Spirek Benton, Brenton J Nicholas
University of Virginia School of Medicine, Charlottesville, Virginia.
Division of Pediatric Neurology, Department of Neurology, University of Virginia, Charlottesville, Virginia.
Pediatr Neurol. 2025 Mar;164:89-96. doi: 10.1016/j.pediatrneurol.2024.12.015. Epub 2025 Jan 3.
Fingolimod and ocrelizumab are approved treatments for adults with multiple sclerosis (MS); however, only fingolimod is approved by the Food and Drug Administration for the treatment of pediatric MS. Currently, there are limited data for the safety and efficacy of ocrelizumab use in children.
This retrospective cohort study included patients with relapsing-remitting MS who started either ocrelizumab or fingolimod before age 18 years. Neuroimaging, electrocardiogram, laboratory evaluation, relapse history, and side effects were recorded at baseline and every six months.
Thirty-six pediatric patients were included (fingolimod, n = 14; ocrelizumab, n = 22). Clinical relapses occurred in 14% of patients treated with fingolimod versus in none of the patients treated with ocrelizumab. Seventy-one percent of patients in the fingolimod group switched or discontinued therapy compared with 9% treated with ocrelizumab (P = 0.0001). From patients with greater than six months of treatment on the given therapy (fingolimod n = 10, ocrelizumab n = 17), 60% on fingolimod exhibited new/enlarged T2-hyperintense lesions on brain magnetic resonance imaging compared with 6% on ocrelizumab (P = 0.004). Of those treated with ocrelizumab, 10 of 22 (45%) had infusion reactions during their initial infusion. Reaction rates decreased to 20% with subsequent infusions.
Ocrelizumab is associated with fewer brain lesions, lower clinical relapse rates, and reduced discontinuation rates compared with fingolimod. Although both therapies have the potential for adverse effects, these are unlikely to prompt discontinuation of therapy in isolation. These findings highlight the benefits of ocrelizumab as a treatment option for children and youth living with MS.
芬戈莫德和奥瑞珠单抗是已获批用于治疗成人多发性硬化症（MS）的药物；然而，只有芬戈莫德获得美国食品药品监督管理局批准用于治疗儿童MS。目前，关于奥瑞珠单抗在儿童中使用的安全性和有效性的数据有限。
这项回顾性队列研究纳入了18岁之前开始使用奥瑞珠单抗或芬戈莫德的复发缓解型MS患者。在基线时以及每六个月记录神经影像学、心电图、实验室评估、复发史和副作用。
纳入了36例儿科患者（芬戈莫德组14例；奥瑞珠单抗组22例）。接受芬戈莫德治疗的患者中有14%发生临床复发，而接受奥瑞珠单抗治疗的患者中无一例复发。芬戈莫德组71%的患者更换或停止了治疗，而奥瑞珠单抗组为9%（P = 0.0001）。在接受给定治疗超过六个月的患者中（芬戈莫德组n = 10，奥瑞珠单抗组n = 17），芬戈莫德组60%的患者在脑磁共振成像上出现新的/扩大的T2高信号病变，而奥瑞珠单抗组为6%（P = 0.004）。在接受奥瑞珠单抗治疗的患者中，22例中有10例（45%）在首次输注期间出现输注反应。后续输注时反应率降至20%。
与芬戈莫德相比，奥瑞珠单抗与更少的脑部病变、更低的临床复发率和更低的停药率相关。虽然两种疗法都有产生不良反应的可能性，但这些不良反应不太可能单独导致治疗中断。这些发现凸显了奥瑞珠单抗作为儿童和青少年MS治疗选择的益处。
