Table 2.
Selection percentage and the average number of patients (shown in parentheses) treated at each dose level under the proposed design and EffTox 1 and 2 designs with dropout rate about 50%. The bolded numbers are target doses.
| Design | Dose level | Duration (months) |
||||
|---|---|---|---|---|---|---|
| 1 | 2 | 3 | 4 | 5 | ||
| Scenario 1 | ||||||
| (πE, πT) | (0.42, 0.05) | (0.45, 0.45) | (0.46, 0.65) | (0.47, 0.75) | (0.48, 0.85) | |
| Proposed | 75.7 (24.5) | 19.8 (22.3) | 0.0 (2.4) | 0.0 (0.2) | 0.0 (0.0) | 21.0 |
| EffTox1 | 78.9 (27.6) | 16.0 (18.6) | 0.0 (2.7) | 0.0 (0.2) | 0.0 (0.0) | 30.6 |
| EffTox2 | 21.2 (9.5) | 52.3 (20.8) | 17.7 (13.6) | 5.8 (4.9) | 1.2 (1.4) | 23.4 |
| Scenario 2 | ||||||
| (πE, πT) | (0.41, 0.03) | (0.48, 0.13) | (0.56, 0.45) | (0.63, 0.77) | (0.7, 0.89) | |
| Proposed | 3.6 (5.0) | 82.6 (28.3) | 13.1 (15.9) | 0.0 (1.6) | 0.0 (0.0) | 21.9 |
| EffTox1 | 10.2 (7.1) | 81.0 (28.0) | 7.9 (13.4) | 0.2 (2.1) | 0.0 (0.1) | 29.7 |
| EffTox2 | 2.3 (3.6) | 54.8 (17.3) | 41.4 (24.1) | 1.0 (5.0) | 0.0 (0.8) | 26.7 |
| Scenario 3 | ||||||
| (πE, πT) | (0.3, 0.01) | (0.36, 0.05) | (0.43, 0.17) | (0.51, 0.45) | (0.6, 0.77) | |
| Proposed | 2.4 (4.1) | 13.7 (8.8) | 71.7 (24.8) | 11.6 (11.8) | 0.0 (1.2) | 23.4 |
| EffTox1 | 4.7 (5.2) | 27.9 (13.5) | 62.1 (21.5) | 4.0 (8.6) | 0.0 (1.7) | 31.8 |
| EffTox2 | 0.2 (3.1) | 3.9 (3.9) | 50.1 (15.6) | 42.1 (20.7) | 3.6 (7.6) | 25.5 |
| Scenario 4 | ||||||
| (πE, πT) | (0.21, 0.01) | (0.26, 0.04) | (0.32, 0.1) | (0.38, 0.23) | (0.46, 0.47) | |
| Proposed | 1.1 (3.7) | 6.9 (6.1) | 20.2 (10.5) | 62.9 (21.7) | 7.1 (8.5) | 24.9 |
| EffTox1 | 3.0 (4.7) | 15.0 (8.6) | 29.2 (12.7) | 44.0 (17.0) | 5.2 (7.2) | 35.4 |
| EffTox2 | 0.5 (3.3) | 2.9 (3.9) | 14.8 (7.2) | 43.3 (15.9) | 38.5 (20.7) | 30.6 |
| Scenario 5 | ||||||
| (πE, πT) | (0.11, 0.03) | (0.17, 0.04) | (0.24, 0.04) | (0.34, 0.05) | (0.45, 0.06) | |
| Proposed | 0.0 (3.5) | 3.5 (4.6) | 7.5 (6.3) | 14.1 (8.0) | 71.6 (27.7) | 25.5 |
| EffTox1 | 0.5 (3.5) | 3.0 (4.7) | 11.2 (7.3) | 16.8 (8.8) | 64.3 (25.4) | 35.4 |
| EffTox2 | 1.1 (3.5) | 4.0 (4.3) | 7.9 (5.5) | 27.0 (7.8) | 59.5 (29.7) | 35.1 |
| Scenario 6 | ||||||
| (πE, πT) | (0.4, 0.03) | (0.4, 0.3) | (0.4, 0.5) | (0.4, 0.65) | (0.4, 0.8) | |
| Proposed | 23.4 (11.6) | 70.8 (30.4) | 2.9 (7.1) | 0.0 (0.9) | 0.0 (0.1) | 22.2 |
| EffTox1 | 29.5 (12.6) | 64.4 (28.2) | 2.2 (7.3) | 0.0 (1.2) | 0.0 (0.1) | 33.3 |
| EffTox2 | 10.5 (6.8) | 66.5 (24.9) | 20.2 (13.8) | 2.2 (4.3) | 0.2 (1.0) | 33.6 |
| Scenario 7 | ||||||
| (πE, πT) | (0.12, 0.03) | (0.3, 0.04) | (0.57, 0.04) | (0.3, 0.05) | (0.12, 0.06) | |
| Proposed | 0.5 (3.9) | 22.6 (11.5) | 62.4 (19.9) | 8.3 (7.9) | 1.7 (6.7) | 24.3 |
| EffTox1 | 1.9 (4.1) | 26.3 (13.1) | 52.8 (17.7) | 10.3 (7.3) | 1.8 (6.7) | 33.6 |
| EffTox2 | 2.0 (3.6) | 23.3 (7.6) | 48.4 (14.3) | 23.3 (10.7) | 2.5 (14.7) | 35.1 |
| Scenario 8 | ||||||
| (πE, πT) | (0.27, 0.02) | (0.52, 0.03) | (0.3, 0.05) | (0.24, 0.06) | (0.2, 0.07) | |
| Proposed | 8.8 (7.1) | 55.7 (18.7) | 19.6 (11.3) | 8.3 (6.2) | 5.9 (7.4) | 24.9 |
| EffTox1 | 7.7 (6.9) | 50.6 (18.1) | 23.7 (11.2) | 8.0 (6.2) | 6.5 (7.6) | 35.1 |
| EffTox2 | 4.2 (5.0) | 35.7 (11.5) | 35.6 (11.2) | 19.9 (9.4) | 4.1 (13.7) | 36.6 |
| Scenario 9 | ||||||
| (πE, πT) | (0.01, 0.01) | (0.02, 0.01) | (0.03, 0.01) | (0.04, 0.01) | (0.05, 0.3) | |
| Proposed | 0.0 (3.3) | 0.0 (3.5) | 0.0 (3.4) | 0.2 (4.1) | 0.4 (8.0) | 19.5 |
| EffTox1 | 0.7 (3.3) | 0.5 (3.3) | 0.1 (3.2) | 0.0 (3.7) | 2.9 (7.8) | 20.7 |
| EffTox2 | 1.8 (3.4) | 3.6 (3.8) | 6.1 (3.9) | 8.8 (5.4) | 20.1 (19.9) | 33.3 |
| Scenario 10 | ||||||
| (πE, πT) | (0.1, 0.01) | (0.3, 0.01) | (0.05, 0.01) | (0.05, 0.01) | (0.05, 0.3) | |
| Proposed | 5.6 (8.3) | 37.8 (14.4) | 0.5 (5.2) | 0.3 (4.4) | 1.0 (7.0) | 24.9 |
| EffTox1 | 7.2 (8.0) | 25.8 (10.8) | 0.6 (4.2) | 0.1 (4.0) | 1.9 (6.3) | 30.9 |
| EffTox2 | 11.7 (7.5) | 52.3 (15.2) | 7.6 (5.6) | 4.5 (5.2) | 7.1 (13.5) | 42.0 |