Background Immunosuppressive therapy may potentially alter the natural disease course of scleroderma. There have been reports of using mycophenolate mofetil (MMF) for the treatment of scleroderma skin disease.
Objective To analyse the experience of using MMF for the treatment of active diffuse cutaneous scleroderma.
Methods The authors compared the change in mean modified Rodnan skin scores (mRSS) in an MMF cohort at baseline with scores at 3, 6, 9 and 12 months and with those of historical controls from a pooled analysis of three multicentre randomised clinical trials of recombinant human relaxin, d-penicillamine and oral bovine type I collagen.
Results Improvement in mRSS after treatment with MMF compared with baseline was seen as early as 3 months and continued through the 12-month follow-up. The mRSS of the MMF cohort was not different from that of the historical controls at 6 months (MMF −3.05±7.4 vs relaxin −4.83±6.99, p=0.059), but was significantly lower at 12 months (MMF −7.59±10.1 vs d-penicillamine −2.47±8.6, p<0.001; collagen −3.4±7.12, p=0.002). General and muscle severity scores and quality of life measures also improved compared with baseline. Pulmonary function remained stable.
Conclusions MMF may benefit skin disease in patients with diffuse scleroderma, but prospective studies are required to determine its role.
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Funding AAS receives funding from the American College of Rheumatology Research and Education Foundation's Clinical Investigator Fellowship Award and from the NIH grant P30 AR058885. FB is supported by the NIH K23AR055667 grant.
Competing interests None.
Ethics approval This study was conducted with the approval of The John Hopkins University School of Medicine.
Provenance and peer review Not commissioned; externally peer reviewed.