Mycophenolate mofetil in the treatment of scleroderma-associated diffuse skin disease: Results from the Australian scleroderma cohort study.

Abstract

Aim: Recent small studies have reported an improvement in skin scores in patients with diffuse systemic sclerosis (dSSc) treated with mycophenolate mofetil (MMF). We report our findings of the efficacy and tolerability of MMF in early dSSc in patients from the Australian Scleroderma Cohort Study (ASCS). Method(s): Patients with dSSc commenced on MMF within 2.5 years of skin disease onset were identified from the ASCS database. Modified Rodnan skin scores (mRSS) at baseline (date of MMF commencement) and subsequent annual assessments were retrieved. The paired t-test was used to compare baseline mRSS with annual skin scores through to 72 months. Result(s): 34 patients with dSSc met inclusion criteria and received MMF for skin disease for at least 12 months. The mean duration of skin disease prior to MMF commencement was 1.74 +/- 1.56 years. Mean age at commencement was 44.49 +/- 12.88 years and mean duration of MMF treatment was 3.21 +/- 2.49 years. Mean MMF dose was 1933 +/- 430 g/day. The mean mRSS was 23.32 at baseline, 20.51 at 12 months, 19.21 at 36 months, 17.63 at 48 months, 16.36 at 60 months and 14.20 at 72 months. A significant reduction in mRSS was not seen until 48 months (p = 0.002) and 60 months (p = 0.001) with a change from baseline of 6.5 and 11.6 respectively. No significant change was seen in oral aperture or finger-palm distance. MMF was well tolerated with 2 patients reporting gastrointestinal disturbance. Conclusion(s): Treatment withMMFin dSSc produced only modest improvement in skin scores at 48 months. This result is less favourable than that seen in previous studies and may be due to the longer duration of disease prior to MMF treatment in our cohort. Given the tendency for spontaneous improvement in mRSS in some patients, further placebo-controlled studies of patients with early, progressive disease are required to determine if MMF is of benefit.