Arthritis & Rheumatism, Volume 62,
November 2010 Abstract Supplement
Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Atlanta, Georgia November 6-11, 2010.
Long-Term Benefit of Mycophenolate Mofetil for Treatment of Diffuse Cutaneous Systemic Sclerosis.
Le3, Elizabeth, Wigley1, Fredrick M., Shah2, Ami A., Boin2, Francesco, Hummers2, Laura K.
No drug has been demonstrated to successfully control active diffuse cutaneous scleroderma, but immunosuppressive therapy, particularly if given at the early inflammatory phase of diffuse skin disease, could alter the natural course of the disease. In this observational study, we analyzed our Scleroderma Center's experience using mycophenolate mofetil (MMF) for treatment of diffuse skin disease and compared changes in skin score with data from historical controls.
Scleroderma patients in our Center's database started on MMF primarily for active skin disease were included in the analysis. Historical controls were obtained from 3 large multicenter randomized clinical trials (RCTs) of other medications (D-penicillamine (D-pen), Recombinant Human Relaxin (Relaxin), and Oral Bovine Type I Collagen (Collagen) Trials). Data from these 3 RCTs were pooled and analyzed irrespective of treatment assignments because none of the primary and secondary outcomes differed in the active agent groups compared with the placebo or control groups. The overall mean change in modified Rodnan skin scores (mRSS) at 6 months for the Relaxin group, and at 12 months for the D-pen and Collagen groups were compared to those of our MMF cohort at 6 and 12 months, respectively, using Student's t-tests.
Of 2571 patients in our database, 99 patients satisfied our inclusion criteria and were included in the primary analysis. Patients were mostly female (82%) of white (76%) or black (14%) race. The mean age at MMF initiation was 48.2 ± 11.2 years, and the mean scleroderma duration at MMF initiation was 21.8 ± 77.5 months with a median of 12 months (IQR 823). The median MMF dose was 3 grams daily (range 0.53 gm daily). The mean baseline mRSS of the MMF cohort (24.2 ± 9.5) was not significantly different from that of the Collagen group (26.1 ± 7.8, p= 0.08), but was significantly higher than the D-pen group (21.0 ± 8, p=0.006) and lower than the Relaxin group (27.3 ± 6.9, p=0.001). The mean mRSS of the MMF cohort progressively decreased over time and was statistically significant compared to baseline at 6 months (21.4 ± 10.6, p=0.0003), 9 months (17.5 ± 10.3, p<0.0001), and 12 months (17.3 ± 10.5, p<0.0001), but not at 3 months (23.4 +/- 10.1). Although the change in mean mRSS between the control population (Relaxin: -4.83 ± 6.99) and the MMF cohort (-3.07 ± 7.4) at 6 months was similar (p=0.06), a statistically significant improvement in mRSS was detected at 12 months in the MMF cohort (-7.55 ± 10.0) compared to controls (D-pen: -2.47 ± 8.6, p 0.0009; Collagen: -3.4 ± 7.12, p 0.001). Compared to baseline, at 12 months there was also a statistically significant improvement in quality of life (p<0.0001) as indicated by the Health Assessment Questionnaire disability index and in the general (p=0.006) and muscle (p=0.01) Medsger severity scores. Forced vital capacity and diffusing capacity remained stable at 1 year.
This study suggests that mycophenolate mofetil can be an effective therapy for active diffuse skin disease in scleroderma, but the full benefits of the drug may be evident after long-term therapy.
To cite this abstract, please use the following information:
Le, Elizabeth, Wigley, Fredrick M., Shah, Ami A., Boin, Francesco, Hummers, Laura K.; Long-Term Benefit of Mycophenolate Mofetil for Treatment of Diffuse Cutaneous Systemic Sclerosis. [abstract]. Arthritis Rheum 2010;62 Suppl 10 :2192