Telitacicept for refractory AChR-positive generalized myasthenia gravis: a retrospective real-world study

Background and aims: Treating refractory acetylcholine receptor-positive generalized myasthenia gravis (AChR+ gMG) remains challenging, especially for patients requiring long-term immunosuppressive therapy. Current treatments often lack specificity and pose significant long-term risks, underscoring the need for alternatives. Telitacicept, a novel dual inhibitor of B lymphocyte stimulator (BLyS) and proliferation-inducing ligand (APRIL), offers a promising targeted therapeutic approach. This study aimed to evaluate the efficacy and safety of telitacicept in the treatment of refractory AChR+ generalized myasthenia gravis. Methods: This retrospective study included 42 patients with refractory AChR+ gMG who received telitacicept. The primary outcomes assessed were changes from baseline in Quantitative Myasthenia Gravis (QMG) scores, analyzed using mixed-effects models. Secondary outcomes comprised cumulative response rates, reductions in concomitant immunosuppressive medications, and safety events. Results: A total of 42 refractory MG patients with MGFA class II-IV were enrolled. Significant improvements were observed in the QMG total score (least-squares [LS] mean change at month 5: -2.24, 95% CI -3.34 to -1.15, p