[Pubmed] Clinical effectiveness of efgartigimod in a broad population of patients with generalized myasthenia gravis: su

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J Neurol. 2026 Jun 3;273(6):363. doi: 10.1007/s00415-026-13877-z.

ABSTRACT

BACKGROUND: The ADAPT phase 3 trial (NCT03669588) showed efgartigimod was well tolerated and efficacious in acetylcholine receptor antibody-positive (AChR-Ab +) patients with generalized myasthenia gravis (gMG). This analysis utilized data from the ADAPT trial to investigate the efficacy and safety of efgartigimod in different patient subgroups.

METHODS: ADAPT included a broad population of AChR-Ab + participants who received a stable dose of ≥ 1 (any) treatment for gMG and were randomized 1:1 to efgartigimod (10 mg/kg) or placebo (administered as four once-weekly infusions per cycle) for 26 weeks. Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) responder rates in cycles 1 and 2, and rates of treatment-emergent adverse events (TEAEs) were analyzed in the following patient subgroups: history of nonsteroidal immunosuppressive treatment (NSIST), concomitant use of gMG treatments throughout the study, and baseline patient and disease characteristics, including time since diagnosis, age, baseline MG-ADL score, body mass index (BMI), sex, and prior thymectomy.

RESULTS: In total, 129 AChR-Ab + participants were included (efgartigimod, n = 65; placebo, n = 64). Across all subgroups, higher MG-ADL and QMG responder rates were observed in participants treated with efgartigimod versus placebo during cycles 1 and 2. Rates of TEAEs were similar between participants treated with efgartigimod and placebo, regardless of concomitant gMG treatment type.

CONCLUSION: Similar to outcomes observed in the ADAPT overall population, efgartigimod was well tolerated and efficacious across a broad range of patients, regardless of NSIST treatment history, concomitant use of gMG treatments, or baseline patient and disease characteristics.

TRIAL REGISTRATION: ClinicalTrials.gov: NCT03669588 (registered September 13, 2018).

PMID:42234197 | DOI:10.1007/s00415-026-13877-z


Source: https://pubmed.ncbi.nlm.nih.gov/4223419 ... 3&v=2.20.0