Rituximab Therapy As A First-Line Immunosuppressive Treatment In Children With Neuromyelitis Optica Spectrum Disorder: A Single-Center Experience. (P7.325)
Bibliographic record
Abstract
OBJECTIVE: To report the therapeutic response to a standardized B-cell targeted immunosuppressive protocol in children with neuromyelitis optica spectrum disorders (NMOSDs). BACKGROUND: NMOSDs are inflammatory disorders of the central nervous system generally characterized by poor motor and visual outcomes. Recent observations suggest that rituximab, a monoclonal antibody targeting the CD20 antigen of B-lymphocytes, may ameliorate the course of this disorder in the pediatric population, but no standardized treatment approach has been proposed thus far. DESIGN/METHODS: We retrospectively analyzed data from nine consecutive NMOSD patients followed at The Hospital for Sick Children, Toronto, Canada. Inclusion criteria were: 1) positive anti-aquaporin 4 IgG; 2) first-line immunosuppressive treatment with rituximab (induction therapy: 500 mg/m2 infused twice 2 weeks apart; re-treatment using the same induction course at CD19+ B-cell reconstitution) in combination with an oral prednisone taper. RESULTS: Five patients (4 females; median age at onset 12.9 years, range 10.9-17.0; median follow-up 0.9 years, range 0.5-1.9) were included. The median time from onset to treatment with rituximab was 0.27 years (range 0.24-0.33). After induction therapy, two patients were relapse-free until the last follow-up, two presented one further attack before CD19+ B-lymphocyte therapeutic depletion, and one showed a relapse during B-cell elevation before re-treatment. Adverse events included urticaria (one patient) and facial rash (two patients). The median EDSS score of the patients at 6 months from onset and at last visit were 2 and 0.8, respectively. CONCLUSIONS: A standardized immunosuppressive protocol with rituximab and an oral prednisone taper appears to be a well-tolerated and effective strategy for reducing relapses in treatment-naive pediatric NMOSDs. The retrospective nature of the study, the small size of the cohort and the short follow-up limit our results. More studies are needed to assess efficacy, safety, and optimal dosage of the suggested treatment protocol.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".