Methotrexate and Mycophenolate Mofetil and Clinical Response in Juvenile Localized Scleroderma

Area of Science:

• Rheumatology
• Pediatrics
• Dermatology

Background:

• Juvenile localized scleroderma (JLS) lacks FDA-approved treatments.
• Methotrexate (MTX) is a common first-line therapy for JLS.
• Mycophenolate mofetil (MMF) shows promise as an alternative treatment due to potential benefits in tolerability and adherence.

Purpose of the Study:

• To compare the clinical outcomes of JLS patients treated with MTX versus MMF.
• To evaluate the efficacy and tolerability of MMF compared to MTX in managing JLS.

Main Methods:

• Retrospective cohort study of JLS patients from January 2010 to January 2023.
• Patients received MTX monotherapy, MMF monotherapy, or combination therapy (CT).
• Disease activity was assessed using the Localized Scleroderma Cutaneous Assessment Tool; flare rates were analyzed using Kaplan-Meier analysis.

Main Results:

• No significant differences in disease activity reduction or flare rates were observed between MTX and MMF groups.
• Patients treated with MTX reported significantly higher rates of fatigue and nausea compared to MMF.
• MMF group showed comparable treatment response with improved tolerability.

Conclusions:

• MMF demonstrates similar efficacy to MTX in treating JLS.
• MMF offers potential advantages in tolerability and adherence for JLS patients.
• Further randomized controlled trials are needed to confirm MMF as a first-line JLS treatment.