Pohl D, Waubant E, Banwell B, Chabas D, Chitnis T, Weinstock-Guttman B, Tenembaum S
Department of Pediatrics and Pediatric Neurology, Georg-August-University Goettingen, Germany.
Neurology. 2007 Apr 17;68(16 Suppl 2):S54-65. doi: 10.1212/01.wnl.0000259407.40023.ab.
Studies in adult patients with multiple sclerosis (MS) suggest significant benefit of early treatment initiation. However, there are no approved therapies for children and adolescents with MS. For adult MS, tolerability and efficacy of several immunomodulatory and immunosuppressive drugs have been demonstrated. Guidelines for the use of these MS therapies in children do not exist. Several small cohort studies of the safety and tolerability of disease-modifying therapies (DMT) in children and adolescents with MS have been recently reported. The side effects of interferon beta (IFNB) and glatiramer acetate (GA) appear to be similar to those reported by adults. The long-term tolerability and safety have yet to be established and efficacy data have yet to be studied. In view of the potential for significant long-term physical and cognitive disability in children with MS, and recent evidence that initiation of immunomodulatory therapy early in the course of MS improves long-term prognosis, an increasing number of children and adolescents with MS are being offered the DMT approved for adults. This review summarizes current knowledge of DMT in pediatric MS and experience in several centers treating pediatric MS and MS variants such as neuromyelitis optica or Devic disease, Balo concentric sclerosis, Marburg acute MS, and Schilder disease (myelinoclastic diffuse sclerosis). Finally, an overview of symptomatic MS therapies and experiences with these treatments in pediatric patients is provided.
针对成年多发性硬化症（MS）患者的研究表明，早期开始治疗有显著益处。然而，目前尚无获批用于儿童和青少年MS患者的疗法。对于成年MS患者，已证实几种免疫调节和免疫抑制药物的耐受性和疗效。目前不存在关于在儿童中使用这些MS疗法的指南。最近有几项关于儿童和青少年MS患者疾病修饰疗法（DMT）安全性和耐受性的小型队列研究报告。干扰素β（IFNB）和醋酸格拉替雷（GA）的副作用似乎与成人报告的相似。其长期耐受性和安全性尚未确立，疗效数据也有待研究。鉴于MS患儿有出现严重长期身体和认知残疾的可能性，且近期有证据表明在MS病程早期开始免疫调节治疗可改善长期预后，越来越多的MS患儿和青少年正在接受获批用于成人的DMT治疗。本综述总结了儿科MS中DMT的现有知识以及几个治疗儿科MS和MS变异型（如视神经脊髓炎或Devic病、巴洛同心圆性硬化、马尔堡急性MS和席尔德病（髓鞘破坏弥漫性硬化））中心的经验。最后，提供了对症MS疗法的概述以及这些治疗方法在儿科患者中的经验。
