Table 1.
Patient characteristics
| Trial patients (N = 572) | PBC study patients | P* | |||
|---|---|---|---|---|---|
| All (N = 151) | ICE arm, n = 82 (54.3 %) | HDS arm, n = 69 (45.7%) | |||
| Age, median (range), y | 52 (16-73) | 53 (20-73) | 59.5 (28-73) | 47 (20-72) | <.0001 |
| Males, n (%) | 301 (52.6) | 78 (51.7) | 44 (53.66) | 34 (49.28) | .5913 |
| ECOG performance score, n (%) | .6338 | ||||
| 0 | 292 (51.1) | 77 (51.0) | 39 (47.56) | 38 (55.07) | |
| 1 | 232 (40.6) | 63 (41.7) | 37 (45.12) | 26 (37.68) | |
| 2-3 | 48 (8.3) | 11 (7.3) | 6 (7.32) | 5 (7.25) | |
| White blood cells, median (range), ×109/L | 10.5 (0.5-990) | 16.8 (1.0-282.0) | 13.3 (1.2-282.0) | 20.8 (1.0-260.0) | .3361 |
| Hemoglobin, median (range), g/dL | 9.1 (3-15.8) | 9.2 (5.1-15.8) | 9.5 (5.3-14.1) | 8.8 (5.1-15.8) | .5376 |
| Platelets, median (range), ×109/L | 53 (2-852) | 51.0 (2.0-852.0) | 53.0 (2.0-373.0) | 46.0 (10.0-852.0) | .2334 |
| Diagnosis, n (%) | .6510 | ||||
| De novo AML | 494 (86.4) | 140 (92.7) | 75 (91.46) | 65 (94.20) | |
| Secondary (MPN/MDS) | 49 (8.6) | 9 (6.0) | 5 (6.10) | 4 (5.80) | |
| Therapy related | 29 (5.1) | 2 (1.3) | 2 (2.44) | 0 (0.00) | |
| Cytogenetics, n (%) | .1748 | ||||
| SR | 52 (9.1) | 22 (14.6) | 9 (10.98) | 13 (18.84) | |
| t(8,21) | 20 (3.5) | 10 (6.6) | 6 (7.32) | 4 (5.80) | |
| Inv(16) | 31 (5.4) | 11 (7.3) | 3 (3.66) | 8 (11.59) | |
| del(16) | 1 (0.2) | 1 (0.7) | 0 (0.00) | 1 (1.45) | |
| Normal (46 XX/XY) | 272 (47.6) | 70 (46.4) | 45 (54.88) | 25 (36.23) | |
| Intermediate risk | 21 (3.7) | 8 (5.3) | 4 (4.88) | 4 (5.80) | |
| HR | 170 (29.7) | 42 (27.8) | 21 (25.61) | 21 (30.44) | |
| Unknown | 57 (10.0) | 9 (6.0) | 3 (3.66) | 6 (8.70) | |
| Genetics | |||||
| RUNX1-RUNX1T1, n | 558 | 144 | 75 | 69 | .7481 |
| Rearranged, n (%) | 27 (4.8) | 10 (6.9) | 6 (8.0) | 4 (5.8) | |
| CBF-MYH11, n | 557 | 144 | 75 | 69 | .0221 |
| Rearranged n (%) | 41 (7.4) | 14 (9.7) | 3 (4.0) | 11 (15.9) | |
| NPM1, n | 551 | 143 | 76 | 67 | .1617 |
| Mutant n (%) | 167 (30.3) | 47 (32.9) | 28 (36.8) | 19 (28.4) | |
| FLT3-ITD, n | 569 | 145 | 76 | 69 | .7990 |
| Mutant n (%) | 103 (18.1) | 35 (24.1) | 19 (25.0) | 16 (23.2) | |
| MLL-PTD, n | 397 | 119 | 68 | 51 | .0445 |
| Mutant n (%) | 24 (6.0) | 12 (10.1) | 6 (8.8) | 6 (11.8) | |
| CEBPA, n | 361 | 150 | 81 | 69 | .0239 |
| Mutant (double mutation) n (%) | 19 (5.3) | 8 (5.3) | 1 (1.2) | 7 (10.1) | |
| NILG risk classification, n (%) | .1048 | ||||
| SR | 156 (27.3) | 51 (33.8) | 23 (28.0) | 28 (40.6) | |
| HR | 416 (72.7) | 100 (66.2) | 59 (72.0) | 41 (59.4) | |
| ELN 2010 risk classification, n | 518 | 144 | 80 | 64 | .1688 |
| Favorable, n (%) | 166 (32.0) | 56 (38.9) | 27 (33.8) | 29 (45.3) | |
| Intermediate 1, n (%) | 147 (28.4) | 35 (24.3) | 25 (31.2) | 10 (15.6) | |
| Intermediate 2, n (%) | 70 (13.5) | 18 (12.5) | 9 (11.3) | 9 (14.1) | |
| Adverse, n (%) | 135 (26.1) | 35 (24.3) | 19 (23.7) | 16 (25.0) | |
PBC study patients represented 26.3% of all trial patients and expressed comparable clinico-biologic and risk features. Among PBC study patients, those randomized to HDS chemotherapy were significantly younger than the control patients.
ECOG, Eastern Conference Oncology Group; ITD, internal tandem duplication.
*
ICE vs HDS. Differences between treatment groups were evaluated using the Mann-Whitney U test for continuous variables and Pearson’s χ2 test (or Fisher’s exact tests for cells <10) for categorical variables.