Long-term experience of mycophenolate mofetil for treatment of diffuse cutaneous systemic sclerosis.

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.