Naxitamab (NAX) treatment for refractory/relapsed (R/R) high-risk neuroblastoma (HR-NB): Response data and efficacy in patient (pt) subgroups.
Bibliographic record
Abstract
e22019 Background: Almost half of pts with NB present with high-risk disease, most of whom experience persistence of disease or disease progression. Further, residual disease in the bone/bone marrow (BM) can drive relapse. NAX is a humanized GD2-binding monoclonal antibody indicated under accelerated approval in the US with GM-CSF for the treatment of R/R HR-NB in the bone/BM in pts ≥1 year of age with a partial response (PR), minor response (MR), or stable disease (SD) to prior therapy. Here we characterize the response data of NAX overall and efficacy in pt subgroups. Methods: Trial 201 is an ongoing registrational phase II trial evaluating NAX in pts with R/R HR-NB. Pts with progressive or residual soft tissue disease were excluded. NAX was administered over 30-60 min in the outpatient setting on Days 1/3/5 at 3 mg/kg/day with GM-CSF at 250 µg/m2/day on Days –4 to 0 and 500 µg/m2/day on Days 1 to 5; cycles were every 4 wks. Efficacy was evaluated by independent pathology and radiology review per revised International Neuroblastoma Response Criteria. Results: Results presented here are from an ad hoc interim analysis (Aug 5, 2020) of 48 pts (safety population) and 36 (efficacy population). Pts in the efficacy population received a median of 7 cycles (range 1–11). Overall response rate (ORR; complete response [CR] + PR) was 58% (44% CR; Table). Of the 9 responders who had PR initially, 4 improved to CR. Median number of cycles before onset of response (CR or PR) was 2 (range 2–8) and median time to onset of response (CR or PR) was 7.3 wks (95% CI 6–17). Median duration of response was 24.9 wks (95% CI 25–not estimable). The most common adverse events (CTCAE graded) were infusion-related reactions including hypotension and pain, which occurred in 98% and 96% of pts, respectively. Adverse events were managed with established guidelines. Conclusions: NAX provided a clinically meaningful response in pts with R/R HR-NB limited to bone/BM – frequent sites of residual disease. Response rates were generally consistent across subgroups. These efficacy results combined with a manageable safety profile and use in the outpatient setting demonstrate that NAX addresses a significant unmet medical need. Clinical trial information: NCT03363373. [Table: see text]
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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 | 0.001 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.003 | 0.001 |
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".