Novel approaches in the treatment of myositis and myopathies

Abstract

The inflammatory myopathies are a heterogeneous group of disorders characterized by muscle weakness and inflammation. Although no standard therapeutic guidelines exist, traditional treatment has included corticosteroids and a variety of second-line immunosuppressants. As treatment of refractory disease has been difficult, newer agents and approaches have been used with varying response. The advent of standardized treatment response criteria by the International Myositis Assessment and Clinical Studies (IMACS) group has helped investigators to evaluate and compare clinical trial outcomes in a more rigorous fashion. The use of intravenous immunoglobulin (IVIG), rituximab, biologic agents including tumor necrosis factor (TNF) inhibitors, stem-cell transplantation, gene therapy, and vascular occlusion resistance training are reviewed here. As our understanding of disease pathogenesis at the immunologic, genetic, and molecular level expands, the discovery of novel therapeutic targets hold promise for the successful treatment of these conditions.

Keywords: IVIG; gene therapy; idiopathic inflammatory myopathies; novel approaches; rituximab; stem-cell transplantation.