Utility and safety of rituximab in pediatric autoimmune and inflammatory CNS disease
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Abstract
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.
GLOSSARY
- AE=
- adverse events;
- CMV=
- cytomegalovirus;
- CTCAE=
- Common Terminology Criteria for Adverse Events;
- DoD=
- duration of disease;
- mRS=
- modified Rankin Scale;
- NMDAR=
- NMDA receptor;
- NMO=
- neuromyelitis optica;
- NMOSD=
- neuromyelitis optica spectrum disorders;
- OMAS=
- opsoclonus myoclonus ataxia syndrome;
- PML=
- progressive multifocal leukoencephalopathy
Footnotes
Go to Neurology.org for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.
Editorial, page 111
Supplemental data at Neurology.org
- Received October 15, 2013.
- Accepted in final form February 11, 2014.
- © 2014 American Academy of Neurology
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