Table 1.
Simulation study comparing the complete-data CRM (CRMcomp), the CRM based on the observed toxicity data only (CRMobs), time-to-event CRM (TITE-CRM) and the proposed data augmentation CRM (DA-CRM) with the sample size 36, the cohort size 3, and the A/I ratio = 6.
| Time to toxicity |
Design | Recommendation percentage at dose level | NMTD+ | Duration (months) |
||||||
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 2 | 3 | 4 | 5 | 6 | None | ||||
| Scenario 1 | Pr(toxicity) | 0.1 | 0.15 | 0.3 | 0.45 | 0.6 | 0.7 | |||
| CRMcomp | 0.6 | 13.8 | 61.9 | 22.9 | 0.6 | 0.0 | 0.2 | 9.0 | 36.4 | |
| # patients | 4.8 | 7.2 | 14.9 | 7.6 | 1.3 | 0.1 | ||||
| Weibull | CRMobs | 0.4 | 7.5 | 48.4 | 27.4 | 2.4 | 0.1 | 13.7 | 4.0 | 8.2 |
| # patients | 7.5 | 8.3 | 13.2 | 3.0 | 0.9 | 0.1 | ||||
| TITE-CRM | 3.4 | 23.1 | 55.9 | 16.5 | 0.6 | 0.0 | 0.5 | 15.5 | 9.0 | |
| # patients | 5.1 | 6.3 | 9.0 | 8.1 | 4.9 | 2.5 | ||||
| DA-CRM | 0.9 | 14.7 | 56.4 | 25.1 | 1.5 | 0.0 | 1.2 | 10.4 | 8.9 | |
| # patients | 9.4 | 7.6 | 8.3 | 6.0 | 3.0 | 1.3 | ||||
| Log-logistic | CRMobs | 0.3 | 7.8 | 48.2 | 27.4 | 2.7 | 0.1 | 13.6 | 4.0 | 8.2 |
| # patients | 7.4 | 8.4 | 13.2 | 3.0 | 0.9 | 0.1 | ||||
| TITE-CRM | 3.6 | 22.6 | 56.3 | 16.5 | 0.6 | 0.0 | 0.4 | 15.4 | 9.0 | |
| # patients | 5.1 | 6.4 | 9.1 | 8.2 | 4.8 | 2.4 | ||||
| DA-CRM | 0.9 | 13.9 | 58.1 | 23.8 | 1.9 | 0.0 | 1.3 | 10.3 | 8.9 | |
| # patients | 9.5 | 7.6 | 8.2 | 6.0 | 3.0 | 1.3 | ||||
| Uniform | CRMobs | 0.1 | 4.7 | 38.0 | 30.5 | 2.9 | 0.1 | 23.6 | 2.8 | 7.5 |
| # patients | 8.6 | 8.2 | 10.9 | 2.3 | 0.5 | 0.0 | ||||
| TITE-CRM | 2.1 | 20.4 | 56.6 | 19.1 | 1.4 | 0.0 | 0.4 | 13.0 | 9.0 | |
| # patients | 6.3 | 7.0 | 9.7 | 8.0 | 3.7 | 1.3 | ||||
| DA-CRM | 0.6 | 11.2 | 56.9 | 27.6 | 1.7 | 0.0 | 1.9 | 8.7 | 8.9 | |
| # patients | 10.8 | 7.6 | 8.5 | 5.7 | 2.2 | 0.7 | ||||
| Scenario 2 | Pr(toxicity) | 0.08 | 0.1 | 0.2 | 0.3 | 0.45 | 0.6 | |||
| CRMcomp | 0.0 | 1.4 | 23 | 55.9 | 18.8 | 0.8 | 0.1 | 6.6 | 36.4 | |
| # patients | 4.1 | 4.1 | 9.0 | 12.2 | 5.5 | 1.0 | ||||
| Weibull | CRMobs | 0.0 | 1.0 | 18.9 | 48.5 | 22.2 | 1.8 | 7.6 | 2.9 | 8.5 |
| # patients | 5.7 | 6.7 | 13.8 | 5.2 | 2.3 | 0.7 | ||||
| TITE-CRM | 0.1 | 2.6 | 29.2 | 52.4 | 14.9 | 0.8 | 0.1 | 11.2 | 9.0 | |
| # patients | 4.2 | 4.7 | 7.1 | 8.8 | 6.8 | 4.4 | ||||
| DA-CRM | 0.0 | 1.5 | 24.4 | 54.0 | 17.7 | 1.4 | 1.1 | 7.3 | 8.9 | |
| # patients | 8.4 | 6.3 | 7.0 | 6.7 | 4.5 | 2.8 | ||||
| Log-logistic | CRMobs | 0.0 | 0.9 | 19.0 | 48.5 | 22.0 | 1.9 | 7.7 | 2.9 | 8.5 |
| # patients | 5.7 | 6.7 | 13.8 | 5.1 | 2.3 | 0.7 | ||||
| TITE-CRM | 0.1 | 2.6 | 29.0 | 52.3 | 15.1 | 0.8 | 0.1 | 11.1 | 9.0 | |
| # patients | 4.1 | 4.7 | 7.2 | 8.8 | 6.8 | 4.3 | ||||
| DA-CRM | 0.0 | 1.4 | 23.3 | 54.0 | 18.7 | 1.6 | 1.0 | 7.5 | 8.9 | |
| # patients | 8.3 | 6.2 | 6.9 | 6.8 | 4.6 | 2.9 | ||||
| Uniform | CRMobs | 0.0 | 0.7 | 14.9 | 45.3 | 23.3 | 2.3 | 13.5 | 2.0 | 8.1 |
| # patients | 6.9 | 7.1 | 12.3 | 4.5 | 1.7 | 0.4 | ||||
| TITE-CRM | 0.1 | 2.2 | 26.9 | 54.4 | 15.4 | 0.9 | 0.1 | 9.6 | 9.0 | |
| # patients | 4.9 | 5.0 | 7.6 | 8.8 | 6.2 | 3.3 | ||||
| DA-CRM | 0.0 | 1.3 | 23.7 | 54.0 | 18.8 | 1.1 | 1.2 | 6.2 | 8.9 | |
| # patients | 9.3 | 6.3 | 7.1 | 6.8 | 4.2 | 2.0 | ||||