Mycophenolate mofetil (MMF) demonstrated comparable effectiveness to methotrexate (MTX) in treating juvenile localized scleroderma while causing significantly fewer adverse effects, according to a retrospective cohort study of 114 patients published in JAMA Dermatology. Researchers at the University of Pittsburgh analyzed data from the National Registry of Childhood Onset Scleroderma collected between 2010 and 2023, comparing outcomes among patients receiving MTX monotherapy, MMF monotherapy, or combination therapy. All three groups achieved inactive disease status (median modified Localized Scleroderma Skin Severity Index score of zero) by 12 months, with no statistically significant differences in disease activity trajectories or flare rates between treatments. However, tolerability differed markedly: patients on MTX experienced significantly higher rates of nausea (60% vs 7%), fatigue (47% vs 11%), anxiety (29% vs 7%), and anticipatory vomiting (18% vs 0%) compared with those on MMF. The researchers concluded that these findings support MMF as a potential first-line treatment option for juvenile localized scleroderma, though prospective randomized trials are needed to confirm noninferiority.

Source: JAMA Dermatology