Mycophenolate shows efficacy, tolerability in morphea

A retrospective analysis showed that patients with recalcitrant morphea benefited from mycophenolate therapy, with tolerable adverse events.

“First-line systemic therapy for morphea includes methotrexate with or without systemic corticosteroids,” Megan Arthur, MD, of the department of dermatology at the University of Nebraska, Omaha, and colleagues wrote. “When this regimen is ineffective, not tolerated or contraindicated, a trial of mycophenolate mofetil or mycophenolic acid —referred to herein as mycophenolate — is recommended; however, evidence to support this recommendation remains weak.”

The researchers conducted the retrospective cohort study to assess the efficacy and tolerability of mycophenolate in a cohort of 77 patients from eight institutions. Data for patients treated between Jan. 1, 1999, and Dec. 31, 2018, were included in the analysis.

Morphea disease activity, severity and response at 0 months, 3 to 6 months and 9 to 12 months of treatment with mycophenolate served as the primary endpoint. Tolerability served as the secondary endpoint.

The cohort was 79% female, with a median age at disease onset of 36 years (interquartile range, 16-53 years). Patients also experienced a delay of an average of 8 months (IQR, 4-14 months) for diagnosis.

The most frequently reported form of morphea was generalized disease, which occurred in 48% of the cohort, followed by pansclerotic morphea (16%) and linear morphea of the trunk and/or extremities (12%).

An associated functional impairment was reported in 53%, while 64% experienced severe disease.

Mycophenolate monotherapy or in combination with another therapy was used as first-line treatment in 12 patients. By comparison, 65 patients were treated with mycophenolate after first-line therapy was ineffective (50 patients) or poorly tolerated (21 patients). Initial treatment regimens in this group included methotrexate (74%), systemic corticosteroids (65%), hydroxychloroquine (31%) and/or phototherapy (22%).

Findings at 3 to 6 months showed that 22 patients who were treated with mycophenolate had stable disease, while 44 had improved disease.

By 9 to 12 months, 14 patients in the mycophenolate group had stable disease and 33 had improved disease.

The disease remission rate at the completion of the study was 35%.

Most patients who were treated with mycophenolate were also treated with other therapies.

Safety data showed good tolerability of mycophenolate, with 31% of patients reporting gastrointestinal events, 4% experiencing cytopenia and 3% reporting infections.

“This study suggests that mycophenolate is a well-tolerated and beneficial treatment of recalcitrant, severe morphea,” the researchers said.

Carla N. Cruz-Diaz, MD, and Anna K. Haemel, MD, both of the department of dermatology at the University of California, San Francisco, noted in an accompanying editorial that this is largest study to date demonstrating the efficacy and safety of mycophenolate in morphea. They added, however, that the Localized Scleroderma Assessment Tool score could not be used because the analysis was retrospective.

“These data provide further evidence for adding mycophenolate to the treatment armamentarium in morphea, especially in cases where methotrexate is contraindicated or ineffective,” they wrote. “Mycophenolate can also be considered as a first-line treatment in combination with systemic corticosteroids in severe morphea.” – by Rob Volansky

Disclosures: Arthur and Cruz-Diaz report no relevant financial disclosures. Please see the reports for all other authors’ relevant financial disclosures.