Table 2. Efficacy endpoints.
| Endpoints | Arm 1 (n=16) | Arm 2 (n=14) | Arm 3 (n=5) | Arm 4 (n=18) | Arm 5 (n=17) | Total (n=70) | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| No (%) | 95% CI | No (%) | 95% CI | No (%) | 95% CI | No (%) | 95% CI | No (%) | 95% CI | No (%) | 95% CI | ||
| Objective response rate* | 8 (50.00) | 24.65, 75.35 | 6 (42.86) | 17.66, 71.14 | 1 (20.00) | 0.51, 71.64 | 6 (33.33) | 13.34, 59.01 | 5 (29.41) | 10.31, 55.96 | 26 (37.14) | 25.89, 49.52 | |
| Best overall response | |||||||||||||
| CR | 0 (0.00) | 0 (0.00) | 1 (20.00) | 0 (0.00) | 1 (5.88) | 2 (2.86) | |||||||
| PR | 8 (50.00) | 6 (42.86) | 0 (0.00) | 6 (33.33) | 4 (23.53) | 24 (34.29) | |||||||
| Stable disease | 2 (12.50) | 4 (28.57) | 2 (40.00) | 3 (16.67) | 6 (35.29) | 17 (24.29) | |||||||
| PD | 3 (18.75) | 2 (14.29) | 2 (40.00) | 7 (38.89) | 4 (23.53) | 18 (25.71) | |||||||
| NE | 3 (18.75) | 2 (14.29) | 0 (0.00) | 2 (11.11) | 2 (11.76) | 9 (12.86) | |||||||
| Disease control rate* | 10 (62.50) | 35.43, 84.80 | 10 (71.43) | 41.90, 91.61 | 3 (60.00) | 14.66, 94.73 | 9 (50.00) | 26.02, 73.98 | 11 (64.71) | 38.33, 85.79 | 43 (61.43) | 49.03, 72.83 | |
| Disease control rate at 24 wk* | 9 (56.25) | 29.88, 80.25 | 9 (64.29) | 35.14, 87.24 | 2 (40.00) | 5.27, 85.34 | 7 (38.89) | 17.30, 64.25 | 10 (58.82) | 32.92, 81.56 | 37 (52.86) | 40.55, 64.91 | |
| Median time to response† (wk) | 11.39 | 11.10, 12.53 | 17.23 | 11.25, 35.59 | 12.81 | 11.10 | 11.10, 23.92 | 11.25 | 11.10, 23.06 | 11.32 | 11.10, 12.53 | ||
| Median duration of response† (wk) | 24.06 | 3.27, 59.08 | 24.49 | 6.69, 36.45 | 23.21 | 3.70, ∞ | 24.20 | 24.20, ∞ | 24.20 | 12.67, 36.45 | |||
| Median PFS† (mo) | 5.62 | 2.60, 10.48 | 5.36 | 2.63, 13.83 | 3.68 | 1.61, ∞ | 3.98 | 2.60, 9.49 | 5.13 | 2.60, 8.12 | 4.76 | 3.38, 8.08 | |
| 6-month PFS rate‡ (%) | 43.27 | 17.98, 66.43 | 47.62 | 20.26, 70.83 | 40.00 | 5.20, 75.28 | 31.67 | 11.60, 54.14 | 45.75 | 21.47, 67.21 | 41.10 | 28.93, 52.86 | |
| Median OS† (mo) | 18.53 | 4.17, ∞ | 15.51 | 7.33, 15.51 | 19.65 | 3.55, ∞ | 15.51 | 12.29, ∞ | |||||
| 6-month OS rate‡ (%) | 77.92 | 45.90, 92.32 | 92.86 | 59.08, 98.96 | 75.00 | 12.79, 96.05 | 83.92 | 49.40, 95.73 | 81.93 | 53.77, 93.80 | 83.31 | 71.09, 90.69 | |
| Median duration of follow-up (mo) | 9.81 | 7.95, 17.62 | 7.41 | 6.76, 16.93 | 4.63 | −2.64, 27.11 | 7.87 | 7.46, 18.82 | 8.08 | 6.14, 10.43 | 8.31 | 9.41, 13.70 | |
The denominator of percentages is the number of patients in each treatment group.
CI, confidence interval; CR, complete response; NE, not evaluable; OS, overall survival; PD, progressive disease; PFS, progression-free survival; PR, partial response.
*95% CI using Clopper-Pearson exact method; †95% CI using the method known as the log cumulative hazard transformation method; ‡95% CI using the Kaplan-Meier method.