CTP-16. ONC201 (Dordaviprone) in the treatment of Diffuse Midline Glioma of the Pons in children: single center experience from Canada
Bibliographic record
Abstract
Abstract INTRODUCTION Diffuse midline glioma of the pons (DMG-P) is a uniformly fatal brain tumor in children. Radiation therapy (RT) is the current standard of care with only transient benefits. Therapy at progression is very limited and offers minimal survival advantage. ONC201 (Dordaviprone), a brain penetrant dopamine receptor D2 antagonist has shown efficacy in recent clinical trials of DMG-P. We present our experience of DMG-P patients treated with ONC201 from a tertiary care center in Canada. METHODS We analyzed clinical data of patients diagnosed with DMG-P from 2012-2024 and compared outcomes of patients treated with and without ONC201. RESULTS There were nineteen patients diagnosed with DMG-P in the study period; Thirteen (N=13/19) patients were diagnosed based on clinical / MRI findings; six patients had biopsy confirmed DMG-P and all six patients had H3.3-K27M mutation. All but one patient were treated with RT. Six (n=6/19, 31%) patients were treated with ONC201 through the expanded access program from Chimerix: Two patients started ONC201 post-RT, prior to recurrence and four patients after first recurrence post-RT. Four of the six (N=4/6, 66%) patients also received repeat RT (RT2) at first recurrence. ONC201 was tolerated well without any dose modifying toxicities. Median progression free survival (PFS) from RT1 and median overall survival (OS) from initial diagnosis in the ONC201 vs non-ONC201 patients was 9.05 months (9.05 vs 4.6 mths, p=0.001) and 19.2 months (19.2 vs 8.2 mths, p=0.002) respectively. Patients treated with ONC201 experienced longer survival when compared to the non-ONC201 treated group (HR = 0.012, 95% CI: 0.000, 0.282; p = 0.005). Two patients on ONC201 are still alive (at 14 and 36 months) on therapy. CONCLUSION Our data suggest that ONC201 as monotherapy (Post-RT, prior to recurrence) or in combination with repeat RT(RT2) in the recurrent setting has clinical efficacy in the treatment of DMG-P tumors in children.
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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 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 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".