Low-dose intrathecal rituximab is a safe and potentially effective treatment for pediatric neuroimmunologic disorders

J Neuroimmunol. 2021 Oct 15:359:577687. doi: 10.1016/j.jneuroim.2021.577687. Epub 2021 Jul 31.

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.

Keywords: Intrathecal; Neuroimmunology; Pediatric; Refractory; Rituximab.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Anti-N-Methyl-D-Aspartate Receptor Encephalitis / diagnosis
  • Anti-N-Methyl-D-Aspartate Receptor Encephalitis / drug therapy*
  • Child
  • Child, Preschool
  • Dose-Response Relationship, Drug
  • Encephalitis / diagnosis
  • Encephalitis / drug therapy*
  • Female
  • Humans
  • Immunologic Factors / administration & dosage*
  • Infant
  • Injections, Spinal*
  • Male
  • Opsoclonus-Myoclonus Syndrome / diagnosis
  • Opsoclonus-Myoclonus Syndrome / drug therapy*
  • Rituximab / administration & dosage*
  • Treatment Outcome

Substances

  • Immunologic Factors
  • Rituximab