Table 1. Characteristics of the entire and RP2D study cohort.
| Parameter | Entire cohort, n= 52 | At/above R2PD cohort, n=46 |
|---|---|---|
|
| ||
| Median age (range), years | 55 (19-72) | 55 (19-72) |
|
| ||
| Male gender, n (%) | 29 (55.8%) | 24 (52.2%) |
|
| ||
| Disease-type | ||
| AML | 45 (86.5%) | 41 (89.1%) |
| With recurrent genetic abnormalities | 9 (17.3%) | 7 (15.2%) |
| With mutated NPM1 | 5 (9.6%) | 5 (10.9%) |
| With myelodysplasia-related changes | 19 (36.5%) | 17 (36.9%) |
| Treatment-related myeloid neoplasms | 1 (1.9%) | 1 (2.2%) |
| AML, not otherwise specified | 11 (21.2%) | 11 (23.9%) |
| MDS-RAEB2 | 7 (13.5%) | 5 (10.9%) |
|
| ||
| Secondary disease* | 9 (17.3%) | 7 (15.2%) |
|
| ||
| Disease status, n (%) | ||
| Primary refractory | 19 (36.6%) | 18 (39.1%) |
| Relapse | 33 (63.4%) | 28 (60.9%) |
| Median CR1 duration (range), months | 5 (1-19) | 5 (1-19) |
| Prior HCT | 13 (25.0%) | 13 (28.3%) |
| Median number of prior therapies (range) | 2 (1-7) | 2 (1-7) |
| Prior receipt of intermediate/high-dose cytarabine** | 39 (75.0%) | 35 (76.1%) |
|
| ||
| Median TRM score (range) | 3.15 (0.07-9.05) | 3.17 (0.07-9.05) |
|
| ||
| Performance status, n (%) | ||
| 0 | 7 (13.5%) | 6 (13.0%) |
| 1 | 39 (75.0%) | 34 (73.9%) |
| 2 | 6 (11.5%) | 6 (13.0%) |
|
| ||
| Cytogenetic risk, n (%) | ||
| Favorable | 3 (5.6%) | 1 (2.2%) |
| Intermediate | 28 (53.8%) | 25 (54.3%) |
| Adverse | 21 (40.4%) | 20 (43.5%) |
|
| ||
| Mutational status, n (%) | ||
| FLT3-ITD | ||
| No | 32 (61.5%) | 30 (65.2%) |
| Yes | 5 (9.6%) | 5 (10.9%) |
| Unknown | 15 (28.8%) | 11 (23.9%) |
| NPM1 | ||
| No | 31 (59.6%) | 29 (63.0%) |
| Yes | 5 (9.6%) | 5 (10.9%) |
| Unknown | 16 (30.8%) | 12 (26.1%) |
|
| ||
| Laboratory findings at baseline, median (range) | ||
| WBC (x 109L) | 2.12 (0.05-59.4) | 1.90 (0.05-59.4) |
| Peripheral blood blasts (%) | 8 (0-83) | 8 (0-83) |
| Hemoglobin (g/dL) | 9.4 (5.5-14.0) | 9.3 (5.5-14.0) |
| Platelets (x 109L) | 30 (4-457) | 34 (4-447) |
| Creatinine (mg/dL) | 0.80 (0.41-1.33) | 0.79 (0.41-1.33) |
| Total bilirubin (mg/dL) | 0.5 (0.2-2.7) | 0.6 (0.2-2.7) |
| AST (U/L) | 26 (11-191) | 25 (11-117) |
| ALT (U/L) | 27 (10-440) | 27 (10-161) |
*
AML transformed from antecedent hematologic disorder or AML/MDS after prior cytotoxic therapy
**
Regimen containing ≥500 mg/m2 cytarabine/day: most common prior regimens included HiDAC (high-dose cytarabine), G-CLAM, G-CLAC, IAP (idarubicin, high-dose cytarabine, pravastatin), and FLAG-Ida
Abbreviations: CR1, first complete remission; RP2D, recommended phase 2 dose; TRM, treatment-related mortality