Rituximab in paediatric onset multiple sclerosis: a case series
Rituximab in paediatric onset multiple sclerosis: a case series
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DOI：
10.1007/s00415-015-7979-x
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发表时间：
2016-02
影响因子：
6
通讯作者：
J. Salzer;J. Lycke;R. Wickström;H. Naver;F. Piehl;A. Svenningsson
中科院分区：
文献类型：
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作者：
J. Salzer;J. Lycke;R. Wickström;H. Naver;F. Piehl;A. Svenningsson
儿童发病的多发性硬化症 (POMS) 的特点是高炎症活性。尚未批准针对 POMS 的疾病修饰治疗。本报告的目的是报告利妥昔单抗（一种 B 细胞耗竭单克隆抗 CD20 抗体）在 POMS 中的使用。这是瑞典四个专业多发性硬化症中心的回顾性病例系列。参与者是通过瑞典 MS 登记处和我们自己的患者库存确定的。通过医疗图表审查收集数据。我们确定了 14 名接受静脉注射治疗的 POMS 患者。每 6 至 12 个月服用 500–1000 mg 利妥昔单抗。发病时的中位年龄为 14.7 岁，开始利妥昔单抗治疗时的中位年龄为 16.5 岁，中位治疗持续时间为 23.6 个月。在利妥昔单抗治疗期间，14 例患者中有 13 例没有报告复发，EDSS 评分保持稳定或下降。开始利妥昔单抗治疗 6 个月后，MRI 仅检测到 1 个新病变。没有报告严重的 AE。药物存活率为 86%。我们的数据表明，利妥昔单抗治疗对于多发性硬化症儿童来说是安全、有效且耐受性良好的。此前已发表过 9 例用利妥昔单抗治疗的 POMS 病例。他们在使用利妥昔单抗前具有较高的疾病活动度，但治疗后的安全性和疗效结果相似。利妥昔单抗治疗 POMS 的随机对照试验是有必要的。
Paediatric onset multiple sclerosis (POMS) is characterized by high inflammatory activity. No disease modifying treatment has been approved for POMS. The objective of this report was to report the use of rituximab, a B cell depleting monoclonal anti-CD20-antibody, in POMS. This is a retrospective case series at four specialized MS centres in Sweden. Participants were identified through the Swedish MS-registry and our own patient stocks. Data were collected through medical charts review. We identified 14 POMS patients treated with i.v. rituximab 500–1000 mg every 6th to 12th months. Median age at disease onset was 14.7 years, median age at rituximab treatment initiation was 16.5 years, and median treatment duration was 23.6 months. No relapses were reported, and the EDSS scores remained stable or decreased in 13 of 14 cases during rituximab treatment. Beyond 6 months from initiating rituximab treatment, only one new lesion was detected on MRI. No serious AEs were reported. The drug survival was 86 %. Our data indicate that rituximab treatment is safe, effective and well tolerated in children with MS. Nine POMS cases treated with rituximab have previously been published. They had higher disease activity pre-rituximab, but similar safety and efficacy outcomes after treatment. An RCT of rituximab in POMS is warranted.