Mycophenolate mofetil in diffuse cutaneous systemic sclerosis--a retrospective analysis.

OBJECTIVES

We have assessed indications, duration and tolerability of treatment with mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc), and compared clinical outcome with a control cohort treated with other immunosuppressive drugs.

METHODS

The clinical records of 109 patients treated with MMF and 63 control subjects receiving other immunosuppressive drugs were reviewed. Data covering a 5-yr period from commencement of treatment or until last assessment date were collected.

RESULTS

MMF and control groups were well-matched in terms of basic demographic and clinical parameters. Treatment with MMF was very well tolerated. Of all patients, 12% experienced adverse reactions with gastrointestinal (GI) tract disturbances and infections being most frequent. MMF was discontinued due to disease stabilization in 9%, side effects in 8% and no effect on the disease activity in 14% of the patients. There was a significantly lower frequency of clinically significant pulmonary fibrosis in the MMF-treated cohort (P = 0.037) and significantly better 5-yr survival from disease onset and from commencement of treatment (P = 0.027 and P = 0.012, respectively). There was no significant difference between the two groups in terms of modified Rodnan skin score and forced vital capacity (FVC) change.

CONCLUSIONS

MMF is very well tolerated and appears to be at least as effective as the other current therapies for dcSSc. Our results provide support for further evaluation of MMF in a prospective trial.