The efficacy and safety of rituximab for the treatment of pediatric autoimmune neuroinflammatory disorders at a single center

Purpose: Rituximab is increasingly used as a second-line treatment of neuroinflammatory disorders to improve clinical outcomes in cases refractory to conventional immunotherapy and to reduce relapses. This study aimed to demonstrate the efficacy and safety of rituximab used for pediatric autoimmune neuroinflammatory disorders. Methods: We retrospectively reviewed the medical records of 32 patients (median age, 8.5 years; range, 1.1 to 17.1; 23 girls) who received rituximab treatment at Seoul National University Children’s Hospital. The disease subgroups were anti-N-methyl-D-aspartate (NMDA) receptor (anti-NMDAR) encephalitis (n=11), opsoclonus-myoclonus ataxia syndrome (OMAS) (n=10), other suspected autoimmune encephalitis (n=5), neuromyelitis optica spectrum disorder (n=4), and chronic inflammatory demyelinating polyneuropathy (n=2). Efficacy was measured by modified Rankin Scale (mRS) scores at the initiation of rituximab administration, at 2 months after initiation, and at the last follow-up. A favorable clinical outcome was defined as an improvement of ≥2 in the mRS score or achievement of an mRS score ≤2. Safety was assessed by reviewing infusion-related adverse events and infectious complications, including progressive multifocal leukoencephalopathy. Results: Two months after the initiation of rituximab therapy, 21patients (65.6%) had a favorable response, while 26 (82.1%) had a favorable response at the last follow-up. Among the disease subgroups, anti-NMDAR encephalitis and OMAS showed especially good responses. Rituximab infusion-related adverse events were identified in nine patients (28.1%). All complications recovered spontaneously or with only symptomatic treatment. Conclusion: Rituximab can be used safely for various pediatric autoimmune neuroinflammatory diseases. Rituximab is expected to improve clinical outcomes in pediatric patients with anti-NMDAR encephalitis and OMAS.