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

Abstract

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.

• Authors: Daniel Boulos, Gene-Siew Ngian, Anton Rajadurai, Kathleen Elford, Wendy Stevens, Susanna Proudman, Claire Owen, Janet Roddy, Mandana Nikpour, Peter Youssef, Catherine Hill, Joanne Sahhar
• Journal: International journal of rheumatic diseases (Int J Rheum Dis) Vol. 20 Issue 4 Pg. 481-488 (Apr 2017) ISSN: 1756-185X [Electronic] England
• PMID: 28337853 (Publication Type: Journal Article, Multicenter Study, Observational Study)
• Copyright: © 2017 Asia Pacific League of Associations for Rheumatology and John Wiley & Sons Australia, Ltd.

Chemical References

• Immunosuppressive Agents
• Mycophenolic Acid

Topics

• Adult
• Australia
• Female
• Humans
• Immunosuppressive Agents (adverse effects, therapeutic use)
• Longitudinal Studies
• Male
• Middle Aged
• Mycophenolic Acid (adverse effects, therapeutic use)
• Prospective Studies
• Remission Induction
• Scleroderma, Diffuse (diagnosis, drug therapy, immunology)
• Time Factors
• Treatment Outcome