Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease. [International Journal of Rheumatic Diseases]

Abstract

Objectives: To assess the long-term efficacy and tolerability of mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc). Method(s): Patients enrolled in the Australian Scleroderma Cohort study with dcSSc and baseline modified Rodnan skin score (mRSS) >= 12 who were treated for a minimum of 12 months with MMF for the primary indication of skin disease were included and their prospectively collected data retrieved. Change in mRSS, the proportion with a clinically significant improvement (reduction in mRSS >= 5 from baseline) and adverse effects due to therapy were determined. Result(s): Seventy-four participants treated with MMF were identified and of these, 42 met inclusion criteria. The mean age was 53 +/- 12 years, with mean disease duration at MMF commencement of 4.8 +/- 4.3 years. Twenty-one participants (50%) commenced MMF within 2 years of disease onset and the mean duration of therapy was 2.7 +/- 1.7 years. The mean mRSS at baseline was 25.9 +/- 9.2 with a reduction of 3.7 +/- 7.1 (P = 0.07) after 1 year of therapy, 7.6 +/- 8.3 after 2 years (P = 0.01) and 10.5 +/- 10.3 after 5 years (P < 0.01). Response to treatment was not affected by disease duration at MMF commencement or baseline skin score. Eighteen participants (43%) demonstrated clinically significant improvement after 1 year, increasing to 92% after 4 years. Two participants (5%) ceased MMF due to adverse effects. Conclusion(s): MMF was associated with a modest improvement in mRSS and was well tolerated in the treatment of dcSSc. Given the natural history of dcSSc where skin involvement can spontaneously improve, randomized, placebo-controlled studies are required to confirm whether improvement can be attributed to MMF therapy.Copyright © 2017 Asia Pacific League of Associations for Rheumatology and John Wiley & Sons Australia, Ltd