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Low-dose intrathecal rituximab is a safe and potentially effective treatment for pediatric neuroimmunologic disorders
Journal of Neuroimmunology ( IF 3.3 ) Pub Date : 2021-07-31 , DOI: 10.1016/j.jneuroim.2021.577687
Jason A Santiago 1 , Mary Babico 2 , Gideon Stitt 2 , Christopher A Thomas 2 , Sudhen Desai 3 , Scott Willard 3 , Ted Swing 4 , Michael C Kruer 1
Affiliation  

Historically, treatment options for refractory neuroimmunologic disorders have been limited. Use of intrathecal rituximab has been described in a few case reports but experience in pediatric patients is limited. Here, we report our experience with intrathecal rituximab in 5 pediatric patients with refractory neuroimmunologic conditions. Patients were identified based on treatment-refractory symptoms despite first and second-line therapies and treated according to a standardized protocol. Although individual outcomes varied, intrathecal rituximab showed a favorable safety profile and was well-tolerated. Three out of five patients showed evidence of a positive clinical response assessed by modified Rankin score or Mitchell-Pike Opsoclonus-Myoclonus score. Findings from this retrospective observational study suggest that intrathecal rituximab is a safe and potentially effective therapy in carefully selected patients with refractory neuroimmunologic disorders despite appropriate first and second-line therapies.



中文翻译:

小剂量鞘内注射利妥昔单抗是一种安全且潜在有效的儿科神经免疫疾病治疗方法

从历史上看,难治性神经免疫疾病的治疗选择是有限的。少数病例报告中描述了鞘内注射利妥昔单抗的使用,但在儿科患者中的经验有限。在这里,我们报告了我们在 5 名患有难治性神经免疫疾病的儿科患者中鞘内注射利妥昔单抗的经验。尽管接受了一线和二线治疗,但根据治疗难治性症状确定患者,并根据标准化方案进行治疗。尽管个体结果不同,鞘内注射利妥昔单抗显示出良好的安全性并且耐受性良好。五分之三的患者显示出通过改良 Rankin 评分或 Mitchell-Pike Opsoclonus-Myoclonus 评分评估的阳性临床反应的证据。

更新日期:2021-08-05
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