OBJECTIVES: Myasthenia gravis (MG) is a chronic autoimmune neurological disorder characterized byfatigable muscle weakness resulting from defective transmission at the neuromuscular junction. Weassessed evidence gaps in the literature for the burden of disease and treatment of MG.
METHODS: A targeted review of literature published between May 4, 2013, and May 4, 2023 wasconducted in PubMed, Embase, and the Cochrane Library using a predefined search strategy for articlesdescribing the disease; epidemiology; clinical, humanistic, and economic burden; and treatment patternsof MG. Online searches were also conducted for health technology assessments, prescribing information,treatment guidelines and clinical trials.
RESULTS: We identified and reviewed 251 articles. Our analysis indicated that previous epidemiologicestimates may not reflect the current disease landscape. Symptoms and comorbidities of MG are wellstudied, but differences by autoantibody subtype, age of onset, and geography are not reportedadequately. Various instruments have been used to assess the humanistic burden, including tools specificfor MG, neurological disorders, psychological disorders, and fatigue, as well as generic instruments.However, detailed studies on differences across subgroups are needed. Studies reporting cost in MG varydue to differences in health care resources included in the analyses. The cost of an initial exacerbation inthe United States exceeded $43,000, with additional MG-related events costing over $24,000. Recentdevelopments in the therapeutic landscape necessitate updates to these analyses. Besides traditionalnon-specific immunosuppressive agents, recently available biologics (e.g., eculizumab, ravulizumab,efgartigimod, rozanolixizumab, zilucoplan) are increasingly used in MG to target specific components ofthe immune pathway. Therefore, treatment pattern studies are needed to assess the real-worldeffectiveness of emerging therapies.
CONCLUSIONS: We identified several gaps in the literature, including the need for comprehensive studiesto advance our understanding of the epidemiology, humanistic and economic burden, as well as theevolving treatment pathways in MG.
