129P Naxitamab efficacy in patients with refractory/relapsed high-risk neuroblastoma and bone metastases as assessed by Curie score
Bibliographic record
Abstract
High-risk neuroblastoma (HR-NB) commonly metastasizes to the bone, a frequent site of resistant disease. Naxitamab, a humanized GD2-binding monoclonal antibody, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), has previously been shown to be effective in the treatment of bone metastases. Here we report the maximum change in Curie score (CS) from baseline based on Trial 201 prespecified interim analysis. Trial 201 (NCT03363373, data cutoff 31 Dec 2021) is a Phase 2 study evaluating naxitamab in combination with GM-CSF in patients (pts) with HR-NB with an incomplete response in bone/bone marrow after induction or relapse therapy. Naxitamab was administered intravenously (3 mg/kg/day) on days 1, 3, and 5, and GM-CSF was administered subcutaneously on days -4 to 5 in 4-week cycles. CS and treatment response were evaluated by use of 123I-MIBG scans per independent review in accordance with the International Neuroblastoma Response Criteria. The current analysis reports on the maximum change in CS from baseline in the overall efficacy population and in relapsed and refractory subgroups. A total of 48 pts with evaluable disease in bone and at least 1 postbaseline CS were eligible for assessment. A summary of baseline and maximum CS change for the overall and relapsed/refractory subgroups are presented in the Table. Among the 20 (42%) pts with a 100% decrease in CS, the median and mean baseline CSs were 2.5 and 4.8, respectively. Notably, 46/48 (96%) pts achieved a decreased or stable CS. Related grade ≥3 adverse events reported in ≥10% of pts included pain, hypotension, urticaria, and bronchospasm and were manageable with appropriate interventions in place.Table 129POverall (n=48)Refractory to induction (n=25)Refractory to relapse therapy (n=23)Baseline CSMedian (range)3 (1-20)3 (1-20)3 (1-19)Mean5.55.45.7Maximum CS changeMedian (range)-2 (-18 to 14)-2 (-18 to 0)-1 (-17 to 14)Mean-3.3-4.3-2.3Maximum % CS changeMedian-69-85-67Mean (SD)-55 (59.2)-68 (37.5)-41 (74.5) Open table in a new tab The maximum change in CS provides a measure of best bone response and demonstrates that naxitamab has the potential to significantly reduce the disease burden in the bones of pts with HR-NB. Safety was in line with prior observations.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.001 | 0.002 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.000 | 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 teacher head, 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".