .0031).

“Patients subject to less sun exposure require a lower dose than patients with high sun exposure, and this was present and persisted when controlling for potential confounders,” the researchers wrote. “Although robustly demonstrated, the difference in doses seen here was small, and so may not directly impact at a health economic level, as the difference would not necessarily change the number of vials used. However, it may be of relevance to training and protocolization of treatments. Rigid protocols about doses and distributions may lead to undertreatment if applied in sunnier climates.”

They acknowledged certain limitations of their study, including its unblinded design and the fact that they did not evaluate or control for ethnicity. They also characterized the population of Malta as “very homogeneous, mainly made up of Maltese with less than 5% foreigners,” while the demographics of the United Kingdom and especially London, where the injections were performed, “are much more diverse.”

Asked to comment on the results, Pooja Sodha, MD, director of the Center for Laser and Cosmetic Dermatology at George Washington University, Washington, DC, said that the study highlights the importance of tailoring neuromodulator treatment to the individual patient based not just on gender but also on lifestyle and climate. “The conclusion [of the study] is logical, but it’s encouraging that the data supports this,” Dr. Sodha told this news organization. “The potential confounders, such as injection technique (5 point vs 3 point), nonblinding of the evaluator, history of prior treatments, and variation in treatment effect by different botulinum toxin products may be important as well in how we consider this data in practice.”

This study did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors. Neither the researchers nor Dr. Sodha reported having financial disclosures.

A version of this article appeared on Medscape.com.

Recent research on popular weight loss drugs has uncovered surprising benefits beyond their intended use, like lowering the risk of fatal heart attacks. And now there may be another unforeseen advantage: People with type 2 diabetes who took these drugs had a lower risk of having 10 out of 13 obesity-related cancers, compared to those who used insulin therapy.

That’s according to a study published July 5 in JAMA Network Open where researchers studied glucagon-like peptide receptor agonists (known as GLP-1RAs), a class of drugs used to treat diabetes and obesity. Ozempic, Wegovy, Mounjaro, and Zepbound, which have become well-known recently because they are linked to rapid weight loss, contain GLP-1RAs.

For the study, they looked at electronic health records of 1.7 million patients who had type 2 diabetes, no prior diagnosis of obesity-related cancers, and had been prescribed GLP-1RAs, insulins, or metformin from March 2005 to November 2018.

The scientists found that compared to patients who took insulin, people who took GLP-1RAs had a “significant risk reduction” in 10 of 13 obesity-related cancers. Those 10 cancers were esophageal, colorectal, endometrial, gallbladder, kidney, liver, ovarian, and pancreatic cancers, as well as meningioma and multiple myeloma.

Compared with patients taking insulin, patients taking GLP-1RAs showed no statistically significant reduction in stomach cancer and no reduced risk of breast and thyroid cancers, the study said.

But the study found no decrease in cancer risk with GLP-1RAs compared with metformin.

While the study results suggest that these drugs may reduce the risk of certain obesity-related cancers better than insulins, more research is needed, they said.

A version of this article appeared on WebMD.com.

Recent research on popular weight loss drugs has uncovered surprising benefits beyond their intended use, like lowering the risk of fatal heart attacks. And now there may be another unforeseen advantage: People with type 2 diabetes who took these drugs had a lower risk of having 10 out of 13 obesity-related cancers, compared to those who used insulin therapy.

That’s according to a study published July 5 in JAMA Network Open where researchers studied glucagon-like peptide receptor agonists (known as GLP-1RAs), a class of drugs used to treat diabetes and obesity. Ozempic, Wegovy, Mounjaro, and Zepbound, which have become well-known recently because they are linked to rapid weight loss, contain GLP-1RAs.

For the study, they looked at electronic health records of 1.7 million patients who had type 2 diabetes, no prior diagnosis of obesity-related cancers, and had been prescribed GLP-1RAs, insulins, or metformin from March 2005 to November 2018.

The scientists found that compared to patients who took insulin, people who took GLP-1RAs had a “significant risk reduction” in 10 of 13 obesity-related cancers. Those 10 cancers were esophageal, colorectal, endometrial, gallbladder, kidney, liver, ovarian, and pancreatic cancers, as well as meningioma and multiple myeloma.

Compared with patients taking insulin, patients taking GLP-1RAs showed no statistically significant reduction in stomach cancer and no reduced risk of breast and thyroid cancers, the study said.

But the study found no decrease in cancer risk with GLP-1RAs compared with metformin.

While the study results suggest that these drugs may reduce the risk of certain obesity-related cancers better than insulins, more research is needed, they said.

A version of this article appeared on WebMD.com.

Recent research on popular weight loss drugs has uncovered surprising benefits beyond their intended use, like lowering the risk of fatal heart attacks. And now there may be another unforeseen advantage: People with type 2 diabetes who took these drugs had a lower risk of having 10 out of 13 obesity-related cancers, compared to those who used insulin therapy.

That’s according to a study published July 5 in JAMA Network Open where researchers studied glucagon-like peptide receptor agonists (known as GLP-1RAs), a class of drugs used to treat diabetes and obesity. Ozempic, Wegovy, Mounjaro, and Zepbound, which have become well-known recently because they are linked to rapid weight loss, contain GLP-1RAs.

For the study, they looked at electronic health records of 1.7 million patients who had type 2 diabetes, no prior diagnosis of obesity-related cancers, and had been prescribed GLP-1RAs, insulins, or metformin from March 2005 to November 2018.

The scientists found that compared to patients who took insulin, people who took GLP-1RAs had a “significant risk reduction” in 10 of 13 obesity-related cancers. Those 10 cancers were esophageal, colorectal, endometrial, gallbladder, kidney, liver, ovarian, and pancreatic cancers, as well as meningioma and multiple myeloma.

Compared with patients taking insulin, patients taking GLP-1RAs showed no statistically significant reduction in stomach cancer and no reduced risk of breast and thyroid cancers, the study said.

But the study found no decrease in cancer risk with GLP-1RAs compared with metformin.

While the study results suggest that these drugs may reduce the risk of certain obesity-related cancers better than insulins, more research is needed, they said.

A version of this article appeared on WebMD.com.

Mount Sinai Health System in New York City is forcing part-time physicians to sign employment contracts that violate their labor rights, according to a June 2024 complaint by the National Labor Relations Board (NLRB). 

The complaint stems from no-poaching and confidentiality clauses in the agreements required as a condition of employment, NLRB officials alleged.

The contracts state that, for 1 year following termination, part-time physicians may not recruit, solicit, or induce to terminate the employment of any hospital system employee or independent contractor, according to a copy of the terms included in NLRB’s June 18 complaint. 

By requiring the agreements, NLRB officials claimed, Mount Sinai is “interfering with, restraining, and coercing employees” in violation of the National Labor Relations Act. The health system’s “unfair labor practices” affects commerce as outlined under the law, according to the NLRB. The Act bans employers from burdening or obstructing commerce or the free flow of commerce.

Mount Sinai did not respond to requests for comment.

The NLRB’s complaint follows a landmark decision by the Federal Trade Commission (FTC) to ban noncompete agreements nationwide. In April 2024, the FTC voted to prohibit noncompetes indefinitely in an effort to protect workers.

“Noncompete clauses keep wages low, suppress new ideas, and rob the American economy of dynamism, including from the more than 8500 new startups that would be created a year once noncompetes are banned,” FTC Chair Lina M. Khan said in a statement. “The FTC’s final rule to ban noncompetes will ensure Americans have the freedom to pursue a new job, start a new business, or bring a new idea to market.”

Business groups and agencies have since sued to challenge against the ban, including the Chamber of Commerce. The Chamber and other business groups argue that noncompete agreements are important for companies to protect trade secrets, shield recruiting investments, and hide confidential information. The lawsuits are ongoing. 
 

A Physician Blows the Whistle

An anonymous physician first alerted the NLRB to the contract language in November 2023. The doctor was required the sign the hospital system’s agreement for part-time physicians. The complaint does not say if the employee is still employed by the hospital system. 

To remedy the unfair labor practices alleged, the NLRB seeks an order requiring the health system to rescind the contract language, stop any actions against current or former employees to enforce the provisions, and make whole any employees who suffered financial losses related to the contract terms. 

The allegation against Mount Sinai is among a rising number of grievances filed with the NLRB that claim unfair labor practices. During the first 6 months of fiscal year 2024, unfair labor practice charges filed across the NLRB’s field offices increased 7% — from 9612 in 2023 to 10,278 in 2024, according to a news release. 

NLRB, meanwhile has been cracking down on anticompetitive labor practices and confidentiality provisions that prevent employees from speaking out. 

In a February 2023 decision for instance, NLRB ruled that an employer violates the National Labor Relations Act by offering severance agreements to workers that include restrictive confidentiality and nondisparagement terms. In 2022, the NLRB and the Federal Trade Commission forged a partnership to more widely combat unfair, anticompetitive, and deceptive business practices. 

“Noncompete provisions reasonably tend to chill employees in the exercise of Section 7 rights when the provisions could reasonably be construed by employees to deny them the ability to quit or change jobs by cutting off their access to other employment opportunities that they are qualified for,” NLRB General Counsel Jennifer Abruzzo said in a 2023 release. 

Ms. Abruzzo stressed in a memo that NLR Act is committed to an interagency approach to restrictions on the exercise of employee rights, “including limits to workers’ job mobility, information sharing, and referrals to other agencies.” 

Mount Sinai Health System must respond to the NLRB’s complaint by July 16, and an administrative law judge is scheduled to hear the case on September 24.

A version of this article first appeared on Medscape.com.

Mount Sinai Health System in New York City is forcing part-time physicians to sign employment contracts that violate their labor rights, according to a June 2024 complaint by the National Labor Relations Board (NLRB). 

The complaint stems from no-poaching and confidentiality clauses in the agreements required as a condition of employment, NLRB officials alleged.

The contracts state that, for 1 year following termination, part-time physicians may not recruit, solicit, or induce to terminate the employment of any hospital system employee or independent contractor, according to a copy of the terms included in NLRB’s June 18 complaint. 

By requiring the agreements, NLRB officials claimed, Mount Sinai is “interfering with, restraining, and coercing employees” in violation of the National Labor Relations Act. The health system’s “unfair labor practices” affects commerce as outlined under the law, according to the NLRB. The Act bans employers from burdening or obstructing commerce or the free flow of commerce.

Mount Sinai did not respond to requests for comment.

The NLRB’s complaint follows a landmark decision by the Federal Trade Commission (FTC) to ban noncompete agreements nationwide. In April 2024, the FTC voted to prohibit noncompetes indefinitely in an effort to protect workers.

“Noncompete clauses keep wages low, suppress new ideas, and rob the American economy of dynamism, including from the more than 8500 new startups that would be created a year once noncompetes are banned,” FTC Chair Lina M. Khan said in a statement. “The FTC’s final rule to ban noncompetes will ensure Americans have the freedom to pursue a new job, start a new business, or bring a new idea to market.”

Business groups and agencies have since sued to challenge against the ban, including the Chamber of Commerce. The Chamber and other business groups argue that noncompete agreements are important for companies to protect trade secrets, shield recruiting investments, and hide confidential information. The lawsuits are ongoing. 
 

A Physician Blows the Whistle

An anonymous physician first alerted the NLRB to the contract language in November 2023. The doctor was required the sign the hospital system’s agreement for part-time physicians. The complaint does not say if the employee is still employed by the hospital system. 

To remedy the unfair labor practices alleged, the NLRB seeks an order requiring the health system to rescind the contract language, stop any actions against current or former employees to enforce the provisions, and make whole any employees who suffered financial losses related to the contract terms. 

The allegation against Mount Sinai is among a rising number of grievances filed with the NLRB that claim unfair labor practices. During the first 6 months of fiscal year 2024, unfair labor practice charges filed across the NLRB’s field offices increased 7% — from 9612 in 2023 to 10,278 in 2024, according to a news release. 

NLRB, meanwhile has been cracking down on anticompetitive labor practices and confidentiality provisions that prevent employees from speaking out. 

In a February 2023 decision for instance, NLRB ruled that an employer violates the National Labor Relations Act by offering severance agreements to workers that include restrictive confidentiality and nondisparagement terms. In 2022, the NLRB and the Federal Trade Commission forged a partnership to more widely combat unfair, anticompetitive, and deceptive business practices. 

“Noncompete provisions reasonably tend to chill employees in the exercise of Section 7 rights when the provisions could reasonably be construed by employees to deny them the ability to quit or change jobs by cutting off their access to other employment opportunities that they are qualified for,” NLRB General Counsel Jennifer Abruzzo said in a 2023 release. 

Ms. Abruzzo stressed in a memo that NLR Act is committed to an interagency approach to restrictions on the exercise of employee rights, “including limits to workers’ job mobility, information sharing, and referrals to other agencies.” 

Mount Sinai Health System must respond to the NLRB’s complaint by July 16, and an administrative law judge is scheduled to hear the case on September 24.

A version of this article first appeared on Medscape.com.

Mount Sinai Health System in New York City is forcing part-time physicians to sign employment contracts that violate their labor rights, according to a June 2024 complaint by the National Labor Relations Board (NLRB). 

The complaint stems from no-poaching and confidentiality clauses in the agreements required as a condition of employment, NLRB officials alleged.

The contracts state that, for 1 year following termination, part-time physicians may not recruit, solicit, or induce to terminate the employment of any hospital system employee or independent contractor, according to a copy of the terms included in NLRB’s June 18 complaint. 

By requiring the agreements, NLRB officials claimed, Mount Sinai is “interfering with, restraining, and coercing employees” in violation of the National Labor Relations Act. The health system’s “unfair labor practices” affects commerce as outlined under the law, according to the NLRB. The Act bans employers from burdening or obstructing commerce or the free flow of commerce.

Mount Sinai did not respond to requests for comment.

The NLRB’s complaint follows a landmark decision by the Federal Trade Commission (FTC) to ban noncompete agreements nationwide. In April 2024, the FTC voted to prohibit noncompetes indefinitely in an effort to protect workers.

“Noncompete clauses keep wages low, suppress new ideas, and rob the American economy of dynamism, including from the more than 8500 new startups that would be created a year once noncompetes are banned,” FTC Chair Lina M. Khan said in a statement. “The FTC’s final rule to ban noncompetes will ensure Americans have the freedom to pursue a new job, start a new business, or bring a new idea to market.”

Business groups and agencies have since sued to challenge against the ban, including the Chamber of Commerce. The Chamber and other business groups argue that noncompete agreements are important for companies to protect trade secrets, shield recruiting investments, and hide confidential information. The lawsuits are ongoing. 
 

A Physician Blows the Whistle

An anonymous physician first alerted the NLRB to the contract language in November 2023. The doctor was required the sign the hospital system’s agreement for part-time physicians. The complaint does not say if the employee is still employed by the hospital system. 

To remedy the unfair labor practices alleged, the NLRB seeks an order requiring the health system to rescind the contract language, stop any actions against current or former employees to enforce the provisions, and make whole any employees who suffered financial losses related to the contract terms. 

The allegation against Mount Sinai is among a rising number of grievances filed with the NLRB that claim unfair labor practices. During the first 6 months of fiscal year 2024, unfair labor practice charges filed across the NLRB’s field offices increased 7% — from 9612 in 2023 to 10,278 in 2024, according to a news release. 

NLRB, meanwhile has been cracking down on anticompetitive labor practices and confidentiality provisions that prevent employees from speaking out. 

In a February 2023 decision for instance, NLRB ruled that an employer violates the National Labor Relations Act by offering severance agreements to workers that include restrictive confidentiality and nondisparagement terms. In 2022, the NLRB and the Federal Trade Commission forged a partnership to more widely combat unfair, anticompetitive, and deceptive business practices. 

“Noncompete provisions reasonably tend to chill employees in the exercise of Section 7 rights when the provisions could reasonably be construed by employees to deny them the ability to quit or change jobs by cutting off their access to other employment opportunities that they are qualified for,” NLRB General Counsel Jennifer Abruzzo said in a 2023 release. 

Ms. Abruzzo stressed in a memo that NLR Act is committed to an interagency approach to restrictions on the exercise of employee rights, “including limits to workers’ job mobility, information sharing, and referrals to other agencies.” 

Mount Sinai Health System must respond to the NLRB’s complaint by July 16, and an administrative law judge is scheduled to hear the case on September 24.

A version of this article first appeared on Medscape.com.

On any given day, oncologist Mark Lewis, MD, feels like he’s seesawing between two eras of technology. 

One minute, he’s working on sequencing a tumor genome. The next, he’s sifting through pages of disorganized data from a device that has been around for decades: the fax machine. 

“If two doctors’ offices aren’t on the same electronic medical record, one of the main ways to transfer records is still by fax,” said Dr. Lewis, director of gastrointestinal oncology at Intermountain Healthcare in Murray, Utah. “I can go from cutting-edge innovation to relying on, at best, 1980s information technology. It just boggles my mind.”

Dr. Lewis, who has posted about his frustration with fax machines, is far from alone. Oncologists are among the many specialists across the country at the mercy of telecopiers. 

According to a 2021 report by the Office of the National Coordinator for Health Information Technology, fax and mail continue to be the most common methods for hospitals and health systems to exchange care record summaries. In 2019, nearly 8 in 10 hospitals used mail or fax to send and receive health information, the report found. 

Fax machines are still commonplace across the healthcare spectrum, said Robert Havasy, MS, senior director for informatics strategy at the Healthcare Information and Management Systems Society (HIMSS). Inertia, cost, and more pressing priorities for hospitals and medical institutions contribute to the technology sticking around, he explained. 

“Post-COVID, my guess is we’re still at over 50% of healthcare practices using fax for some reason, on a daily basis,” Mr. Havasy said in an interview. “A lot of hospitals just don’t have the time, the money, or the staff to fix that problem because there’s always something a little higher up the priority chain they need to focus on.” 

If, for instance, “you’re going to do a process redesign to reduce hospital total acquired infections, your fax machine replacement might be 10th or 12th on the list. It just never gets up to 1 or 2 because it’s ‘not that much of a problem,’ ” he added.

Or is it?

Administrators may not view fax machines as a top concern, but clinicians who deal with the machines daily see it differently. 

“What worries me is we’re taking records out of an electronic storehouse [and] converting them to a paper medium,” Dr. Lewis said. “And then we are scanning into another electronic storehouse. The more steps, the more can be lost.”

And when information is lost, patient care can be compromised. 

Slower Workflows, Care Concerns

Although there are no published data on fax machine use in oncology specifically, this outdated technology does come into play in a variety of ways along the cancer care continuum. 

Radiation oncologist David R. Penberthy, MD, said patients often seek his cancer center’s expertise for second opinions, and that requires collecting patient records from many different practices. 

“Ideally, it would come electronically, but sometimes it does come by fax,” said Dr. Penberthy, program director of radiation oncology at the University of Virginia School of Medicine in Charlottesville. “The quality of the fax is not always the best. Sometimes it’s literally a fax of a fax. You’re reading something that’s very difficult to read.” 

Orders for new tests are also typically sent and received via fax temporarily while IT teams work to integrate them into the electronic health record (EHR), Dr. Penberthy said. 

Insurers and third-party laboratories often send test results back by fax as well.

“Even if I haven’t actually sent my patient out of our institution, this crucial result may only be entered back into the record as a scanned document from a fax, which is not great because it can get lost in the other results that are reported electronically,” Dr. Lewis said. The risk here is that an ordering physician won’t see these results, which can lead to delayed or overlooked care for patients, he explained.

“To me, it’s like a blind spot,” Dr. Lewis said. “Every time we use a fax, I see it actually as an opportunity for oversight and missed opportunity to collect data.”

Dr. Penberthy said faxing can slow things down at his practice, particularly if he faxes a document to another office but receives no confirmation and has to track down what happened. 

As for cybersecurity, data that are in transit during faxing are generally considered secure and compliant with the Health Insurance Portability and Accountability Act (HIPAA), said Mr. Havasy of HIMSS. However, the Privacy Rule also requires that data remain secure while at rest, which isn’t always possible, he added. 

“That’s where faxes fall down, because generally fax machines are in public, if you will, or open areas in a hospital,” he said. “They just sit on a desk. I don’t know that the next nurse who comes up and looks through that stack was the nurse who was treating the patient.” 

Important decisions or results can also be missed when sent by fax, creating headaches for physicians and care problems for patients. 

Dr. Lewis recently experienced an insurance-related fax mishap over Memorial Day weekend. He believed his patient had access to the antinausea medication he had prescribed. When Dr. Lewis happened to check the fax machine over the weekend, he found a coverage denial for the medication from the insurer but, at that point, had no recourse to appeal because it was a long holiday weekend. 

“Had the denial been sent by an electronic means that was quicker and more readily available, it would have been possible to appeal before the holiday weekend,” he said. 

Hematologist Aaron Goodman, MD, encountered a similar problem after an insurer denied coverage of an expensive cancer drug for a patient and faxed over its reason for the denial. Dr. Goodman was not directly notified that the information arrived and didn’t learn about the denial for a week, he said. 

“There’s no ‘ding’ in my inbox if something is faxed over and scanned,” said Dr. Goodman, associate professor of medicine at UC San Diego Health. “Once I realized it was denied, I was able to rectify it, but it wasted a week of a patient not getting a drug that I felt would be beneficial for them.”

Broader Health Policy Impacts

The use of outdated technology, such as fax machines, also creates ripple effects that burden the health system, health policy experts say. 

Duplicate testing and unnecessary care are top impacts, said Julia Adler-Milstein, PhD, professor of medicine and chief of the division of clinical informatics and digital transformation at the University of California, San Francisco.

Studies show that 20%-30% of the $65 billion spent annually on lab tests is used on unnecessary duplicate tests, and another estimated $30 billion is spent each year on unnecessary duplicate medical imaging. These duplicate tests may be mitigated if hospitals adopt certified EHR technology, research shows.

Still, without EHR interoperability between institutions, new providers may be unaware that tests or past labs for patients exist, leading to repeat tests, said Dr. Adler-Milstein, who researches health IT policy with a focus on EHRs. Patients can sometimes fill in the gaps, but not always. 

“Fax machines only help close information gaps if the clinician is aware of where to seek out the information and there is someone at the other organization to locate and transmit the information in a timely manner,” Dr. Adler-Milstein said. 

Old technology and poor interoperability also greatly affect data collection for disease surveillance and monitoring, said Janet Hamilton, MPH, executive director for the Council of State and Territorial Epidemiologists. This issue was keenly demonstrated during the pandemic, Ms. Hamilton said. 

“It was tragic, quite honestly,” she said. “There was such an immense amount of data that needed to be moved quickly, and that’s when computers are at their best.”

But, she said, “we didn’t have the level of systems in place to do it well.”

Specifically, the lack of electronic case reporting in place during the pandemic — where diagnoses are documented in the record and then immediately sent to the public health system — led to reports that were delayed, not made, or had missing or incomplete information, such as patients’ race and ethnicity or other health conditions, Ms. Hamilton said. 

Incomplete or missing data hampered the ability of public health officials and researchers to understand how the virus might affect different patients.

“If you had a chronic condition like cancer, you were less likely to have a positive outcome with COVID,” Ms. Hamilton said. “But because electronic case reporting was not in place, we didn’t get some of those additional pieces of information. We didn’t have people’s underlying oncology status to then say, ‘Here are individuals with these types of characteristics, and these are the things that happen if they also have a cancer.’” 

Slow, but Steady, Improvements

Efforts at the state and federal levels have targeted improved health information exchange, but progress takes time, Dr. Adler-Milstein said.

Most states have some form of health information exchange, such as statewide exchanges, regional health information organizations, or clinical data registries. Maryland is often held up as a notable example for its health information exchange, Dr. Adler-Milstein noted.

According to Maryland law, all hospitals under the jurisdiction of the Maryland Health Care Commission are required to electronically connect to the state-designated health information exchange. In 2012, Maryland became the first state to connect all its 46 acute care hospitals in the sharing of real-time data. 

The Health Information Technology for Economic and Clinical Health (HITECH) Act provided federal-enhanced Medicaid matching funds to states through 2021 to support efforts to advance electronic exchange. Nearly all states used these funds, and most have identified other sources to sustain the efforts, according to a recent US Government Accountability Office (GAO) report. However, GAO found that small and rural providers are less likely to have the financial and technological resources to participate in or maintain electronic exchange capabilities.

Nationally, several recent initiatives have targeted health data interoperability, including for cancer care. The Centers for Disease Control and Prevention’s Data Modernization Initiative is a multiyear, multi–billion-dollar effort to improve data sharing across the federal and state public health landscape. 

Meanwhile, in March 2024, the Biden-Harris administration launched United States Core Data for Interoperability Plus Cancer. The program will define a recommended minimum set of cancer-related data to be included in a patient’s EHR to enhance data exchange for research and clinical care. 

EHR vendors are also key to improving the landscape, said Dr. Adler-Milstein. Vendors such as Epic have developed strong sharing capabilities for transmitting health information from site to site, but of course, that only helps if providers have Epic, she said. 

“That’s where these national frameworks should help, because we don’t want it to break down by what EHR vendor you have,” she said. “It’s a patchwork. You can go to some places and hear success stories because they have Epic or a state health information exchange, but it’s very heterogeneous. In some places, they have nothing and are using a fax machine.”

Mr. Havasy believes fax machines will ultimately go extinct, particularly as a younger, more digitally savvy generation enters the healthcare workforce. He also foresees that the growing use of artificial intelligence will help eradicate the outdated technology. 

But, Ms. Hamilton noted, “unless we have consistent, ongoing, sustained funding, it is very hard to move off [an older] technology that can work. That’s one of the biggest barriers.” 

“Public health is about protecting the lives of every single person everywhere,” Ms. Hamilton said, “but when we don’t have the data that comes into the system, we can’t achieve our mission.”
 

A version of this article appeared on Medscape.com.

On any given day, oncologist Mark Lewis, MD, feels like he’s seesawing between two eras of technology. 

One minute, he’s working on sequencing a tumor genome. The next, he’s sifting through pages of disorganized data from a device that has been around for decades: the fax machine. 

“If two doctors’ offices aren’t on the same electronic medical record, one of the main ways to transfer records is still by fax,” said Dr. Lewis, director of gastrointestinal oncology at Intermountain Healthcare in Murray, Utah. “I can go from cutting-edge innovation to relying on, at best, 1980s information technology. It just boggles my mind.”

Dr. Lewis, who has posted about his frustration with fax machines, is far from alone. Oncologists are among the many specialists across the country at the mercy of telecopiers. 

According to a 2021 report by the Office of the National Coordinator for Health Information Technology, fax and mail continue to be the most common methods for hospitals and health systems to exchange care record summaries. In 2019, nearly 8 in 10 hospitals used mail or fax to send and receive health information, the report found. 

Fax machines are still commonplace across the healthcare spectrum, said Robert Havasy, MS, senior director for informatics strategy at the Healthcare Information and Management Systems Society (HIMSS). Inertia, cost, and more pressing priorities for hospitals and medical institutions contribute to the technology sticking around, he explained. 

“Post-COVID, my guess is we’re still at over 50% of healthcare practices using fax for some reason, on a daily basis,” Mr. Havasy said in an interview. “A lot of hospitals just don’t have the time, the money, or the staff to fix that problem because there’s always something a little higher up the priority chain they need to focus on.” 

If, for instance, “you’re going to do a process redesign to reduce hospital total acquired infections, your fax machine replacement might be 10th or 12th on the list. It just never gets up to 1 or 2 because it’s ‘not that much of a problem,’ ” he added.

Or is it?

Administrators may not view fax machines as a top concern, but clinicians who deal with the machines daily see it differently. 

“What worries me is we’re taking records out of an electronic storehouse [and] converting them to a paper medium,” Dr. Lewis said. “And then we are scanning into another electronic storehouse. The more steps, the more can be lost.”

And when information is lost, patient care can be compromised. 

Slower Workflows, Care Concerns

Although there are no published data on fax machine use in oncology specifically, this outdated technology does come into play in a variety of ways along the cancer care continuum. 

Radiation oncologist David R. Penberthy, MD, said patients often seek his cancer center’s expertise for second opinions, and that requires collecting patient records from many different practices. 

“Ideally, it would come electronically, but sometimes it does come by fax,” said Dr. Penberthy, program director of radiation oncology at the University of Virginia School of Medicine in Charlottesville. “The quality of the fax is not always the best. Sometimes it’s literally a fax of a fax. You’re reading something that’s very difficult to read.” 

Orders for new tests are also typically sent and received via fax temporarily while IT teams work to integrate them into the electronic health record (EHR), Dr. Penberthy said. 

Insurers and third-party laboratories often send test results back by fax as well.

“Even if I haven’t actually sent my patient out of our institution, this crucial result may only be entered back into the record as a scanned document from a fax, which is not great because it can get lost in the other results that are reported electronically,” Dr. Lewis said. The risk here is that an ordering physician won’t see these results, which can lead to delayed or overlooked care for patients, he explained.

“To me, it’s like a blind spot,” Dr. Lewis said. “Every time we use a fax, I see it actually as an opportunity for oversight and missed opportunity to collect data.”

Dr. Penberthy said faxing can slow things down at his practice, particularly if he faxes a document to another office but receives no confirmation and has to track down what happened. 

As for cybersecurity, data that are in transit during faxing are generally considered secure and compliant with the Health Insurance Portability and Accountability Act (HIPAA), said Mr. Havasy of HIMSS. However, the Privacy Rule also requires that data remain secure while at rest, which isn’t always possible, he added. 

“That’s where faxes fall down, because generally fax machines are in public, if you will, or open areas in a hospital,” he said. “They just sit on a desk. I don’t know that the next nurse who comes up and looks through that stack was the nurse who was treating the patient.” 

Important decisions or results can also be missed when sent by fax, creating headaches for physicians and care problems for patients. 

Dr. Lewis recently experienced an insurance-related fax mishap over Memorial Day weekend. He believed his patient had access to the antinausea medication he had prescribed. When Dr. Lewis happened to check the fax machine over the weekend, he found a coverage denial for the medication from the insurer but, at that point, had no recourse to appeal because it was a long holiday weekend. 

“Had the denial been sent by an electronic means that was quicker and more readily available, it would have been possible to appeal before the holiday weekend,” he said. 

Hematologist Aaron Goodman, MD, encountered a similar problem after an insurer denied coverage of an expensive cancer drug for a patient and faxed over its reason for the denial. Dr. Goodman was not directly notified that the information arrived and didn’t learn about the denial for a week, he said. 

“There’s no ‘ding’ in my inbox if something is faxed over and scanned,” said Dr. Goodman, associate professor of medicine at UC San Diego Health. “Once I realized it was denied, I was able to rectify it, but it wasted a week of a patient not getting a drug that I felt would be beneficial for them.”

Broader Health Policy Impacts

The use of outdated technology, such as fax machines, also creates ripple effects that burden the health system, health policy experts say. 

Duplicate testing and unnecessary care are top impacts, said Julia Adler-Milstein, PhD, professor of medicine and chief of the division of clinical informatics and digital transformation at the University of California, San Francisco.

Studies show that 20%-30% of the $65 billion spent annually on lab tests is used on unnecessary duplicate tests, and another estimated $30 billion is spent each year on unnecessary duplicate medical imaging. These duplicate tests may be mitigated if hospitals adopt certified EHR technology, research shows.

Still, without EHR interoperability between institutions, new providers may be unaware that tests or past labs for patients exist, leading to repeat tests, said Dr. Adler-Milstein, who researches health IT policy with a focus on EHRs. Patients can sometimes fill in the gaps, but not always. 

“Fax machines only help close information gaps if the clinician is aware of where to seek out the information and there is someone at the other organization to locate and transmit the information in a timely manner,” Dr. Adler-Milstein said. 

Old technology and poor interoperability also greatly affect data collection for disease surveillance and monitoring, said Janet Hamilton, MPH, executive director for the Council of State and Territorial Epidemiologists. This issue was keenly demonstrated during the pandemic, Ms. Hamilton said. 

“It was tragic, quite honestly,” she said. “There was such an immense amount of data that needed to be moved quickly, and that’s when computers are at their best.”

But, she said, “we didn’t have the level of systems in place to do it well.”

Specifically, the lack of electronic case reporting in place during the pandemic — where diagnoses are documented in the record and then immediately sent to the public health system — led to reports that were delayed, not made, or had missing or incomplete information, such as patients’ race and ethnicity or other health conditions, Ms. Hamilton said. 

Incomplete or missing data hampered the ability of public health officials and researchers to understand how the virus might affect different patients.

“If you had a chronic condition like cancer, you were less likely to have a positive outcome with COVID,” Ms. Hamilton said. “But because electronic case reporting was not in place, we didn’t get some of those additional pieces of information. We didn’t have people’s underlying oncology status to then say, ‘Here are individuals with these types of characteristics, and these are the things that happen if they also have a cancer.’” 

Slow, but Steady, Improvements

Efforts at the state and federal levels have targeted improved health information exchange, but progress takes time, Dr. Adler-Milstein said.

Most states have some form of health information exchange, such as statewide exchanges, regional health information organizations, or clinical data registries. Maryland is often held up as a notable example for its health information exchange, Dr. Adler-Milstein noted.

According to Maryland law, all hospitals under the jurisdiction of the Maryland Health Care Commission are required to electronically connect to the state-designated health information exchange. In 2012, Maryland became the first state to connect all its 46 acute care hospitals in the sharing of real-time data. 

The Health Information Technology for Economic and Clinical Health (HITECH) Act provided federal-enhanced Medicaid matching funds to states through 2021 to support efforts to advance electronic exchange. Nearly all states used these funds, and most have identified other sources to sustain the efforts, according to a recent US Government Accountability Office (GAO) report. However, GAO found that small and rural providers are less likely to have the financial and technological resources to participate in or maintain electronic exchange capabilities.

Nationally, several recent initiatives have targeted health data interoperability, including for cancer care. The Centers for Disease Control and Prevention’s Data Modernization Initiative is a multiyear, multi–billion-dollar effort to improve data sharing across the federal and state public health landscape. 

Meanwhile, in March 2024, the Biden-Harris administration launched United States Core Data for Interoperability Plus Cancer. The program will define a recommended minimum set of cancer-related data to be included in a patient’s EHR to enhance data exchange for research and clinical care. 

EHR vendors are also key to improving the landscape, said Dr. Adler-Milstein. Vendors such as Epic have developed strong sharing capabilities for transmitting health information from site to site, but of course, that only helps if providers have Epic, she said. 

“That’s where these national frameworks should help, because we don’t want it to break down by what EHR vendor you have,” she said. “It’s a patchwork. You can go to some places and hear success stories because they have Epic or a state health information exchange, but it’s very heterogeneous. In some places, they have nothing and are using a fax machine.”

Mr. Havasy believes fax machines will ultimately go extinct, particularly as a younger, more digitally savvy generation enters the healthcare workforce. He also foresees that the growing use of artificial intelligence will help eradicate the outdated technology. 

But, Ms. Hamilton noted, “unless we have consistent, ongoing, sustained funding, it is very hard to move off [an older] technology that can work. That’s one of the biggest barriers.” 

“Public health is about protecting the lives of every single person everywhere,” Ms. Hamilton said, “but when we don’t have the data that comes into the system, we can’t achieve our mission.”
 

A version of this article appeared on Medscape.com.

On any given day, oncologist Mark Lewis, MD, feels like he’s seesawing between two eras of technology. 

One minute, he’s working on sequencing a tumor genome. The next, he’s sifting through pages of disorganized data from a device that has been around for decades: the fax machine. 

“If two doctors’ offices aren’t on the same electronic medical record, one of the main ways to transfer records is still by fax,” said Dr. Lewis, director of gastrointestinal oncology at Intermountain Healthcare in Murray, Utah. “I can go from cutting-edge innovation to relying on, at best, 1980s information technology. It just boggles my mind.”

Dr. Lewis, who has posted about his frustration with fax machines, is far from alone. Oncologists are among the many specialists across the country at the mercy of telecopiers. 

According to a 2021 report by the Office of the National Coordinator for Health Information Technology, fax and mail continue to be the most common methods for hospitals and health systems to exchange care record summaries. In 2019, nearly 8 in 10 hospitals used mail or fax to send and receive health information, the report found. 

Fax machines are still commonplace across the healthcare spectrum, said Robert Havasy, MS, senior director for informatics strategy at the Healthcare Information and Management Systems Society (HIMSS). Inertia, cost, and more pressing priorities for hospitals and medical institutions contribute to the technology sticking around, he explained. 

“Post-COVID, my guess is we’re still at over 50% of healthcare practices using fax for some reason, on a daily basis,” Mr. Havasy said in an interview. “A lot of hospitals just don’t have the time, the money, or the staff to fix that problem because there’s always something a little higher up the priority chain they need to focus on.” 

If, for instance, “you’re going to do a process redesign to reduce hospital total acquired infections, your fax machine replacement might be 10th or 12th on the list. It just never gets up to 1 or 2 because it’s ‘not that much of a problem,’ ” he added.

Or is it?

Administrators may not view fax machines as a top concern, but clinicians who deal with the machines daily see it differently. 

“What worries me is we’re taking records out of an electronic storehouse [and] converting them to a paper medium,” Dr. Lewis said. “And then we are scanning into another electronic storehouse. The more steps, the more can be lost.”

And when information is lost, patient care can be compromised. 

Slower Workflows, Care Concerns

Although there are no published data on fax machine use in oncology specifically, this outdated technology does come into play in a variety of ways along the cancer care continuum. 

Radiation oncologist David R. Penberthy, MD, said patients often seek his cancer center’s expertise for second opinions, and that requires collecting patient records from many different practices. 

“Ideally, it would come electronically, but sometimes it does come by fax,” said Dr. Penberthy, program director of radiation oncology at the University of Virginia School of Medicine in Charlottesville. “The quality of the fax is not always the best. Sometimes it’s literally a fax of a fax. You’re reading something that’s very difficult to read.” 

Orders for new tests are also typically sent and received via fax temporarily while IT teams work to integrate them into the electronic health record (EHR), Dr. Penberthy said. 

Insurers and third-party laboratories often send test results back by fax as well.

“Even if I haven’t actually sent my patient out of our institution, this crucial result may only be entered back into the record as a scanned document from a fax, which is not great because it can get lost in the other results that are reported electronically,” Dr. Lewis said. The risk here is that an ordering physician won’t see these results, which can lead to delayed or overlooked care for patients, he explained.

“To me, it’s like a blind spot,” Dr. Lewis said. “Every time we use a fax, I see it actually as an opportunity for oversight and missed opportunity to collect data.”

Dr. Penberthy said faxing can slow things down at his practice, particularly if he faxes a document to another office but receives no confirmation and has to track down what happened. 

As for cybersecurity, data that are in transit during faxing are generally considered secure and compliant with the Health Insurance Portability and Accountability Act (HIPAA), said Mr. Havasy of HIMSS. However, the Privacy Rule also requires that data remain secure while at rest, which isn’t always possible, he added. 

“That’s where faxes fall down, because generally fax machines are in public, if you will, or open areas in a hospital,” he said. “They just sit on a desk. I don’t know that the next nurse who comes up and looks through that stack was the nurse who was treating the patient.” 

Important decisions or results can also be missed when sent by fax, creating headaches for physicians and care problems for patients. 

Dr. Lewis recently experienced an insurance-related fax mishap over Memorial Day weekend. He believed his patient had access to the antinausea medication he had prescribed. When Dr. Lewis happened to check the fax machine over the weekend, he found a coverage denial for the medication from the insurer but, at that point, had no recourse to appeal because it was a long holiday weekend. 

“Had the denial been sent by an electronic means that was quicker and more readily available, it would have been possible to appeal before the holiday weekend,” he said. 

Hematologist Aaron Goodman, MD, encountered a similar problem after an insurer denied coverage of an expensive cancer drug for a patient and faxed over its reason for the denial. Dr. Goodman was not directly notified that the information arrived and didn’t learn about the denial for a week, he said. 

“There’s no ‘ding’ in my inbox if something is faxed over and scanned,” said Dr. Goodman, associate professor of medicine at UC San Diego Health. “Once I realized it was denied, I was able to rectify it, but it wasted a week of a patient not getting a drug that I felt would be beneficial for them.”

Broader Health Policy Impacts

The use of outdated technology, such as fax machines, also creates ripple effects that burden the health system, health policy experts say. 

Duplicate testing and unnecessary care are top impacts, said Julia Adler-Milstein, PhD, professor of medicine and chief of the division of clinical informatics and digital transformation at the University of California, San Francisco.

Studies show that 20%-30% of the $65 billion spent annually on lab tests is used on unnecessary duplicate tests, and another estimated $30 billion is spent each year on unnecessary duplicate medical imaging. These duplicate tests may be mitigated if hospitals adopt certified EHR technology, research shows.

Still, without EHR interoperability between institutions, new providers may be unaware that tests or past labs for patients exist, leading to repeat tests, said Dr. Adler-Milstein, who researches health IT policy with a focus on EHRs. Patients can sometimes fill in the gaps, but not always. 

“Fax machines only help close information gaps if the clinician is aware of where to seek out the information and there is someone at the other organization to locate and transmit the information in a timely manner,” Dr. Adler-Milstein said. 

Old technology and poor interoperability also greatly affect data collection for disease surveillance and monitoring, said Janet Hamilton, MPH, executive director for the Council of State and Territorial Epidemiologists. This issue was keenly demonstrated during the pandemic, Ms. Hamilton said. 

“It was tragic, quite honestly,” she said. “There was such an immense amount of data that needed to be moved quickly, and that’s when computers are at their best.”

But, she said, “we didn’t have the level of systems in place to do it well.”

Specifically, the lack of electronic case reporting in place during the pandemic — where diagnoses are documented in the record and then immediately sent to the public health system — led to reports that were delayed, not made, or had missing or incomplete information, such as patients’ race and ethnicity or other health conditions, Ms. Hamilton said. 

Incomplete or missing data hampered the ability of public health officials and researchers to understand how the virus might affect different patients.

“If you had a chronic condition like cancer, you were less likely to have a positive outcome with COVID,” Ms. Hamilton said. “But because electronic case reporting was not in place, we didn’t get some of those additional pieces of information. We didn’t have people’s underlying oncology status to then say, ‘Here are individuals with these types of characteristics, and these are the things that happen if they also have a cancer.’” 

Slow, but Steady, Improvements

Efforts at the state and federal levels have targeted improved health information exchange, but progress takes time, Dr. Adler-Milstein said.

Most states have some form of health information exchange, such as statewide exchanges, regional health information organizations, or clinical data registries. Maryland is often held up as a notable example for its health information exchange, Dr. Adler-Milstein noted.

According to Maryland law, all hospitals under the jurisdiction of the Maryland Health Care Commission are required to electronically connect to the state-designated health information exchange. In 2012, Maryland became the first state to connect all its 46 acute care hospitals in the sharing of real-time data. 

The Health Information Technology for Economic and Clinical Health (HITECH) Act provided federal-enhanced Medicaid matching funds to states through 2021 to support efforts to advance electronic exchange. Nearly all states used these funds, and most have identified other sources to sustain the efforts, according to a recent US Government Accountability Office (GAO) report. However, GAO found that small and rural providers are less likely to have the financial and technological resources to participate in or maintain electronic exchange capabilities.

Nationally, several recent initiatives have targeted health data interoperability, including for cancer care. The Centers for Disease Control and Prevention’s Data Modernization Initiative is a multiyear, multi–billion-dollar effort to improve data sharing across the federal and state public health landscape. 

Meanwhile, in March 2024, the Biden-Harris administration launched United States Core Data for Interoperability Plus Cancer. The program will define a recommended minimum set of cancer-related data to be included in a patient’s EHR to enhance data exchange for research and clinical care. 

EHR vendors are also key to improving the landscape, said Dr. Adler-Milstein. Vendors such as Epic have developed strong sharing capabilities for transmitting health information from site to site, but of course, that only helps if providers have Epic, she said. 

“That’s where these national frameworks should help, because we don’t want it to break down by what EHR vendor you have,” she said. “It’s a patchwork. You can go to some places and hear success stories because they have Epic or a state health information exchange, but it’s very heterogeneous. In some places, they have nothing and are using a fax machine.”

Mr. Havasy believes fax machines will ultimately go extinct, particularly as a younger, more digitally savvy generation enters the healthcare workforce. He also foresees that the growing use of artificial intelligence will help eradicate the outdated technology. 

But, Ms. Hamilton noted, “unless we have consistent, ongoing, sustained funding, it is very hard to move off [an older] technology that can work. That’s one of the biggest barriers.” 

“Public health is about protecting the lives of every single person everywhere,” Ms. Hamilton said, “but when we don’t have the data that comes into the system, we can’t achieve our mission.”
 

A version of this article appeared on Medscape.com.

Results from the latest survey by the National Comprehensive Cancer Network (NCCN) showed that numerous critical systemic anticancer therapies, primarily generic drugs, are currently in shortage.

Nearly 90% of the 28 NCCN member centers who responded to the survey, conducted between May 28 and June 11, said they were experiencing a shortage of at least one drug.

“Many drugs that are currently in shortage form the backbones of effective multiagent regimens across both curative and palliative treatment settings,” NCCN’s CEO Crystal S. Denlinger, MD, said in an interview.

The good news is that carboplatin and cisplatin shortages have fallen dramatically since 2023. At the peak of the shortage in 2023, 93% of centers surveyed reported experiencing a shortage of carboplatin and 70% were experiencing a shortage of cisplatin, whereas in 2024, only 11% reported a carboplatin shortage and 7% reported a cisplatin shortage.

“Thankfully, the shortages for carboplatin and cisplatin are mostly resolved at this time,” Dr. Denlinger said.

However, all three NCCN surveys conducted in the past year, including the most recent one, have found shortages of various chemotherapies and supportive care medications, which suggests this is an ongoing issue affecting a significant spectrum of generic drugs.

“The acute crisis associated with the shortage of carboplatin and cisplatin was a singular event that brought the issue into the national spotlight,” but it’s “important to note that the current broad drug shortages found on this survey are not new,” said Dr. Denlinger.

In the latest survey, 89% of NCCN centers continue to report shortages of one or more drugs, and 75% said they are experiencing shortages of two or more drugs.

Overall, 57% of centers are short on vinblastine, 46% are short on etoposide, and 43% are short on topotecan. Other common chemotherapy and supportive care agents in short supply include dacarbazine (18% of centers) as well as 5-fluorouracil (5-FU) and methotrexate (14% of centers).

In 2023, however, shortages of methotrexate and 5-FU were worse, with 67% of centers reporting shortages of methotrexate and 26% of 5-FU.

In the current survey, 75% of NCCN centers also noted they were aware of drug shortages within community practices in their area, and more than one in four centers reported treatment delays requiring additional prior authorization.

Cancer drug shortages impact not only routine treatments but also clinical trials. The recent survey found that 43% of respondents said drug shortages disrupted clinical trials at their center. The biggest issues centers flagged included greater administrative burdens, lower patient enrollment, and fewer open trials.

How are centers dealing with ongoing supply issues?

Top mitigation strategies include reducing waste, limiting use of current stock, and adjusting the timing and dosage within evidence-based ranges.

“The current situation underscores the need for sustainable, long-term solutions that ensure a stable supply of high-quality cancer medications,” Alyssa Schatz, MSW, NCCN senior director of policy and advocacy, said in a news release.

Three-quarters (75%) of survey respondents said they would like to see economic incentives put in place to encourage the high-quality manufacturing of medications, especially generic versions that are often in short supply. Nearly two-thirds (64%) cited a need for a broader buffer stock payment, and the same percentage would like to see more information on user experiences with various generic suppliers to help hospitals contract with those engaging in high-quality practices.

The NCCN also continues to work with federal regulators, agencies, and lawmakers to implement long-term solutions to cancer drug shortages.

“The federal government has a key role to play in addressing this issue,” Ms. Schatz said. “Establishing economic incentives, such as tax breaks or manufacturing grants for generic drugmakers, will help support a robust and resilient supply chain — ultimately safeguarding care for people with cancer across the country.”

A version of this article appeared on Medscape.com.

Results from the latest survey by the National Comprehensive Cancer Network (NCCN) showed that numerous critical systemic anticancer therapies, primarily generic drugs, are currently in shortage.

Nearly 90% of the 28 NCCN member centers who responded to the survey, conducted between May 28 and June 11, said they were experiencing a shortage of at least one drug.

“Many drugs that are currently in shortage form the backbones of effective multiagent regimens across both curative and palliative treatment settings,” NCCN’s CEO Crystal S. Denlinger, MD, said in an interview.

The good news is that carboplatin and cisplatin shortages have fallen dramatically since 2023. At the peak of the shortage in 2023, 93% of centers surveyed reported experiencing a shortage of carboplatin and 70% were experiencing a shortage of cisplatin, whereas in 2024, only 11% reported a carboplatin shortage and 7% reported a cisplatin shortage.

“Thankfully, the shortages for carboplatin and cisplatin are mostly resolved at this time,” Dr. Denlinger said.

However, all three NCCN surveys conducted in the past year, including the most recent one, have found shortages of various chemotherapies and supportive care medications, which suggests this is an ongoing issue affecting a significant spectrum of generic drugs.

“The acute crisis associated with the shortage of carboplatin and cisplatin was a singular event that brought the issue into the national spotlight,” but it’s “important to note that the current broad drug shortages found on this survey are not new,” said Dr. Denlinger.

In the latest survey, 89% of NCCN centers continue to report shortages of one or more drugs, and 75% said they are experiencing shortages of two or more drugs.

Overall, 57% of centers are short on vinblastine, 46% are short on etoposide, and 43% are short on topotecan. Other common chemotherapy and supportive care agents in short supply include dacarbazine (18% of centers) as well as 5-fluorouracil (5-FU) and methotrexate (14% of centers).

In 2023, however, shortages of methotrexate and 5-FU were worse, with 67% of centers reporting shortages of methotrexate and 26% of 5-FU.

In the current survey, 75% of NCCN centers also noted they were aware of drug shortages within community practices in their area, and more than one in four centers reported treatment delays requiring additional prior authorization.

Cancer drug shortages impact not only routine treatments but also clinical trials. The recent survey found that 43% of respondents said drug shortages disrupted clinical trials at their center. The biggest issues centers flagged included greater administrative burdens, lower patient enrollment, and fewer open trials.

How are centers dealing with ongoing supply issues?

Top mitigation strategies include reducing waste, limiting use of current stock, and adjusting the timing and dosage within evidence-based ranges.

“The current situation underscores the need for sustainable, long-term solutions that ensure a stable supply of high-quality cancer medications,” Alyssa Schatz, MSW, NCCN senior director of policy and advocacy, said in a news release.

Three-quarters (75%) of survey respondents said they would like to see economic incentives put in place to encourage the high-quality manufacturing of medications, especially generic versions that are often in short supply. Nearly two-thirds (64%) cited a need for a broader buffer stock payment, and the same percentage would like to see more information on user experiences with various generic suppliers to help hospitals contract with those engaging in high-quality practices.

The NCCN also continues to work with federal regulators, agencies, and lawmakers to implement long-term solutions to cancer drug shortages.

“The federal government has a key role to play in addressing this issue,” Ms. Schatz said. “Establishing economic incentives, such as tax breaks or manufacturing grants for generic drugmakers, will help support a robust and resilient supply chain — ultimately safeguarding care for people with cancer across the country.”

A version of this article appeared on Medscape.com.

Results from the latest survey by the National Comprehensive Cancer Network (NCCN) showed that numerous critical systemic anticancer therapies, primarily generic drugs, are currently in shortage.

Nearly 90% of the 28 NCCN member centers who responded to the survey, conducted between May 28 and June 11, said they were experiencing a shortage of at least one drug.

“Many drugs that are currently in shortage form the backbones of effective multiagent regimens across both curative and palliative treatment settings,” NCCN’s CEO Crystal S. Denlinger, MD, said in an interview.

The good news is that carboplatin and cisplatin shortages have fallen dramatically since 2023. At the peak of the shortage in 2023, 93% of centers surveyed reported experiencing a shortage of carboplatin and 70% were experiencing a shortage of cisplatin, whereas in 2024, only 11% reported a carboplatin shortage and 7% reported a cisplatin shortage.

“Thankfully, the shortages for carboplatin and cisplatin are mostly resolved at this time,” Dr. Denlinger said.

However, all three NCCN surveys conducted in the past year, including the most recent one, have found shortages of various chemotherapies and supportive care medications, which suggests this is an ongoing issue affecting a significant spectrum of generic drugs.

“The acute crisis associated with the shortage of carboplatin and cisplatin was a singular event that brought the issue into the national spotlight,” but it’s “important to note that the current broad drug shortages found on this survey are not new,” said Dr. Denlinger.

In the latest survey, 89% of NCCN centers continue to report shortages of one or more drugs, and 75% said they are experiencing shortages of two or more drugs.

Overall, 57% of centers are short on vinblastine, 46% are short on etoposide, and 43% are short on topotecan. Other common chemotherapy and supportive care agents in short supply include dacarbazine (18% of centers) as well as 5-fluorouracil (5-FU) and methotrexate (14% of centers).

In 2023, however, shortages of methotrexate and 5-FU were worse, with 67% of centers reporting shortages of methotrexate and 26% of 5-FU.

In the current survey, 75% of NCCN centers also noted they were aware of drug shortages within community practices in their area, and more than one in four centers reported treatment delays requiring additional prior authorization.

Cancer drug shortages impact not only routine treatments but also clinical trials. The recent survey found that 43% of respondents said drug shortages disrupted clinical trials at their center. The biggest issues centers flagged included greater administrative burdens, lower patient enrollment, and fewer open trials.

How are centers dealing with ongoing supply issues?

Top mitigation strategies include reducing waste, limiting use of current stock, and adjusting the timing and dosage within evidence-based ranges.

“The current situation underscores the need for sustainable, long-term solutions that ensure a stable supply of high-quality cancer medications,” Alyssa Schatz, MSW, NCCN senior director of policy and advocacy, said in a news release.

Three-quarters (75%) of survey respondents said they would like to see economic incentives put in place to encourage the high-quality manufacturing of medications, especially generic versions that are often in short supply. Nearly two-thirds (64%) cited a need for a broader buffer stock payment, and the same percentage would like to see more information on user experiences with various generic suppliers to help hospitals contract with those engaging in high-quality practices.

The NCCN also continues to work with federal regulators, agencies, and lawmakers to implement long-term solutions to cancer drug shortages.

“The federal government has a key role to play in addressing this issue,” Ms. Schatz said. “Establishing economic incentives, such as tax breaks or manufacturing grants for generic drugmakers, will help support a robust and resilient supply chain — ultimately safeguarding care for people with cancer across the country.”

A version of this article appeared on Medscape.com.

In a survey of adults with moderate to severe atopic dermatitis (AD) participating in a patient support program for upadacitinib in the United States, 86.8% reported that their itch was “very much” or “much” improved from taking the drug, while 7.8% rated their itch as minimally improved.

Also, 27.5% reported itch improvement within one day of taking upadacitinib (Rinvoq), an oral Janus kinase inhibitor that was approved to treat moderate to severe AD in adults and children aged ≥ 12 years in January 2022.

“We have a lot of data about upadacitinib from clinical trials, but sometimes there’s a concern that when you start using a medication in the real world, the effectiveness doesn’t match up with the efficacy observed in clinical trials,” the study’s first author, Jonathan I. Silverberg, MD, PhD, professor of dermatology at George Washington University, Washington, said in an interview after the Revolutionizing Atopic Dermatitis conference, where the study was presented during a late-breaking abstract session. “We always want to confirm or reaffirm clinical trial results with real-world data.”

Dr. Silverberg

In SCALE-UP, 6191 adults with moderate to severe AD participating in the patient support program for upadacitinib in the United States were invited to complete a one-time online survey about their experience with upadacitinib, including the degree of and time to itch improvement and skin clearance. The researchers reported on 204 patients who completed the survey questions, for a response rate of 3.3%. The mean age of respondents was 45.3 years, their mean age when diagnosed with AD was 30.3 years; 70.1% were women, and 37% were using topical corticosteroids. In addition, 68.6% were White individuals, 12.3% were Black individuals, 8.8% were Asian individuals or Pacific Islanders, and 0.5% were Native Americans/Alaska Natives.

Duration of upadacitinib treatment was 2-6 months for 50.5% of the patients and 7-12 months for the remaining patients. Starting upadacitinib dose was 15 mg for about 95% of patients and 30 mg for nearly 4% of patients. At the time of the survey, 79.4% of patients were receiving upadacitinib 15 mg once a day, and 19.6% were receiving upadacitinib 30 mg once a day.
 

Improvements in Itch, Skin Clearance

Nearly all experienced improvements in itch, with 86.8% reporting “very much” or “much” improved itch. Relief was rapid, with 87% noticing improvement in itch within 7 days and 27.5% noticing improvement within 1 day. “This is something I have clinically seen,” Dr. Silverberg said.

After receiving upadacitinib, 87% and 86% of patients indicated they were “extremely” or “very” satisfied with the degree and speed of itch improvement, respectively.

In findings related to skin clearance, 90.7% of respondents reported clearer skin after initiating upadacitinib, with 81.4% reporting “very much” or “much” clearer skin. Skin clearance occurred rapidly, with 30.8% of patients noticing clearer skin within 3 days of starting upadacitinib and 89.2% of patients noticing clearer skin within 14 days. The proportions of patients who were “extremely” or “very” satisfied with the degree and speed of skin clearance were 83.8% and 83.2%, respectively.

“What we’re seeing is that the real-world effectiveness [of upadacitinib] aligns with the clinical trial efficacy,” Dr. Silverberg told this news organization. “This study adds even more data to help inform shared decision-making discussion with our patients in trying to decide what medication is best for them.”

He acknowledged certain limitations of the survey, including the lack of a control group of other treatments for comparison, a low response rate, and the potential for response bias. “That said, I think the results remain important, but we value having even more real-world data in the future from prospective registries,” he said. “Those kinds of studies are ongoing, and we look forward to getting more real-world data readouts.”

AbbVie, the manufacturer of upadacitinib, funded the study. Dr. Silverberg reported having served as an advisor, consultant, speaker, and/or investigator for several pharmaceutical companies, including AbbVie. Two authors are AbbVie employees.

A version of this article appeared on Medscape.com.

In a survey of adults with moderate to severe atopic dermatitis (AD) participating in a patient support program for upadacitinib in the United States, 86.8% reported that their itch was “very much” or “much” improved from taking the drug, while 7.8% rated their itch as minimally improved.

Also, 27.5% reported itch improvement within one day of taking upadacitinib (Rinvoq), an oral Janus kinase inhibitor that was approved to treat moderate to severe AD in adults and children aged ≥ 12 years in January 2022.

“We have a lot of data about upadacitinib from clinical trials, but sometimes there’s a concern that when you start using a medication in the real world, the effectiveness doesn’t match up with the efficacy observed in clinical trials,” the study’s first author, Jonathan I. Silverberg, MD, PhD, professor of dermatology at George Washington University, Washington, said in an interview after the Revolutionizing Atopic Dermatitis conference, where the study was presented during a late-breaking abstract session. “We always want to confirm or reaffirm clinical trial results with real-world data.”

Dr. Silverberg

In SCALE-UP, 6191 adults with moderate to severe AD participating in the patient support program for upadacitinib in the United States were invited to complete a one-time online survey about their experience with upadacitinib, including the degree of and time to itch improvement and skin clearance. The researchers reported on 204 patients who completed the survey questions, for a response rate of 3.3%. The mean age of respondents was 45.3 years, their mean age when diagnosed with AD was 30.3 years; 70.1% were women, and 37% were using topical corticosteroids. In addition, 68.6% were White individuals, 12.3% were Black individuals, 8.8% were Asian individuals or Pacific Islanders, and 0.5% were Native Americans/Alaska Natives.

Duration of upadacitinib treatment was 2-6 months for 50.5% of the patients and 7-12 months for the remaining patients. Starting upadacitinib dose was 15 mg for about 95% of patients and 30 mg for nearly 4% of patients. At the time of the survey, 79.4% of patients were receiving upadacitinib 15 mg once a day, and 19.6% were receiving upadacitinib 30 mg once a day.
 

Improvements in Itch, Skin Clearance

Nearly all experienced improvements in itch, with 86.8% reporting “very much” or “much” improved itch. Relief was rapid, with 87% noticing improvement in itch within 7 days and 27.5% noticing improvement within 1 day. “This is something I have clinically seen,” Dr. Silverberg said.

After receiving upadacitinib, 87% and 86% of patients indicated they were “extremely” or “very” satisfied with the degree and speed of itch improvement, respectively.

In findings related to skin clearance, 90.7% of respondents reported clearer skin after initiating upadacitinib, with 81.4% reporting “very much” or “much” clearer skin. Skin clearance occurred rapidly, with 30.8% of patients noticing clearer skin within 3 days of starting upadacitinib and 89.2% of patients noticing clearer skin within 14 days. The proportions of patients who were “extremely” or “very” satisfied with the degree and speed of skin clearance were 83.8% and 83.2%, respectively.

“What we’re seeing is that the real-world effectiveness [of upadacitinib] aligns with the clinical trial efficacy,” Dr. Silverberg told this news organization. “This study adds even more data to help inform shared decision-making discussion with our patients in trying to decide what medication is best for them.”

He acknowledged certain limitations of the survey, including the lack of a control group of other treatments for comparison, a low response rate, and the potential for response bias. “That said, I think the results remain important, but we value having even more real-world data in the future from prospective registries,” he said. “Those kinds of studies are ongoing, and we look forward to getting more real-world data readouts.”

AbbVie, the manufacturer of upadacitinib, funded the study. Dr. Silverberg reported having served as an advisor, consultant, speaker, and/or investigator for several pharmaceutical companies, including AbbVie. Two authors are AbbVie employees.

A version of this article appeared on Medscape.com.

In a survey of adults with moderate to severe atopic dermatitis (AD) participating in a patient support program for upadacitinib in the United States, 86.8% reported that their itch was “very much” or “much” improved from taking the drug, while 7.8% rated their itch as minimally improved.

Also, 27.5% reported itch improvement within one day of taking upadacitinib (Rinvoq), an oral Janus kinase inhibitor that was approved to treat moderate to severe AD in adults and children aged ≥ 12 years in January 2022.

“We have a lot of data about upadacitinib from clinical trials, but sometimes there’s a concern that when you start using a medication in the real world, the effectiveness doesn’t match up with the efficacy observed in clinical trials,” the study’s first author, Jonathan I. Silverberg, MD, PhD, professor of dermatology at George Washington University, Washington, said in an interview after the Revolutionizing Atopic Dermatitis conference, where the study was presented during a late-breaking abstract session. “We always want to confirm or reaffirm clinical trial results with real-world data.”

Dr. Silverberg

In SCALE-UP, 6191 adults with moderate to severe AD participating in the patient support program for upadacitinib in the United States were invited to complete a one-time online survey about their experience with upadacitinib, including the degree of and time to itch improvement and skin clearance. The researchers reported on 204 patients who completed the survey questions, for a response rate of 3.3%. The mean age of respondents was 45.3 years, their mean age when diagnosed with AD was 30.3 years; 70.1% were women, and 37% were using topical corticosteroids. In addition, 68.6% were White individuals, 12.3% were Black individuals, 8.8% were Asian individuals or Pacific Islanders, and 0.5% were Native Americans/Alaska Natives.

Duration of upadacitinib treatment was 2-6 months for 50.5% of the patients and 7-12 months for the remaining patients. Starting upadacitinib dose was 15 mg for about 95% of patients and 30 mg for nearly 4% of patients. At the time of the survey, 79.4% of patients were receiving upadacitinib 15 mg once a day, and 19.6% were receiving upadacitinib 30 mg once a day.
 

Improvements in Itch, Skin Clearance

Nearly all experienced improvements in itch, with 86.8% reporting “very much” or “much” improved itch. Relief was rapid, with 87% noticing improvement in itch within 7 days and 27.5% noticing improvement within 1 day. “This is something I have clinically seen,” Dr. Silverberg said.

After receiving upadacitinib, 87% and 86% of patients indicated they were “extremely” or “very” satisfied with the degree and speed of itch improvement, respectively.

In findings related to skin clearance, 90.7% of respondents reported clearer skin after initiating upadacitinib, with 81.4% reporting “very much” or “much” clearer skin. Skin clearance occurred rapidly, with 30.8% of patients noticing clearer skin within 3 days of starting upadacitinib and 89.2% of patients noticing clearer skin within 14 days. The proportions of patients who were “extremely” or “very” satisfied with the degree and speed of skin clearance were 83.8% and 83.2%, respectively.

“What we’re seeing is that the real-world effectiveness [of upadacitinib] aligns with the clinical trial efficacy,” Dr. Silverberg told this news organization. “This study adds even more data to help inform shared decision-making discussion with our patients in trying to decide what medication is best for them.”

He acknowledged certain limitations of the survey, including the lack of a control group of other treatments for comparison, a low response rate, and the potential for response bias. “That said, I think the results remain important, but we value having even more real-world data in the future from prospective registries,” he said. “Those kinds of studies are ongoing, and we look forward to getting more real-world data readouts.”

AbbVie, the manufacturer of upadacitinib, funded the study. Dr. Silverberg reported having served as an advisor, consultant, speaker, and/or investigator for several pharmaceutical companies, including AbbVie. Two authors are AbbVie employees.

A version of this article appeared on Medscape.com.

Patients with small melanoma in situ (MIS) on low-risk body sites managed with 5-mm margins had a local recurrence rate of 0.9%, results from a retrospective case series from a single dermatology practice in Australia showed. This approach has the potential to reduce morbidity and cost associated with treatment “without compromising patient outcomes in a selected population of lesions,” the authors say. 

“Currently, there is uncertainty regarding the optimal excision margin for MIS, with different guidelines recommending a range between 5 and 10 mm,” corresponding author Cong Sun, MD, of Mater Hospital Brisbane Raymond Terrace, South Brisbane, Queensland, Australia, and colleagues wrote in the study, which was published in JAMA Dermatology. “In addition, studies using the Mohs micrographic surgery technique have suggested that wider margins, up to 18 mm, may be required for MIS in some settings.”

To further examine the use of 5-mm margins for excision of small MIS on low-risk sites, the researchers retrospectively evaluated 351 MIS lesions diagnosed in 292 patients between January 1, 2011, and November 30, 2018. Lesions were eligible for analysis if a 5-mm excisional margin was documented on the operation report and if there was more than 5 years of site-specific follow-up after wide local excision. Lesions with undocumented margins were excluded from analysis, as were those with fewer than 5 years of follow-up, and those that required more than one wide local excision.

The mean age of patients was 60.3 years, 55.5% were female, and the mean dimensions of the lesions was 6 × 5 mm. The most common subtype of melanoma diagnosed was superficial spreading melanoma (50.4% of lesions), followed by lentigo maligna (30.5%) and lentiginous MIS (19.1%). Nearly half of the lesions were on the trunk (47.9%), followed by the upper limb (27.4%), lower limb (16.8%), neck (4%), face (3.4%), and scalp (0.6%). As for the size of lesions, 78.1% were < 10 mm long and 88.9% were < 10 mm wide. 

Nearly 71% (248) of the lesions were treated with an initial excisional biopsy, and 29.3% (103) underwent an initial shave excision. Median follow-up was 7 years.

Only three of the 351 lesions (0.9%) had a local recurrence, with no regional recurrence or metastatic spread, and 99.1% had no recurrence. The recurrences were reexcised “with clear margins” and after at least 5 years of follow-up, no further recurrences were reported, the authors said. 

In Mohs surgery studies, reported recurrence rates for MIS have been “between 0.26% and 1.1%, with excisional margins between 6 and 12 mm required,” the authors noted. “This study demonstrated a comparable 0.9% recurrence rate achieved with a conservative 5-mm excisional margin. This shows that using a 5-mm margin for MIS of smaller size (< 10 mm) may reduce morbidity and cost associated with treatment without compromising patient outcomes in a selected population of lesions.” 

The researchers recommended additional studies to confirm their findings and acknowledged certain limitations of their analysis, including its retrospective, single-center design and the predominantly small sizes of the lesions. 

In an accompanying editorial, John A. Zitelli, MD, of the University of Pittsburgh, Pittsburgh, Pennsylvania, said that the margin measurement used by the researchers was another limitation. “Before the excision with a 5-mm margin was performed, the diagnosis of MIS was obtained by shave biopsy or excisional biopsy with a 2- to 3-mm margin of clinically normal skin,” Dr. Zitelli wrote. “Therefore, in patients without a 2- to 3-mm biopsy margin, a minimum surgical margin of 7-8 mm would be required to achieve a similar true negative excision margin.” 

Also, he continued, the exclusion of lesions with wide subclinical extension that required wider margins “weakens the conclusion that 5 mm would be an effective treatment for all MIS.” 

Hugh Greenway, MD, head of Mohs micrographic surgery and director of cutaneous oncology at Scripps Cancer Center, San Diego, who was asked to comment on the study, said that clinicians continue to search for the optimum smaller surgical margin for MIS. “This can be challenging with the variability of MIS based on location and other factors,” Dr. Greenway told this news organization. “This Australian retrospective study notes that for selected, well-defined 6 × 5 mm lesions of low-risk body sites (mainly torso and limbs), a 5-mm surgical margin can provide a high cure rate. The authors note further studies are indicated. Thus, for selected lesions in selected locations, the 5-mm surgical margin may be appropriate for MIS.”

The study authors, Dr. Zitelli, and Dr. Greenway reported no financial disclosures.

A version of this article appeared on Medscape.com.

Patients with small melanoma in situ (MIS) on low-risk body sites managed with 5-mm margins had a local recurrence rate of 0.9%, results from a retrospective case series from a single dermatology practice in Australia showed. This approach has the potential to reduce morbidity and cost associated with treatment “without compromising patient outcomes in a selected population of lesions,” the authors say. 

“Currently, there is uncertainty regarding the optimal excision margin for MIS, with different guidelines recommending a range between 5 and 10 mm,” corresponding author Cong Sun, MD, of Mater Hospital Brisbane Raymond Terrace, South Brisbane, Queensland, Australia, and colleagues wrote in the study, which was published in JAMA Dermatology. “In addition, studies using the Mohs micrographic surgery technique have suggested that wider margins, up to 18 mm, may be required for MIS in some settings.”

To further examine the use of 5-mm margins for excision of small MIS on low-risk sites, the researchers retrospectively evaluated 351 MIS lesions diagnosed in 292 patients between January 1, 2011, and November 30, 2018. Lesions were eligible for analysis if a 5-mm excisional margin was documented on the operation report and if there was more than 5 years of site-specific follow-up after wide local excision. Lesions with undocumented margins were excluded from analysis, as were those with fewer than 5 years of follow-up, and those that required more than one wide local excision.

The mean age of patients was 60.3 years, 55.5% were female, and the mean dimensions of the lesions was 6 × 5 mm. The most common subtype of melanoma diagnosed was superficial spreading melanoma (50.4% of lesions), followed by lentigo maligna (30.5%) and lentiginous MIS (19.1%). Nearly half of the lesions were on the trunk (47.9%), followed by the upper limb (27.4%), lower limb (16.8%), neck (4%), face (3.4%), and scalp (0.6%). As for the size of lesions, 78.1% were < 10 mm long and 88.9% were < 10 mm wide. 

Nearly 71% (248) of the lesions were treated with an initial excisional biopsy, and 29.3% (103) underwent an initial shave excision. Median follow-up was 7 years.

Only three of the 351 lesions (0.9%) had a local recurrence, with no regional recurrence or metastatic spread, and 99.1% had no recurrence. The recurrences were reexcised “with clear margins” and after at least 5 years of follow-up, no further recurrences were reported, the authors said. 

In Mohs surgery studies, reported recurrence rates for MIS have been “between 0.26% and 1.1%, with excisional margins between 6 and 12 mm required,” the authors noted. “This study demonstrated a comparable 0.9% recurrence rate achieved with a conservative 5-mm excisional margin. This shows that using a 5-mm margin for MIS of smaller size (< 10 mm) may reduce morbidity and cost associated with treatment without compromising patient outcomes in a selected population of lesions.” 

The researchers recommended additional studies to confirm their findings and acknowledged certain limitations of their analysis, including its retrospective, single-center design and the predominantly small sizes of the lesions. 

In an accompanying editorial, John A. Zitelli, MD, of the University of Pittsburgh, Pittsburgh, Pennsylvania, said that the margin measurement used by the researchers was another limitation. “Before the excision with a 5-mm margin was performed, the diagnosis of MIS was obtained by shave biopsy or excisional biopsy with a 2- to 3-mm margin of clinically normal skin,” Dr. Zitelli wrote. “Therefore, in patients without a 2- to 3-mm biopsy margin, a minimum surgical margin of 7-8 mm would be required to achieve a similar true negative excision margin.” 

Also, he continued, the exclusion of lesions with wide subclinical extension that required wider margins “weakens the conclusion that 5 mm would be an effective treatment for all MIS.” 

Hugh Greenway, MD, head of Mohs micrographic surgery and director of cutaneous oncology at Scripps Cancer Center, San Diego, who was asked to comment on the study, said that clinicians continue to search for the optimum smaller surgical margin for MIS. “This can be challenging with the variability of MIS based on location and other factors,” Dr. Greenway told this news organization. “This Australian retrospective study notes that for selected, well-defined 6 × 5 mm lesions of low-risk body sites (mainly torso and limbs), a 5-mm surgical margin can provide a high cure rate. The authors note further studies are indicated. Thus, for selected lesions in selected locations, the 5-mm surgical margin may be appropriate for MIS.”

The study authors, Dr. Zitelli, and Dr. Greenway reported no financial disclosures.

A version of this article appeared on Medscape.com.

Patients with small melanoma in situ (MIS) on low-risk body sites managed with 5-mm margins had a local recurrence rate of 0.9%, results from a retrospective case series from a single dermatology practice in Australia showed. This approach has the potential to reduce morbidity and cost associated with treatment “without compromising patient outcomes in a selected population of lesions,” the authors say. 

“Currently, there is uncertainty regarding the optimal excision margin for MIS, with different guidelines recommending a range between 5 and 10 mm,” corresponding author Cong Sun, MD, of Mater Hospital Brisbane Raymond Terrace, South Brisbane, Queensland, Australia, and colleagues wrote in the study, which was published in JAMA Dermatology. “In addition, studies using the Mohs micrographic surgery technique have suggested that wider margins, up to 18 mm, may be required for MIS in some settings.”

To further examine the use of 5-mm margins for excision of small MIS on low-risk sites, the researchers retrospectively evaluated 351 MIS lesions diagnosed in 292 patients between January 1, 2011, and November 30, 2018. Lesions were eligible for analysis if a 5-mm excisional margin was documented on the operation report and if there was more than 5 years of site-specific follow-up after wide local excision. Lesions with undocumented margins were excluded from analysis, as were those with fewer than 5 years of follow-up, and those that required more than one wide local excision.

The mean age of patients was 60.3 years, 55.5% were female, and the mean dimensions of the lesions was 6 × 5 mm. The most common subtype of melanoma diagnosed was superficial spreading melanoma (50.4% of lesions), followed by lentigo maligna (30.5%) and lentiginous MIS (19.1%). Nearly half of the lesions were on the trunk (47.9%), followed by the upper limb (27.4%), lower limb (16.8%), neck (4%), face (3.4%), and scalp (0.6%). As for the size of lesions, 78.1% were < 10 mm long and 88.9% were < 10 mm wide. 

Nearly 71% (248) of the lesions were treated with an initial excisional biopsy, and 29.3% (103) underwent an initial shave excision. Median follow-up was 7 years.

Only three of the 351 lesions (0.9%) had a local recurrence, with no regional recurrence or metastatic spread, and 99.1% had no recurrence. The recurrences were reexcised “with clear margins” and after at least 5 years of follow-up, no further recurrences were reported, the authors said. 

In Mohs surgery studies, reported recurrence rates for MIS have been “between 0.26% and 1.1%, with excisional margins between 6 and 12 mm required,” the authors noted. “This study demonstrated a comparable 0.9% recurrence rate achieved with a conservative 5-mm excisional margin. This shows that using a 5-mm margin for MIS of smaller size (< 10 mm) may reduce morbidity and cost associated with treatment without compromising patient outcomes in a selected population of lesions.” 

The researchers recommended additional studies to confirm their findings and acknowledged certain limitations of their analysis, including its retrospective, single-center design and the predominantly small sizes of the lesions. 

In an accompanying editorial, John A. Zitelli, MD, of the University of Pittsburgh, Pittsburgh, Pennsylvania, said that the margin measurement used by the researchers was another limitation. “Before the excision with a 5-mm margin was performed, the diagnosis of MIS was obtained by shave biopsy or excisional biopsy with a 2- to 3-mm margin of clinically normal skin,” Dr. Zitelli wrote. “Therefore, in patients without a 2- to 3-mm biopsy margin, a minimum surgical margin of 7-8 mm would be required to achieve a similar true negative excision margin.” 

Also, he continued, the exclusion of lesions with wide subclinical extension that required wider margins “weakens the conclusion that 5 mm would be an effective treatment for all MIS.” 

Hugh Greenway, MD, head of Mohs micrographic surgery and director of cutaneous oncology at Scripps Cancer Center, San Diego, who was asked to comment on the study, said that clinicians continue to search for the optimum smaller surgical margin for MIS. “This can be challenging with the variability of MIS based on location and other factors,” Dr. Greenway told this news organization. “This Australian retrospective study notes that for selected, well-defined 6 × 5 mm lesions of low-risk body sites (mainly torso and limbs), a 5-mm surgical margin can provide a high cure rate. The authors note further studies are indicated. Thus, for selected lesions in selected locations, the 5-mm surgical margin may be appropriate for MIS.”

The study authors, Dr. Zitelli, and Dr. Greenway reported no financial disclosures.

A version of this article appeared on Medscape.com.