Background

RTX is used off-label in several neurological inflammatory diseases in adults children patients. We conducted a study to assess indications and safety of rituximab (RTX) for children and to identify risk factors for early B-cell repopulation.

Methods

A single-center retrospective study of children treated with RTX for a neurological disease between May 31, 2010, and May 31, 2020, was performed.

Results

A total of 77 children (median age, 8.9 years) were included. RTX was mostly used as second-line therapy in all groups of diseases (68%). Median dose was 1500 mg/m² for each patient. There were 13 clinical relapses (17%), 5 when B-cell depletion was complete. Adverse events were present in 6% of the cases. The factors influencing early B-cell repopulation were the recent infusion of intravenous Ig (P < 0.01) and the administration of less than 1500 mg/m² during the first RTX treatment (P = 0.04). The median time to B-cell repopulation seemed to be shorter (160 vs 186 days) when patients had plasmapheresis even when a 48-hour delay was observed with RTX infusions.

Conclusions

This study confirms the good tolerance of RTX in the treatment of specific neurological disorders in a pediatric population. It also highlights risk factors for early B-cell repopulation and underlines the importance of B-cell monitoring.

中文翻译：

---

利妥昔单抗在小儿神经病学中的适应症和安全性：一项 10 年回顾性研究

背景

RTX 在成年儿童患者的几种神经炎症性疾病中超说明书使用。我们进行了一项研究，以评估利妥昔单抗 (RTX) 对儿童的适应症和安全性，并确定早期 B 细胞再增殖的风险因素。

方法

对 2010 年 5 月 31 日至 2020 年 5 月 31 日期间接受 RTX 治疗神经系统疾病的儿童进行了一项单中心回顾性研究。

结果

总共包括 77 名儿童（中位年龄，8.9 岁）。RTX 主要用作所有疾病组的二线治疗 (68%)。每位患者的中位剂量为 1500 mg/m ²。有 13 例临床复发 (17%)，其中 5 例发生在 B 细胞完全耗尽时。6% 的病例出现不良事件。影响早期 B 细胞再增殖的因素是最近静脉输注 Ig ( P < 0.01) 和第一次 RTX 治疗期间 给予低于 1500 mg/m ^{2 (} P  = 0.04)。当患者进行血浆置换时，B 细胞再增殖的中位时间似乎更短（160 天对 186 天），即使 RTX 输注观察到 48 小时延迟也是如此。

结论

这项研究证实了 RTX 在治疗儿科人群特定神经系统疾病方面的良好耐受性。它还强调了早期 B 细胞再增殖的风险因素，并强调了 B 细胞监测的重要性。