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芬戈莫德治疗儿童复发缓解型多发性硬化症的有效性:两例病例报告。

Fingolimod as an effective therapeutic strategy for pediatric relapsing-remitting multiple sclerosis: two case reports.

机构信息

Neurology Unit, IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132, Milan, Italy.

Neurorehabilitation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

出版信息

Neurol Sci. 2021 May;42(Suppl 1):9-13. doi: 10.1007/s10072-021-05270-7. Epub 2021 Apr 27.

DOI:10.1007/s10072-021-05270-7
PMID:33904006
Abstract

Approximately 3-10% of patients with multiple sclerosis (MS) have onset during childhood. Pediatric MS is characterized by a relapsing-remitting course and a high relapse rate. In 2010, fingolimod (Gilenya®) was approved in the USA for the treatment of relapsing-remitting MS in adults. In 2018, both the United States Food and Drug Administration and the European Medicines Agency expanded the approved indications of fingolimod to include its use in children with relapsing MS, and the drug was approved in Italy for this indication in September 2020. We describe two cases of children with relapsing-remitting MS who were treated with fingolimod at IRCCS Ospedale San Raffaele Multiple Sclerosis Center (Milan, Italy) for more than 2 years. Our real-world data confirm that fingolimod is an effective therapeutic strategy for children with relapsing MS, and its use could be considered in pediatric patients with active disease.

摘要

约 3-10%的多发性硬化症(MS)患者在儿童时期发病。儿科 MS 的特征是复发缓解病程和高复发率。2010 年,芬戈莫德(Gilenya®)在美国获得批准,用于治疗成人复发缓解型 MS。2018 年,美国食品和药物管理局和欧洲药品管理局扩大了芬戈莫德的批准适应证,将其用于复发型 MS 儿童,该药于 2020 年 9 月在意大利获得该适应证批准。我们描述了在意大利米兰圣拉斐尔多发性硬化症中心(IRCCS Ospedale San Raffaele)接受芬戈莫德治疗超过 2 年的 2 例复发缓解型 MS 儿童的病例。我们的真实世界数据证实,芬戈莫德是治疗复发型 MS 儿童的有效治疗策略,可考虑在活动期儿科患者中使用。

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SAGE Open Med. 2023 May 8;11:20503121231171996. doi: 10.1177/20503121231171996. eCollection 2023.
2
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Neurol Sci. 2022 Apr;43(4):2641-2649. doi: 10.1007/s10072-021-05623-2. Epub 2021 Oct 1.

本文引用的文献

1
Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis.真实世界中初始疾病修正疗法在儿科多发性硬化症中的疗效。
Ann Neurol. 2020 Jul;88(1):42-55. doi: 10.1002/ana.25737. Epub 2020 May 14.
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Trial of Fingolimod versus Interferon Beta-1a in Pediatric Multiple Sclerosis.芬戈莫德与干扰素β-1a 在儿科多发性硬化症中的对比试验。
N Engl J Med. 2018 Sep 13;379(11):1017-1027. doi: 10.1056/NEJMoa1800149.
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Pediatric multiple sclerosis: a review.小儿多发性硬化症:综述
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Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents: A multicentre, Italian, retrospective, observational study.儿童多发性硬化症患者采用注射用一线药物治疗的长期随访:一项多中心、意大利、回顾性、观察性研究。
Mult Scler. 2019 Mar;25(3):399-407. doi: 10.1177/1352458518754364. Epub 2018 Jan 24.
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Multiple Sclerosis in Pediatrics: Current Concepts and Treatment Options.小儿多发性硬化症:当前概念与治疗选择
Neurol Ther. 2016 Dec;5(2):131-143. doi: 10.1007/s40120-016-0052-6. Epub 2016 Sep 17.
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Pediatric multiple sclerosis: Conventional first-line treatment and general management.儿童多发性硬化症:传统一线治疗与综合管理
Neurology. 2016 Aug 30;87(9 Suppl 2):S97-S102. doi: 10.1212/WNL.0000000000002823.
7
Elevated relapse rates in pediatric compared to adult MS persist for at least 6 years.与成人多发性硬化症相比,儿童多发性硬化症的复发率升高至少持续6年。
Mult Scler Relat Disord. 2014 Mar;3(2):186-93. doi: 10.1016/j.msard.2013.06.004. Epub 2013 Jul 12.