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Utility and safety of rituximab in pediatric autoimmune and inflammatory CNS disease
Russell C Dale1, Fabienne Brilot2, Lisa V Duffy2
1From the Neuroimmunology Group (R.C.D., F.B.), Children's Hospital at Westmead, University of Sydney, Australia; Boston Children's Hospital (L.V.D., M.P.G.), MA; the Division of Rheumatology (M.T., S.M.B.), The Hospital for Sick Children, University of Toronto, Canada; Children's Hospital of Philadelphia (A.T.W., S.N., B.B.), University of Pennsylvania; Paediatric Neurology and Rheumatology (E.M., A.S.), Texas Children's Hospital, Baylor College of Medicine, Houston; Assistance Publique-Hopitaux de Paris (K.D., M.T.), Hôpitaux Universitaires Paris-Sud, National Referral Center for Neuro-Inflammatory Diseases in Children, Pediatric Neurology Department and Université Paris-Sud, Inserm U1012, Le Kremlin-Bicêtre, France; Royal Children's Hospital Melbourne (E.A., A.K.), Australia; Great Ormond Street Hospital NHS Trust (M.R.E., C.H.), London; UCL Institute of Child Health (D.E., P.A.B.), London; Alder Hey Children's NHS Foundation Trust and the Institute of Infection and Global Health (R.K.), University of Liverpool, UK; Children's Hospital of Pittsburgh (G.A.), University of Pittsburgh, PA; Hacettepe University Child Neurology (B.A.), Turkey; Birmingham Children's Hospital (E.W.); John Radcliffe Hospital (K.H.), Oxford, UK; Neurosciences Unit and Mater Medical Research Institute (C.J.R.), Mater Children's Hospital, South Brisbane, University of Queensland, Australia; Alberta Children's Hospital (S.M.B.), Calgary, University of Calgary, Canada; and Evelina Children's Hospital (M.L.), London, UK. russell.dale@health.nsw.gov.au.
Insights
Rituximab shows benefit for pediatric CNS inflammatory disorders, improving outcomes in 87% of patients. However, a 7.6% risk of serious infections necessitates careful use in severe cases.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Autoimmune Disorders
Background:
- Autoimmune and inflammatory disorders of the central nervous system (CNS) in children are rare but can cause significant morbidity.
- Rituximab, a monoclonal antibody targeting B cells, has shown promise in adult neuroinflammatory conditions.
- Its utility and safety in pediatric populations remain less defined.
Purpose of the Study:
- To evaluate the effectiveness and safety of rituximab for treating various autoimmune and inflammatory CNS disorders in pediatric patients.
- To assess the impact of rituximab on neurological outcomes and functional status.
Main Methods:
- A multicenter retrospective study involving 144 children and adolescents.
- Patients diagnosed with NMDA receptor encephalitis, opsoclonus myoclonus ataxia syndrome, neuromyelitis optica spectrum disorders, neuropsychiatric systemic lupus erythematosus, and other neuroinflammatory conditions were included.
- Data on disease characteristics, rituximab treatment, adverse events, and clinical outcomes were analyzed.
Main Results:
- Rituximab treatment led to a significant improvement in functional outcomes, with 73.9% of patients achieving a modified Rankin Scale (mRS) score of 0-2 at outcome, compared to 17.4% at initiation.
- A benefit was observed in 87% of patients, with earlier treatment correlating with greater improvement.
- Adverse events included infusion reactions (12.5%) and infectious complications (7.6%), with two grade 5 infectious adverse events (deaths). No cases of progressive multifocal leukoencephalopathy were reported.
Conclusions:
- Rituximab demonstrates significant clinical utility in pediatric autoimmune and inflammatory CNS disorders, improving neurologic outcomes.
- The observed rate of serious infectious complications highlights the need for cautious use, reserving rituximab for conditions with substantial morbidity and mortality.
- Further prospective studies are warranted to confirm these findings and optimize treatment protocols.
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.
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