Abstract
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<0.001), with sustained benefits across ocular, limb, and bulbar areas. Cumulative response rates reached 69.9% for MGFA-PIS and 73.8% for QMG improvement (≥3-point reduction) by 6 months. Notable decreases in prednisone (LS mean -10.17 mg/day, p<0.001) and immunosuppressant use were also seen. The therapy demonstrated a promising safety profile. CONCLUSIONS: Telitacicept demonstrated significant efficacy in refractory AChR+ gMG and may reduce dependence on traditional immunosuppressants. These real-world findings support its use as a valuable treatment choice for this challenging patient group.