Understanding the burden of refractory myasthenia gravis

• Published in: Therapeutic advances in neurological disorders Vol. 12; p. 1756286419832242
• Main Authors: Schneider-Gold, Christiane, Hagenacker, Tim, Melzer, Nico, Ruck, Tobias
• Format: Journal Article
• Language: English
• Published: London, England SAGE Publications 2019; SAGE PUBLICATIONS, INC; SAGE Publishing
• Subjects: Acetylcholine receptors; Autoantibodies; Immunoglobulins; Immunomodulation; Intravenous administration; Mental disorders; Myasthenia; Myasthenia gravis; Neuromuscular junctions; Patients; Quality of life; Review; Skeletal muscle; treatment; unmet need; disability; tolerability; side effects; definition; refractory myasthenia gravis; quality of life; burden
• ISSN: 1756-2864; 1756-2856; 1756-2864
• DOI: 10.1177/1756286419832242

Myasthenia gravis (MG) is an autoantibody-mediated disease that compromises the acetylcholine receptors or associated structures of the postsynaptic membrane of the neuromuscular junction. This leads to impaired neuromuscular transmission and subsequent fluctuating fatigability and weakness of ocular, bulbar, and limb skeletal muscles. Over the past few decades, there have been significant advances in our understanding of the disease pathophysiology and improvements in prognosis due to intensive care medicine and immunomodulation. Despite this, an estimated 10–20% of patients with MG do not achieve an adequate response, are intolerant to conventional treatment, or require chronic treatment with intravenous immunoglobulins or plasma separation procedures. Such patients are regarded as having MG that is ‘refractory’ to treatment and may represent a distinct clinical subgroup. Because the majority of patients with MG have well-controlled disease, the burden of illness in the minority with refractory disease is poorly understood and may be underestimated. However, clinically these patients are liable to experience extreme fatigue, considerable disability owing to uncontrolled symptoms, and frequent myasthenic crises and hospitalizations. Both acute adverse effects and an increased risk of comorbidity from treatment regimens may contribute to reduced quality of life. As yet, little is known concerning the impact of refractory MG on mental health and health-related quality of life. This review aims to highlight the burden of disease and unmet needs in patients with refractory MG.