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Rituximab study in pediatric autoimmune and inflammatory CNS disease


Senior Member

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.

Jonathan Edwards

I have not seen the full paper, although I may be able to access it. I would just note two things. Firstly, neuropsychiatric lupus is a very severe condition with a high mortality, also very commonly associated with severe infection. Rituximab tends to be given in lupus when other treatments have failed and the patient is critically ill, particularly in children. So I am not quite sure what to make of the rate of complications. Lupus patients are also about the only people who have antibodies against rituximab before actually receiving the drug. Otherwise the severe infusion reactions sound atypical. The other point is that immunoglobulins behave quite differently in young children following rituximab. For both reasons I am not sure on the basis of the abstract that one should draw any conclusions from the side effects reported here for other contexts. The irony is that it does not necessarily make sense to restrict rituximab to those with significant morbidity and mortality - they are the ones most likely to get side effects. For those with more benign disease (in the sense of organ damage - I realise that ME is hardly 'benign' in other ways) rituximab seems much safer. I am not saying there are not occasional severe problems but I think the conclusion may give an unbalanced impression.