Mycophenolate mofetil: A new therapeutic option in the treatment of blistering autoimmune diseases

Background: Mycophenolate mofetil (MMF), an ester of mycophenolic acid (MPA ), was approved by the Food and Drug Administration in 1995 and is currentl y primarily indicated for the prophylaxis of rejection in renal transplant patients. The drug seems also to be of value in the treatment of psoriasis and rheumatic arthritis. Recently there have been 6 reported cases of succe ssful treatment of blistering autoimmune diseases with MMF in combination w ith high dose prednisone therapy. Objective: On the basis of these: reports we: administered this new treatme nt regimen to several patients with blistering autoimmune diseases. Besides using a combination of MMF and high-dose prednisone we wanted to evaluate whether MMF monotherapy is also effective in the treatment of blistering au toimmune diseases. Methods: We administered MMF to 5 patients who had severe pemphigus vulgari s or bullous pemphigoid. Two patients received MMF in combination with high -dose prednisone therapy and 3 patients received MMF monotherapy. To our kn owledge, this is the first report of successful treatment of pemphigus vulg aris and bullous pemphigoid with MMF monotherapy. Results: All patients were completely free of symptoms within 8 to 11 weeks of therapy. Patients who had received MMF monotherapy responded as well to treatment as those who received a combination of MMF and high-dose prednis one. Conclusion: Our experiences strongly suggest that MMF monotherapy may be ef fective for patients even with severe pemphigus vulgaris and bullous pemphi goid. In addition, MMF monotherapy, at least over the short term, offers th e advantage of fewer side effects in comparison to immunosuppressive combin ation therapy and was well tolerated by our patients.