Division of Neurology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, 3501 Civic Center Boulevard, Colket Translational Research Building, 10th floor, Philadelphia, PA, 19104, USA,
Curr Treat Options Neurol. 2015 Mar;17(3):336. doi: 10.1007/s11940-014-0336-z.
The past 10 years have borne witness to increased recognition and diagnosis of pediatric multiple sclerosis (MS). Additionally, during this time period, the number of treatment options available for MS patients has increased significantly, as has the number of studies evaluating the use of these therapies in children. Though the U.S. Food and Drug Administration has not formally approved any of these therapies for use in pediatric MS, a number of injectable, oral, and intravenous treatments are currently being used off-label in these children. Disease modifying therapy should be initiated promptly following a diagnosis of MS. The patient and family should be engaged in the choice of therapy as this is likely to promote adherence. First-line options include any of the injectable therapies (glatiramer acetate, interferon beta), which have roughly similar efficacy (approximately 30 % reduction of clinical relapses). If a patient has breakthrough disease or persistent, unmanageable side effects, transition to a different first-line therapy or escalation to a second-line therapy, such as natalizumab, should be considered. Though the efficacy of second-line agents is higher, the potential risk of serious adverse effects also increases. New therapies, including oral agents, are now being rigorously studied with pediatric clinical trials and may provide safe alternatives for patients that are either unresponsive or intolerant to currently available medications. When necessary, acute exacerbations can be treated with corticosteroids. Intravenous methylprednisolone at a dosage of 30 mg/kg/day (maximum dose 1000 mg/day) for 3-5 days is recommended with severe attacks. If patients are unresponsive to corticosteroids, treatment with either intravenous immunoglobulin or plasma exchange may be required. Fatigue, spasticity, and pain can also occur in pediatric patients with MS. Medications are needed if symptoms are severe and impact quality of life.
过去 10 年来，小儿多发性硬化症（MS）的识别和诊断率有所提高。此外，在此期间，MS 患者可用的治疗选择数量显著增加，评估这些疗法在儿童中应用的研究数量也有所增加。尽管美国食品和药物管理局尚未正式批准这些疗法中的任何一种用于小儿 MS，但目前许多注射剂、口服和静脉用治疗方法正在这些儿童中被超适应证使用。确诊 MS 后应立即开始进行疾病修正治疗。应让患者及其家属参与治疗选择，因为这可能会提高治疗的依从性。一线治疗选择包括任何一种注射用治疗药物（醋酸格拉替雷、干扰素β），它们的疗效大致相似（大约可使临床复发减少 30%）。如果患者发生突破性疾病或持续存在无法耐受的不良反应，应考虑转为另一种一线治疗或升级为二线治疗，如那他珠单抗。虽然二线药物的疗效更高，但严重不良反应的潜在风险也会增加。现在，新的治疗方法，包括口服药物，正在进行严格的儿科临床试验，可能为那些对现有药物无反应或不耐受的患者提供安全的替代方案。必要时，可使用皮质类固醇治疗急性恶化。建议严重发作时给予 30mg/kg/天（最大剂量 1000mg/天）的静脉用甲泼尼龙治疗 3-5 天。如果患者对皮质类固醇无反应，可能需要使用静脉用免疫球蛋白或血浆置换进行治疗。小儿 MS 患者还可能出现疲劳、痉挛和疼痛。如果症状严重并影响生活质量，则需要使用药物治疗。
