De Rosas EC, et al.JAMA Dermatology. 2026
Mycophenolate mofetil has been shown to be equally effective and more tolerable than methotrexate in the treatment of juvenile localized scleroderma.
Juvenile localized scleroderma (JLS), also known as morphea, is an autoimmune disease that inflames the skin and can progress to sclerosis and muscle atrophy in children. Currently, according to the study, there are no FDA-approved treatments for JLS, but methotrexate is typically the initial drug prescribed to these patients.
Methotrexate has historically been considered the first-line therapy, but in clinical practice we often see children struggling with treatment-limiting side effects, which affect adherence and quality of life.
For the retrospective study, Torok and colleagues evaluated treatment responses in 114 children with JLS enrolled in the National Registry for Childhood-Onset Scleroderma at UPMC Children's Hospital (median age at onset: 8.3 years; 67.5% female) treated with methotrexate (n = 68), mycophenolate mofetil (MMF; n = 28), or combination therapy (n = 18).
The results showed that methotrexate and MMF have similar efficacy rates in controlling disease activity and preventing JLS flare-ups. In both treatment groups, patients experienced a reduction in disease activity (≥0.14; 95% CI: 0.62-0.33) with no differences in disease flare rates over time (HR = 0.85; 95% CI: 0.51-1.33), according to Kaplan-Meier analysis.
The profile of adverse events differed between drugs. Compared with patients treated with MMF, patients treated with methotrexate reported significantly higher rates of fatigue (47% vs. 11%, P = 0.001) and nausea (60% vs. 7%; P = 0.001).