Mycophenolate mofetil for methotrexate-resistant juvenile localized scleroderma

Rheumatology (Oxford). 2021 Mar 2;60(3):1387-1391. doi: 10.1093/rheumatology/keaa392.


Objectives: To investigate safety and efficacy of MMF in patients with severe or MTX-refractory juvenile localized scleroderma.

Methods: Consecutive juvenile localized scleroderma patients undergoing systemic treatment were included in a retrospective longitudinal study. Patients treated with MMF because they were refractory or intolerant to MTX (MMF-group) were compared with responders to MTX (MTX-group). Disease activity was assessed by Localized Scleroderma Cutaneous Assessment Tool and thermography. Disease course was established on the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan-Meier analysis.

Results: MMF and MTX groups included 22 and 47 patients, respectively. No significant difference in demographics, follow-up duration and treatment before diagnosis was observed between groups. The most represented clinical subtypes in the MMF-group were pansclerotic morphea and mixed subtype (P = 0.008 and P = 0.029, respectively), and linear scleroderma of the face in the MTX-group (P = 0.048). MMF was started because of MTX resistance (18 patients), relapse during MTX tapering/withdrawal (3 patients) and anaphylaxis to MTX (1 patient). After mean 9.4 years of follow-up, 90.9% of patients on MMF and 100% of those on MTX had inactive disease. No significant difference in relapse-free survival between the groups was found (P = 0.066, log-rank test), although MMF likely induced more persistent remission. MMF was well tolerated and combination of MMF and MTX did not increase its efficacy.

Conclusion: The present study adds strong evidence on the efficacy and tolerance of MMF in severe and/or MTX-refractory juvenile localized scleroderma. Further controlled studies are needed to prove its efficacy as first line treatment.

Keywords: Mychophenolate mofetil; juvenile; localized scleroderma; methotrexate; treatment.

MeSH terms

  • Antirheumatic Agents / therapeutic use*
  • Child
  • Female
  • Humans
  • Kaplan-Meier Estimate
  • Longitudinal Studies
  • Male
  • Methotrexate / therapeutic use*
  • Mycophenolic Acid / therapeutic use*
  • Retrospective Studies
  • Scleroderma, Localized / diagnosis
  • Scleroderma, Localized / drug therapy*
  • Scleroderma, Localized / pathology
  • Thermography
  • Treatment Failure
  • Treatment Outcome


  • Antirheumatic Agents
  • Mycophenolic Acid
  • Methotrexate