163TiP Single-institution, single-arm, open-label phase II basket study of mutation and/or elevated hedgehog (Hh) expression-directed smoothened inhibitor sonidegib in patients with advanced solid malignancies
Bibliographic record
Abstract
as partial response (PR) + complete response (CR), and disease control rate (DCR), defined as CR + PR + stable disease (SD).Results: 525 patients were treated on 98 EPCCTs.All patients received at least one dose of treatment with median of 4 cycles.93% of patients were off trial at data collection, 73.1% due to progression, 11% from toxicity.470 patients were evaluable for response.The ORR was 14.3% and DCR was 63.9%.Of the 238 patients with SD, 48.7% had duration of >6 months.The DCR in monotherapy (66.2%) and combination (62.8%) trials was similar.The difference in response rates due to sex (p 0.016), presence of liver metastasis (p¼0.0001),Royal Marsden Hospital prognostic score (p¼0.0001),Gustave Roussy Immune Score (p¼0.0001), and LDH (p¼0.0001) were all statistically significant.The median progression free survival was 4 months (SE 0.2, 95% CI 3.4-4.5)and median overall survival 10 months (SE 0.63, 95% CI 8.7-11.2).Patients with SD at 10 weeks have a 12-month survival probability of 0.53 (0.47-0.61) versus 0.12 (0.07-0.19) for PD.Table: 161P Category N % Age Median 60 years (18-83) Sex Female 256 48.8 Male 269 51.2 Ethnicity White 444 84.6 Asian 11 2.1 Black 4 0.8 Other 2 0.4 Unknown 64 12.2 Disease type Lung 131 25.0 Lower GI 86 16.4 Urological 63 12.0 Gynaecological 47 9.0 Breast 46 8.8 Other 152 28.9 Number of metastases 0 7 1.3 1 137 26.1 2 174 33.1 3+ 207 39.4 Liver metastases No 323 61.5 Yes 202 38.5 ECOG 0 160 30.5 1 359 68.4 2 6 1.1 Number of prior treatment lines 0 40 7.6 1 144 27.4 2 134 25.5 3+ 207 39.4 Trial treatment Monotherapy 353 67.2 Combination 172 32.8 Type of trial drug Chemotherapy 62 11.8 Drug conjugate 64 12.2 Immunotherapy 92 17.5 Targeted 229 43.6 Targeted + chemotherapy 24 4.6 Targeted + immunotherapy 38 7.2 Other 16 3.0Conclusions: EPCCTs are a valid treatment approach for patients with advanced cancer with two thirds of patients having DCR.Factors including LDH, presence of liver metastasis and sex contribute to the differences in response rates seen.
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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 | 0.000 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.003 |
| Insufficient payload (model declined to judge) | 0.006 | 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".