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在青少年多发性硬化症患者中从干扰素 β-1a 转换为芬戈莫德的疗效:病例报告。

Efficacy of fingolimod after switching from interferon β-1a in an adolescent with multiple sclerosis: case report.

机构信息

Department of Clinical and Experimental Medicine, Neurology Unit, Azienda Ospedaliero Universitaria Pisana, University of Pisa, via Roma 67, 56126, Pisa, Italy.

出版信息

Neurol Sci. 2021 May;42(Suppl 1):5-7. doi: 10.1007/s10072-021-05170-w. Epub 2021 Mar 16.

DOI:10.1007/s10072-021-05170-w
PMID:33723709
Abstract

Pediatric-onset multiple sclerosis (POMS) accounts for approximately 2-10% of all cases of multiple sclerosis (MS) and is associated with higher levels of disease activity than adult-onset MS, including higher rates of clinical relapse and a greater incidence of new T2 lesions on magnetic resonance imaging (MRI). First-line therapy for POMS usually includes interferon β or glatiramer acetate; however, there is limited evidence from randomized trials regarding the safety and efficacy of these disease-modifying drugs in pediatric patients. Fingolimod represents a second-line therapy option for relapsing-remitting MS in pediatric patients. Here, we report the case of a 14-year-old girl with a diagnosis of POMS who started interferon β-1a as first-line therapy and then switched to fingolimod after 12 months due to radiologic progression and clinical relapse. The patient subsequently experienced clinical stability and showed minimal radiologic activity on follow-up MRI. Our case demonstrates the real-world clinical effectiveness and safety of fingolimod in pediatric MS and is in line with the results of previous randomized and observational studies.

摘要

儿科发病多发性硬化症(POMS)约占所有多发性硬化症(MS)病例的 2-10%,其疾病活动水平高于成人发病 MS,包括更高的临床复发率和磁共振成像(MRI)上新 T2 病变的更高发生率。POMS 的一线治疗通常包括干扰素β或那他珠单抗;然而,关于这些疾病修正药物在儿科患者中的安全性和疗效,随机试验的证据有限。芬戈莫德是儿科患者复发缓解型 MS 的二线治疗选择。在这里,我们报告了一例 14 岁女孩的病例,该患者被诊断为 POMS,最初接受干扰素β-1a 作为一线治疗,12 个月后因影像学进展和临床复发而改用芬戈莫德。此后,该患者的临床状况稳定,随访 MRI 显示影像学活动最小。我们的病例表明了在儿科 MS 中使用芬戈莫德的真实世界临床疗效和安全性,与之前的随机和观察性研究结果一致。

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2
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本文引用的文献

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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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Therapy of highly active pediatric multiple sclerosis.小儿活动性多发性硬化症的治疗。
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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
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Use of Disease-Modifying Therapies in Pediatric MS.儿科多发性硬化症中的疾病修正治疗的应用。
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Fingolimod Prescribed for the Treatment of Multiple Sclerosis in Patients Younger Than Age 18 Years.芬戈莫德被用于治疗18岁以下患者的多发性硬化症。
Pediatr Neurol. 2015 Aug;53(2):166-8. doi: 10.1016/j.pediatrneurol.2015.03.024. Epub 2015 Apr 2.
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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.
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Difference in disease burden and activity in pediatric patients on brain magnetic resonance imaging at time of multiple sclerosis onset vs adults.多发性硬化症发病时小儿患者与成人患者脑磁共振成像的疾病负担和活动差异。
Arch Neurol. 2009 Aug;66(8):967-71. doi: 10.1001/archneurol.2009.135.
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Increased relapse rate in pediatric-onset compared with adult-onset multiple sclerosis.与成人起病的多发性硬化症相比,儿童起病的多发性硬化症复发率更高。
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