Major Finding: At the end of the 12-month study, 17 of 24 patients randomized to receive placebo and prednisone experienced a relapse, compared with 15 of 46 patients randomized to methotrexate and prednisone.
Data Source: A randomized, double-blind, placebo-controlled trial of 70 children with juvenile localized scleroderma.
Disclosures: Dr. Zulian said he had no relevant financial disclosures.
ATLANTA — Methotrexate was an effective, well-tolerated treatment for juvenile localized scleroderma in a randomized, double-blind, placebo-controlled trial of 70 patients with active disease.
At the end of the 12-month study, 31 of 46 patients randomized to receive methotrexate had responded to treatment and were flare-free, compared with 7 of 24 patients given placebo, Dr. Francesco Zulian reported.
Participants were aged 6–17 years, and had active localized scleroderma of linear, generalized, or deep subtypes with onset before age 16.
Methotrexate patients received an oral dose of 15 mg/m
Patients' lesions were evaluated using a computerized scoring system, and changes were quantified using a skin score rate. Active lesions were monitored by clinical exam and serial thermography. Mean skin score rates fell significantly from 1.0 at baseline to 0.79 in the patients on methotrexate, but did not decrease in the placebo patients. The mean target lesion temperature on thermography decreased by 44% in the methotrexate group and by 12% in the placebo group, a significant difference, he said.
Adverse events occurred in 26 of 46 patients on methotrexate, and in 11 of 24 on placebo. In the methotrexate group, adverse events included alopecia, nausea, headache, fatigue, hepatotoxicity, weight gain, and striae rubra. The only adverse events in the placebo group were weight gain and striae rubra.
Interestingly, weight gain of more than 5% of body weight occurred in 11% of the methotrexate patients and 42% of the placebo patients, Dr. Zulian said, noting that this may suggest there is a “biological or pathophysiological link” between prednisone and methotrexate that tempers this steroid-related side effect.
None of the side effects was severe enough to stop treatment, but both groups had a high drop-out rate due to relapse or lack of response: 31 of 46 patients on methotrexate, and 7 of 24 in the placebo group, completed the study.
Mean skin score fell from 1.0 at baseline to 0.79 in the methotrexate group, but did not fall in the placebo group.
Source DR. ZULIAN