神经系统疾病

H.P. Acthar® Gel in Dermatomyositis and Polymyositis Treatment Registry: An Interim Analysis

Abstract

Todd Levine, Justin Malone, Petros Efthimiou, Rup Tandan, Ara Dikranian, Aidan Levine and David Saperstein

Introduction: Therapies for dermatomyositis and polymyositis (DM/PM) include corticosteroids, immunosuppressants, and intravenous immunoglobulin (IVIg). A high proportion of patients with DM/PM are refractory to therapy. H.P. Acthar® Gel, repository corticotropin injection (RCI) is a potential anti-inflammatory treatment.

Methods: Patients enrolled in the Acthar in Dermatomyositis and Polymyositis Treatment registry were monitored for changes in the clinical parameters of DM/PM after initiation of RCI twice weekly in doses up to 80 IU per subcutaneous injection.

Results: RCI treatment effectively altered the clinical course of DM/PM in 14 of 24 patients. Positive responses to treatment were associated with disease activity at baseline and duration of treatment. Mild to moderate adverse events were reported.

Conclusions: This is the largest observational study of RCI in treatment of DM/PM to date and results indicate that RCI may be an effective, tolerable treatment for refractory patients. Controlled studies are necessary to identify any additional associations between disease state and response to RCI treatment.