Brain and behavior · 2026
Add-On Telitacicept Significantly Improves Outcome of Patients With Refractory Ocular Myasthenia Gravis a Real-World Case Series.
СтатьяНазвание и аннотация: Перевод в очереди
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Lin Jing, Li Yue, Gui Mengcui, Bu Bitao, Li Zhijun
Аннотация
INTRODUCTION: Refractory ocular myasthenia gravis (MG) represents a significant therapeutic challenge, as conventional immunotherapies often prove ineffective. Telitacicept, a recombinant B-lymphocyte stimulator receptor-antibody fusion protein, offers a novel immunomodulatory approach. This retrospective study evaluates its efficacy and safety in patients with MG-associated refractory ocular symptoms.
METHODS: This is a single-center retrospective cohort study. evaluated patients with refractory ocular symptoms who received telitacicept weekly between August 2024 and January 2025. We evaluated treatment efficacy using Myasthenia Gravis Foundation of America post-intervention status (MGFA-PIS), Myasthenia Gravis Impairment Index-patient-reported outcome (MGII-PRO), quantitative MG (QMG), and MG activity of daily living (ADL) scores, as well as the reduction in daily prednisone dosage at baseline and each month following treatment. The safety assessment was also evaluated.
RESULTS: Seven MGFA class 1 patients (5 females, 2 males) were enrolled, with a median onset age of 6 years (interquartile range [IQR] 2-21) and a median disease duration of 98 months (IQR 46-121). MG-ADL scores showed significant reduction with prolonged follow-up. 6/7 patients achieved ≥2-point MG-ADL improvement by the third follow-up. 4/6 patients reached minimal symptom expression at the fifth follow-up. The average ocular MG-ADL, MGII-PRO, and QMG scores revealed statistically significant improvements at the third month of follow-up, sustained through the sixth month. The regimen exhibited excellent tolerability, with no severe adverse events (e.g., hypersensitivity reactions, infections) reported.
CONCLUSION: Our preliminary findings indicate that telitacicept represents a promising and well-tolerated adjunct therapy for refractory ocular symptoms in MG, providing significant clinical evidence to support this novel therapeutic approach.
METHODS: This is a single-center retrospective cohort study. evaluated patients with refractory ocular symptoms who received telitacicept weekly between August 2024 and January 2025. We evaluated treatment efficacy using Myasthenia Gravis Foundation of America post-intervention status (MGFA-PIS), Myasthenia Gravis Impairment Index-patient-reported outcome (MGII-PRO), quantitative MG (QMG), and MG activity of daily living (ADL) scores, as well as the reduction in daily prednisone dosage at baseline and each month following treatment. The safety assessment was also evaluated.
RESULTS: Seven MGFA class 1 patients (5 females, 2 males) were enrolled, with a median onset age of 6 years (interquartile range [IQR] 2-21) and a median disease duration of 98 months (IQR 46-121). MG-ADL scores showed significant reduction with prolonged follow-up. 6/7 patients achieved ≥2-point MG-ADL improvement by the third follow-up. 4/6 patients reached minimal symptom expression at the fifth follow-up. The average ocular MG-ADL, MGII-PRO, and QMG scores revealed statistically significant improvements at the third month of follow-up, sustained through the sixth month. The regimen exhibited excellent tolerability, with no severe adverse events (e.g., hypersensitivity reactions, infections) reported.
CONCLUSION: Our preliminary findings indicate that telitacicept represents a promising and well-tolerated adjunct therapy for refractory ocular symptoms in MG, providing significant clinical evidence to support this novel therapeutic approach.