1. Low-dose intrathecal rituximab is a safe and potentially effective treatment for pediatric neuroimmunologic disorders
- Author
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Sudhen B. Desai, Mary Babico, Scott D Willard, Ted Swing, Michael C. Kruer, Christopher A. Thomas, Gideon Stitt, and Jason Santiago
- Subjects
Male ,Pediatrics ,medicine.medical_specialty ,Adolescent ,Immunology ,Intrathecal ,Refractory ,Humans ,Immunologic Factors ,Immunology and Allergy ,Medicine ,Effective treatment ,Child ,Injections, Spinal ,Anti-N-Methyl-D-Aspartate Receptor Encephalitis ,Opsoclonus-Myoclonus Syndrome ,Dose-Response Relationship, Drug ,business.industry ,Modified rankin score ,Low dose ,Infant ,Retrospective cohort study ,Safety profile ,Treatment Outcome ,Neurology ,Child, Preschool ,Encephalitis ,Female ,Rituximab ,Neurology (clinical) ,business ,medicine.drug - Abstract
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.
- Published
- 2021
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