Effectiveness and safety of telitacicept in refractory ocular myasthenia gravis: a retrospective, real-world study.

INTRODUCTION: Refractory ocular myasthenia gravis (OMG) remains a significant therapeutic challenge with limited evidence-based biologic therapies. This study aimed to evaluate the effectiveness and safety of telitacicept, a recombinant B-lymphocyte stimulator receptor-antibody fusion protein, in refractory OMG. METHODS: This retrospective, single-center observational study assessed clinical outcomes, immunological biomarkers, and safety following add-on telitacicept therapy in refractory OMG. Disease severity was evaluated using the Myasthenia Gravis Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), and Myasthenia Gravis Impairment Index (MGII) scales. Serum immunoglobulins, complement components, and CD19+ B-cell counts were measured, and adverse events were systematically recorded. RESULTS: Twelve patients with refractory OMG were included. Median baseline MG-ADL, QMG, and MGII scores were 5.5 (interquartile range [IQR] 5.0-6.0), 7.0 (IQR 5.3-8.0), and 16.0 (IQR 12.0-18.0), respectively. Statistically significant improvement was observed as early as week 1 (all p < 0.05). By week 12, MG-ADL scores decreased by a median of 3.0 (95% CI: 2.0-4.0; p < 0.001, r = 0.89), with consistent improvements in QMG and MGII scores (all p < 0.01, all r > 0.85). Minimal symptom expression (MSE) was achieved in 7 of 12 patients (58.3%) within the 12-week period. The median daily prednisone dose was reduced from 15.0 mg (IQR 10.0-22.5) to 9.0 mg (IQR 5.0-13.8) by week 12 (median decrease: 7.5 mg, 95% CI: 2.5-13.5; p = 0.031, r = 0.79), with 1 patient discontinuing corticosteroids by week 8. Telitacicept treatment was associated with reductions in serum IgG, IgA, and IgM levels, alongside increased levels of complement components. No serious adverse events, infections, or treatment discontinuations occurred. CONCLUSION: Telitacicept was associated with rapid, sustained clinical improvement and a favorable safety profile in refractory OMG, which supports its potential role as an add-on therapeutic option.