Efficacy and Safety of Telitacicept (BAFF/APRIL Inhibition) in Generalized Myasthenia Gravis: A Systematic Review and Meta-analysis of Randomized and Real-world Evidence
Shankar Biswas1, Sindhu Vasireddy3, Yashasvi Srivastava2, Jignen Prajapati4, Simran Arora5, Anagha Shankar6, Sai Pratibha Yandamuri7
1Internal Medicine, Ivano Frankivsk National Medical University, 2Ivano Frankivsk National Medical University, 3NMC Specialty Hospital, 4Internal Medicine, Gujarat University, 5University of Debrecen, 6Humanitas University, 7Tbilisi State Medical University
Objective:
To conduct the first systematic review and meta-analysis of telitacicept in generalized myasthenia gravis, pooling randomized and real-world evidence with pre-specified subgroup analyses for refractory status and dose.
Background:
To conduct the first systematic review and meta-analysis of telitacicept in generalized myasthenia gravis, pooling randomized and real-world evidence with pre-specified subgroup analyses for refractory status and dose.
Design/Methods:
PubMed, Embase, and Scopus were searched through April 2026 for studies of telitacicept in adult gMG. Two reviewers independently extracted data and assessed risk of bias. Random-effects meta-analyses (frequentist restricted maximum likelihood and Bayesian) were performed for change from baseline in Quantitative Myasthenia Gravis (QMG) score (primary outcome) and MG-Activities of Daily Living (MG-ADL). Pre-specified subgroup analyses examined refractory status and dose. Certainty of evidence was rated by GRADE.
Results:
Eight studies were included (one phase 2 randomized trial and seven retrospective cohorts; 152 telitacicept-exposed patients). At week 24, the pooled QMG change was −6.28 points (95% CI −8.91 to −3.66; I² = 89.5%; Bayesian posterior probability exceeding the minimum clinically important difference = 0.967), and the pooled MG-ADL change was −4.39 points (95% CI −5.27 to −3.51). The effect was substantially smaller in refractory cohorts (−2.6 versus −8.0 points; meta-regression p = 0.011). The 95% prediction interval for QMG at week 24 was −12.3 to −0.3 points. Safety was favorable, with no deaths and one serious adverse event (pneumonia) across 152 patients. GRADE certainty was LOW for primary efficacy outcomes.
Conclusions:
Telitacicept was associated with clinically meaningful improvements in QMG and MG-ADL in generalized myasthenia gravis, with greater benefit in non-refractory disease and a favorable short-term safety profile. Predominantly Chinese retrospective evidence, substantial between-study heterogeneity, and the absence of a peer-reviewed phase 3 publication tempered certainty.
Generative AI Usage
No, did not use generative AI in the drafting or editing in this abstract.
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