Objective: To summarize the evidence for the use of mycophenolate mofetil (MMF) in non-renal manifestations of systemic lupus erythematosus (SLE).
Methods: Treatment trials in human SLE from 1990 to 2006 that have been published in the English literature were searched by Medline using the keywords 'lupus', 'mycophenolate', 'neuropsychiatric', 'neurological', 'hematological', 'dermatological', 'cutaneous', 'skin', 'hemolytic' and 'thrombocytopenia'. Laboratory studies were excluded.
Results: Twenty relevant studies were summarized. All were case series or open-labelled trials. The main indications for MMF were refractory haematological and dermatological lupus. Data regarding MMF in neuropsychiatric SLE were scant. Favourable results were reported with haematological disease. Evidence regarding the efficacy of MMF in refractory lupus skin lesions was conflicting. The efficacy of MMF in neuropsychiatric lupus was only modest and could not be separated from that of concomitant therapies. In one uncontrolled study, MMF was reported to be effective in preventing clinical flares in patients with persistently active serological markers. MMF was well tolerated in these reports.
Conclusions: Limited evidence suggests that MMF may be effective in refractory haematological and dermatological manifestations of SLE. Because of the possibility of publication bias, the efficacy of MMF in these manifestations has to be confirmed with controlled trials. The efficacy of MMF in neuropsychiatric SLE is unproven and should be restricted to those patients who are refractory and intolerant to, or reluctant for, cyclophosphamide. Finally, the current level of evidence does not support treating serology alone in SLE by MMF.