Mycophenolate mofetil as an effective corticosteroid-sparing therapy for recalcitrant dermatomyositis.

BACKGROUND Dermatomyositis (DM) is a multisystem idiopathic inflammatory disorder that most commonly affects the muscles and skin. Systemic corticosteroids are the mainstay of therapy but are limited by their long-term adverse effects. OBSERVATIONS We sought to evaluate the effectiveness of oral mycophenolate mofetil in patients with cutaneous lesions of DM recalcitrant to other therapies through an open-label retrospective medical chart review of patients in a university-affiliated private practice setting. Twelve patients with DM who had skin lesions recalcitrant to traditional therapies or who developed toxic effects from traditional therapies began mycophenolate mofetil treatment at doses ranging from 500 mg to 1 g twice a day. Response was based on improvement in skin disease as judged clinically, an increase in strength, and/or an ability to decrease or discontinue concomitant therapies. Improvement was seen in 10 of the 12 patients, most within 4 to 8 weeks. Most patients tolerated mycophenolate mofetil treatment without problem; however, 1 patient developed a B-cell lymphoma of the central nervous system, and another developed abnormal levels of hepatic enzymes along with urinary symptoms. Resolution of these toxic reactions occurred with cessation of mycophenolate mofetil treatment in each patient. CONCLUSION Mycophenolate mofetil may be an effective corticosteroid-sparing therapy for the treatment of some patients with DM.