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Meta-analysis of efgartigimod for myasthenia gravis reports efficacy and safety outcomes

Meta-analysis of efgartigimod for myasthenia gravis reports efficacy and safety outcomes
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Key Takeaway
Consider the reported efficacy and safety of efgartigimod in myasthenia gravis, noting gaps in AChR-Ab-negative subtype data.

This is a meta-analysis of intravenous efgartigimod in patients with myasthenia gravis, synthesizing data from 1594 patients. The authors report that 83% of patients achieved clinically meaningful improvement, defined as a 2-point reduction in the MG-ADL score, and 36% achieved minimal symptom expression. Significant decreases were observed in the MG-ADL score (effect size -4.3 points, 95% CI: -4.99 to -3.61) and the Quantitative MG score (effect size -3.6 points, 95% CI: -4.28 to -2.91).

Reductions were noted in MG-QoL15r, IgG levels, and corticosteroid use, but these findings were reported only for the AChR-Ab-positive subtype; outcomes were not reported for the AChR-Ab-negative subtype. The analysis found no significant difference between AChR-Ab-positive and AChR-Ab-negative subtypes for CMI and MSE, and no difference for MG-ADL score reduction.

Serious adverse events occurred in 4.42% of patients. The authors acknowledge limitations, including the lack of reported outcomes for the AChR-Ab-negative subtype. Practice relevance was not reported, and the findings should be interpreted with caution given the observational nature of the synthesized data.

Study Details

Study typeMeta analysis
Sample sizen = 1,594
EvidenceLevel 1
PublishedMay 2026
View Original Abstract ↓
OBJECTIVE: To assess the effectiveness and safety of intravenous efgartigimod in patients with myasthenia gravis (MG) and to compare treatment responses between anti-acetylcholine receptor antibody (AChR-Ab)-positive and -negative subtypes. METHODS: A comprehensive search was conducted in the PubMed, Scopus, Web of Science, and Cochrane CENTRAL databases up to 15 October 2025. Clinical trials and cohort studies evaluating the effectiveness and safety of efgartigimod in patients with MG were included. A random-effects model was used to pool mean differences (MDs) for continuous outcomes and proportions for categorical outcomes, with corresponding 95% confidence intervals (CIs). Subgroup analyses were performed based on study design and MG subtype. RESULTS: Twenty-nine studies (1594 patients) were included. Overall, 83% of patients achieved clinically meaningful improvement (CMI; ≥ 2-point reduction in MG Activities of Daily Living [MG-ADL] score), and 36% achieved minimal symptom expression (MSE; MG-ADL score of 0 or 1) with no significant difference between AChR-Ab-positive and AChR-Ab-negative subtypes. MG-ADL score significantly decreased from baseline (MD: -4.3 points, 95% CI: -4.99 to -3.61), with no difference between the MG subtypes. Quantitative MG score (QMG; MD: -3.6 points, 95% CI: -4.28 to -2.91), MG Quality of Life 15-item revised scale (MG-QoL15r), IgG levels, and corticosteroid use showed significant reductions in the AChR-Ab-positive subtype; however, these outcomes were not reported in the AChR-Ab-negative subtype. Serious adverse events were reported in 4.42% of patients. CONCLUSION: Efgartigimod significantly improved clinical symptoms and quality of life in patients with MG and may offer a steroid-sparing effect, with no significant differences observed between subtypes.
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