Ocrelizumab治疗小儿复发性多发性硬化症

IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY
Raed Alroughani , Malak AlMojel , Jasem Al-Hashel , Samar Farouk Ahmed
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引用次数: 0

摘要

目的评估Ocrelizumab治疗儿科起病多发性硬化症(POMS)的有效性和安全性。背景与成人起病多发性硬化症相比,POMS患者在更早的年龄有更高的复发和进展至继发性进展病程。文献中关于其在POMS亚群中的有效性和安全性的数据有限。方法对年龄小于18岁开始接受Ocrelizumab治疗的POMS患者进行回顾性研究,随访时间至少1年。主要终点为观察期末无复发患者的比例。次要终点是年化复发率(ARR)、扩展残疾状态量表(EDSS)评分的变化以及无MRI活动(新发/扩大的T2病变和钆(Gd)增强病变)的患者比例。结果纳入研究的24例POMS患者中,58.3%为女性,平均年龄16.33岁。在给予Ocrelizumab之前,所有患者均有临床和放射学活动,其中23例为naïve, 1例既往接受过治疗。Ocrelizumab组的中位随访时间为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 & lt;0.001. 尽管如此,只有1名患者(4.2%)在治疗过程中有MRI活动。75%的患者平均EDSS稳定,16.7%的患者改善。结论ocrelizumab可被认为是一种安全有效的治疗伴有RRMS的POMS的方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Ocrelizumab for pediatric relapsing multiple sclerosis

Objective

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.

Method

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).

Results

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.
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来源期刊
CiteScore
5.80
自引率
20.00%
发文量
814
审稿时长
66 days
期刊介绍: Multiple Sclerosis is an area of ever expanding research and escalating publications. Multiple Sclerosis and Related Disorders is a wide ranging international journal supported by key researchers from all neuroscience domains that focus on MS and associated disease of the central nervous system. The primary aim of this new journal is the rapid publication of high quality original research in the field. Important secondary aims will be timely updates and editorials on important scientific and clinical care advances, controversies in the field, and invited opinion articles from current thought leaders on topical issues. One section of the journal will focus on teaching, written to enhance the practice of community and academic neurologists involved in the care of MS patients. Summaries of key articles written for a lay audience will be provided as an on-line resource. A team of four chief editors is supported by leading section editors who will commission and appraise original and review articles concerning: clinical neurology, neuroimaging, neuropathology, neuroepidemiology, therapeutics, genetics / transcriptomics, experimental models, neuroimmunology, biomarkers, neuropsychology, neurorehabilitation, measurement scales, teaching, neuroethics and lay communication.
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