Table 1.
Baseline Patient Demographics and Disease Characteristics
| Characteristic | Primary Efficacy Analysis Set | Final Analysis Set | ||
|---|---|---|---|---|
| P+LDAC (n = 125) | V+LDAC (n = 246) | P+LDAC (n = 222) | V+LDAC (n = 444) | |
| Sex, n (%) | ||||
| Male | 75 (60.0) | 140 (56.9) | 135 (60.8) | 241 (54.3) |
| Female | 50 (40.0) | 106 (43.1) | 87 (39.2) | 203 (45.7) |
| Race, n (%) | ||||
| White | 88 (70.4) | 181 (73.6) | 158 (71.2) | 328 (73.9) |
| Asian | 21 (16.8) | 39 (15.9) | 39 (17.6) | 74 (16.7) |
| Other/missing | 16 (12.8) | 26 (10.6) | 25 (11.3) | 42 (9.5) |
| Age, median (min–max) | 75.0 (65–85) | 75.0 (65–93) | 76.0 (65–88) | 75.0 (65–93) |
| ECOG PS, n (%) | ||||
| 0 | 27 (21.6) | 48 (19.5) | 53 (23.9) | 100 (22.5) |
| 1 | 65 (52.0) | 136 (55.3) | 117 (52.7) | 241 (54.3) |
| 2 | 33 (26.4) | 62 (25.2) | 52 (23.4) | 103 (23.2) |
| WBC count/nL, n (%) | ||||
| <10/nL | 86 (68.8) | 173 (70.3) | 149 (67.1) | 310 (69.8) |
| ≥10/nL and <50/nL | 36 (28.8) | 52 (21.1) | 62 (27.9) | 104 (23.4) |
| ≥50/nL | 3 (2.4) | 21 (8.5) | 11 (5.0) | 30 (6.8) |
| Type of AML, n (%) | ||||
| De novo | 64 (51.2) | 130 (52.8) | 114 (51.4) | 230 (51.8) |
| Secondary AML | 61 (48.8) | 116 (47.2) | 108 (48.6) | 214 (48.2) |
| Preceding MDS | 45 (36.0) | 83 (33.7) | 77 (34.7) | 162 (36.5) |
| Preceding MPS | 8 (6.4) | 17 (6.9) | 18 (8.1) | 28 (6.3) |
| Therapy-relateda | 8 (6.4) | 16 (6.5) | 12 (5.4) | 24 (5.4) |
| Other | 3 (2.4) | 11 (4.5) | 10 (4.5) | 17 (3.8) |
| 2010 ELN genetic group, n (%) | ||||
| Favorable | 13 (10.4) | 28 (11.4) | 21 (9.5) | 47 (10.6) |
| Intermediate I | 38 (30.4) | 80 (32.5) | 71 (32.0) | 144 (32.4) |
| Intermediate II | 33 (26.4) | 42 (17.1) | 46 (20.7) | 75 (16.9) |
| Adverse | 36 (28.8) | 82 (33.3) | 70 (31.5) | 142 (32.0) |
| Missing | 5 (4.0) | 14 (5.7) | 14 (6.3) | 36 (8.1) |
| Mutation types, n (%) | ||||
| NPM1 | 16 (12.8) | 35 (14.2) | 29 (13.1) | 68 (15.3) |
| FLT3 ITD | 6 (4.8) | 13 (5.3) | 8 (3.6) | 22 (5.0) |
aPrior therapy with alkylating agents or topoisomerase II inhibitors.
AML = acute myeloid leukemia; ECOG PS = Eastern Cooperative Oncology Group Performance Status; ELN = European LeukemiaNet; ITD = internal tandem duplication; MDS = myelodysplastic syndrome; MPS = myeloproliferative syndrome; P+LDAC = placebo plus low-dose cytarabine; V+LDAC = volasertib plus low-dose cytarabine; WBC = white blood cell.