Department of Pediatrics (Neurology), University of Toronto, ON, Canada.
Paediatr Drugs. 2012 Jun 1;14(3):165-77. doi: 10.2165/11596330-000000000-00000.
Pediatric onset multiple sclerosis (MS) may be seen in 2-5% of patients with MS. It is characterized by high disease burden. As such, early treatment with preventative therapies should be considered. Although randomized controlled trials have not been conducted on therapies for pediatric MS, there is a growing body of literature suggesting safety of first-line agents approved for use in adult MS, including interferons and glatiramer acetate. The use of second-line therapies, such as natalizumab, cyclophosphamide, and mitoxantrone has been described in a small number of pediatric MS cases. These case series suggest benefit of these agents after limited follow-up. Little information on long-term effects of therapies such as cyclophosphamide, mitoxantrone, or natalizumab is available for this population, although concerns of increased risk for opportunistic infections (progressive multifocal leukoencephalopathy with natalizumab) and secondary hematologic cancers (with mitoxantrone) exist. Finally, although fatigue, motor, cognitive, and psychosocial difficulties are common in this population, no trials have been conducted on pharmacologic or non-pharmacologic interventions for the management of these problems. Therapies for spasticity, including baclofen (including the baclofen pump), diazepam, and botulinum toxin have been evaluated in children with cerebral palsy and may be used safely in children. Psychiatric intervention is often necessary for affective disorders. Interventions for fatigue have not been studied, although evidence in the adult MS literature suggests possible benefit of exercise and modafinil. This article provides a practical guide to the diagnosis and treatment of multiple sclerosis in pediatric patients.
儿科发病的多发性硬化症(MS)可能在 2-5%的 MS 患者中出现。其特点是疾病负担高。因此,应考虑早期使用预防性治疗。尽管尚未对儿科 MS 的治疗进行随机对照试验,但越来越多的文献表明,批准用于成人 MS 的一线药物(包括干扰素和那他珠单抗)是安全的。在少数儿科 MS 病例中已描述了二线治疗药物的使用,如那他珠单抗、环磷酰胺和米托蒽醌。这些病例系列研究表明,在有限的随访后,这些药物有效。对于儿科患者,有关环磷酰胺、米托蒽醌或那他珠单抗等治疗方法的长期效果的信息很少,尽管存在与纳他珠单抗相关的机会性感染(进行性多灶性白质脑病)和继发血液系统癌症(米托蒽醌)风险增加的担忧。最后,尽管疲劳、运动、认知和社会心理问题在该人群中很常见,但尚未对这些问题的药物或非药物干预进行临床试验。已在脑瘫儿童中评估了包括巴氯芬(包括巴氯芬泵)、地西泮和肉毒毒素在内的痉挛治疗药物,并且可以在儿童中安全使用。对于情感障碍,通常需要进行心理干预。尚未研究疲劳干预措施,尽管成人 MS 文献中的证据表明运动和莫达非尼可能有益。本文提供了儿科多发性硬化症诊断和治疗的实用指南。