Table 2.
Demographics and disease characteristics
| Dose escalation/ expansion (N = 69) | Fractionated dosing (N = 12) | NPM1 (N = 13) | Post-alloHSCT (N = 12) | Treatment naive* (N= 27) | |
|---|---|---|---|---|---|
| Median age (range), y | 75 (27-86) | 72.5 (64-83) | 69 (41-80) | 56 (26-72) | 74 (67-89) |
| Sex, male, % | 61 | 67 | 62 | 58 | 48 |
| ECOG score, % | |||||
| 0 | 16 | 25 | 23 | 17 | 37 |
| 1 | 84 | 75 | 77 | 83 | 63 |
| De novo AML, % | 58 | 58 | 92 | 58 | 56 |
| Secondary AML, % | 42 | 42 | 8 | 42 | 44 |
| MRC cytogenetic risk group, % | |||||
| Favorable | 1 | 0 | 0 | 0 | 0 |
| Intermediate | 64 | 50 | 100 | 25 | 70 |
| Adverse | 33 | 50 | 0 | 75 | 26 |
| Underlying myelodysplasia, % | 57 | 58 | 8 | 42 | 48 |
| FLT3 mutation only, % | 4 | 17 | 0 | 17 | 13 |
| NPM1 mutation only, % | 12 | 0 | 92 | 0 | 0 |
| FLT3 and NPM1 mutations, % | 7 | 0 | 8 | 8 | 13 |
| Enrollment BM (range), % | 40 (4-93) | 47 (20-83) | 72.5 (6-99) | 44 (0-90) | 47 (20-96) |
| Baseline WBC (range), × 109/L | 2.4 (0.5-55.1) | 1.7 (0.3-14.4) | 4 (0.7-36.3) | 1.6 (0.5-15.6) | 2.7 (0.5-26.3) |
MRC, Medical Research Council; WBC, white blood cell.
*
Includes the treatment-naive cohort and 3 patients in the 40 µg/kg–dose-escalation cohort who were treatment naive.