Best overall responses and ORRs for patients with AML
| Response category, N (%) . | Part A (N = 54) . | Part B (N = 26) . | Part C (N = 14) . | Part D (N = 21) . | Part E (N = 3) . | Overall (N = 118) . |
|---|---|---|---|---|---|---|
| Best overall response | ||||||
| CR | 2 (3.7) | 0 | 0 | 1 (4.8) | 0 | 3 (2.5) |
| CRi | 1 (1.9) | 1 (3.8) | 1 (7.1) | 0 | 0 | 3 (2.5) |
| MLFS | 1 (1.9) | 1 (3.8) | 0 | 1 (4.8) | 0 | 3 (2.5) |
| PR | 0 | 0 | 0 | 1 (4.8) | 0 | 1 (0.8) |
| Stable disease | 27 (50.0) | 12 (46.2) | 7 (50.0) | 11 (52.4) | 0 | 57 (48.3) |
| Progressive disease | 9 (16.7) | 5 (19.2) | 5 (35.7) | 3 (14.3) | 1 (33.3) | 23 (19.5) |
| NE | 9 (16.7) | 5 (19.2) | 1 (7.1) | 4 (19.0) | 2 (66.7) | 21 (17.8) |
| ORR | 4/49 (8.2) | 2/24 (8.3) | 1/14 (7.1) | 3/21 (14.3) | 0/3 (0.0) | 10/111 (9.0) |
| 95% CI | 2.3, 19.6 | 1.0, 27.0 | 0.2, 33.9 | 3.0, 36.3 | 0.0, 70.8 | 4.4, 15.9 |
| Disease control∗ rate | 31/49 (63.3) | 14/24 (58.3) | 8/14 (57.1) | 14/21 (66.7) | 0/3 (0.0) | 67/111 (60.4) |
| 95% CI | 48.3, 76.6 | 36.6, 77.9 | 28.9, 82.3 | 43.0, 85.4 | 0.0, 70.8 | 50.6, 69.5 |
| Time to treatment discontinuation | ||||||
| Number of events, n (%) | 54 (100) | 26 (100) | 14 (100) | 21 (100) | 3 (100) | 118 (100) |
| Median follow-up | 22.5 | 22.5 | 43.5 | 50.0 | 5.0 | 29.0 |
| Min, max follow-up | 1, 218 | 1, 134 | 15, 121 | 1, 96 | 1, 29 | 1, 218 |
| K-M median (95% CI) | 22.5 (22.0, 50.0) | 22.5 (15.0, 50.0) | 43.5 (22.0, 64.0) | 50.0 (22.0, 78.0) | 5.0 (1.0, NE) | 29.0 (22.0, 50.0) |
| 25%, 75% | 22.0, 78.0 | 8.0, 54.0 | 22.0, 64.0 | 22.0, 79.0 | 1.0, 29.0 | 22.0, 73.0 |
| Time to initial response (of PR or better) | ||||||
| Median (95% CI) | 26.5 (24.0, NE) | 24.5 (24.0, NE) | 26.0 (NE, NE) | 28.0 (25.0, NE) | NE (NE, NE) | 26.0 (24.0, 28.0) |
| 25%, 75% | 25.0, 39.5 | 24.0, 25.0 | 26.0, 26.0 | 25.0, 53.0 | NE, NE | 25.0, 28.0 |
| DoR for responders (d) | ||||||
| n | 4 | 2 | 1 | 3 | 0 | 10 |
| Number of events | 2 (50.0) | 2 (100) | 1 (100) | 1 (33.3) | — | 6 (60.0) |
| Unadjusted mean (SD) | 92.5 (38.48) | 78.0 (29.70) | 57.0 (−) | 65.0 (38.20) | — | 77.8 (33.38) |
| Unadjusted median | 103.0 | 78.0 | 57.0 | 52.0 | — | 76.0 |
| Min, max | 38, 126 | 57, 99 | 57, 57 | 35, 108 | — | 35, 126 |
| K-M median | 118.5 | 78.0 | 57.0 | 108.0 | — | 108.0 |
| Response category, N (%) . | Part A (N = 54) . | Part B (N = 26) . | Part C (N = 14) . | Part D (N = 21) . | Part E (N = 3) . | Overall (N = 118) . |
|---|---|---|---|---|---|---|
| Best overall response | ||||||
| CR | 2 (3.7) | 0 | 0 | 1 (4.8) | 0 | 3 (2.5) |
| CRi | 1 (1.9) | 1 (3.8) | 1 (7.1) | 0 | 0 | 3 (2.5) |
| MLFS | 1 (1.9) | 1 (3.8) | 0 | 1 (4.8) | 0 | 3 (2.5) |
| PR | 0 | 0 | 0 | 1 (4.8) | 0 | 1 (0.8) |
| Stable disease | 27 (50.0) | 12 (46.2) | 7 (50.0) | 11 (52.4) | 0 | 57 (48.3) |
| Progressive disease | 9 (16.7) | 5 (19.2) | 5 (35.7) | 3 (14.3) | 1 (33.3) | 23 (19.5) |
| NE | 9 (16.7) | 5 (19.2) | 1 (7.1) | 4 (19.0) | 2 (66.7) | 21 (17.8) |
| ORR | 4/49 (8.2) | 2/24 (8.3) | 1/14 (7.1) | 3/21 (14.3) | 0/3 (0.0) | 10/111 (9.0) |
| 95% CI | 2.3, 19.6 | 1.0, 27.0 | 0.2, 33.9 | 3.0, 36.3 | 0.0, 70.8 | 4.4, 15.9 |
| Disease control∗ rate | 31/49 (63.3) | 14/24 (58.3) | 8/14 (57.1) | 14/21 (66.7) | 0/3 (0.0) | 67/111 (60.4) |
| 95% CI | 48.3, 76.6 | 36.6, 77.9 | 28.9, 82.3 | 43.0, 85.4 | 0.0, 70.8 | 50.6, 69.5 |
| Time to treatment discontinuation | ||||||
| Number of events, n (%) | 54 (100) | 26 (100) | 14 (100) | 21 (100) | 3 (100) | 118 (100) |
| Median follow-up | 22.5 | 22.5 | 43.5 | 50.0 | 5.0 | 29.0 |
| Min, max follow-up | 1, 218 | 1, 134 | 15, 121 | 1, 96 | 1, 29 | 1, 218 |
| K-M median (95% CI) | 22.5 (22.0, 50.0) | 22.5 (15.0, 50.0) | 43.5 (22.0, 64.0) | 50.0 (22.0, 78.0) | 5.0 (1.0, NE) | 29.0 (22.0, 50.0) |
| 25%, 75% | 22.0, 78.0 | 8.0, 54.0 | 22.0, 64.0 | 22.0, 79.0 | 1.0, 29.0 | 22.0, 73.0 |
| Time to initial response (of PR or better) | ||||||
| Median (95% CI) | 26.5 (24.0, NE) | 24.5 (24.0, NE) | 26.0 (NE, NE) | 28.0 (25.0, NE) | NE (NE, NE) | 26.0 (24.0, 28.0) |
| 25%, 75% | 25.0, 39.5 | 24.0, 25.0 | 26.0, 26.0 | 25.0, 53.0 | NE, NE | 25.0, 28.0 |
| DoR for responders (d) | ||||||
| n | 4 | 2 | 1 | 3 | 0 | 10 |
| Number of events | 2 (50.0) | 2 (100) | 1 (100) | 1 (33.3) | — | 6 (60.0) |
| Unadjusted mean (SD) | 92.5 (38.48) | 78.0 (29.70) | 57.0 (−) | 65.0 (38.20) | — | 77.8 (33.38) |
| Unadjusted median | 103.0 | 78.0 | 57.0 | 52.0 | — | 76.0 |
| Min, max | 38, 126 | 57, 99 | 57, 57 | 35, 108 | — | 35, 126 |
| K-M median | 118.5 | 78.0 | 57.0 | 108.0 | — | 108.0 |
max, maximum; min, minimum; NE, not evaluable; SD, standard deviation.
Disease control is defined as the proportion of patients achieving a best overall response of CR, CRi, MLFS, PR, or stable disease.