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芬戈莫德作为儿童发病多发性硬化症一线治疗的病例报告。

Fingolimod as first-line treatment in pediatric-onset multiple sclerosis: a case report.

机构信息

Department of Neurology and Regional Referral Multiple Sclerosis Centre, University-Hospital San Luigi, Orbassano, Italy.

出版信息

Neurol Sci. 2021 May;42(Suppl 1):25-28. doi: 10.1007/s10072-020-05027-8. Epub 2021 Mar 12.

DOI:10.1007/s10072-020-05027-8
PMID:33712907
Abstract

Pediatric-onset multiple sclerosis (MS) has a highly active and aggressive course, which can have a devastating effect on the physical and cognitive functioning of a child if not treated appropriately with effective disease-modifying drugs. The optimal treatment strategy of pediatric MS is currently unknown and debate continues as to whether treatment escalation or initiation of a highly active therapy provides a better outcome. Here, we present the case of a 16-year-old female diagnosed with highly active relapsing-remitting MS (age at onset: 14 years) who received first-line treatment with fingolimod within 1 year of the first recorded symptom. Since starting fingolimod, the course of the disease has essentially been stable. No new or active lesions were observed in magnetic resonance imaging scans performed at 3 and 12 months after starting fingolimod, and treatment was well tolerated. These data suggest that, in this case, early treatment with first-line fingolimod was able to slow disease progression.

摘要

儿童发病多发性硬化症(MS)具有高度活跃和侵袭性的病程,如果不使用有效的疾病修正药物进行适当治疗,可能会对儿童的身体和认知功能造成毁灭性影响。目前,儿童 MS 的最佳治疗策略尚不清楚,关于是否需要升级治疗或开始高活性治疗以获得更好的结果仍存在争议。在这里,我们报告了一例 16 岁女性患者的病例,该患者被诊断为高度活跃的复发缓解型 MS(发病年龄:14 岁),在首次记录症状后 1 年内接受了芬戈莫德的一线治疗。自开始芬戈莫德治疗以来,疾病的进程基本稳定。在开始芬戈莫德治疗后 3 个月和 12 个月进行的磁共振成像扫描中未观察到新的或活动病变,且治疗耐受性良好。这些数据表明,在这种情况下,早期使用一线芬戈莫德治疗能够减缓疾病进展。

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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

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Cognitive functions in pediatric multiple sclerosis: 2-years follow-up.儿童多发性硬化症的认知功能:两年随访
Neurol Res. 2020 Feb;42(2):159-163. doi: 10.1080/01616412.2019.1710417. Epub 2020 Jan 8.
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Consistent control of disease activity with fingolimod versus IFN β-1a in paediatric-onset multiple sclerosis: further insights from PARADIG.在儿科发病多发性硬化症中,与 IFNβ-1a 相比, fingolimod 能更持续地控制疾病活动:来自 PARADIG 的进一步见解。
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美国儿科多发性硬化症中新型疾病修正疗法的应用。
Neurology. 2018 Nov 6;91(19):e1778-e1787. doi: 10.1212/WNL.0000000000006471. Epub 2018 Oct 17.
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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
BMC Neurol. 2018 Mar 9;18(1):27. doi: 10.1186/s12883-018-1026-3.
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Pediatric Multiple Sclerosis: From Recognition to Practical Clinical Management.小儿多发性硬化症:从识别到实际临床管理
Neurol Clin. 2018 Feb;36(1):135-149. doi: 10.1016/j.ncl.2017.08.005.
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International Pediatric MS Study Group Global Members Symposium report.国际儿科多发性硬化症研究组全球成员研讨会报告
Neurology. 2016 Aug 30;87(9 Suppl 2):S110-6. doi: 10.1212/WNL.0000000000002880.
8
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.
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Treatment of pediatric multiple sclerosis.小儿多发性硬化症的治疗。
Curr Treat Options Neurol. 2015 Mar;17(3):336. doi: 10.1007/s11940-014-0336-z.
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Multiple sclerosis in children: an update on clinical diagnosis, therapeutic strategies, and research.儿童多发性硬化症:临床诊断、治疗策略和研究的最新进展。
Lancet Neurol. 2014 Sep;13(9):936-48. doi: 10.1016/S1474-4422(14)70093-6.