A randomized, double-blind, placebo-controlled trial of infliximab in refractory polymyositis and dermatomyositis.

Objective: To investigate in a pilot study the safety and efficacy of infliximab in patients with refractory dermatomyositis (DM) and polymyositis (PM). Methods: A randomized, double-blind, placebo-controlled trial including subjects with active DM or PM. Participants had stable doses of immunosuppressive medication and prednisone (<= 0.5 mg/kg/day), and exhibited clinical signs of muscle weakness for at least 4 weeks prior to study entry. Participants received infusions of either placebo or infliximab 5 mg/kg at 0, 2, 6, and 14 weeks in blinded manner. The primary outcome was a >= 15% manual muscle strength (MMT) improvement at week 16 compared to week 0. The secondary outcome measures were improvement defined by the International Myositis Assessment and Clinical Studies Group (IMACS) criteria. At week 16, responders in each arm had the option of either continuing the same treatment or changing to the non-responder treatment for that study arm. Non-responders in the 5 mg/kg infliximab arm were increased to infliximab 7.5 mg/kg for weeks 22, 30, and 38. Non-responders in the placebo arm at week 16 received infliximab 5 mg/kg at weeks 16, 18, 22, 30, and 38. Outcomes were reassessed at week 40. Results: Twelve subjects completed the study to week 16. Six of the 12 subjects received infliximab treatment at the dose of 5 mg/kg with only one subject meeting the responder criteria at that dose. Of the remaining five subjects on infliximab, three crossed over to the infliximab 7.5 mg/kg dose. One of those three subjects responded. All six patients in the placebo arm crossed over to the 5 mg/kg dosing regimen after week 16, and two of those responded to infliximab. Conclusions: Infliximab therapy for patients with refractory PM and DM was well tolerated and may benefit a subset of patients. Published by Elsevier Inc.