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Long‐term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease
Author(s) -
Boulos Daniel,
Ngian GeneSiew,
Rajadurai Anton,
Elford Kathleen,
Stevens Wendy,
Proudman Susanna,
Owen Claire,
Roddy Janet,
Nikpour Mandana,
Youssef Peter,
Hill Catherine,
Sahhar Joanne
Publication year - 2017
Publication title -
international journal of rheumatic diseases
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.795
H-Index - 41
eISSN - 1756-185X
pISSN - 1756-1841
DOI - 10.1111/1756-185x.13035
Subject(s) - medicine , tolerability , adverse effect , scleroderma (fungus) , mycophenolate , cohort , gastroenterology , surgery , transplantation , pathology , inoculation
Objectives To assess the long‐term efficacy and tolerability of mycophenolate mofetil ( MMF ) in patients with diffuse cutaneous systemic sclerosis (dc SS c). Methods Patients enrolled in the Australian Scleroderma Cohort study with dc SS c 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 dc SS c. Given the natural history of dc SS c where skin involvement can spontaneously improve, randomized, placebo‐controlled studies are required to confirm whether improvement can be attributed to MMF therapy.