A pilot study was conducted to assess the efficacy of early treatment of severe juvenile dermatomyositis (JDMS) patients with intravenous methylprednisolone (IVMP) and methotrexate (MTX). Twelve children diagnosed with severe JDMS were treated with IVMP and MTX. Six patients were treated early (within 6 weeks of the diagnosis) while in the other six patients, MTX was started 5-72 months after the diagnosis was made. The clinical responses of the patients to treatment, including alterations in muscle strength, muscle enzyme levels and corticosteroid dosage as well as the development of side-effects, were recorded. The indications for starting the treatment were defined and documented. The primary measures of response were resolution of the clinical indications for treatment, decreased activity of the disease manifestations and tapering of the corticosteroids to the minimal dose or discontinuation without clinical or biochemical flare. The main indications for starting IVMP and MTX were dysphagia and severe cutaneous vasculitis. All the patients received MTX orally for at least 8 months, as well as IVMP (30 mg/kg/dose), but none of the patients was on additional second-line treatments. The six patients who were treated early with MTX showed a significant clinical improvement. In five out of the six, the corticosteroid dosage was eventually reduced to <5 mg/day. None of them developed calcinosis. In contrast, two of the six patients who were treated late with MTX developed calcinosis. This study suggests that MTX and IVMP are a useful combination in the early treatment of severe JDMS. Given the fact that our sample was small, further studies in a controlled trial are necessary to confirm these findings.