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Mycophenolate Mofetil for Pemphigus

As a possible alternative to corticosteroids, this agent expands treatment options for patients with pemphigus.

Mycophenolate mofetil is a relatively recent addition to the therapeutic armamentarium of clinical dermatologists. Its predecessor, mycophenolic acid, was poorly tolerated and poorly effective as treatment for psoriasis. Mycophenolate mofetil is used to prevent rejection in transplant recipients and has been used as a replacement for azathioprine in the treatment of many conditions. These investigators measured mycophenolate mofetil's effects in a relatively large series of patients with pemphigus.

Forty-two patients with pemphigus vulgaris (PV; 31 patients) or pemphigus foliaceus (PF; 11 patients) who had steroid-related toxicity or who relapsed while tapering corticosteroid therapy received 35 to 45 mg/kg/day of mycophenolate mofetil. Patients were treated for a median of 22 months, and the median follow-up period was 22 months. Complete remission occurred in 22 PV patients (71%) and 5 PF patients (45%), and partial remission occurred in 1 PV patient (3%) and 4 PF patients (36%). Two patients with PV had initial responses that were not sustainable. The time to remission ranged from 1 to 13 months (median, 9 months). Gastrointestinal upset was the primary adverse effect.

Comment: Long-term use of mycophenolate mofetil is thought to increase the risks for infection and neoplasia. These investigators report successful treatment of most inflammatory conditions with this agent. Mycophenolate mofetil has been useful in my practice, but I have not seen the high level of response that they report. However, this drug is probably less toxic than corticosteroids, and when used to treat conditions other than transplants, may not be associated with levels of immunosuppression resulting in neoplasia. Thus, this drug is worthy of consideration as a potential steroid-sparing agent for patients with pemphigus.

— Jeffrey P. Callen, MD

Published in Journal Watch Dermatology July 29, 2003

Citation(s):

Mimouni D et al. Treatment of pemphigus vulgaris and pemphigus foliaceus with mycophenolate mofetil. Arch Dermatol 2003 Jun; 139:739-42.

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