Treatment outcomes
| Treatment outcomes . | Phase 2 and Exp (n = 60) . | Phase 1b (n = 17) . |
|---|---|---|
| Best response, n (%) | ||
| CR | 22 (37) | 4 (24) |
| CRh | 5 (8)∗ | 2 (12) |
| CRi | 2 (3) | 1 (6) |
| MLFS | 1 (2) | 1 (6) |
| Partial remission | 2 (3) | 1 (6) |
| Stable disease | 21 (35) | 3 (18) |
| Progressive disease | 2 (3) | 0 (0) |
| Treatment failure | 1 (2) | 0 (0) |
| Not evaluated | 4 (7)† | 5 (29)‡ |
| cCR rate [CR/CRi], n (%, 95% CI) | 29 (48, adjusted: 30.3-60.5)§ | 7 (41, 18-67) |
| Median time to best response [CR/CRi] | (n = 29) | (n = 7) |
| Months (range) | 3 (0.9-11) | 3.7 (0.7-6.8) |
| Overall response rate [CR/CRi/MLFS], n (%, 95% CI) | 30 (50, 37-63) | 8 (47, 23-72) |
| Treatment outcomes . | Phase 2 and Exp (n = 60) . | Phase 1b (n = 17) . |
|---|---|---|
| Best response, n (%) | ||
| CR | 22 (37) | 4 (24) |
| CRh | 5 (8)∗ | 2 (12) |
| CRi | 2 (3) | 1 (6) |
| MLFS | 1 (2) | 1 (6) |
| Partial remission | 2 (3) | 1 (6) |
| Stable disease | 21 (35) | 3 (18) |
| Progressive disease | 2 (3) | 0 (0) |
| Treatment failure | 1 (2) | 0 (0) |
| Not evaluated | 4 (7)† | 5 (29)‡ |
| cCR rate [CR/CRi], n (%, 95% CI) | 29 (48, adjusted: 30.3-60.5)§ | 7 (41, 18-67) |
| Median time to best response [CR/CRi] | (n = 29) | (n = 7) |
| Months (range) | 3 (0.9-11) | 3.7 (0.7-6.8) |
| Overall response rate [CR/CRi/MLFS], n (%, 95% CI) | 30 (50, 37-63) | 8 (47, 23-72) |
MLFS, morphologic leukemia-free state.
All 5 CRh also qualified as CRi.
Early death within the first cycle before assessment could be performed.
Went off treatment before finishing the first cycle of combination therapy.
Given the conditional expansion design, which was conditional on the success of 2 prior interim stages, an adjusted 95% CI is provided for the primary end point. The naïve 95% CI on the CR/CRi rate of 48% that does not consider prior interim analyses is 35% to 62%.