Measuring efficacy for myasthenia gravis treatments: A review of biomarkers used in clinical trials.

Myasthenia gravis is an autoimmune disease that impairs neuromuscular transmission through autoantibodies, most commonly targeting acetylcholine receptors (AChR). While clinical scores like the Quantitative Myasthenia Gravis Score (QMGS) and the Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) are commonly used in trials, there is no gold standard biomarker for evaluating treatment efficacy. This scoping review aimed to identify which biomarkers are most frequently used in randomized controlled trials (RCTs) for non-surgical treatments of generalized myasthenia gravis. We searched five databases for English-language RCTs published before May 9, 2024. Of 6685 screened texts, 33 met inclusion criteria. A total of 33 distinct biomarkers were tracked across studies, with AChR antibodies, IgG, and IL-2 most frequently reported. Biomarkers were measured inconsistently and at varied intervals, limiting cross-study comparability. The lack of standardized biomarker use hinders the ability to assess treatment efficacy and perform meta-analyses. We recommend developing consensus guidelines to improve future trial quality.