Utility and safety of rituximab in pediatric autoimmune and inflammatory CNS disease

Neurology. 2014 Jul 8;83(2):142-50. doi: 10.1212/WNL.0000000000000570. Epub 2014 Jun 11.


Objective: To assess the utility and safety of rituximab in pediatric autoimmune and inflammatory disorders of the CNS.

Methods: Multicenter retrospective study.

Results: A total of 144 children and adolescents (median age 8 years, range 0.7-17; 103 female) with NMDA receptor (NMDAR) encephalitis (n = 39), opsoclonus myoclonus ataxia syndrome (n = 32), neuromyelitis optica spectrum disorders (n = 20), neuropsychiatric systemic lupus erythematosus (n = 18), and other neuroinflammatory disorders (n = 35) were studied. Rituximab was given after a median duration of disease of 0.5 years (range 0.05-9.5 years). Infusion adverse events were recorded in 18/144 (12.5%), including grade 4 (anaphylaxis) in 3. Eleven patients (7.6%) had an infectious adverse event (AE), including 2 with grade 5 (death) and 2 with grade 4 (disabling) infectious AE (median follow-up of 1.65 years [range 0.1-8.5]). No patients developed progressive multifocal leukoencephalopathy. A definite, probable, or possible benefit was reported in 125 of 144 (87%) patients. A total of 17.4% of patients had a modified Rankin Scale (mRS) score of 0-2 at rituximab initiation, compared to 73.9% at outcome. The change in mRS 0-2 was greater in patients given rituximab early in their disease course compared to those treated later.

Conclusion: While limited by the retrospective nature of this analysis, our data support an off-label use of rituximab, although the significant risk of infectious complications suggests rituximab should be restricted to disorders with significant morbidity and mortality.

Classification of evidence: This study provides Class IV evidence that in pediatric autoimmune and inflammatory CNS disorders, rituximab improves neurologic outcomes with a 7.6% risk of adverse infections.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Aging / physiology
  • Anti-Inflammatory Agents, Non-Steroidal / therapeutic use*
  • Antibodies, Monoclonal, Murine-Derived / adverse effects*
  • Antibodies, Monoclonal, Murine-Derived / therapeutic use*
  • Autoimmune Diseases / drug therapy*
  • Blood Cell Count
  • Central Nervous System Diseases / drug therapy*
  • Child
  • Child, Preschool
  • Cohort Studies
  • Female
  • Humans
  • Immunity, Cellular / drug effects
  • Immunosuppressive Agents / adverse effects*
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Infections / epidemiology
  • Infections / etiology
  • Inflammation / drug therapy
  • Infusions, Intravenous / adverse effects
  • Male
  • Retrospective Studies
  • Rituximab
  • Treatment Outcome


  • Anti-Inflammatory Agents, Non-Steroidal
  • Antibodies, Monoclonal, Murine-Derived
  • Immunosuppressive Agents
  • Rituximab