News

Advances in understanding the pathophysiology of vitiligo are transforming patient management, offering new hope for individuals with mild, moderate, and even severe forms of the disease, delegates heard at a recent conference, the Dermatology Days of Paris 2024, organized by the French Society of Dermatology.

A Distinct Disease

An estimated 65% of patients with vitiligo in Europe have been told that their disease is untreatable, according to a recent international study, and this figure rises to 75% in France, Julien Seneschal, MD, PhD, professor of dermatology at Bordeaux University Hospital in Bordeaux, France, told the audience during his presentation. 

“This is a message we must change,” he said.

The survey also revealed that in France, even when treatment is offered, 80% of patients do not receive appropriate care. However, treatments do exist, and novel approaches are revolutionizing the management of patients, whatever the degree of severity, he explained.

As a specialist in inflammatory and autoimmune skin diseases, he stressed that these advances are important because vitiligo is a distinct disease and not merely a cosmetic issue. When widespread, it has a significant impact on quality of life and can lead to depression, anxiety, and even suicidal thoughts, even though it does not affect life expectancy.

 

Updated Guidelines

Since October 2023, new international guidelines for vitiligo management have defined a therapeutic algorithm.

“Nowadays, we place the patient at the center of therapeutic decision-making,” Seneschal said. It is essential to educate patients about the disease and take the time to understand their treatment goals.

For patients with mild vitiligo that does not affect quality of life, simple monitoring may suffice.

However, when a decision is made to pursue treatment, its goals should be:

• Halting disease progression and melanocyte loss 
• Achieving repigmentation (a process that can take 6-24 months) 
• Preventing relapse after treatment discontinuation 

For moderate cases affecting less than 10% of the skin surface, localized treatment is recommended. Previously, topical corticosteroids were used for body lesions, while tacrolimus 0.1% (off-label) was often prescribed for the face and neck. However, as of March 2024, tacrolimus has been officially approved for use in patients aged ≥ 2 years.

In more severe, generalized, and/or active cases, oral treatments such as corticosteroids taken twice weekly for 12-24 weeks can stabilize the disease in 80% of cases (off-label use). Other off-label options include methotrexate, cyclosporine, and tetracyclines.

 

Targeted Therapies

Recent targeted therapies have significantly advanced the treatment of moderate to severe vitiligo. Since January 2024, the Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib cream has been available in community pharmacies after previously being restricted to hospital use, Seneschal said, and can have spectacular results if previous treatments have failed.

Ruxolitinib is approved for patients aged > 12 years with nonsegmental vitiligo and facial involvement, covering up to 10% of the body surface area. Treatment typically lasts 6 months to 1 year.

 

Key Findings

The cream-formulated drug has been demonstrated effective in reducing inflammation in two phase 3 clinical trials published in the New England Journal of Medicine that demonstrated its efficacy and safety in patients aged ≥ 12 years. The treatment was well tolerated despite some mild acne-like reactions in 8% of patients. It was shown to be very effective on the face, with a reduction of over 75% in facial lesions in more than 50% of patients, and had good effectiveness on the body, with a 50% decrease in lesions in more than 50% of patients on the body, trunk, arms, and legs, excluding hands and feet.

“Areas like the underarms, hands, and feet are more resistant to treatment,” Seneschal noted.

Although some improvement continues after 1 year, disease recurrence is common if treatment is stopped: Only 40% of patients maintain therapeutic benefits in the year following discontinuation.

“It is therefore important to consider the value of continuing treatment in order to achieve better efficacy or to maintain the repigmentation obtained,” Seneschal said.

He stressed that all treatments should be paired with phototherapy, typically narrowband UVB, to accelerate repigmentation. “There is no increased skin cancer risk in vitiligo patients treated with narrowband UVB,” Seneschal said.

 

New Therapies

Emerging treatments under development, including injectable biologics alone or in combination with phototherapy, show great promise, he said. Oral JAK inhibitors such as ritlecitinib, upadacitinib, and povorcitinib are also under investigation.

In particular, ritlecitinib, a JAK3/TEC pathway inhibitor, has shown significant reductions in affected skin area in severely affected patients in a phase 2b trial. Phase 3 trials are now underway.

On the safety profile of JAK inhibitors, Seneschal said that studies are reassuring but highlighted the need to monitor cardiovascular, thromboembolic, and infectious risks.

“The question of safety is important because vitiligo is a visible but nonsevere condition, and we do not want to expose patients to unnecessary risks,” added Gaëlle Quéreux, MD, PhD, president of the French Society of Dermatology.

This story was translated from Medscape’s French edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Advances in understanding the pathophysiology of vitiligo are transforming patient management, offering new hope for individuals with mild, moderate, and even severe forms of the disease, delegates heard at a recent conference, the Dermatology Days of Paris 2024, organized by the French Society of Dermatology.

A Distinct Disease

An estimated 65% of patients with vitiligo in Europe have been told that their disease is untreatable, according to a recent international study, and this figure rises to 75% in France, Julien Seneschal, MD, PhD, professor of dermatology at Bordeaux University Hospital in Bordeaux, France, told the audience during his presentation. 

“This is a message we must change,” he said.

The survey also revealed that in France, even when treatment is offered, 80% of patients do not receive appropriate care. However, treatments do exist, and novel approaches are revolutionizing the management of patients, whatever the degree of severity, he explained.

As a specialist in inflammatory and autoimmune skin diseases, he stressed that these advances are important because vitiligo is a distinct disease and not merely a cosmetic issue. When widespread, it has a significant impact on quality of life and can lead to depression, anxiety, and even suicidal thoughts, even though it does not affect life expectancy.

 

Updated Guidelines

Since October 2023, new international guidelines for vitiligo management have defined a therapeutic algorithm.

“Nowadays, we place the patient at the center of therapeutic decision-making,” Seneschal said. It is essential to educate patients about the disease and take the time to understand their treatment goals.

For patients with mild vitiligo that does not affect quality of life, simple monitoring may suffice.

However, when a decision is made to pursue treatment, its goals should be:

• Halting disease progression and melanocyte loss 
• Achieving repigmentation (a process that can take 6-24 months) 
• Preventing relapse after treatment discontinuation 

For moderate cases affecting less than 10% of the skin surface, localized treatment is recommended. Previously, topical corticosteroids were used for body lesions, while tacrolimus 0.1% (off-label) was often prescribed for the face and neck. However, as of March 2024, tacrolimus has been officially approved for use in patients aged ≥ 2 years.

In more severe, generalized, and/or active cases, oral treatments such as corticosteroids taken twice weekly for 12-24 weeks can stabilize the disease in 80% of cases (off-label use). Other off-label options include methotrexate, cyclosporine, and tetracyclines.

 

Targeted Therapies

Recent targeted therapies have significantly advanced the treatment of moderate to severe vitiligo. Since January 2024, the Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib cream has been available in community pharmacies after previously being restricted to hospital use, Seneschal said, and can have spectacular results if previous treatments have failed.

Ruxolitinib is approved for patients aged > 12 years with nonsegmental vitiligo and facial involvement, covering up to 10% of the body surface area. Treatment typically lasts 6 months to 1 year.

 

Key Findings

The cream-formulated drug has been demonstrated effective in reducing inflammation in two phase 3 clinical trials published in the New England Journal of Medicine that demonstrated its efficacy and safety in patients aged ≥ 12 years. The treatment was well tolerated despite some mild acne-like reactions in 8% of patients. It was shown to be very effective on the face, with a reduction of over 75% in facial lesions in more than 50% of patients, and had good effectiveness on the body, with a 50% decrease in lesions in more than 50% of patients on the body, trunk, arms, and legs, excluding hands and feet.

“Areas like the underarms, hands, and feet are more resistant to treatment,” Seneschal noted.

Although some improvement continues after 1 year, disease recurrence is common if treatment is stopped: Only 40% of patients maintain therapeutic benefits in the year following discontinuation.

“It is therefore important to consider the value of continuing treatment in order to achieve better efficacy or to maintain the repigmentation obtained,” Seneschal said.

He stressed that all treatments should be paired with phototherapy, typically narrowband UVB, to accelerate repigmentation. “There is no increased skin cancer risk in vitiligo patients treated with narrowband UVB,” Seneschal said.

 

New Therapies

Emerging treatments under development, including injectable biologics alone or in combination with phototherapy, show great promise, he said. Oral JAK inhibitors such as ritlecitinib, upadacitinib, and povorcitinib are also under investigation.

In particular, ritlecitinib, a JAK3/TEC pathway inhibitor, has shown significant reductions in affected skin area in severely affected patients in a phase 2b trial. Phase 3 trials are now underway.

On the safety profile of JAK inhibitors, Seneschal said that studies are reassuring but highlighted the need to monitor cardiovascular, thromboembolic, and infectious risks.

“The question of safety is important because vitiligo is a visible but nonsevere condition, and we do not want to expose patients to unnecessary risks,” added Gaëlle Quéreux, MD, PhD, president of the French Society of Dermatology.

This story was translated from Medscape’s French edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Advances in understanding the pathophysiology of vitiligo are transforming patient management, offering new hope for individuals with mild, moderate, and even severe forms of the disease, delegates heard at a recent conference, the Dermatology Days of Paris 2024, organized by the French Society of Dermatology.

A Distinct Disease

An estimated 65% of patients with vitiligo in Europe have been told that their disease is untreatable, according to a recent international study, and this figure rises to 75% in France, Julien Seneschal, MD, PhD, professor of dermatology at Bordeaux University Hospital in Bordeaux, France, told the audience during his presentation. 

“This is a message we must change,” he said.

The survey also revealed that in France, even when treatment is offered, 80% of patients do not receive appropriate care. However, treatments do exist, and novel approaches are revolutionizing the management of patients, whatever the degree of severity, he explained.

As a specialist in inflammatory and autoimmune skin diseases, he stressed that these advances are important because vitiligo is a distinct disease and not merely a cosmetic issue. When widespread, it has a significant impact on quality of life and can lead to depression, anxiety, and even suicidal thoughts, even though it does not affect life expectancy.

 

Updated Guidelines

Since October 2023, new international guidelines for vitiligo management have defined a therapeutic algorithm.

“Nowadays, we place the patient at the center of therapeutic decision-making,” Seneschal said. It is essential to educate patients about the disease and take the time to understand their treatment goals.

For patients with mild vitiligo that does not affect quality of life, simple monitoring may suffice.

However, when a decision is made to pursue treatment, its goals should be:

• Halting disease progression and melanocyte loss 
• Achieving repigmentation (a process that can take 6-24 months) 
• Preventing relapse after treatment discontinuation 

For moderate cases affecting less than 10% of the skin surface, localized treatment is recommended. Previously, topical corticosteroids were used for body lesions, while tacrolimus 0.1% (off-label) was often prescribed for the face and neck. However, as of March 2024, tacrolimus has been officially approved for use in patients aged ≥ 2 years.

In more severe, generalized, and/or active cases, oral treatments such as corticosteroids taken twice weekly for 12-24 weeks can stabilize the disease in 80% of cases (off-label use). Other off-label options include methotrexate, cyclosporine, and tetracyclines.

 

Targeted Therapies

Recent targeted therapies have significantly advanced the treatment of moderate to severe vitiligo. Since January 2024, the Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib cream has been available in community pharmacies after previously being restricted to hospital use, Seneschal said, and can have spectacular results if previous treatments have failed.

Ruxolitinib is approved for patients aged > 12 years with nonsegmental vitiligo and facial involvement, covering up to 10% of the body surface area. Treatment typically lasts 6 months to 1 year.

 

Key Findings

The cream-formulated drug has been demonstrated effective in reducing inflammation in two phase 3 clinical trials published in the New England Journal of Medicine that demonstrated its efficacy and safety in patients aged ≥ 12 years. The treatment was well tolerated despite some mild acne-like reactions in 8% of patients. It was shown to be very effective on the face, with a reduction of over 75% in facial lesions in more than 50% of patients, and had good effectiveness on the body, with a 50% decrease in lesions in more than 50% of patients on the body, trunk, arms, and legs, excluding hands and feet.

“Areas like the underarms, hands, and feet are more resistant to treatment,” Seneschal noted.

Although some improvement continues after 1 year, disease recurrence is common if treatment is stopped: Only 40% of patients maintain therapeutic benefits in the year following discontinuation.

“It is therefore important to consider the value of continuing treatment in order to achieve better efficacy or to maintain the repigmentation obtained,” Seneschal said.

He stressed that all treatments should be paired with phototherapy, typically narrowband UVB, to accelerate repigmentation. “There is no increased skin cancer risk in vitiligo patients treated with narrowband UVB,” Seneschal said.

 

New Therapies

Emerging treatments under development, including injectable biologics alone or in combination with phototherapy, show great promise, he said. Oral JAK inhibitors such as ritlecitinib, upadacitinib, and povorcitinib are also under investigation.

In particular, ritlecitinib, a JAK3/TEC pathway inhibitor, has shown significant reductions in affected skin area in severely affected patients in a phase 2b trial. Phase 3 trials are now underway.

On the safety profile of JAK inhibitors, Seneschal said that studies are reassuring but highlighted the need to monitor cardiovascular, thromboembolic, and infectious risks.

“The question of safety is important because vitiligo is a visible but nonsevere condition, and we do not want to expose patients to unnecessary risks,” added Gaëlle Quéreux, MD, PhD, president of the French Society of Dermatology.

This story was translated from Medscape’s French edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Merkel cell carcinoma (MCC) is less common and is associated with higher mortality rates than melanoma, according to a study that also reported that male gender, older age, and exposure to ultraviolet radiation (UVR) are significant risk factors.

METHODOLOGY:

• Researchers identified 19,444 MCC cases and 646,619 melanoma cases diagnosed between 2000 and 2021 using data from the Surveillance, Epidemiology, and End Results (SEER) Program.
• Ambient UVR exposure data were obtained from the National Aeronautics and Space Administration’s total ozone mapping spectrometer database.
• Risk factors and cancer-specific mortality rates were evaluated for both cancers.

TAKEAWAY:

• Incidence rates per 100,000 person-years of MCC and melanoma were 0.8 and 27.3, respectively.
• Men (adjusted incidence rate ratio [IRR], 1.72 for MCC and 1.23 for melanoma), older age groups (IRR: 2.69 for MCC and 1.62 for melanoma among those 70-79 years; and 5.68 for MCC and 2.26 for melanoma among those 80 years or older) showed higher incidences of MCC and melanoma. Non-Hispanic White individuals were at higher risk for MCC and melanoma than other racial/ethnic groups.
• Exposure to UVR was associated with higher incidences of melanoma (IRR, 1.24-1.49) and MCC (IRR, 1.15-1.20) in non-Hispanic White individuals, particularly on the head and neck. These associations were unclear among racial/ethnic groups.
• Individuals with MCC had a higher risk for cancer-specific mortality than those with melanoma (adjusted hazard ratio [HR], 2.33; 95% CI, 2.26-2.42). Cancer-specific survival for both cancers improved for cases diagnosed during 2012-2021 vs 2004-2011 (MCC: HR, 0.83; 95% CI, 0.78-0.89; melanoma: HR, 0.75; 95% CI, 0.74-0.76).

IN PRACTICE:

“MCC and melanoma are aggressive skin cancers with similar risk factors including male sex, older age, and UV radiation exposure. Clinicians should be alert to diagnosis of these cancers to allow for prompt treatment,” the authors wrote, adding: “It is encouraging that survival for both cancers has increased in recent years, with the largest gains in survival seen in distant stage melanoma, coinciding with the approval of BRAF and PD-1 inhibitors used for distant stage disease,” although mortality for advanced stage tumors “continues to be very high.”

SOURCE:

The study was led by Jacob T. Tribble, BA, National Cancer Institute, Rockville, Maryland. It was published online on January 5 in the Journal of Investigative Dermatology.

LIMITATIONS:

The study relied on SEER’s general staging system rather than the American Joint Committee on Cancer standard, and UVR exposure estimates did not account for individual sun protection behaviors or prior residential history. Race and ethnicity served as a proxy for UVR sensitivity, which may introduce misclassification bias.

DISCLOSURES:

The research was supported by the Intramural Research Program of the National Cancer Institute, the National Institutes of Health, the American Association for Dental Research, and the Colgate-Palmolive Company. The authors reported no conflict of interests.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Merkel cell carcinoma (MCC) is less common and is associated with higher mortality rates than melanoma, according to a study that also reported that male gender, older age, and exposure to ultraviolet radiation (UVR) are significant risk factors.

METHODOLOGY:

• Researchers identified 19,444 MCC cases and 646,619 melanoma cases diagnosed between 2000 and 2021 using data from the Surveillance, Epidemiology, and End Results (SEER) Program.
• Ambient UVR exposure data were obtained from the National Aeronautics and Space Administration’s total ozone mapping spectrometer database.
• Risk factors and cancer-specific mortality rates were evaluated for both cancers.

TAKEAWAY:

• Incidence rates per 100,000 person-years of MCC and melanoma were 0.8 and 27.3, respectively.
• Men (adjusted incidence rate ratio [IRR], 1.72 for MCC and 1.23 for melanoma), older age groups (IRR: 2.69 for MCC and 1.62 for melanoma among those 70-79 years; and 5.68 for MCC and 2.26 for melanoma among those 80 years or older) showed higher incidences of MCC and melanoma. Non-Hispanic White individuals were at higher risk for MCC and melanoma than other racial/ethnic groups.
• Exposure to UVR was associated with higher incidences of melanoma (IRR, 1.24-1.49) and MCC (IRR, 1.15-1.20) in non-Hispanic White individuals, particularly on the head and neck. These associations were unclear among racial/ethnic groups.
• Individuals with MCC had a higher risk for cancer-specific mortality than those with melanoma (adjusted hazard ratio [HR], 2.33; 95% CI, 2.26-2.42). Cancer-specific survival for both cancers improved for cases diagnosed during 2012-2021 vs 2004-2011 (MCC: HR, 0.83; 95% CI, 0.78-0.89; melanoma: HR, 0.75; 95% CI, 0.74-0.76).

IN PRACTICE:

“MCC and melanoma are aggressive skin cancers with similar risk factors including male sex, older age, and UV radiation exposure. Clinicians should be alert to diagnosis of these cancers to allow for prompt treatment,” the authors wrote, adding: “It is encouraging that survival for both cancers has increased in recent years, with the largest gains in survival seen in distant stage melanoma, coinciding with the approval of BRAF and PD-1 inhibitors used for distant stage disease,” although mortality for advanced stage tumors “continues to be very high.”

SOURCE:

The study was led by Jacob T. Tribble, BA, National Cancer Institute, Rockville, Maryland. It was published online on January 5 in the Journal of Investigative Dermatology.

LIMITATIONS:

The study relied on SEER’s general staging system rather than the American Joint Committee on Cancer standard, and UVR exposure estimates did not account for individual sun protection behaviors or prior residential history. Race and ethnicity served as a proxy for UVR sensitivity, which may introduce misclassification bias.

DISCLOSURES:

The research was supported by the Intramural Research Program of the National Cancer Institute, the National Institutes of Health, the American Association for Dental Research, and the Colgate-Palmolive Company. The authors reported no conflict of interests.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Merkel cell carcinoma (MCC) is less common and is associated with higher mortality rates than melanoma, according to a study that also reported that male gender, older age, and exposure to ultraviolet radiation (UVR) are significant risk factors.

METHODOLOGY:

• Researchers identified 19,444 MCC cases and 646,619 melanoma cases diagnosed between 2000 and 2021 using data from the Surveillance, Epidemiology, and End Results (SEER) Program.
• Ambient UVR exposure data were obtained from the National Aeronautics and Space Administration’s total ozone mapping spectrometer database.
• Risk factors and cancer-specific mortality rates were evaluated for both cancers.

TAKEAWAY:

• Incidence rates per 100,000 person-years of MCC and melanoma were 0.8 and 27.3, respectively.
• Men (adjusted incidence rate ratio [IRR], 1.72 for MCC and 1.23 for melanoma), older age groups (IRR: 2.69 for MCC and 1.62 for melanoma among those 70-79 years; and 5.68 for MCC and 2.26 for melanoma among those 80 years or older) showed higher incidences of MCC and melanoma. Non-Hispanic White individuals were at higher risk for MCC and melanoma than other racial/ethnic groups.
• Exposure to UVR was associated with higher incidences of melanoma (IRR, 1.24-1.49) and MCC (IRR, 1.15-1.20) in non-Hispanic White individuals, particularly on the head and neck. These associations were unclear among racial/ethnic groups.
• Individuals with MCC had a higher risk for cancer-specific mortality than those with melanoma (adjusted hazard ratio [HR], 2.33; 95% CI, 2.26-2.42). Cancer-specific survival for both cancers improved for cases diagnosed during 2012-2021 vs 2004-2011 (MCC: HR, 0.83; 95% CI, 0.78-0.89; melanoma: HR, 0.75; 95% CI, 0.74-0.76).

IN PRACTICE:

“MCC and melanoma are aggressive skin cancers with similar risk factors including male sex, older age, and UV radiation exposure. Clinicians should be alert to diagnosis of these cancers to allow for prompt treatment,” the authors wrote, adding: “It is encouraging that survival for both cancers has increased in recent years, with the largest gains in survival seen in distant stage melanoma, coinciding with the approval of BRAF and PD-1 inhibitors used for distant stage disease,” although mortality for advanced stage tumors “continues to be very high.”

SOURCE:

The study was led by Jacob T. Tribble, BA, National Cancer Institute, Rockville, Maryland. It was published online on January 5 in the Journal of Investigative Dermatology.

LIMITATIONS:

The study relied on SEER’s general staging system rather than the American Joint Committee on Cancer standard, and UVR exposure estimates did not account for individual sun protection behaviors or prior residential history. Race and ethnicity served as a proxy for UVR sensitivity, which may introduce misclassification bias.

DISCLOSURES:

The research was supported by the Intramural Research Program of the National Cancer Institute, the National Institutes of Health, the American Association for Dental Research, and the Colgate-Palmolive Company. The authors reported no conflict of interests.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Tildrakizumab was well tolerated and achieved sustained response in patients with moderate to severe plaque psoriasis of the scalp in a phase 3 study.

METHODOLOGY:

• A 72-week, multicenter, randomized, double-blind, placebo-controlled phase 3b trial enrolled 231 patients with moderate to severe plaque psoriasis of the scalp.
• Patients were randomly assigned to receive placebo (n = 114) or tildrakizumab (n = 117) until week 16, when patients in the placebo group switched to receive tildrakizumab.
• The primary endpoint, Investigator Global Assessment modified 2011 (IGA) scalp response, was defined as a score of 0 (clear) or 1 (almost clear) or an improvement of at least two points at week 16.
• The treatment was stopped at week 52, and participants were observed for another 20 weeks for safety and tolerability.

TAKEAWAY:

• At week 16, the response rate was higher in the tildrakizumab group than in the placebo group (49.4% vs 7.3%; P < .00001), and it increased to 62.9% and 56.1% (after crossover), respectively, at week 52.
• Psoriasis Scalp Severity Index 90 (PSSI 90) response rates were 60.7% and 4.9% at week 16 in the tildrakizumab and placebo groups, rising to 65.2% and 57.3%, respectively, at week 52.
• More than 80% of the week 16 responders maintained IGA and PSSI 90 responses at week 52.
• More than 50% of patients in both groups experienced adverse events, with no treatment-related serious toxicity.

IN PRACTICE:

“Tildrakizumab maintains improvements in scalp psoriasis for up to 52 weeks,” the authors wrote.

SOURCE:

Howard L. Sofen, MD, University of California, Los Angeles, led the study, which was published online on December 22, 2024, in the Journal of the American Academy of Dermatology.

LIMITATIONS:

This study excluded patients with predominantly scalp involvement and minimal whole body psoriasis, who might respond differently to the treatment. Results were obtained under controlled clinical conditions and may not be generalizable to clinical practice.

DISCLOSURES:

This study and analyses were funded by Sun Pharma. Sofen reported serving as a clinical investigator for various pharmaceutical companies, including Sun Pharma. Five authors were current or former employees of Sun Pharma and associated companies. Others also disclosed financial ties outside this work.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Tildrakizumab was well tolerated and achieved sustained response in patients with moderate to severe plaque psoriasis of the scalp in a phase 3 study.

METHODOLOGY:

• A 72-week, multicenter, randomized, double-blind, placebo-controlled phase 3b trial enrolled 231 patients with moderate to severe plaque psoriasis of the scalp.
• Patients were randomly assigned to receive placebo (n = 114) or tildrakizumab (n = 117) until week 16, when patients in the placebo group switched to receive tildrakizumab.
• The primary endpoint, Investigator Global Assessment modified 2011 (IGA) scalp response, was defined as a score of 0 (clear) or 1 (almost clear) or an improvement of at least two points at week 16.
• The treatment was stopped at week 52, and participants were observed for another 20 weeks for safety and tolerability.

TAKEAWAY:

• At week 16, the response rate was higher in the tildrakizumab group than in the placebo group (49.4% vs 7.3%; P < .00001), and it increased to 62.9% and 56.1% (after crossover), respectively, at week 52.
• Psoriasis Scalp Severity Index 90 (PSSI 90) response rates were 60.7% and 4.9% at week 16 in the tildrakizumab and placebo groups, rising to 65.2% and 57.3%, respectively, at week 52.
• More than 80% of the week 16 responders maintained IGA and PSSI 90 responses at week 52.
• More than 50% of patients in both groups experienced adverse events, with no treatment-related serious toxicity.

IN PRACTICE:

“Tildrakizumab maintains improvements in scalp psoriasis for up to 52 weeks,” the authors wrote.

SOURCE:

Howard L. Sofen, MD, University of California, Los Angeles, led the study, which was published online on December 22, 2024, in the Journal of the American Academy of Dermatology.

LIMITATIONS:

This study excluded patients with predominantly scalp involvement and minimal whole body psoriasis, who might respond differently to the treatment. Results were obtained under controlled clinical conditions and may not be generalizable to clinical practice.

DISCLOSURES:

This study and analyses were funded by Sun Pharma. Sofen reported serving as a clinical investigator for various pharmaceutical companies, including Sun Pharma. Five authors were current or former employees of Sun Pharma and associated companies. Others also disclosed financial ties outside this work.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Tildrakizumab was well tolerated and achieved sustained response in patients with moderate to severe plaque psoriasis of the scalp in a phase 3 study.

METHODOLOGY:

• A 72-week, multicenter, randomized, double-blind, placebo-controlled phase 3b trial enrolled 231 patients with moderate to severe plaque psoriasis of the scalp.
• Patients were randomly assigned to receive placebo (n = 114) or tildrakizumab (n = 117) until week 16, when patients in the placebo group switched to receive tildrakizumab.
• The primary endpoint, Investigator Global Assessment modified 2011 (IGA) scalp response, was defined as a score of 0 (clear) or 1 (almost clear) or an improvement of at least two points at week 16.
• The treatment was stopped at week 52, and participants were observed for another 20 weeks for safety and tolerability.

TAKEAWAY:

• At week 16, the response rate was higher in the tildrakizumab group than in the placebo group (49.4% vs 7.3%; P < .00001), and it increased to 62.9% and 56.1% (after crossover), respectively, at week 52.
• Psoriasis Scalp Severity Index 90 (PSSI 90) response rates were 60.7% and 4.9% at week 16 in the tildrakizumab and placebo groups, rising to 65.2% and 57.3%, respectively, at week 52.
• More than 80% of the week 16 responders maintained IGA and PSSI 90 responses at week 52.
• More than 50% of patients in both groups experienced adverse events, with no treatment-related serious toxicity.

IN PRACTICE:

“Tildrakizumab maintains improvements in scalp psoriasis for up to 52 weeks,” the authors wrote.

SOURCE:

Howard L. Sofen, MD, University of California, Los Angeles, led the study, which was published online on December 22, 2024, in the Journal of the American Academy of Dermatology.

LIMITATIONS:

This study excluded patients with predominantly scalp involvement and minimal whole body psoriasis, who might respond differently to the treatment. Results were obtained under controlled clinical conditions and may not be generalizable to clinical practice.

DISCLOSURES:

This study and analyses were funded by Sun Pharma. Sofen reported serving as a clinical investigator for various pharmaceutical companies, including Sun Pharma. Five authors were current or former employees of Sun Pharma and associated companies. Others also disclosed financial ties outside this work.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Changes in Krebs von den Lungen 6 (KL-6) levels after 12 months of treatment with mycophenolate mofetil (MMF) or cyclophosphamide (CYC) are associated with the development of progressive pulmonary fibrosis (PPF) in patients with systemic sclerosis–associated interstitial lung disease (SSc-ILD) in the following year.

METHODOLOGY:

• Despite available treatments, about 25% of patients with SSc-ILD develop PPF, highlighting the need for reliable early treatment response indicators, such as blood biomarkers, which may help predict the risk for PPF.
• Researchers conducted post hoc analyses of a randomized control trial that compared treatment responses to MMF with those to CYC in patients with SSc-ILD. Patients received either oral CYC for 12 months followed by placebo for 12 months or MMF for 24 months.
• A total of 92 patients with complete biomarker measurements at baseline and 12 months were included in the analysis (mean age, 52.2 years; 73.9% women; 68.5% White).
• The analysis included measurement of multiple blood biomarker levels, including C-reactive protein (CRP), interleukin-6, chemokine ligand 4 (CXCL4), CXCL18, and KL-6. Changes in these levels were evaluated from baseline to 12 months.
• The primary outcome was the development of PPF between 12 and 24 months, defined by meeting at least two of these following conditions: Worsening respiratory symptoms, a decline in forced vital capacity ≥ 5% and/or a decline in diffusing capacity for carbon monoxide ≥ 10%, or radiological disease progression.

TAKEAWAY:

• Among 92 patients, 19 developed PPF between 12 and 24 months, with 10 patients in the MMF arm and 9 patients in the CYC arm.
• KL-6 levels increased from baseline to 12 months in patients who developed PPF and decreased in those who did not (mean change, 365.68 vs –207.45 u/mL; P < .001).
• A 0.10-unit increase in KL-6 levels was associated with a 40% increase in the odds of developing PPF in an adjusted analysis (P = .0002).
• In the MMF group, levels of KL-6, CRP, and CXCL4 differed significantly between patients who developed PPF and those who did not (P = .004, P = .04, and P = .038, respectively).

IN PRACTICE:

“Reliable response biomarkers detectable early in the course of SSc-ILD treatment could minimize exposure to toxic therapies that are not conferring benefit and maximize exposure to alternative therapies that do confer benefit,” the authors wrote.

SOURCE:

The study was led by Elizabeth R. Volkmann, MD, MS, University of California, Los Angeles David Geffen School of Medicine. It was published online in Arthritis Care & Research.

LIMITATIONS:

The study population consisted of patients who were treatment-naive to MMF and CYC and had a relatively early disease course, potentially limiting generalizability to patients at later disease stages or with different treatment histories. Additionally, biomarker measurements were conducted at 12 months, when treatment response may be detectable through currently available methods, rather than at earlier timepoints.

DISCLOSURES:

The study was funded by grants from the National Institutes of Health/National Heart, Lung, and Blood Institute and the Department of Defense. MMF was supplied by Hoffmann–La Roche. Some authors reported having financial relationships with pharmaceutical companies, including Hoffmann–La Roche.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Changes in Krebs von den Lungen 6 (KL-6) levels after 12 months of treatment with mycophenolate mofetil (MMF) or cyclophosphamide (CYC) are associated with the development of progressive pulmonary fibrosis (PPF) in patients with systemic sclerosis–associated interstitial lung disease (SSc-ILD) in the following year.

METHODOLOGY:

• Despite available treatments, about 25% of patients with SSc-ILD develop PPF, highlighting the need for reliable early treatment response indicators, such as blood biomarkers, which may help predict the risk for PPF.
• Researchers conducted post hoc analyses of a randomized control trial that compared treatment responses to MMF with those to CYC in patients with SSc-ILD. Patients received either oral CYC for 12 months followed by placebo for 12 months or MMF for 24 months.
• A total of 92 patients with complete biomarker measurements at baseline and 12 months were included in the analysis (mean age, 52.2 years; 73.9% women; 68.5% White).
• The analysis included measurement of multiple blood biomarker levels, including C-reactive protein (CRP), interleukin-6, chemokine ligand 4 (CXCL4), CXCL18, and KL-6. Changes in these levels were evaluated from baseline to 12 months.
• The primary outcome was the development of PPF between 12 and 24 months, defined by meeting at least two of these following conditions: Worsening respiratory symptoms, a decline in forced vital capacity ≥ 5% and/or a decline in diffusing capacity for carbon monoxide ≥ 10%, or radiological disease progression.

TAKEAWAY:

• Among 92 patients, 19 developed PPF between 12 and 24 months, with 10 patients in the MMF arm and 9 patients in the CYC arm.
• KL-6 levels increased from baseline to 12 months in patients who developed PPF and decreased in those who did not (mean change, 365.68 vs –207.45 u/mL; P < .001).
• A 0.10-unit increase in KL-6 levels was associated with a 40% increase in the odds of developing PPF in an adjusted analysis (P = .0002).
• In the MMF group, levels of KL-6, CRP, and CXCL4 differed significantly between patients who developed PPF and those who did not (P = .004, P = .04, and P = .038, respectively).

IN PRACTICE:

“Reliable response biomarkers detectable early in the course of SSc-ILD treatment could minimize exposure to toxic therapies that are not conferring benefit and maximize exposure to alternative therapies that do confer benefit,” the authors wrote.

SOURCE:

The study was led by Elizabeth R. Volkmann, MD, MS, University of California, Los Angeles David Geffen School of Medicine. It was published online in Arthritis Care & Research.

LIMITATIONS:

The study population consisted of patients who were treatment-naive to MMF and CYC and had a relatively early disease course, potentially limiting generalizability to patients at later disease stages or with different treatment histories. Additionally, biomarker measurements were conducted at 12 months, when treatment response may be detectable through currently available methods, rather than at earlier timepoints.

DISCLOSURES:

The study was funded by grants from the National Institutes of Health/National Heart, Lung, and Blood Institute and the Department of Defense. MMF was supplied by Hoffmann–La Roche. Some authors reported having financial relationships with pharmaceutical companies, including Hoffmann–La Roche.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Changes in Krebs von den Lungen 6 (KL-6) levels after 12 months of treatment with mycophenolate mofetil (MMF) or cyclophosphamide (CYC) are associated with the development of progressive pulmonary fibrosis (PPF) in patients with systemic sclerosis–associated interstitial lung disease (SSc-ILD) in the following year.

METHODOLOGY:

• Despite available treatments, about 25% of patients with SSc-ILD develop PPF, highlighting the need for reliable early treatment response indicators, such as blood biomarkers, which may help predict the risk for PPF.
• Researchers conducted post hoc analyses of a randomized control trial that compared treatment responses to MMF with those to CYC in patients with SSc-ILD. Patients received either oral CYC for 12 months followed by placebo for 12 months or MMF for 24 months.
• A total of 92 patients with complete biomarker measurements at baseline and 12 months were included in the analysis (mean age, 52.2 years; 73.9% women; 68.5% White).
• The analysis included measurement of multiple blood biomarker levels, including C-reactive protein (CRP), interleukin-6, chemokine ligand 4 (CXCL4), CXCL18, and KL-6. Changes in these levels were evaluated from baseline to 12 months.
• The primary outcome was the development of PPF between 12 and 24 months, defined by meeting at least two of these following conditions: Worsening respiratory symptoms, a decline in forced vital capacity ≥ 5% and/or a decline in diffusing capacity for carbon monoxide ≥ 10%, or radiological disease progression.

TAKEAWAY:

• Among 92 patients, 19 developed PPF between 12 and 24 months, with 10 patients in the MMF arm and 9 patients in the CYC arm.
• KL-6 levels increased from baseline to 12 months in patients who developed PPF and decreased in those who did not (mean change, 365.68 vs –207.45 u/mL; P < .001).
• A 0.10-unit increase in KL-6 levels was associated with a 40% increase in the odds of developing PPF in an adjusted analysis (P = .0002).
• In the MMF group, levels of KL-6, CRP, and CXCL4 differed significantly between patients who developed PPF and those who did not (P = .004, P = .04, and P = .038, respectively).

IN PRACTICE:

“Reliable response biomarkers detectable early in the course of SSc-ILD treatment could minimize exposure to toxic therapies that are not conferring benefit and maximize exposure to alternative therapies that do confer benefit,” the authors wrote.

SOURCE:

The study was led by Elizabeth R. Volkmann, MD, MS, University of California, Los Angeles David Geffen School of Medicine. It was published online in Arthritis Care & Research.

LIMITATIONS:

The study population consisted of patients who were treatment-naive to MMF and CYC and had a relatively early disease course, potentially limiting generalizability to patients at later disease stages or with different treatment histories. Additionally, biomarker measurements were conducted at 12 months, when treatment response may be detectable through currently available methods, rather than at earlier timepoints.

DISCLOSURES:

The study was funded by grants from the National Institutes of Health/National Heart, Lung, and Blood Institute and the Department of Defense. MMF was supplied by Hoffmann–La Roche. Some authors reported having financial relationships with pharmaceutical companies, including Hoffmann–La Roche.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

In 2022, autoimmune diseases affected over 15 million individuals in the United States, with women nearly twice as likely to be affected as men and more than one third of affected individuals having more than one autoimmune condition.

METHODOLOGY:

• Researchers used electronic health record (EHR) data from six healthcare systems in the United States between 2011 and 2022 to estimate the prevalence of autoimmune diseases according to sex and age.
• They selected 105 autoimmune diseases from the textbook The Autoimmune Diseases and estimated their prevalence in more than 10 million individuals from these healthcare systems; these statistics were subsequently extrapolated to an estimated US population of 333.3 million.
• An individual was considered to have a diagnosis of an autoimmune disease if they had at least two diagnosis codes for the condition, with the codes being at least 30 days apart.
• A software program was developed to compute the prevalence of autoimmune diseases alone and in aggregate, enabling other researchers to replicate or modify the analysis over time.

TAKEAWAY:

• More than 15 million people, accounting for 4.6% of the US population, were diagnosed with at least one autoimmune disease from January 2011 to June 2022; 34% were diagnosed with more than one autoimmune disease.
• Sex-stratified analysis revealed that 63% of patients diagnosed with autoimmune disease were women, and only 37% were men, establishing a female-to-male ratio of 1.7:1; age-stratified analysis revealed increasing prevalence of autoimmune conditions with age, peaking in individuals aged ≥ 65 years.
• Among individuals with autoimmune diseases, 65% of patients had one condition, whereas 24% had two, 8% had three, and 2% had four or more autoimmune diseases (does not add to 100% due to rounding).
• Rheumatoid arthritis emerged as the most prevalent autoimmune disease, followed by psoriasis, type 1 diabetes, Grave’s disease, and autoimmune thyroiditis; 19 of the top 20 most prevalent autoimmune diseases occurred more frequently in women.

IN PRACTICE:

“Accurate data on the prevalence of autoimmune diseases as a category of disease and for individual autoimmune diseases are needed to further clinical and basic research to improve diagnosis, biomarkers, and therapies for these diseases, which significantly impact the US population,” the authors wrote.

SOURCE:

The study was led by Aaron H. Abend, Autoimmune Registry, Guilford, Connecticut, and was published online in The Journal of Clinical Investigation.

LIMITATIONS:

The use of EHR data presented several challenges, including potential inaccuracies in diagnosis codes and the possibility of missing patients with single diagnosis codes because of the two-code requirement. Certain autoimmune diseases evolve over time and involve nonspecific clinical signs and symptoms that can mimic other diseases, potentially resulting in underdiagnosis. Moreover, rare diseases lacking specific diagnosis codes may have been underrepresented.

DISCLOSURES:

The study received support from Autoimmune Registry; the National Institutes of Health National Center for Advancing Translational Sciences; the National Heart, Lung, and Blood Institute; and other sources. Information on potential conflicts of interest was not disclosed.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

In 2022, autoimmune diseases affected over 15 million individuals in the United States, with women nearly twice as likely to be affected as men and more than one third of affected individuals having more than one autoimmune condition.

METHODOLOGY:

• Researchers used electronic health record (EHR) data from six healthcare systems in the United States between 2011 and 2022 to estimate the prevalence of autoimmune diseases according to sex and age.
• They selected 105 autoimmune diseases from the textbook The Autoimmune Diseases and estimated their prevalence in more than 10 million individuals from these healthcare systems; these statistics were subsequently extrapolated to an estimated US population of 333.3 million.
• An individual was considered to have a diagnosis of an autoimmune disease if they had at least two diagnosis codes for the condition, with the codes being at least 30 days apart.
• A software program was developed to compute the prevalence of autoimmune diseases alone and in aggregate, enabling other researchers to replicate or modify the analysis over time.

TAKEAWAY:

• More than 15 million people, accounting for 4.6% of the US population, were diagnosed with at least one autoimmune disease from January 2011 to June 2022; 34% were diagnosed with more than one autoimmune disease.
• Sex-stratified analysis revealed that 63% of patients diagnosed with autoimmune disease were women, and only 37% were men, establishing a female-to-male ratio of 1.7:1; age-stratified analysis revealed increasing prevalence of autoimmune conditions with age, peaking in individuals aged ≥ 65 years.
• Among individuals with autoimmune diseases, 65% of patients had one condition, whereas 24% had two, 8% had three, and 2% had four or more autoimmune diseases (does not add to 100% due to rounding).
• Rheumatoid arthritis emerged as the most prevalent autoimmune disease, followed by psoriasis, type 1 diabetes, Grave’s disease, and autoimmune thyroiditis; 19 of the top 20 most prevalent autoimmune diseases occurred more frequently in women.

IN PRACTICE:

“Accurate data on the prevalence of autoimmune diseases as a category of disease and for individual autoimmune diseases are needed to further clinical and basic research to improve diagnosis, biomarkers, and therapies for these diseases, which significantly impact the US population,” the authors wrote.

SOURCE:

The study was led by Aaron H. Abend, Autoimmune Registry, Guilford, Connecticut, and was published online in The Journal of Clinical Investigation.

LIMITATIONS:

The use of EHR data presented several challenges, including potential inaccuracies in diagnosis codes and the possibility of missing patients with single diagnosis codes because of the two-code requirement. Certain autoimmune diseases evolve over time and involve nonspecific clinical signs and symptoms that can mimic other diseases, potentially resulting in underdiagnosis. Moreover, rare diseases lacking specific diagnosis codes may have been underrepresented.

DISCLOSURES:

The study received support from Autoimmune Registry; the National Institutes of Health National Center for Advancing Translational Sciences; the National Heart, Lung, and Blood Institute; and other sources. Information on potential conflicts of interest was not disclosed.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

In 2022, autoimmune diseases affected over 15 million individuals in the United States, with women nearly twice as likely to be affected as men and more than one third of affected individuals having more than one autoimmune condition.

METHODOLOGY:

• Researchers used electronic health record (EHR) data from six healthcare systems in the United States between 2011 and 2022 to estimate the prevalence of autoimmune diseases according to sex and age.
• They selected 105 autoimmune diseases from the textbook The Autoimmune Diseases and estimated their prevalence in more than 10 million individuals from these healthcare systems; these statistics were subsequently extrapolated to an estimated US population of 333.3 million.
• An individual was considered to have a diagnosis of an autoimmune disease if they had at least two diagnosis codes for the condition, with the codes being at least 30 days apart.
• A software program was developed to compute the prevalence of autoimmune diseases alone and in aggregate, enabling other researchers to replicate or modify the analysis over time.

TAKEAWAY:

• More than 15 million people, accounting for 4.6% of the US population, were diagnosed with at least one autoimmune disease from January 2011 to June 2022; 34% were diagnosed with more than one autoimmune disease.
• Sex-stratified analysis revealed that 63% of patients diagnosed with autoimmune disease were women, and only 37% were men, establishing a female-to-male ratio of 1.7:1; age-stratified analysis revealed increasing prevalence of autoimmune conditions with age, peaking in individuals aged ≥ 65 years.
• Among individuals with autoimmune diseases, 65% of patients had one condition, whereas 24% had two, 8% had three, and 2% had four or more autoimmune diseases (does not add to 100% due to rounding).
• Rheumatoid arthritis emerged as the most prevalent autoimmune disease, followed by psoriasis, type 1 diabetes, Grave’s disease, and autoimmune thyroiditis; 19 of the top 20 most prevalent autoimmune diseases occurred more frequently in women.

IN PRACTICE:

“Accurate data on the prevalence of autoimmune diseases as a category of disease and for individual autoimmune diseases are needed to further clinical and basic research to improve diagnosis, biomarkers, and therapies for these diseases, which significantly impact the US population,” the authors wrote.

SOURCE:

The study was led by Aaron H. Abend, Autoimmune Registry, Guilford, Connecticut, and was published online in The Journal of Clinical Investigation.

LIMITATIONS:

The use of EHR data presented several challenges, including potential inaccuracies in diagnosis codes and the possibility of missing patients with single diagnosis codes because of the two-code requirement. Certain autoimmune diseases evolve over time and involve nonspecific clinical signs and symptoms that can mimic other diseases, potentially resulting in underdiagnosis. Moreover, rare diseases lacking specific diagnosis codes may have been underrepresented.

DISCLOSURES:

The study received support from Autoimmune Registry; the National Institutes of Health National Center for Advancing Translational Sciences; the National Heart, Lung, and Blood Institute; and other sources. Information on potential conflicts of interest was not disclosed.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Tumor necrosis factor (TNF) inhibitors led to no significant difference in survival or respiratory-related hospitalizations, compared with non-TNF inhibitors, in patients with rheumatoid arthritis–associated interstitial lung disease (RA-ILD).

METHODOLOGY:

• Guidelines from the American College of Rheumatology and the American College of Chest Physicians conditionally advise against the use of TNF inhibitors for treating ILD in patients with RA-ILD, with persisting uncertainty about the safety of TNF inhibitors.
• Researchers conducted a retrospective cohort study using data from the US Department of Veterans Affairs, with a focus on comparing outcomes in patients with RA-ILD who initiated TNF or non-TNF inhibitors between 2006 and 2018.
• A total of 1047 US veterans with RA-ILD were included, with 237 who initiated TNF inhibitors propensity matched in a 1:1 ratio with 237 who initiated non-TNF inhibitors (mean age, 68 years; 92% men).
• The primary composite outcome was time to death or respiratory-related hospitalization over a follow-up period of up to 3 years.
• The secondary outcomes included all-cause mortality, respiratory-related mortality, and respiratory-related hospitalization, with additional assessments over a 1-year period.

TAKEAWAY:

• No significant difference was observed in the composite outcome of death or respiratory-related hospitalization between the TNF and non-TNF inhibitor groups (adjusted hazard ratio, 1.21; 95% CI, 0.92-1.58).
• No significant differences in the risk for respiratory-related hospitalization and all-cause or respiratory-related mortality were found between the TNF and non-TNF inhibitor groups. Similar findings were observed for all the outcomes during 1 year of follow-up.
• The mean duration of medication use prior to discontinuation, the time to discontinuation, and the mean predicted forced vital capacity percentage were similar for both groups.
• In a subgroup analysis of patients aged ≥ 65 years, those treated with non-TNF inhibitors had a higher risk for the composite outcome and all-cause and respiratory-related mortality than those treated with TNF inhibitors. No significant differences in outcomes were observed between the two treatment groups among patients aged < 65 years.

IN PRACTICE:

“Our results do not suggest that systematic avoidance of TNF inhibitors is required in all patients with rheumatoid arthritis–associated ILD. However, given disease heterogeneity and imprecision of our estimates, some subpopulations of patients with rheumatoid arthritis–associated ILD might benefit from specific biological or targeted synthetic DMARD [disease-modifying antirheumatic drug] treatment strategies,” the authors wrote.

SOURCE:

The study was led by Bryant R. England, MD, PhD, University of Nebraska Medical Center, Omaha It was published online on January 7, 2025, in The Lancet Rheumatology.

LIMITATIONS:

Administrative algorithms were used for identifying RA-ILD, potentially leading to misclassification and limiting phenotyping accuracy. Even with the use of propensity score methods, there might still be residual selection bias or unmeasured confounding. The study lacked comprehensive measures of posttreatment forced vital capacity and other indicators of ILD severity. The study population, predominantly men and those with a smoking history, may limit the generalizability of the findings to other groups.

DISCLOSURES:

The study was primarily funded by the US Department of Veterans Affairs. Some authors reported having financial relationships with pharmaceutical companies unrelated to the submitted work.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Tumor necrosis factor (TNF) inhibitors led to no significant difference in survival or respiratory-related hospitalizations, compared with non-TNF inhibitors, in patients with rheumatoid arthritis–associated interstitial lung disease (RA-ILD).

METHODOLOGY:

• Guidelines from the American College of Rheumatology and the American College of Chest Physicians conditionally advise against the use of TNF inhibitors for treating ILD in patients with RA-ILD, with persisting uncertainty about the safety of TNF inhibitors.
• Researchers conducted a retrospective cohort study using data from the US Department of Veterans Affairs, with a focus on comparing outcomes in patients with RA-ILD who initiated TNF or non-TNF inhibitors between 2006 and 2018.
• A total of 1047 US veterans with RA-ILD were included, with 237 who initiated TNF inhibitors propensity matched in a 1:1 ratio with 237 who initiated non-TNF inhibitors (mean age, 68 years; 92% men).
• The primary composite outcome was time to death or respiratory-related hospitalization over a follow-up period of up to 3 years.
• The secondary outcomes included all-cause mortality, respiratory-related mortality, and respiratory-related hospitalization, with additional assessments over a 1-year period.

TAKEAWAY:

• No significant difference was observed in the composite outcome of death or respiratory-related hospitalization between the TNF and non-TNF inhibitor groups (adjusted hazard ratio, 1.21; 95% CI, 0.92-1.58).
• No significant differences in the risk for respiratory-related hospitalization and all-cause or respiratory-related mortality were found between the TNF and non-TNF inhibitor groups. Similar findings were observed for all the outcomes during 1 year of follow-up.
• The mean duration of medication use prior to discontinuation, the time to discontinuation, and the mean predicted forced vital capacity percentage were similar for both groups.
• In a subgroup analysis of patients aged ≥ 65 years, those treated with non-TNF inhibitors had a higher risk for the composite outcome and all-cause and respiratory-related mortality than those treated with TNF inhibitors. No significant differences in outcomes were observed between the two treatment groups among patients aged < 65 years.

IN PRACTICE:

“Our results do not suggest that systematic avoidance of TNF inhibitors is required in all patients with rheumatoid arthritis–associated ILD. However, given disease heterogeneity and imprecision of our estimates, some subpopulations of patients with rheumatoid arthritis–associated ILD might benefit from specific biological or targeted synthetic DMARD [disease-modifying antirheumatic drug] treatment strategies,” the authors wrote.

SOURCE:

The study was led by Bryant R. England, MD, PhD, University of Nebraska Medical Center, Omaha It was published online on January 7, 2025, in The Lancet Rheumatology.

LIMITATIONS:

Administrative algorithms were used for identifying RA-ILD, potentially leading to misclassification and limiting phenotyping accuracy. Even with the use of propensity score methods, there might still be residual selection bias or unmeasured confounding. The study lacked comprehensive measures of posttreatment forced vital capacity and other indicators of ILD severity. The study population, predominantly men and those with a smoking history, may limit the generalizability of the findings to other groups.

DISCLOSURES:

The study was primarily funded by the US Department of Veterans Affairs. Some authors reported having financial relationships with pharmaceutical companies unrelated to the submitted work.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Tumor necrosis factor (TNF) inhibitors led to no significant difference in survival or respiratory-related hospitalizations, compared with non-TNF inhibitors, in patients with rheumatoid arthritis–associated interstitial lung disease (RA-ILD).

METHODOLOGY:

• Guidelines from the American College of Rheumatology and the American College of Chest Physicians conditionally advise against the use of TNF inhibitors for treating ILD in patients with RA-ILD, with persisting uncertainty about the safety of TNF inhibitors.
• Researchers conducted a retrospective cohort study using data from the US Department of Veterans Affairs, with a focus on comparing outcomes in patients with RA-ILD who initiated TNF or non-TNF inhibitors between 2006 and 2018.
• A total of 1047 US veterans with RA-ILD were included, with 237 who initiated TNF inhibitors propensity matched in a 1:1 ratio with 237 who initiated non-TNF inhibitors (mean age, 68 years; 92% men).
• The primary composite outcome was time to death or respiratory-related hospitalization over a follow-up period of up to 3 years.
• The secondary outcomes included all-cause mortality, respiratory-related mortality, and respiratory-related hospitalization, with additional assessments over a 1-year period.

TAKEAWAY:

• No significant difference was observed in the composite outcome of death or respiratory-related hospitalization between the TNF and non-TNF inhibitor groups (adjusted hazard ratio, 1.21; 95% CI, 0.92-1.58).
• No significant differences in the risk for respiratory-related hospitalization and all-cause or respiratory-related mortality were found between the TNF and non-TNF inhibitor groups. Similar findings were observed for all the outcomes during 1 year of follow-up.
• The mean duration of medication use prior to discontinuation, the time to discontinuation, and the mean predicted forced vital capacity percentage were similar for both groups.
• In a subgroup analysis of patients aged ≥ 65 years, those treated with non-TNF inhibitors had a higher risk for the composite outcome and all-cause and respiratory-related mortality than those treated with TNF inhibitors. No significant differences in outcomes were observed between the two treatment groups among patients aged < 65 years.

IN PRACTICE:

“Our results do not suggest that systematic avoidance of TNF inhibitors is required in all patients with rheumatoid arthritis–associated ILD. However, given disease heterogeneity and imprecision of our estimates, some subpopulations of patients with rheumatoid arthritis–associated ILD might benefit from specific biological or targeted synthetic DMARD [disease-modifying antirheumatic drug] treatment strategies,” the authors wrote.

SOURCE:

The study was led by Bryant R. England, MD, PhD, University of Nebraska Medical Center, Omaha It was published online on January 7, 2025, in The Lancet Rheumatology.

LIMITATIONS:

Administrative algorithms were used for identifying RA-ILD, potentially leading to misclassification and limiting phenotyping accuracy. Even with the use of propensity score methods, there might still be residual selection bias or unmeasured confounding. The study lacked comprehensive measures of posttreatment forced vital capacity and other indicators of ILD severity. The study population, predominantly men and those with a smoking history, may limit the generalizability of the findings to other groups.

DISCLOSURES:

The study was primarily funded by the US Department of Veterans Affairs. Some authors reported having financial relationships with pharmaceutical companies unrelated to the submitted work.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

Children who were severely ill with multisystem inflammatory syndrome in children (MIS-C) related to COVID-19 infection appear to show excellent cardiovascular and noncardiovascular outcomes by 6 months, according to data published in JAMA Pediatrics.

MIS-C is a life-threatening complication of COVID-19 infection and data on outcomes are limited, wrote the authors, led by Dongngan T. Truong, MD, MSSI, with Children’s Healthcare of Atlanta Cardiology, Emory University School of Medicine in Atlanta, Georgia. These 6-month results are from the Long-Term Outcomes After the Multisystem Inflammatory Syndrome in Children (MUSIC) study, sponsored by the National Heart, Lung, and Blood Institute.

Researchers found in this cohort study of 1204 participants that by 6 months after hospital discharge, 99% had normalization of left ventricular systolic function, and 92.3% had normalized coronary artery dimensions. More than 95% reported being more than 90% back to baseline health.

Patient-Reported Outcomes Measurement Information Systems (PROMIS) Global Health scores were at least equivalent to prepandemic population normative values. PROMIS Global Health parent/guardian proxy median T scores for fatigue, global health, and pain interference improved significantly from 2 weeks to 6 months: fatigue, 56.1 vs 48.9; global health, 48.8 vs 51.3; pain interference, 53.0 vs 43.3 (P < .001).

The most common symptoms reported at 2 weeks were fatigue (15.9%) and low stamina/energy (9.2%); both decreased to 3.4% and 3.3%, respectively, by 6 months. The most common cardiovascular symptom at 2 weeks was palpitations (1.5%), which decreased to 0.6%.

 

Chest Pain Increased Over Time

Reports of chest pain, however, reportedly increased over time, with 1.3% reporting chest pain at rest at 2 weeks and 2.2% at 6 months. Although gastrointestinal symptoms were common during the acute MIS-C, only 5.3% of respondents reported those symptoms at 2 weeks.

Children in the cohort had a median age of 9 years, and 60% were men. They self-identified with the following races and ethnicities: American Indian or Alaska Native (0.1%), Asian (3.3%), Black (27.0%), Hawaiian Native or Other Pacific Islander (0.2%), Hispanic or Latino (26.9%), multiracial (2.7%), White (31.2%), other (1.0%), and unknown or refused to specify (7.6%). Authors wrote that the cohort was followed-up to 2 years after illness onset and long-term results are not yet known.

 

Time to Exhale

David J. Goldberg, MD, with the Cardiac Center, Children’s Hospital of Philadelphia, Pennsylvania, and colleagues, wrote in an accompanying editorial that “the decreased frequency of the disease along (with) the reassuring reports on midterm outcomes can allow the pediatric community a moment of collective exhale.”

The editorialists note that of those who initially presented with myocardial dysfunction, all but one patient evaluated had a normal ejection fraction at follow-up. Energy, sleep, appetite, cognition, and mood also normalized by midterm.

“The results of the MUSIC study add to the emerging midterm outcomes data suggesting a near-complete cardiovascular recovery in the overwhelming majority of patients who develop MIS-C,” Goldberg and colleagues wrote. “Despite initial concerns, driven by the severity of acute presentation at diagnosis and longer-term questions that remain (for example, does coronary microvascular dysfunction persist even after normalization of coronary artery z score?), these data suggest an encouraging outlook for the long-term health of affected children.”

The Centers for Disease Control and Prevention and other agencies have reported a declining overall incidence of MIS-C and highlighted the protective value of vaccination. 

The editorialists add, however, that while the drop in MIS-C cases is encouraging, cases are still reported, especially amid high viral activity periods, “and nearly half of affected children continue to require intensive care in the acute phase of illness.”

Truong reported grants from the National Institutes of Health and serving as coprincipal investigator for Pfizer for research on COVID-19 vaccine-associated myocarditis funded by Pfizer and occurring through the framework of the National Heart, Lung, and Blood Institute’s Pediatric Heart Network outside the submitted work. One coauthor reported grants from Pfizer and Boston Scientific outside the submitted work. One coauthor reported receiving grants from Additional Ventures Foundation outside the submitted work. One coauthor reported receiving consultant fees from Amryt Pharma, Chiesi, Esperion, and Ultragenyx outside the submitted work. A coauthor reported receiving consultant fees from Larimar Therapeutics for mitochondrial therapies outside the submitted work. One coauthor reported being an employee of Takeda Pharmaceuticals since July 2023. One editorialist reported grants from Childhood Arthritis and Rheumatology Research Alliance and the Arthritis Foundation, Academy Health, and the Gordon and Betty Moore Foundation during the conduct of the study.

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

Children who were severely ill with multisystem inflammatory syndrome in children (MIS-C) related to COVID-19 infection appear to show excellent cardiovascular and noncardiovascular outcomes by 6 months, according to data published in JAMA Pediatrics.

MIS-C is a life-threatening complication of COVID-19 infection and data on outcomes are limited, wrote the authors, led by Dongngan T. Truong, MD, MSSI, with Children’s Healthcare of Atlanta Cardiology, Emory University School of Medicine in Atlanta, Georgia. These 6-month results are from the Long-Term Outcomes After the Multisystem Inflammatory Syndrome in Children (MUSIC) study, sponsored by the National Heart, Lung, and Blood Institute.

Researchers found in this cohort study of 1204 participants that by 6 months after hospital discharge, 99% had normalization of left ventricular systolic function, and 92.3% had normalized coronary artery dimensions. More than 95% reported being more than 90% back to baseline health.

Patient-Reported Outcomes Measurement Information Systems (PROMIS) Global Health scores were at least equivalent to prepandemic population normative values. PROMIS Global Health parent/guardian proxy median T scores for fatigue, global health, and pain interference improved significantly from 2 weeks to 6 months: fatigue, 56.1 vs 48.9; global health, 48.8 vs 51.3; pain interference, 53.0 vs 43.3 (P < .001).

The most common symptoms reported at 2 weeks were fatigue (15.9%) and low stamina/energy (9.2%); both decreased to 3.4% and 3.3%, respectively, by 6 months. The most common cardiovascular symptom at 2 weeks was palpitations (1.5%), which decreased to 0.6%.

 

Chest Pain Increased Over Time

Reports of chest pain, however, reportedly increased over time, with 1.3% reporting chest pain at rest at 2 weeks and 2.2% at 6 months. Although gastrointestinal symptoms were common during the acute MIS-C, only 5.3% of respondents reported those symptoms at 2 weeks.

Children in the cohort had a median age of 9 years, and 60% were men. They self-identified with the following races and ethnicities: American Indian or Alaska Native (0.1%), Asian (3.3%), Black (27.0%), Hawaiian Native or Other Pacific Islander (0.2%), Hispanic or Latino (26.9%), multiracial (2.7%), White (31.2%), other (1.0%), and unknown or refused to specify (7.6%). Authors wrote that the cohort was followed-up to 2 years after illness onset and long-term results are not yet known.

 

Time to Exhale

David J. Goldberg, MD, with the Cardiac Center, Children’s Hospital of Philadelphia, Pennsylvania, and colleagues, wrote in an accompanying editorial that “the decreased frequency of the disease along (with) the reassuring reports on midterm outcomes can allow the pediatric community a moment of collective exhale.”

The editorialists note that of those who initially presented with myocardial dysfunction, all but one patient evaluated had a normal ejection fraction at follow-up. Energy, sleep, appetite, cognition, and mood also normalized by midterm.

“The results of the MUSIC study add to the emerging midterm outcomes data suggesting a near-complete cardiovascular recovery in the overwhelming majority of patients who develop MIS-C,” Goldberg and colleagues wrote. “Despite initial concerns, driven by the severity of acute presentation at diagnosis and longer-term questions that remain (for example, does coronary microvascular dysfunction persist even after normalization of coronary artery z score?), these data suggest an encouraging outlook for the long-term health of affected children.”

The Centers for Disease Control and Prevention and other agencies have reported a declining overall incidence of MIS-C and highlighted the protective value of vaccination. 

The editorialists add, however, that while the drop in MIS-C cases is encouraging, cases are still reported, especially amid high viral activity periods, “and nearly half of affected children continue to require intensive care in the acute phase of illness.”

Truong reported grants from the National Institutes of Health and serving as coprincipal investigator for Pfizer for research on COVID-19 vaccine-associated myocarditis funded by Pfizer and occurring through the framework of the National Heart, Lung, and Blood Institute’s Pediatric Heart Network outside the submitted work. One coauthor reported grants from Pfizer and Boston Scientific outside the submitted work. One coauthor reported receiving grants from Additional Ventures Foundation outside the submitted work. One coauthor reported receiving consultant fees from Amryt Pharma, Chiesi, Esperion, and Ultragenyx outside the submitted work. A coauthor reported receiving consultant fees from Larimar Therapeutics for mitochondrial therapies outside the submitted work. One coauthor reported being an employee of Takeda Pharmaceuticals since July 2023. One editorialist reported grants from Childhood Arthritis and Rheumatology Research Alliance and the Arthritis Foundation, Academy Health, and the Gordon and Betty Moore Foundation during the conduct of the study.

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

Children who were severely ill with multisystem inflammatory syndrome in children (MIS-C) related to COVID-19 infection appear to show excellent cardiovascular and noncardiovascular outcomes by 6 months, according to data published in JAMA Pediatrics.

MIS-C is a life-threatening complication of COVID-19 infection and data on outcomes are limited, wrote the authors, led by Dongngan T. Truong, MD, MSSI, with Children’s Healthcare of Atlanta Cardiology, Emory University School of Medicine in Atlanta, Georgia. These 6-month results are from the Long-Term Outcomes After the Multisystem Inflammatory Syndrome in Children (MUSIC) study, sponsored by the National Heart, Lung, and Blood Institute.

Researchers found in this cohort study of 1204 participants that by 6 months after hospital discharge, 99% had normalization of left ventricular systolic function, and 92.3% had normalized coronary artery dimensions. More than 95% reported being more than 90% back to baseline health.

Patient-Reported Outcomes Measurement Information Systems (PROMIS) Global Health scores were at least equivalent to prepandemic population normative values. PROMIS Global Health parent/guardian proxy median T scores for fatigue, global health, and pain interference improved significantly from 2 weeks to 6 months: fatigue, 56.1 vs 48.9; global health, 48.8 vs 51.3; pain interference, 53.0 vs 43.3 (P < .001).

The most common symptoms reported at 2 weeks were fatigue (15.9%) and low stamina/energy (9.2%); both decreased to 3.4% and 3.3%, respectively, by 6 months. The most common cardiovascular symptom at 2 weeks was palpitations (1.5%), which decreased to 0.6%.

 

Chest Pain Increased Over Time

Reports of chest pain, however, reportedly increased over time, with 1.3% reporting chest pain at rest at 2 weeks and 2.2% at 6 months. Although gastrointestinal symptoms were common during the acute MIS-C, only 5.3% of respondents reported those symptoms at 2 weeks.

Children in the cohort had a median age of 9 years, and 60% were men. They self-identified with the following races and ethnicities: American Indian or Alaska Native (0.1%), Asian (3.3%), Black (27.0%), Hawaiian Native or Other Pacific Islander (0.2%), Hispanic or Latino (26.9%), multiracial (2.7%), White (31.2%), other (1.0%), and unknown or refused to specify (7.6%). Authors wrote that the cohort was followed-up to 2 years after illness onset and long-term results are not yet known.

 

Time to Exhale

David J. Goldberg, MD, with the Cardiac Center, Children’s Hospital of Philadelphia, Pennsylvania, and colleagues, wrote in an accompanying editorial that “the decreased frequency of the disease along (with) the reassuring reports on midterm outcomes can allow the pediatric community a moment of collective exhale.”

The editorialists note that of those who initially presented with myocardial dysfunction, all but one patient evaluated had a normal ejection fraction at follow-up. Energy, sleep, appetite, cognition, and mood also normalized by midterm.

“The results of the MUSIC study add to the emerging midterm outcomes data suggesting a near-complete cardiovascular recovery in the overwhelming majority of patients who develop MIS-C,” Goldberg and colleagues wrote. “Despite initial concerns, driven by the severity of acute presentation at diagnosis and longer-term questions that remain (for example, does coronary microvascular dysfunction persist even after normalization of coronary artery z score?), these data suggest an encouraging outlook for the long-term health of affected children.”

The Centers for Disease Control and Prevention and other agencies have reported a declining overall incidence of MIS-C and highlighted the protective value of vaccination. 

The editorialists add, however, that while the drop in MIS-C cases is encouraging, cases are still reported, especially amid high viral activity periods, “and nearly half of affected children continue to require intensive care in the acute phase of illness.”

Truong reported grants from the National Institutes of Health and serving as coprincipal investigator for Pfizer for research on COVID-19 vaccine-associated myocarditis funded by Pfizer and occurring through the framework of the National Heart, Lung, and Blood Institute’s Pediatric Heart Network outside the submitted work. One coauthor reported grants from Pfizer and Boston Scientific outside the submitted work. One coauthor reported receiving grants from Additional Ventures Foundation outside the submitted work. One coauthor reported receiving consultant fees from Amryt Pharma, Chiesi, Esperion, and Ultragenyx outside the submitted work. A coauthor reported receiving consultant fees from Larimar Therapeutics for mitochondrial therapies outside the submitted work. One coauthor reported being an employee of Takeda Pharmaceuticals since July 2023. One editorialist reported grants from Childhood Arthritis and Rheumatology Research Alliance and the Arthritis Foundation, Academy Health, and the Gordon and Betty Moore Foundation during the conduct of the study.

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

TOPLINE:

The use of valaciclovir as prophylaxis prevents herpes zoster (HZ) in patients with systemic lupus erythematosus (SLE) receiving anifrolumab treatment, with no cases of zoster reported during the follow-up period in patients receiving valaciclovir.

METHODOLOGY:

• Anifrolumab, a human monoclonal antibody binding to type I interferon receptor subunit 1, increases the risk for HZ in patients with SLE; however, specific recommendations to prevent HZ are currently nonexistent for patients with SLE receiving anifrolumab.
• Researchers conducted a multicenter observational study in France from November 2021 to July 2024 to evaluate the prophylactic benefits of valaciclovir in 132 patients with SLE (mean age, 42 years; 92% women) treated with anifrolumab for ≥ 3 months.
• Among these patients, 87 received either 500 mg/d valaciclovir (n = 69) or 1000 mg/d valaciclovir (n = 18) as prophylaxis, whereas 45 did not receive valaciclovir.
• The patients were followed up for a median duration of 234 days under anifrolumab treatment, with monitoring for the development of herpes zoster.

TAKEAWAY:

• The risk for HZ was significantly lower in patients who received valaciclovir than in those who did not (hazard ratio, 0.08; P = .01).
• None of the patients treated with valaciclovir developed HZ during the survey period.
• The frequency of HZ in patients who did not receive valaciclovir increased progressively from 2.2% at 3 months to 6.2% at 6 months, reaching 23% at 12 months.
• None of the reported cases of HZ required hospitalization or led to anifrolumab discontinuation, although one patient developed neuralgia.

IN PRACTICE:

“Prophylactic treatment with valaciclovir is effective for preventing HZ [herpes zoster] infection in SLE patients treated with anifrolumab,” the authors wrote. “This finding is particularly relevant for SLE patients who cannot receive the recombinant HZ vaccine or for whom it is unavailable,” they added.

SOURCE:

The study was led by Ludovic Trefond, MD, PhD, Centre Hospitalier Universitaire de Clermont-Ferrand in France. It was published online on January 4, 2025, in RMD Open.

LIMITATIONS:

The observational design of the study and the low number of herpes zoster events during the follow-up period may have affected the robustness of the findings.

DISCLOSURES:

The authors did not receive any specific grants. Some authors reported having financial relationships with various pharmaceutical companies.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

The use of valaciclovir as prophylaxis prevents herpes zoster (HZ) in patients with systemic lupus erythematosus (SLE) receiving anifrolumab treatment, with no cases of zoster reported during the follow-up period in patients receiving valaciclovir.

METHODOLOGY:

• Anifrolumab, a human monoclonal antibody binding to type I interferon receptor subunit 1, increases the risk for HZ in patients with SLE; however, specific recommendations to prevent HZ are currently nonexistent for patients with SLE receiving anifrolumab.
• Researchers conducted a multicenter observational study in France from November 2021 to July 2024 to evaluate the prophylactic benefits of valaciclovir in 132 patients with SLE (mean age, 42 years; 92% women) treated with anifrolumab for ≥ 3 months.
• Among these patients, 87 received either 500 mg/d valaciclovir (n = 69) or 1000 mg/d valaciclovir (n = 18) as prophylaxis, whereas 45 did not receive valaciclovir.
• The patients were followed up for a median duration of 234 days under anifrolumab treatment, with monitoring for the development of herpes zoster.

TAKEAWAY:

• The risk for HZ was significantly lower in patients who received valaciclovir than in those who did not (hazard ratio, 0.08; P = .01).
• None of the patients treated with valaciclovir developed HZ during the survey period.
• The frequency of HZ in patients who did not receive valaciclovir increased progressively from 2.2% at 3 months to 6.2% at 6 months, reaching 23% at 12 months.
• None of the reported cases of HZ required hospitalization or led to anifrolumab discontinuation, although one patient developed neuralgia.

IN PRACTICE:

“Prophylactic treatment with valaciclovir is effective for preventing HZ [herpes zoster] infection in SLE patients treated with anifrolumab,” the authors wrote. “This finding is particularly relevant for SLE patients who cannot receive the recombinant HZ vaccine or for whom it is unavailable,” they added.

SOURCE:

The study was led by Ludovic Trefond, MD, PhD, Centre Hospitalier Universitaire de Clermont-Ferrand in France. It was published online on January 4, 2025, in RMD Open.

LIMITATIONS:

The observational design of the study and the low number of herpes zoster events during the follow-up period may have affected the robustness of the findings.

DISCLOSURES:

The authors did not receive any specific grants. Some authors reported having financial relationships with various pharmaceutical companies.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

The use of valaciclovir as prophylaxis prevents herpes zoster (HZ) in patients with systemic lupus erythematosus (SLE) receiving anifrolumab treatment, with no cases of zoster reported during the follow-up period in patients receiving valaciclovir.

METHODOLOGY:

• Anifrolumab, a human monoclonal antibody binding to type I interferon receptor subunit 1, increases the risk for HZ in patients with SLE; however, specific recommendations to prevent HZ are currently nonexistent for patients with SLE receiving anifrolumab.
• Researchers conducted a multicenter observational study in France from November 2021 to July 2024 to evaluate the prophylactic benefits of valaciclovir in 132 patients with SLE (mean age, 42 years; 92% women) treated with anifrolumab for ≥ 3 months.
• Among these patients, 87 received either 500 mg/d valaciclovir (n = 69) or 1000 mg/d valaciclovir (n = 18) as prophylaxis, whereas 45 did not receive valaciclovir.
• The patients were followed up for a median duration of 234 days under anifrolumab treatment, with monitoring for the development of herpes zoster.

TAKEAWAY:

• The risk for HZ was significantly lower in patients who received valaciclovir than in those who did not (hazard ratio, 0.08; P = .01).
• None of the patients treated with valaciclovir developed HZ during the survey period.
• The frequency of HZ in patients who did not receive valaciclovir increased progressively from 2.2% at 3 months to 6.2% at 6 months, reaching 23% at 12 months.
• None of the reported cases of HZ required hospitalization or led to anifrolumab discontinuation, although one patient developed neuralgia.

IN PRACTICE:

“Prophylactic treatment with valaciclovir is effective for preventing HZ [herpes zoster] infection in SLE patients treated with anifrolumab,” the authors wrote. “This finding is particularly relevant for SLE patients who cannot receive the recombinant HZ vaccine or for whom it is unavailable,” they added.

SOURCE:

The study was led by Ludovic Trefond, MD, PhD, Centre Hospitalier Universitaire de Clermont-Ferrand in France. It was published online on January 4, 2025, in RMD Open.

LIMITATIONS:

The observational design of the study and the low number of herpes zoster events during the follow-up period may have affected the robustness of the findings.

DISCLOSURES:

The authors did not receive any specific grants. Some authors reported having financial relationships with various pharmaceutical companies.

This article was created using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

Children with asthma scored significantly lower than those without asthma on measures of episodic memory, based on longitudinal data from nearly 500 individuals.

Animal models have shown associations between asthma and memory problems, but data for children are lacking, wrote Nicholas J. Christopher-Hayes, MA, of the University of California, Davis, and colleagues.

“Asthma is very frequent among children, and there is mounting evidence from rodent models that asthma may result in neural injury in the hippocampus, which in turn may cause memory loss,” Christopher-Hayes said in an interview. “Although there is also a good amount of research with older adults, very little research has been done with children, the period that is most frequently linked to asthma onset,” he said. Therefore, the researchers leveraged a large national study on child development to examine development of memory as a function of asthma exposure.

In this study published in JAMA Network Open, the researchers conducted both a longitudinal and cross-sectional analysis of data from the Adolescent Brain Cognitive Development Study, which began in 2015. Children were enrolled at ages 9-10 years with a follow-up assessment 1-2 years later.

The participants were categorized as early childhood-onset asthma (asthma at baseline and follow-up), later childhood-onset asthma (asthma at follow-up only), or no asthma history. The primary outcome of the longitudinal analysis was episodic memory. Approximately half of the participants were boys, and slightly more than half were White.

Among 474 children reviewed in the longitudinal analysis, 135 had early-onset asthma, 102 had later-onset asthma, and 237 had no asthma and served as control individuals. Overall, those with early-onset asthma showed significantly lower rates of longitudinal memory improvements at follow-up compared with the comparison group (P < .01).

Developmental memory improvement in children with later-onset asthma was not significantly different from the control individuals. 

Secondary outcomes included processing speed and inhibition, and attention. In a cross-sectional analysis with a larger sample of 2062 children from the same database (1031 with any asthma), those with asthma scored significantly lower on measures not only of episodic memory but also processing speed and inhibition/attention than children with no asthma, with P values of .04, .01, and .02, respectively.

The results were limited by several factors, including the reliance on parent reports for indicators of asthma and the lack of data on the potential effect of prescription corticosteroid use on neurocognitive development, the researchers noted.

The mechanism behind the association remains unclear; the inflammation associated with asthma may disrupt neural processing and manifest as cognitive dysfunction, as has been seen in rodent models of asthma, the researchers wrote. “It is possible that associations between asthma and developmental trajectories emerge earlier for memory, perhaps due to its sensitivity to subtle hippocampal injury,” they noted.

Longer follow-up studies are needed to fully understand how childhood asthma predicts memory declines or difficulties in childhood and beyond, said Christopher-Hayes. “We also need additional studies to understand why children who were diagnosed earlier and had asthma for longer seem to be particularly affected,” he said.

The results of this study were consistent with previous findings and therefore not surprising, senior author Simona Ghetti, PhD, a professor of psychology at the University of California, Davis, said in an interview. However, the finding that the extent of exposure to asthma was associated with slower memory improvement in childhood was striking, she said. That children with an earlier asthma onset who had disease indicators for a longer period showed a slower development of memory over time, suggests that asthma exposure may affect the developmental trajectory of memory, Ghetti noted. 

“Recommendations to clinicians are premature because we need a better understanding of the boundary conditions, such as the minimal level of asthma exposure that might generate memory difficulties,” said Ghetti.

“Nevertheless, our results underscore the importance of looking at asthma as a potential source of cognitive difficulty in children,” she said.

 

Asthma’s Extensive Effect

Evidence is mounting that a diagnosis of asthma may have implications outside the pulmonary system, Diego J. Maselli, MD, professor and chief of the Division of Pulmonary Diseases & Critical Care at UT Health, San Antonio, said in an interview. 

“Asthmatics may be at risk of nasal polyps, allergic rhinitis, and other allergic conditions, but there is emerging of evidence inflammation associated with asthma may affect other organ systems,” said Maselli, who was not involved in the study.

“For example, chronic inflammation in asthmatics may increase the risk of cardiovascular disease,” he said.

Although less is known about the effects of asthma on the nervous system, animal models suggest that inflammation associated with asthma may result in neuronal injury and potential effects on memory, said Maselli.

The findings of this study provide evidence of potential detrimental effects on the memory of children with asthma but should be interpreted with caution, Maselli said. “Children with chronic medical conditions may have an inherent disadvantage compared with their peers due to the burden of their disease, medication utilization and side effects, absenteeism from school, physical limitations, and other disease-specific circumstances,” he noted.

“Uncontrolled asthma, in particular, has strong links to low socioeconomic factors that are closely tied to access to adequate medical care, nutrition, educational institutions, and other relevant contributors to normal cognitive development,” Maselli said. Although the authors account for some of these socioeconomic factors by evaluating income and race, other variables may have influenced the results, he added.

Overall, this study’s findings suggested that the diagnosis of asthma in children may be associated with memory deficits and may influence neurodevelopment; however, more research is needed to determine whether the findings are replicated in other cohorts, said Maselli. “In particular, evaluating the effects of the severity of asthma and different asthma endotypes would be crucial to identify children with a higher risk of memory or cognitive deficits and confirm these associations,” he said.

This study was funded by the Memory and Plasticity Program at the University of California, Davis, and by a Learning, Memory, and Plasticity Training Program Fellowship grant from the National Institutes of Health. The researchers and Maselli had no financial conflicts to disclose. 

 

A version of this article appeared on Medscape.com.

Children with asthma scored significantly lower than those without asthma on measures of episodic memory, based on longitudinal data from nearly 500 individuals.

Animal models have shown associations between asthma and memory problems, but data for children are lacking, wrote Nicholas J. Christopher-Hayes, MA, of the University of California, Davis, and colleagues.

“Asthma is very frequent among children, and there is mounting evidence from rodent models that asthma may result in neural injury in the hippocampus, which in turn may cause memory loss,” Christopher-Hayes said in an interview. “Although there is also a good amount of research with older adults, very little research has been done with children, the period that is most frequently linked to asthma onset,” he said. Therefore, the researchers leveraged a large national study on child development to examine development of memory as a function of asthma exposure.

In this study published in JAMA Network Open, the researchers conducted both a longitudinal and cross-sectional analysis of data from the Adolescent Brain Cognitive Development Study, which began in 2015. Children were enrolled at ages 9-10 years with a follow-up assessment 1-2 years later.

The participants were categorized as early childhood-onset asthma (asthma at baseline and follow-up), later childhood-onset asthma (asthma at follow-up only), or no asthma history. The primary outcome of the longitudinal analysis was episodic memory. Approximately half of the participants were boys, and slightly more than half were White.

Among 474 children reviewed in the longitudinal analysis, 135 had early-onset asthma, 102 had later-onset asthma, and 237 had no asthma and served as control individuals. Overall, those with early-onset asthma showed significantly lower rates of longitudinal memory improvements at follow-up compared with the comparison group (P < .01).

Developmental memory improvement in children with later-onset asthma was not significantly different from the control individuals. 

Secondary outcomes included processing speed and inhibition, and attention. In a cross-sectional analysis with a larger sample of 2062 children from the same database (1031 with any asthma), those with asthma scored significantly lower on measures not only of episodic memory but also processing speed and inhibition/attention than children with no asthma, with P values of .04, .01, and .02, respectively.

The results were limited by several factors, including the reliance on parent reports for indicators of asthma and the lack of data on the potential effect of prescription corticosteroid use on neurocognitive development, the researchers noted.

The mechanism behind the association remains unclear; the inflammation associated with asthma may disrupt neural processing and manifest as cognitive dysfunction, as has been seen in rodent models of asthma, the researchers wrote. “It is possible that associations between asthma and developmental trajectories emerge earlier for memory, perhaps due to its sensitivity to subtle hippocampal injury,” they noted.

Longer follow-up studies are needed to fully understand how childhood asthma predicts memory declines or difficulties in childhood and beyond, said Christopher-Hayes. “We also need additional studies to understand why children who were diagnosed earlier and had asthma for longer seem to be particularly affected,” he said.

The results of this study were consistent with previous findings and therefore not surprising, senior author Simona Ghetti, PhD, a professor of psychology at the University of California, Davis, said in an interview. However, the finding that the extent of exposure to asthma was associated with slower memory improvement in childhood was striking, she said. That children with an earlier asthma onset who had disease indicators for a longer period showed a slower development of memory over time, suggests that asthma exposure may affect the developmental trajectory of memory, Ghetti noted. 

“Recommendations to clinicians are premature because we need a better understanding of the boundary conditions, such as the minimal level of asthma exposure that might generate memory difficulties,” said Ghetti.

“Nevertheless, our results underscore the importance of looking at asthma as a potential source of cognitive difficulty in children,” she said.

 

Asthma’s Extensive Effect

Evidence is mounting that a diagnosis of asthma may have implications outside the pulmonary system, Diego J. Maselli, MD, professor and chief of the Division of Pulmonary Diseases & Critical Care at UT Health, San Antonio, said in an interview. 

“Asthmatics may be at risk of nasal polyps, allergic rhinitis, and other allergic conditions, but there is emerging of evidence inflammation associated with asthma may affect other organ systems,” said Maselli, who was not involved in the study.

“For example, chronic inflammation in asthmatics may increase the risk of cardiovascular disease,” he said.

Although less is known about the effects of asthma on the nervous system, animal models suggest that inflammation associated with asthma may result in neuronal injury and potential effects on memory, said Maselli.

The findings of this study provide evidence of potential detrimental effects on the memory of children with asthma but should be interpreted with caution, Maselli said. “Children with chronic medical conditions may have an inherent disadvantage compared with their peers due to the burden of their disease, medication utilization and side effects, absenteeism from school, physical limitations, and other disease-specific circumstances,” he noted.

“Uncontrolled asthma, in particular, has strong links to low socioeconomic factors that are closely tied to access to adequate medical care, nutrition, educational institutions, and other relevant contributors to normal cognitive development,” Maselli said. Although the authors account for some of these socioeconomic factors by evaluating income and race, other variables may have influenced the results, he added.

Overall, this study’s findings suggested that the diagnosis of asthma in children may be associated with memory deficits and may influence neurodevelopment; however, more research is needed to determine whether the findings are replicated in other cohorts, said Maselli. “In particular, evaluating the effects of the severity of asthma and different asthma endotypes would be crucial to identify children with a higher risk of memory or cognitive deficits and confirm these associations,” he said.

This study was funded by the Memory and Plasticity Program at the University of California, Davis, and by a Learning, Memory, and Plasticity Training Program Fellowship grant from the National Institutes of Health. The researchers and Maselli had no financial conflicts to disclose. 

 

A version of this article appeared on Medscape.com.

Children with asthma scored significantly lower than those without asthma on measures of episodic memory, based on longitudinal data from nearly 500 individuals.

Animal models have shown associations between asthma and memory problems, but data for children are lacking, wrote Nicholas J. Christopher-Hayes, MA, of the University of California, Davis, and colleagues.

“Asthma is very frequent among children, and there is mounting evidence from rodent models that asthma may result in neural injury in the hippocampus, which in turn may cause memory loss,” Christopher-Hayes said in an interview. “Although there is also a good amount of research with older adults, very little research has been done with children, the period that is most frequently linked to asthma onset,” he said. Therefore, the researchers leveraged a large national study on child development to examine development of memory as a function of asthma exposure.

In this study published in JAMA Network Open, the researchers conducted both a longitudinal and cross-sectional analysis of data from the Adolescent Brain Cognitive Development Study, which began in 2015. Children were enrolled at ages 9-10 years with a follow-up assessment 1-2 years later.

The participants were categorized as early childhood-onset asthma (asthma at baseline and follow-up), later childhood-onset asthma (asthma at follow-up only), or no asthma history. The primary outcome of the longitudinal analysis was episodic memory. Approximately half of the participants were boys, and slightly more than half were White.

Among 474 children reviewed in the longitudinal analysis, 135 had early-onset asthma, 102 had later-onset asthma, and 237 had no asthma and served as control individuals. Overall, those with early-onset asthma showed significantly lower rates of longitudinal memory improvements at follow-up compared with the comparison group (P < .01).

Developmental memory improvement in children with later-onset asthma was not significantly different from the control individuals. 

Secondary outcomes included processing speed and inhibition, and attention. In a cross-sectional analysis with a larger sample of 2062 children from the same database (1031 with any asthma), those with asthma scored significantly lower on measures not only of episodic memory but also processing speed and inhibition/attention than children with no asthma, with P values of .04, .01, and .02, respectively.

The results were limited by several factors, including the reliance on parent reports for indicators of asthma and the lack of data on the potential effect of prescription corticosteroid use on neurocognitive development, the researchers noted.

The mechanism behind the association remains unclear; the inflammation associated with asthma may disrupt neural processing and manifest as cognitive dysfunction, as has been seen in rodent models of asthma, the researchers wrote. “It is possible that associations between asthma and developmental trajectories emerge earlier for memory, perhaps due to its sensitivity to subtle hippocampal injury,” they noted.

Longer follow-up studies are needed to fully understand how childhood asthma predicts memory declines or difficulties in childhood and beyond, said Christopher-Hayes. “We also need additional studies to understand why children who were diagnosed earlier and had asthma for longer seem to be particularly affected,” he said.

The results of this study were consistent with previous findings and therefore not surprising, senior author Simona Ghetti, PhD, a professor of psychology at the University of California, Davis, said in an interview. However, the finding that the extent of exposure to asthma was associated with slower memory improvement in childhood was striking, she said. That children with an earlier asthma onset who had disease indicators for a longer period showed a slower development of memory over time, suggests that asthma exposure may affect the developmental trajectory of memory, Ghetti noted. 

“Recommendations to clinicians are premature because we need a better understanding of the boundary conditions, such as the minimal level of asthma exposure that might generate memory difficulties,” said Ghetti.

“Nevertheless, our results underscore the importance of looking at asthma as a potential source of cognitive difficulty in children,” she said.

 

Asthma’s Extensive Effect

Evidence is mounting that a diagnosis of asthma may have implications outside the pulmonary system, Diego J. Maselli, MD, professor and chief of the Division of Pulmonary Diseases & Critical Care at UT Health, San Antonio, said in an interview. 

“Asthmatics may be at risk of nasal polyps, allergic rhinitis, and other allergic conditions, but there is emerging of evidence inflammation associated with asthma may affect other organ systems,” said Maselli, who was not involved in the study.

“For example, chronic inflammation in asthmatics may increase the risk of cardiovascular disease,” he said.

Although less is known about the effects of asthma on the nervous system, animal models suggest that inflammation associated with asthma may result in neuronal injury and potential effects on memory, said Maselli.

The findings of this study provide evidence of potential detrimental effects on the memory of children with asthma but should be interpreted with caution, Maselli said. “Children with chronic medical conditions may have an inherent disadvantage compared with their peers due to the burden of their disease, medication utilization and side effects, absenteeism from school, physical limitations, and other disease-specific circumstances,” he noted.

“Uncontrolled asthma, in particular, has strong links to low socioeconomic factors that are closely tied to access to adequate medical care, nutrition, educational institutions, and other relevant contributors to normal cognitive development,” Maselli said. Although the authors account for some of these socioeconomic factors by evaluating income and race, other variables may have influenced the results, he added.

Overall, this study’s findings suggested that the diagnosis of asthma in children may be associated with memory deficits and may influence neurodevelopment; however, more research is needed to determine whether the findings are replicated in other cohorts, said Maselli. “In particular, evaluating the effects of the severity of asthma and different asthma endotypes would be crucial to identify children with a higher risk of memory or cognitive deficits and confirm these associations,” he said.

This study was funded by the Memory and Plasticity Program at the University of California, Davis, and by a Learning, Memory, and Plasticity Training Program Fellowship grant from the National Institutes of Health. The researchers and Maselli had no financial conflicts to disclose. 

 

A version of this article appeared on Medscape.com.

The latest glucagon-like peptide 1 (GLP-1) receptor agonists have been heralded for their potential to not only boost weight loss and glucose control but also improve cardiovascular, gastric, hepatic, and renal values.

Throughout 2024, research has also indicated GLP-1 drugs may reduce risks for obesity-related cancer.

In a US study of more than 1.6 million patients with type 2 diabetes, cancer researchers found that patients who took a GLP-1 drug had significant risk reductions for 10 of 13 obesity-associated cancers, as compared with patients who only took insulin.

The research team found a reduction in esophageal, colorectal, endometrial, gallbladder, kidney, liver, ovarian, and pancreatic cancers, as well as meningioma and multiple myeloma. They also saw a declining risk for stomach cancer, though it wasn’t considered statistically significant, but not a reduced risk for postmenopausal breast cancer or thyroid cancer.

The associations make sense, particularly because GLP-1 drugs have unexpected effects on modulating immune functions linked to obesity-associated cancers.

“The protective effects of GLP-1s against obesity-associated cancers likely stem from multiple mechanisms,” said lead author Lindsey Wang, a medical student and research scholar at Case Western Reserve University in Cleveland.

“These drugs promote substantial weight loss, reducing obesity-related cancer risks,” she said. “They also enhance insulin sensitivity and lower insulin levels, decreasing cancer cell growth signals.”

 

Additional GLP-1 Studies

The Case Western team also published a study in December 2023 that found people with type 2 diabetes who took GLP-1s had a 44% lower risk for colorectal cancer than those who took insulin and a 25% lower risk than those who took metformin. The research suggested even greater risk reductions among those with overweight or obesity, with GLP-1 users having a 50% lower risk than those who took insulin and a 42% lower risk than those who took metformin.

In another recent Case Western study, both bariatric surgery and GLP-1 drugs reduced the risk for obesity-related cancers. While those who had bariatric surgery had a 22% risk reduction over 10 years, as compared with those who received no treatment, those taking GLP-1 had a 39% risk reduction.

Other studies worldwide have looked at GLP-1 drugs and tumor effects among various cancer cell lines. In a study using pancreatic cancer cell lines, GLP-1 liraglutide suppressed cancer cell growth and led to cell death. Similarly, a study using breast cancer cells found liraglutide reduced cancer cell viability and the ability for cells to migrate.

As researchers identify additional links between GLP-1s and improvements across organ systems, the knock-on effects could lead to lower cancer risks as well. For example, studies presented at The Liver Meeting in San Diego in November pointed to GLP-1s reducing fatty liver disease, which can slow the progression to liver cancer.

“Separate from obesity, having higher levels of body fat is associated with an increased risk of several forms of cancer,” said Neil Iyengar, MD, an oncologist at Memorial Sloan Kettering Cancer Center in New York City. Iyengar researches the relationship between obesity and cancer.

“I foresee that this class of drugs will revolutionize obesity and the cancer burden that comes with it, if people can get access,” he said. “This really is an exciting development.”

 

Ongoing GLP-1 Research

On the other hand, cancer researchers have also expressed concerns about potential associations between GLP-1s and increased cancer risks. In the obesity-associated cancer study by Case Western researchers, patients with type 2 diabetes taking a GLP-1 drug appeared to have a slightly higher risk for kidney cancer than those taking metformin.

In addition, GLP-1 studies in animals have indicated that the drugs may increase the risks for medullary thyroid cancer and pancreatic cancer. However, the data on increased risks in humans remain inconclusive, and more recent studies refute these findings.

For instance, cancer researchers in India conducted a systematic review and meta-analysis of semaglutide and cancer risks, finding that 37 randomized controlled trials and 19 real-world studies didn’t find increased risks for any cancer, including pancreatic and thyroid cancers.

In another systematic review by Brazilian researchers, 50 trials found GLP-1s didn’t increase the risk for breast cancer or benign breast neoplasms.

In 2025, new retrospective studies will show more nuanced data, especially as more patients — both with and without type 2 diabetes — take semaglutide, tirzepatide, and new GLP-1 drugs in the research pipeline.

“The holy grail has always been getting a medication to treat obesity,” said Anne McTiernan, MD, PhD, an epidemiologist and obesity researcher at the Fred Hutchinson Cancer Center in Seattle.

“There have been trials focused on these medications’ effects on diabetes and cardiovascular disease treatment, but no trials have tested their effects on cancer risk,” she said. “Usually, many years of follow-up of large numbers of patients are needed to see cancer effects of a carcinogen or cancer-preventing intervention.”

Those clinical trials are likely coming soon, she said. Researchers will need to conduct prospective clinical trials to examine the direct relationship between GLP-1 drugs and cancer risks, as well as the underlying mechanisms linked to cancer cell growth, activation of immune cells, and anti-inflammatory properties.

Because GLP-1 medications aren’t intended to be taken forever, researchers will also need to consider the associations with long-term cancer risks. Even so, weight loss and other obesity-related improvements could contribute to overall lower cancer risks in the end.

“If taking these drugs for a limited amount of time can help people lose weight and get on an exercise plan, then that’s helping lower cancer risk long-term,” said Sonali Thosani, MD, associate professor of endocrine neoplasia and hormonal disorders at the University of Texas MD Anderson Cancer Center in Houston.

“But it all comes back to someone making lifestyle changes and sticking to them, even after they stop taking the drugs,” she said. “If they can do that, then you’ll probably see a net positive for long-term cancer risks and other long-term health risks.”

 

A version of this article appeared on Medscape.com.

The latest glucagon-like peptide 1 (GLP-1) receptor agonists have been heralded for their potential to not only boost weight loss and glucose control but also improve cardiovascular, gastric, hepatic, and renal values.

Throughout 2024, research has also indicated GLP-1 drugs may reduce risks for obesity-related cancer.

In a US study of more than 1.6 million patients with type 2 diabetes, cancer researchers found that patients who took a GLP-1 drug had significant risk reductions for 10 of 13 obesity-associated cancers, as compared with patients who only took insulin.

The research team found a reduction in esophageal, colorectal, endometrial, gallbladder, kidney, liver, ovarian, and pancreatic cancers, as well as meningioma and multiple myeloma. They also saw a declining risk for stomach cancer, though it wasn’t considered statistically significant, but not a reduced risk for postmenopausal breast cancer or thyroid cancer.

The associations make sense, particularly because GLP-1 drugs have unexpected effects on modulating immune functions linked to obesity-associated cancers.

“The protective effects of GLP-1s against obesity-associated cancers likely stem from multiple mechanisms,” said lead author Lindsey Wang, a medical student and research scholar at Case Western Reserve University in Cleveland.

“These drugs promote substantial weight loss, reducing obesity-related cancer risks,” she said. “They also enhance insulin sensitivity and lower insulin levels, decreasing cancer cell growth signals.”

 

Additional GLP-1 Studies

The Case Western team also published a study in December 2023 that found people with type 2 diabetes who took GLP-1s had a 44% lower risk for colorectal cancer than those who took insulin and a 25% lower risk than those who took metformin. The research suggested even greater risk reductions among those with overweight or obesity, with GLP-1 users having a 50% lower risk than those who took insulin and a 42% lower risk than those who took metformin.

In another recent Case Western study, both bariatric surgery and GLP-1 drugs reduced the risk for obesity-related cancers. While those who had bariatric surgery had a 22% risk reduction over 10 years, as compared with those who received no treatment, those taking GLP-1 had a 39% risk reduction.

Other studies worldwide have looked at GLP-1 drugs and tumor effects among various cancer cell lines. In a study using pancreatic cancer cell lines, GLP-1 liraglutide suppressed cancer cell growth and led to cell death. Similarly, a study using breast cancer cells found liraglutide reduced cancer cell viability and the ability for cells to migrate.

As researchers identify additional links between GLP-1s and improvements across organ systems, the knock-on effects could lead to lower cancer risks as well. For example, studies presented at The Liver Meeting in San Diego in November pointed to GLP-1s reducing fatty liver disease, which can slow the progression to liver cancer.

“Separate from obesity, having higher levels of body fat is associated with an increased risk of several forms of cancer,” said Neil Iyengar, MD, an oncologist at Memorial Sloan Kettering Cancer Center in New York City. Iyengar researches the relationship between obesity and cancer.

“I foresee that this class of drugs will revolutionize obesity and the cancer burden that comes with it, if people can get access,” he said. “This really is an exciting development.”

 

Ongoing GLP-1 Research

On the other hand, cancer researchers have also expressed concerns about potential associations between GLP-1s and increased cancer risks. In the obesity-associated cancer study by Case Western researchers, patients with type 2 diabetes taking a GLP-1 drug appeared to have a slightly higher risk for kidney cancer than those taking metformin.

In addition, GLP-1 studies in animals have indicated that the drugs may increase the risks for medullary thyroid cancer and pancreatic cancer. However, the data on increased risks in humans remain inconclusive, and more recent studies refute these findings.

For instance, cancer researchers in India conducted a systematic review and meta-analysis of semaglutide and cancer risks, finding that 37 randomized controlled trials and 19 real-world studies didn’t find increased risks for any cancer, including pancreatic and thyroid cancers.

In another systematic review by Brazilian researchers, 50 trials found GLP-1s didn’t increase the risk for breast cancer or benign breast neoplasms.

In 2025, new retrospective studies will show more nuanced data, especially as more patients — both with and without type 2 diabetes — take semaglutide, tirzepatide, and new GLP-1 drugs in the research pipeline.

“The holy grail has always been getting a medication to treat obesity,” said Anne McTiernan, MD, PhD, an epidemiologist and obesity researcher at the Fred Hutchinson Cancer Center in Seattle.

“There have been trials focused on these medications’ effects on diabetes and cardiovascular disease treatment, but no trials have tested their effects on cancer risk,” she said. “Usually, many years of follow-up of large numbers of patients are needed to see cancer effects of a carcinogen or cancer-preventing intervention.”

Those clinical trials are likely coming soon, she said. Researchers will need to conduct prospective clinical trials to examine the direct relationship between GLP-1 drugs and cancer risks, as well as the underlying mechanisms linked to cancer cell growth, activation of immune cells, and anti-inflammatory properties.

Because GLP-1 medications aren’t intended to be taken forever, researchers will also need to consider the associations with long-term cancer risks. Even so, weight loss and other obesity-related improvements could contribute to overall lower cancer risks in the end.

“If taking these drugs for a limited amount of time can help people lose weight and get on an exercise plan, then that’s helping lower cancer risk long-term,” said Sonali Thosani, MD, associate professor of endocrine neoplasia and hormonal disorders at the University of Texas MD Anderson Cancer Center in Houston.

“But it all comes back to someone making lifestyle changes and sticking to them, even after they stop taking the drugs,” she said. “If they can do that, then you’ll probably see a net positive for long-term cancer risks and other long-term health risks.”

 

A version of this article appeared on Medscape.com.

The latest glucagon-like peptide 1 (GLP-1) receptor agonists have been heralded for their potential to not only boost weight loss and glucose control but also improve cardiovascular, gastric, hepatic, and renal values.

Throughout 2024, research has also indicated GLP-1 drugs may reduce risks for obesity-related cancer.

In a US study of more than 1.6 million patients with type 2 diabetes, cancer researchers found that patients who took a GLP-1 drug had significant risk reductions for 10 of 13 obesity-associated cancers, as compared with patients who only took insulin.

The research team found a reduction in esophageal, colorectal, endometrial, gallbladder, kidney, liver, ovarian, and pancreatic cancers, as well as meningioma and multiple myeloma. They also saw a declining risk for stomach cancer, though it wasn’t considered statistically significant, but not a reduced risk for postmenopausal breast cancer or thyroid cancer.

The associations make sense, particularly because GLP-1 drugs have unexpected effects on modulating immune functions linked to obesity-associated cancers.

“The protective effects of GLP-1s against obesity-associated cancers likely stem from multiple mechanisms,” said lead author Lindsey Wang, a medical student and research scholar at Case Western Reserve University in Cleveland.

“These drugs promote substantial weight loss, reducing obesity-related cancer risks,” she said. “They also enhance insulin sensitivity and lower insulin levels, decreasing cancer cell growth signals.”

 

Additional GLP-1 Studies

The Case Western team also published a study in December 2023 that found people with type 2 diabetes who took GLP-1s had a 44% lower risk for colorectal cancer than those who took insulin and a 25% lower risk than those who took metformin. The research suggested even greater risk reductions among those with overweight or obesity, with GLP-1 users having a 50% lower risk than those who took insulin and a 42% lower risk than those who took metformin.

In another recent Case Western study, both bariatric surgery and GLP-1 drugs reduced the risk for obesity-related cancers. While those who had bariatric surgery had a 22% risk reduction over 10 years, as compared with those who received no treatment, those taking GLP-1 had a 39% risk reduction.

Other studies worldwide have looked at GLP-1 drugs and tumor effects among various cancer cell lines. In a study using pancreatic cancer cell lines, GLP-1 liraglutide suppressed cancer cell growth and led to cell death. Similarly, a study using breast cancer cells found liraglutide reduced cancer cell viability and the ability for cells to migrate.

As researchers identify additional links between GLP-1s and improvements across organ systems, the knock-on effects could lead to lower cancer risks as well. For example, studies presented at The Liver Meeting in San Diego in November pointed to GLP-1s reducing fatty liver disease, which can slow the progression to liver cancer.

“Separate from obesity, having higher levels of body fat is associated with an increased risk of several forms of cancer,” said Neil Iyengar, MD, an oncologist at Memorial Sloan Kettering Cancer Center in New York City. Iyengar researches the relationship between obesity and cancer.

“I foresee that this class of drugs will revolutionize obesity and the cancer burden that comes with it, if people can get access,” he said. “This really is an exciting development.”

 

Ongoing GLP-1 Research

On the other hand, cancer researchers have also expressed concerns about potential associations between GLP-1s and increased cancer risks. In the obesity-associated cancer study by Case Western researchers, patients with type 2 diabetes taking a GLP-1 drug appeared to have a slightly higher risk for kidney cancer than those taking metformin.

In addition, GLP-1 studies in animals have indicated that the drugs may increase the risks for medullary thyroid cancer and pancreatic cancer. However, the data on increased risks in humans remain inconclusive, and more recent studies refute these findings.

For instance, cancer researchers in India conducted a systematic review and meta-analysis of semaglutide and cancer risks, finding that 37 randomized controlled trials and 19 real-world studies didn’t find increased risks for any cancer, including pancreatic and thyroid cancers.

In another systematic review by Brazilian researchers, 50 trials found GLP-1s didn’t increase the risk for breast cancer or benign breast neoplasms.

In 2025, new retrospective studies will show more nuanced data, especially as more patients — both with and without type 2 diabetes — take semaglutide, tirzepatide, and new GLP-1 drugs in the research pipeline.

“The holy grail has always been getting a medication to treat obesity,” said Anne McTiernan, MD, PhD, an epidemiologist and obesity researcher at the Fred Hutchinson Cancer Center in Seattle.

“There have been trials focused on these medications’ effects on diabetes and cardiovascular disease treatment, but no trials have tested their effects on cancer risk,” she said. “Usually, many years of follow-up of large numbers of patients are needed to see cancer effects of a carcinogen or cancer-preventing intervention.”

Those clinical trials are likely coming soon, she said. Researchers will need to conduct prospective clinical trials to examine the direct relationship between GLP-1 drugs and cancer risks, as well as the underlying mechanisms linked to cancer cell growth, activation of immune cells, and anti-inflammatory properties.

Because GLP-1 medications aren’t intended to be taken forever, researchers will also need to consider the associations with long-term cancer risks. Even so, weight loss and other obesity-related improvements could contribute to overall lower cancer risks in the end.

“If taking these drugs for a limited amount of time can help people lose weight and get on an exercise plan, then that’s helping lower cancer risk long-term,” said Sonali Thosani, MD, associate professor of endocrine neoplasia and hormonal disorders at the University of Texas MD Anderson Cancer Center in Houston.

“But it all comes back to someone making lifestyle changes and sticking to them, even after they stop taking the drugs,” she said. “If they can do that, then you’ll probably see a net positive for long-term cancer risks and other long-term health risks.”

 

A version of this article appeared on Medscape.com.