Study: Mycophenolate Mofetil Effective as First-Line Option for Juvenile Localized Scleroderma

Key Takeaways

• Mycophenolate mofetil (MMF) demonstrated similar efficacy to methotrexate (MTX) in reducing disease activity in juvenile localized scleroderma.

• Flare rates did not significantly differ between MTX and MMF treatment groups.

• MMF was associated with lower rates of fatigue and nausea.

A new study comparing methotrexate (MTX) and mycophenolate mofetil (MMF) suggests MMF may offer a comparable alternative with better patient-reported tolerability outcomes.

Researchers analyzed data from 114 patients (MTX [n=68], MMF [n=28], and CT [n=18]) in the National Registry of Childhood Onset Scleroderma. Participants had disease onset before age 18 and were receiving MTX, MMF, or combination therapy (CT). The primary study endpoint was disease activity measured using the Localized Scleroderma Cutaneous Assessment Tool.

According to the data, all treatment groups experienced reductions in disease activity over time (β = −0.14; 95% CI, −0.62 to 0.33). There was no significant difference in flare rates between MTX and MMF groups (HR = 0.85; 95% CI, 0.51 to 1.33). The authors noted that tolerability varied. Fatigue was reported in 47% of MTX patients vs. 11% in the MMF group (P = 0.001), and nausea in 60% vs. 7%, respectively (P = 0.001).

“The study results suggest that MMF demonstrated a similar response to treatment as MTX in reducing disease activity in JLS, with comparable flare rates and improved tolerability,” the authors wrote. “These initial findings support MMF as a potential candidate for first-line treatment of JLS. Prospective, randomized, noninferiority trials are warranted to confirm these results and guide future treatment recommendations.”

Source: de Rosas E, et al. JAMA Dermatology. 2026. doi:10.1001/jamadermatol.2025.5662