[Pubmed] Prospective study of efgartigimod in severe myasthenia gravis exacerbations requiring ventilatory or enteral su

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J Neuromuscul Dis. 2026 Aug 4:22143602261450402. doi: 10.1177/22143602261450402. Online ahead of print.

ABSTRACT

Background and objectivesTo evaluate the safety and clinical efficacy of efgartigimod in adults with severe generalized myasthenia gravis (MG) exacerbations requiring ventilatory or enteral support.MethodsProspective, single-arm, open-label study conducted between November 2024 and April 2025. Sixteen consecutive adults with AChR- or MuSK-antibody-positive generalized MG requiring invasive or non-invasive ventilatory support or enteral feeding were enrolled; all met 2016 MG Guidelines criteria for impending myasthenic crisis. MGFA classification included Class IIIb (n=14), IIIa (n=1), and IVb (n=1). The primary endpoint was clinically meaningful improvement (CMI) in MG Activities of Daily Living (MG-ADL), defined as ≥2-point reduction from baseline at weeks 4 and 8.ResultsAll 16 participants (mean age 58.5±17.4 years; 8 women) completed eight weeks of follow-up. At week 8, 12 patients (75%; 95% CI 47-91%) achieved the primary endpoint. Median MG-ADL decreased from 11 (IQR 9-13) to 7 (IQR 6-9) (median change -5 points; p=0.003). Median QMG decreased from 21 (IQR 19-23) to 13 (IQR 11-16) (median change -7.5 points; p

Source: https://pubmed.ncbi.nlm.nih.gov/4255144 ... t5+40e1b98