Immunotherapy in neuromuscular disorders

Hamza Malek MD (Dr. Malek of Northwestern University Feinberg School of Medicine has no financial relationships to disclose.)
Jinny O Tavee MD (Dr. Tavee of Northwestern University Feinberg School of Medicine was a clinical investigator for Araim Pharmaceuticals.)
Emma Ciafaloni MD, editor. (

Dr. Ciafaloni of the University of Rochester received consulting fees from Biogen and a research grant from Sarepta.

)
Originally released September 8, 2011; last updated January 16, 2018; expires January 16, 2021

This article includes discussion of immunotherapy in neuromuscular disorders, chronic inflammatory polyradiculoneuropathy, multifocal motor neuropathy, paraproteinemic neuropathies, myasthenia gravis, and inflammatory myopathies. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.

Overview

An increasing number of immunotherapies are being used to treat the heterogeneous group of neuromuscular diseases believed to have an autoimmune pathogenesis. Current treatment options are discussed for the most frequent of these diseases: inflammatory neuropathies, myasthenia gravis, and the inflammatory myopathies. New therapies used in these diseases, including biological agents (monoclonal antibodies or recombinant proteins), are also reviewed.

Key points

 

• Classic immunosuppressants remain the most beneficial and widely used drugs for immune-mediated neuromuscular diseases.

 

• Most patients with immune-mediated neuromuscular diseases experience improvement of symptoms and quality-of-life measures with appropriate treatment.

 

• Several specific therapies have emerged as potential treatments for immune-mediated neuromuscular diseases.

 

• Subcutaneous immunoglobulin is a less expensive and better tolerated option than intravenous immunoglobulin.

 

• Biological agents have emerged as effective therapies for patients with treatment-resistant immune-mediated neuromuscular diseases.

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