1. In patients with different types of morphea unresponsive to phototherapy and methotrexate, mycophenolate appears to be a safe and beneficial treatment option.
Evidence Rating: 2 (Good)
Morphea, otherwise known as localized scleroderma, is a rare sclerosing disorder of the skin affecting patients of all ages. Currently, first-line systemic therapy typically involves methotrexate with or without systemic corticosteroids when phototherapy is ineffective. Escalation of therapy for patients unresponsive to methotrexate is mycophenolate, but this recommendation is based primarily on extrapolation of literature from studies on systemic sclerosis, as there are limited studies actually investigating the efficacy of mycophenolate for morphea. In this multicenter retrospective cohort study, data from 77 patients obtained between 1999 to 2018 was analyzed to investigate mycophenolate use for morphea. The primary outcomes were morphea disease activity, severity, and response at 0, 3 to 6, and 9 to 12 months post treatment. Sixty-one female patients and 16 male patients were included in the study, with median age at disease onset being 36 years. Generalized morphea (48% of cases), pansclerotic morphea (16%), linear morphea of the trunk and/or extremities (12%) were the most common subtypes identified, with most patients having moderate to severe disease severity. Mycophenolate appeared to be an effective treatment, with 66 of 73 patients achieving stable (n=22) or improved (n=44) condition at 3 to 6 months, and 47 of 54 patients with stable (n=14) or improved (n=33) condition at 9 to 12 months, and 27 patients achieving disease remission at study completion. Thirty-four (44%) of patients experienced an adverse event during treatment, with the most common being GI distress. This study represents the largest study to date evaluating the efficacy and safety of mycophenolate for morphea, and appears to support current recommendations for the drug to be a beneficial and largely safe agent for administration in patients unresponsive to phototherapy and methotrexate.