TABLE 3.
Results of Simulation A, including the selection percentage (selection %), the average number of patients treated at each dose (No. of patients), and the percentage of early stopping. The optimal biological dose (OBD) is bolded. In scenario 8, the OBD does not exist, and thus the percentage of early stopping is bolded.
| Design | Dose Level |
% of early stopping | |||||
|---|---|---|---|---|---|---|---|
| 1 | 2 | 3 | 4 | 5 | |||
| Scenario 1 | |||||||
| DLT rate | 0.02 | 0.15 | 0.30 | 0.45 | 0.60 | ||
| Efficacy rate | 0.20 | 0.65 | 0.65 | 0.65 | 0.65 | ||
| Utility | 43.0 | 69.0 | 63.0 | 56.0 | 50.0 | ||
| EffTox | Selection % | 2.0 | 50.0 | 45.0 | 2.0 | 0.0 | 0.0 |
| No. of patients | 4.3 | 22.7 | 23.8 | 2.8 | 0.4 | ||
| U-BOIN | Selection % | 1.7 | 72.9 | 22.4 | 2.8 | 0.0 | 0.2 |
| No. of patients | 6.2 | 29.9 | 13.8 | 3.5 | 0.5 | ||
| Scenario 2 | |||||||
| DLT rate | 0.03 | 0.08 | 0.15 | 0.28 | 0.40 | ||
| Efficacy rate | 0.10 | 0.22 | 0.60 | 0.60 | 0.60 | ||
| Utility | 36.0 | 43.0 | 66.0 | 60.0 | 55.0 | ||
| EffTox | Selection % | 0.0 | 4.0 | 60.0 | 29.0 | 7.0 | 0.0 |
| No. of patients | 3.4 | 4.9 | 26.4 | 14.2 | 5.1 | ||
| U-BOIN | Selection % | 1.1 | 3.2 | 65.7 | 24.9 | 4.3 | 0.8 |
| No. of patients | 4.9 | 7.5 | 24.4 | 12.7 | 4.3 | ||
| Scenario 3 | |||||||
| DLT rate | 0.05 | 0.15 | 0.30 | 0.45 | 0.60 | ||
| Efficacy rate | 0.08 | 0.46 | 0.25 | 0.20 | 0.10 | ||
| Utility | 34.0 | 56.0 | 37.0 | 29.0 | 18.0 | ||
| EffTox | Selection % | 12.0 | 70.0 | 10.0 | 1.0 | 0.0 | 7.0 |
| No. of patients | 10.8 | 25.5 | 11.5 | 2.9 | 1.0 | ||
| U-BOIN | Selection % | 1.2 | 92.2 | 4.0 | 0.4 | 0.0 | 2.1 |
| No. of patients | 6.6 | 34.7 | 8.9 | 2.6 | 0.4 | ||
| Scenario 4 | |||||||
| DLT rate | 0.15 | 0.25 | 0.40 | 0.45 | 0.50 | ||
| Efficacy rate | 0.15 | 0.45 | 0.30 | 0.25 | 0.20 | ||
| Utility | 36.0 | 52.0 | 36.0 | 32.0 | 27.0 | ||
| EffTox | Selection % | 36.0 | 47.0 | 5.0 | 2.0 | 2.0 | 9.0 |
| No. of patients | 19.4 | 19.0 | 7.5 | 2.6 | 2.3 | ||
| U-BOIN | Selection % | 11.9 | 74.1 | 3.7 | 0.4 | 0.0 | 9.9 |
| No. of patients | 15.0 | 29.5 | 5.1 | 1.1 | 0.2 | ||
| Scenario 5 | |||||||
| DLT rate | 0.10 | 0.30 | 0.50 | 0.55 | 0.65 | ||
| Efficacy rate | 0.45 | 0.45 | 0.45 | 0.45 | 0.45 | ||
| Utility | 58.0 | 50.0 | 42.0 | 40.0 | 36.0 | ||
| EffTox | Selection % | 69.0 | 27.0 | 2.0 | 0.0 | 0.0 | 2.0 |
| No. of patients | 29.7 | 17.3 | 4.9 | 1.0 | 0.3 | ||
| U-BOIN | Selection % | 75.4 | 22.8 | 1.5 | 0.2 | 0.0 | 0.2 |
| No. of patients | 33.1 | 16.5 | 3.7 | 0.5 | 0.1 | ||
| Scenario 6 | |||||||
| DLT rate | 0.05 | 0.07 | 0.10 | 0.12 | 0.16 | ||
| Efficacy rate | 0.35 | 0.45 | 0.50 | 0.55 | 0.75 | ||
| Utility | 53.0 | 59.0 | 61.0 | 64.0 | 75.0 | ||
| EffTox | Selection % | 10.0 | 12.0 | 26.0 | 24.0 | 29.0 | 0.0 |
| No. of patients | 8.6 | 7.9 | 14.2 | 11.1 | 12.1 | ||
| U-BOIN | Selection % | 5.9 | 11.7 | 13.1 | 13.6 | 55.7 | 0.0 |
| No. of patients | 7.0 | 8.8 | 9.0 | 9.1 | 20.1 | ||
| Scenario 7 | |||||||
| DLT rate | 0.03 | 0.16 | 0.27 | 0.45 | 0.55 | ||
| Efficacy rate | 0.15 | 0.38 | 0.45 | 0.60 | 0.70 | ||
| Utility | 40.0 | 51.0 | 51.0 | 53.0 | 55.0 | ||
| EffTox | Selection % | 1.0 | 33.0 | 54.0 | 9.0 | 1.0 | 2.0 |
| No. of patients | 4.2 | 13.8 | 25.9 | 7.0 | 2.3 | ||
| U-BOIN | Selection % | 2.0 | 45.0 | 41.0 | 10.0 | 1.0 | 1.0 |
| No. of patients | 5.1 | 20.4 | 20.0 | 6.7 | 1.2 | ||
| Scenario 8 | |||||||
| DLT rate | 0.22 | 0.45 | 0.55 | 0.65 | 0.70 | ||
| Efficacy rate | 0.03 | 0.10 | 0.20 | 0.35 | 0.40 | ||
| Utility | 25.0 | 23.0 | 25.0 | 30.0 | 31.0 | ||
| EffTox | Selection % | 1.0 | 6.0 | 6.0 | 0.0 | 0.0 | 87.0 |
| No. of patients | 4.6 | 6.2 | 6.3 | 2.3 | 1.2 | ||
| U-BOIN | Selection % | 0.8 | 5.5 | 1.7 | 0.0 | 0.0 | 92.0 |
| No. of patients | 14.3 | 9.7 | 1.6 | 0.1 | 0.0 | ||