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


CONCLUSIONS
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.
Department of Rheumatology, Monash Health, Melbourne, Victoria, Australia.
Daniel
D
Boulos
Department of Rheumatology, Monash Health, Melbourne, Victoria, Australia.
Gene-Siew
GS
Ngian
Department of Rheumatology, Monash Health, Melbourne, Victoria, Australia.
Anton
A
Rajadurai
Department of Rheumatology, Monash Health, Melbourne, Victoria, Australia.
Kathleen
K
Elford
Department of Rheumatology, St Vincent's Health, Melbourne, Victoria, Australia.
Wendy
W
Stevens
Department of Rheumatology, Royal Adelaide Hospital, Adelaide, South Australia, Australia.|Discipline of Medicine, University of Adelaide, Adelaide, South Australia, Australia.
Susanna
S
Proudman
Department of Rheumatology, Austin Health, Melbourne, Victoria, Australia.|Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.
Claire
C
Owen
Department of Rheumatology, Royal Perth Hospital, Perth, Western Australia, Australia.
Janet
J
Roddy
Department of Rheumatology, St Vincent's Health, Melbourne, Victoria, Australia.|Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.
Mandana
M
Nikpour
Department of Rheumatology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.
Peter
P
Youssef
Discipline of Medicine, University of Adelaide, Adelaide, South Australia, Australia.|Department of Rheumatology, Queen Elizabeth Hospital, Adelaide, South Australia, Australia.
Catherine
C
Hill
Department of Rheumatology, Monash Health, Melbourne, Victoria, Australia.|Department of Medicine, Monash University, Melbourne, Victoria, Australia.
Joanne
J
Sahhar
D000328:Adult; D001315:Australia; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000627:therapeutic use*; D011446:Prospective Studies; D012074:Remission Induction; D045743:Scleroderma, Diffuse / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology; D013997:Time Factors; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid
diffuse; mycophenolate mofetil; scleroderma; systemic sclerosis