Alroughani Raed, AlMojel Malak, Al-Hashel Jasem, Ahmed Samar Farouk
Division of Neurology, Amiri Hospital, Arabian Gulf Street, Sharq 13041, Kuwait.
Division of Neurology, Amiri Hospital, Arabian Gulf Street, Sharq 13041, Kuwait.
Mult Scler Relat Disord. 2025 Aug;100:106539. doi: 10.1016/j.msard.2025.106539. Epub 2025 May 19.
To evaluate the effectiveness and safety of Ocrelizumab treatment for Pediatric-onset multiple sclerosis (POMS) BACKGROUND: POMS patients tend to have a higher rate of relapses and progression to secondary progressive course at earlier age compared to adult-onset MS. Although Ocrelizumab, a recombinant humanized anti-CD20 monoclonal IgG1, approved by FDA and EMA for adult patients with multiple sclerosis (AOMS), limited data in the literature is found in regards to its efficacy and safety in POMS subsets.
A retrospective study was conducted on POMS patients who had Ocrelizumab treatment initiated at an age younger than 18 years old with at least one year of follow-up were included. The primary end-point was the proportion of relapse-free patients at the end of observational period. The secondary end-points were the change of annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and proportion of patients with no MRI activity (new/enlarging T2 lesions and gadolinium (Gd) enhancing lesions).
Of the 24 POMS patients enrolled in the study, 58.3 % were females with a mean age of 16.33 years. Prior to the administration of Ocrelizumab, all patients had clinical and radiological activities, of whom 23 were naïve and one had prior therapy. The median duration of follow-up under Ocrelizumab was 15 months. At the end of follow-up visits, 91.7 % of patients were relapse-free, with a decrease in the mean ARR from 1.08 to 0.08; p < 0.001. Nonetheless, only one patient (4.2 %) had MRI activity during the treatment course. Mean EDSS was stabilized in 75 % of patient and improved in 16.7 % patients. None of the patients had serious side effects CONCLUSION: Ocrelizumab can be considered a safe and effective treatment for POMS with RRMS.
评估奥瑞珠单抗治疗儿童多发性硬化症（POMS）的有效性和安全性。背景：与成人起病的多发性硬化症（AOMS）相比，POMS患者往往复发率更高，且在更年轻时就进展为继发进展型病程。尽管奥瑞珠单抗是一种重组人源化抗CD20单克隆IgG1，已被美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）批准用于治疗成人多发性硬化症（AOMS），但关于其在POMS亚组中的疗效和安全性，文献中的数据有限。
对年龄小于18岁且接受奥瑞珠单抗治疗并至少随访一年的POMS患者进行回顾性研究。主要终点是观察期末无复发患者的比例。次要终点包括年化复发率（ARR）的变化、扩展残疾状态量表（EDSS）评分以及无MRI活动（新的/扩大的T2病变和钆（Gd）增强病变）患者的比例。
在该研究纳入的24例POMS患者中，58.3%为女性，平均年龄为16.33岁。在使用奥瑞珠单抗之前，所有患者均有临床和影像学活动，其中23例为初治患者，1例曾接受过治疗。奥瑞珠单抗治疗的中位随访时间为15个月。随访结束时，91.7%的患者无复发，平均ARR从1.08降至0.08；p<0.001。然而，治疗过程中只有1例患者（4.2%）有MRI活动。75%的患者平均EDSS稳定，16.7%的患者病情改善。所有患者均无严重副作用。结论：奥瑞珠单抗可被认为是治疗复发缓解型多发性硬化症（RRMS）的POMS的一种安全有效的治疗方法。
