Real-World Clinical Outcomes and Corticosteroid Tapering in Telitacicept-Based Therapy for Ocular Myasthenia Gravis: An Exploratory Analysis by Prior Treatment History.

Abstract

Introduction: Ocular myasthenia gravis (OMG) is an autoantibody-mediated disease characterized by extraocular muscle weakness. Telitacicept has shown potential in treating autoimmune diseases, but its efficacy and corticosteroid-tapering outcomes in OMG remain unexamined.

Methods: This retrospective, multicenter study evaluated the efficacy and corticosteroid-tapering outcomes of telitacicept in 19 OMG patients (11 with refractory OMG and eight with newly/reinitiated treatment OMG). The primary endpoint was the proportion of patients achieving minimal symptom expression (MSE) within 6 months. Secondary endpoints included changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores and reductions in daily prednisone dosage, cumulative response rate (CRR), and short-term tolerability.

Results: Following 6 months of telitacicept treatment, 68.42% of the overall cohort achieved MSE, with the median MG-ADL score significantly decreasing from 3.00 to 0.00 (p < 0.0001). Daily prednisone dosage substantially decreased: the median daily prednisone dose fell from 20.00 to 5.00 mg (p < 0.0001), and 84.21% of patients received prednisone at ≤5 mg/day at month 6. All patients met the predefined initial response criterion within 5 weeks. The refractory and newly/reinitiated treatment groups achieved final MSE rates of 72.7% and 62.5%, respectively, and both groups showed significant reductions in prednisone dependence. This cohort had no serious adverse events.

Conclusion: In this exploratory cohort, telitacicept-based therapy may represent a promising option for OMG, with favorable clinical outcomes, substantial prednisone dose reduction, and acceptable short-term tolerability in patients with different prior treatment histories. Further prospective controlled studies are warranted.