MMF Shows Promise in Juvenile Scleroderma
Mycophenolate mofetil demonstrated comparable disease control with fewer adverse effects.
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By
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Kathryn Wighton
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February 2, 2026
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Objective:
To compare the effectiveness and tolerability of mycophenolate mofetil (MMF) and methotrexate (MTX) in treating juvenile localized scleroderma (JLS).
Key Findings:
- Both MMF and MTX achieved inactive disease status within 12 months with no significant differences in improvement rates.
- MMF demonstrated significantly fewer adverse effects compared to MTX, including lower rates of nausea, fatigue, anxiety, and anticipatory vomiting.
- Disease flare occurred in 25% of patients overall, with no significant differences in flare-free survival between treatment groups.
Interpretation:
These findings suggest that MMF may be a viable first-line treatment option for JLS, offering similar efficacy to MTX with improved tolerability.
Limitations:
- Retrospective, nonrandomized design with unequal treatment group sizes.
- MMF was often initiated after longer disease duration or MTX intolerance, potentially biasing response estimates.
- Adverse effects were assessed through clinical interviews rather than validated measures.
- The study was not powered to detect small differences in efficacy.
Conclusion:
Prospective, randomized, noninferiority trials are needed to confirm these findings and inform future treatment guidelines.