Rituximab Improves Recovery Trajectories in Pediatric N-Methyl-D-Aspartate Receptor Encephalitis
Alexandra Kornbluh1, Ilana Kahn2, Leigh Sepeta3, Zhulin He4, James Brenton5, Jennifer Yang6, Coral Stredny7, Ryan Kammeyer8, Kristen Fisher9, Alexander Sandweiss9, Ayush Gupta10, Michael Sweeney11, Varun Kannan12, Catherine Otten13, NgocHanh Vu14, Jonathan Santoro15, Karla Robles Lopez16, Robert Goodrich17, Scott Otallah18, Janetta Arellano19, Lydia Marcus20, Lileth Joy Mondok21, Andrew Christiana22, Morgan Morris4, Mark Gorman23, Yike Jiang24, Melissa Wright25, Timothy Erickson26, Eyal Muscal9, Kristy Murray9, Manikum Moodley27, Duriel Hardy27, Claude Steriade22, Grace Gombolay28
1Children's National Hospital, 2Childrens National Medical Center, 3Children'S National Health System, 4Emory University, 5University of Virginia Health System, 6Rady Childrens Hospital/UCSD, 7Children's Hospital Boston, 8Childrens Hospital Colorado, 9Baylor College of Medicine, 10University of Nebraska Medical Center, 11University of Louisville, 12Emory/CHOA, 13Seattle Children's Hospital, 14Vanderbilt University, Child Neurology, 15Department of Neurology, Children's Hospital Los Angeles, 16University of Texas at Austin/DMS, 17Wake Forest University, 18Wake Forest, 19CHOC, 20University of Alabama Medical Center, Child Neurology, 21MCW - Children'S Hospital of Wisconsin, 22NYU, 23Boston Children's Hospital, 24Duke, 25University of Utah, 26Texas A&M University, 27Dell Children's Medical Center, 28Emory University/Children'S Healthcare of Atlanta
Objective:
Early first-line immunotherapy improves outcomes in pediatric N-methyl-D-aspartate receptor encephalitis (pNMDARE), while consensus guidelines reserve second-line therapies (e.g., rituximab) for refractory cases.
Background:
We evaluated outcomes in pNMDARE with rituximab treatment.
Design/Methods:
Retrospective pNMDARE cohort study across 18 institutions from the CONNECT (CONquering Neuroinflammation and Epilepsies ConsorTium) registry. Modified Rankin Scale (mRS) was evaluated at presentation, 3, 6, 12, and 24 months. Exact matching (2:1 without replacement) accounted for corticosteroids, intravenous immunoglobulin, plasmapheresis, cyclophosphamide, and presenting mRS. Generalized linear mixed models were used; adjusted models incorporated time and nested random effects.
Results:
Among 219 patients, 103 were matched (65 rituximab-treated, 38 no-rituximab). Baseline characteristics and unadjusted comparisons of mRS at each timepoint were similar. Severe disability (mRS 5) at 24 months occurred only in the no-rituximab group (n=2). In adjusted analyses across follow-ups (n=335 observations), rituximab-treated patients improved more over time (β=-1.47, 95%CI [-2.71, -0.22], p=.022). Rituximab effect varied by corticosteroid use (β=1.81, 95%CI [0.53, 3.08], p=0.006) and over time (β=-.23 per month, 95%CI [-0.45, -0.01], p=0.044). In post-hoc contrasts versus neither treatment, rituximab-alone showed the greatest mRS improvement (-2.30; 95%CI [-3.30, -1.30], p<.001), followed by rituximab + corticosteroids (-2.16; 95%CI [-3.14, -1.18], p<.001), and corticosteroids-alone (-1.66; 95%CI [-2.90, -0.43], p=.009). mRS did not differ between rituximab + corticosteroids versus rituximab-alone (0.14; 95%CI [-0.20, 0.48], p=.413) or corticosteroids-alone (-0.50; 95%CI [-1.44, 0.45], p=.297).
Conclusions:
Rituximab treatment in pNMDARE improves recovery trajectory, although benefits may be modulated by other treatment effects. Prospective large-scale studies are needed to validate findings and examine effects of individual immunotherapies, timing of treatments, and relapse rates.
Generative AI Usage
No, did not use generative AI in the drafting or editing in this abstract.
Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.