Table 5.
ORRs of the ITT patient population
| Response rate in the ITT cohort, % (95% CI) | ||||
|---|---|---|---|---|
| ORR | CR | CRi | PR | |
| Total patients (N = 64)* | 50 (37-63) | 31 (20-44) | 8 (3-17) | 11 (5-21) |
| AML subtype | ||||
| De novo AML (n = 36) | 53 (35-70) | 33 (19-51) | 8 (2-22) | 11 (3-26) |
| Secondary AML (n = 28) | 46 (28-66) | 29 (13-49) | 7 (1-24) | 11 (2-28) |
| Bone marrow blast count | ||||
| <30% (n = 25) | 52 (31-72) | 28 (12-49) | 12 (3-31) | 12 (3-31) |
| ≥30% (n = 39) | 49 (32-65) | 33 (19-50) | 5 (1-17) | 10 (3-24) |
| Cytogenetic risk | ||||
| Intermediate (n = 32) | 44 (26-62) | 28 (14-47) | 3 (0-16) | 13 (4-29) |
| Adverse (n = 18) | 44 (22-69) | 28 (10-53) | 11 (1-35) | 6 (0-27) |
| AZA + PEV exposure | ||||
| <6 cycles (n = 41) | 32 (18-48) | 15 (6-29) | 7 (2-20) | 10 (3-23) |
| ≥6 cycles (n = 23) | 83 (61-95) | 61 (39-80) | 9 (1-28) | 13 (3-34) |
*
Of the 3 patients who were treated at the 30-mg/m2 PEV dose, 1 patient discontinued following a best response of SD (which lasted ∼1 month) due to an SAE of grade 3 pneumonia; 1 patient achieved a CR lasting ∼4 months, at which point this patient discontinued treatment due to progressive disease; and 1 patient had a DLT of grade 4 AST/ALT elevation on day 1; further dosing was held on days 3 and 5, and the patient discontinued due to symptomatic deterioration on day 8 prior to the first postbaseline assessment.