Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease

Daniel Boulos, Gene-Siew Ngian, Anton Rajadurai, Kathleen Elford, Wendy Stevens, Susanna M Proudman, Claire Owen, Janet Roddy, Mandana Nikpour, Peter Paul Youssef, Catherine Hill, Joanne Sahhar

Research output: Contribution to journalArticleResearchpeer-review

8 Citations (Scopus)


Objectives: To assess the long-term efficacy and tolerability of mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc). Methods: 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. Results: 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: 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.

Original languageEnglish
Pages (from-to)481-488
Number of pages8
JournalInternational Journal of Rheumatic Diseases
Issue number4
Publication statusPublished - 1 Apr 2017


  • diffuse
  • mycophenolate mofetil
  • scleroderma
  • systemic sclerosis

Cite this