Table 1 ∣.
Baseline characteristics at screening across the different eligible patient groups
| Characteristic | All eligible patients (n = 395) |
Beat AML sub-study (n = 224) |
SOC therapy (n = 103) |
investigational therapy (n = 28) |
Palliative care/unknown (n = 40) |
P1/P2 |
|---|---|---|---|---|---|---|
| Age (years) | 0.009/0.21 | |||||
| Median (range) | 72 (60–92) | 72(60–92) | 71 (60–87) | 71 (61–83) | 77.5 (60–91) | |
| Age ≥75 years, n (%) | 152 (38) | 87 (39) | 36 (35) | 7 (25) | 22 (55) | 0.07/0.54 |
| Sex, n (%) | 0.79/0.90 | |||||
| Female | 175 (44) | 101 (45) | 45 (44) | 10 (36) | 19 (48) | |
| Male | 220 (56) | 123 (55) | 58 (56) | 18 (64) | 21 (53) | |
| Ethnic group, n (%) | 0.94/1.00 | |||||
| White | 347 (90) | 195 (89) | 89 (89) | 26 (93) | 37 (93) | |
| African American | 18 (5) | 10 (5) | 6 (6) | 0 (0) | 2 (5) | |
| Asian | 13 (3) | 8 (4) | 3 (3) | 2 (7) | 0 (0) | |
| Other | 8 (2) | 5 (2) | 2 (2) | 0 (0) | 1 (3) | |
| Unknown | 9 | 6 | 3 | 0 | 0 | |
| Performance status, n (%) | 0.10/0.25 | |||||
| 0 | 83 (23) | 48 (22) | 23 (26) | 10 (36) | 2 (6) | |
| 1 | 183 (50) | 118 (54) | 38 (43) | 11 (39) | 16 (50) | |
| 2 | 90 (25) | 50 (23) | 23 (26) | 6 (21) | 11 (34) | |
| 3 | 9 (2) | 2 (1) | 4 (5) | 1 (4) | 2 (6) | |
| 4 | 1 (<1) | 0 (0) | 0 (0) | 0 (0) | 1 (3) | |
| Not assessed/unknown | 29 | 6 | 15 | 0 | 8 | |
| Hemoglobin (g dl−1) | 0.18/0.61 | |||||
| Median (range) | 8.5 (3.9–15.0) | 8.5 (6.1–13.9) | 8.6 (4.9–15.0) | 8.7 (6.7–12.1) | 8.15 (3.9–12.0) | |
| Not assessed/unknown | 1 | 1 | 0 | 0 | 0 | |
| Platelet (109 l−1) | 0.71/0.45 | |||||
| Median (range) | 60 (5–656) | 59 (7–656) | 65 (5–427) | 60.5 (11–213) | 58.5 (12–320) | |
| Not assessed/unknown | 3 | 3 | 0 | 0 | 0 | |
| WBC (109 l−1) | 0.01/0.16 | |||||
| Median (range) | 4.85 (0.40–231.2) | 4.7 (0.6–194.1) | 6.0 (0.40–231.2) | 1.85 (0.5–49.9) | 8.5 (0.6–168.2) | |
| Not assessed/unknown | 1 | 1 | 0 | 0 | 0 | |
| WBC >50, n (%) | 40 (10) | 20 (9) | 16 (16) | 0 (0) | 4 (10) | 0.07/0.09 |
| Blood blasts, % | 0.21/0.25 | |||||
| Median (range) | 13.9 (0–98.4) (0–98) | 12 (0–96) | 21 (0–98) | 6(0–62) | 18 (0–85) | |
| Not assessed/unknown | 48 | 25 | 14 | 4 | 5 | |
| Bone marrow blasts (%) | 0.10/0.06 | |||||
| Median (range) | 46 (1–99) | 45 (1–98) | 51 (15–99) | 40.5 (3–76) | 42.5 (11–93) | |
| Not assessed/unknown | 26 | 13 | 7 | 2 | 4 | |
| Alanine aminotransferase, U l−1 | 0.76/0.31 | |||||
| Median (range) | 18 (3–203) | 17 (3–180) | 18 (5–203) | 18 (9–106) | 19.5 (5–50) | |
| Not assessed/unknown | 21 | 10 | 6 | 1 | 4 | |
| Aspartate aminotransferase, U l−1 | 0.15/0.93 | |||||
| Median (range) | 21 (6–256) | 21 (8–203) | 21 (6–256) | 18 (11–42) | 24 (9–79) | |
| Not assessed/unknown | 13 | 7 | 4 | 0 | 2 | |
| Bilirubin (mg dl−1) | 0.08/0.62 | |||||
| Median (range) | 0.6 (0.1–10.4) | 0.6 (0.1–2.9) | 0.6 (0.3–10.4) | 0.6 (0.3–3.5) | 0.8 (0.2–3.1) | |
| Not assessed/unknown | 10 | 5 | 3 | 0 | 2 | |
| Creatinine (mg dl−1) | 0.99/0.70 | |||||
| Median (range) | 0.89 (0.34–2.79) | 0.88 (0.34–2.50) | 0.91 (0.39–2.78) | 0.90 (0.44–2.32) | 0.84 (0.47–2.79) | |
| Not assessed/unknown | 7 | 6 | 0 | 0 | 1 | |
| Treatment-related AML, n (%) | 0.57/0.63 | |||||
| No | 331 (84) | 188 (84) | 83 (81) | 26 (93) | 34 (85) | |
| Yes | 63 (16) | 36 (16) | 19 (19) | 2 (7) | 6 (15) | |
| Unknown | 1 | 0 | 1 | 0 | 0 | |
| CBF, n (%) | 0.56/0.46 | |||||
| Absent | 369 (98) | 213 (98) | 91 (96) | 27 (100) | 38 (100) | |
| Present | 9 (2) | 5 (2) | 4 (4) | 0 (0) | 0 (0) | |
| Unknown | 17 | 6 | 8 | 1 | 2 | |
| MLL, n (%) | 0.20/0.14 | |||||
| Absent | 369 (97) | 215 (98) | 91 (95) | 27 (100) | 36 (95) | |
| Present | 11 (3) | 4 (2) | 5 (5) | 0 (0) | 2 (5) | |
| Unknown | 15 | 5 | 7 | 1 | 2 | |
| Complex cytogenetics, n (%) | 0.13/0.78 | |||||
| Absent | 245 (68) | 143 (69) | 63 (71) | 20 (77) | 19 (51) | |
| Present | 115 (32) | 65 (31) | 26 (29) | 6 (23) | 18 (49) | |
| Unknown | 35 | 16 | 14 | 2 | 3 | |
| FLT3-ITD, n (%) | 0.30/0.10 | |||||
| Absent | 357 (90) | 207 (92) | 89 (86) | 26 (93) | 35 (88) | |
| Present | 38 (10) | 17 (8) | 14 (14) | 2 (7) | 5 (13) | |
| NPM1 cytoplasmic, n (%) | 0.61/0.64 | |||||
| WT (VAF < 20%) | 326 (83) | 183 (82) | 87 (84) | 25 (89) | 31 (78) | |
| Mutated (VAF ≥ 20%) | 69 (17) | 41 (18) | 16 (16) | 3 (11) | 9 (23) | |
| IDH2, n (%) | 0.04/0.17 | |||||
| WT (VAF < 20%) | 332 (84) | 179 (80) | 89 (86) | 26 (93) | 38 (95) | |
| Mutated (VAF ≥ 20%) | 63 (16) | 45 (20) | 14 (14) | 2 (7) | 2 (5) | |
| IDH1, n (%) | 0.02/1.00 | |||||
| WT (VAF < 20%) | 357 (90) | 206 (92) | 95 (92) | 20 (71) | 36 (90) | |
| Mutated (VAF ≥ 20%) | 38 (10) | 18 (8) | 8 (8) | 8 (29) | 4 (10) | |
| TP53, n (%) | 0.57/1.00 | |||||
| WT (VAF < 20%) | 311 (79) | 178 (79) | 82 (80) | 23 (82) | 28 (70) | |
| Mutated (VAF ≥ 20%) | 84 (21) | 46 (21) | 21 (20) | 5 (18) | 12 (30) | |
| FLT3-TKD, n (%) | 0.88/0.73 | |||||
| WT (VAF < 20%) | 384 (97) | 220 216 (98) (96) | 101 (98) | 28 (100) | 39 (98) | |
| Mutated (VAF ≥ 20%) | 11 (3) | 4 8 (2) (4) | 2 (2) | 0 (0) | 1 (3) | |
| FLT3 (other), n (%) | 0.80/1.00 | |||||
| WT (VAF < 20%) | 389 (98) | 220 (98) | 102 (99) | 28 (100) | 39 (98) | |
| Mutated (VAF ≥ 20%) | 6 (2) | 4 (2) | 1 (<1) | 0 (0) | 1 (3) | |
| TET2, n (%) | 0.75/0.77 | |||||
| WT (VAF < 20%) | 312 (79) | 179 (80) | 81 (79) | 20 (71) | 32 (80) | |
| Mutated (VAF ≥ 20%) | 83 (21) | 45 (20) | 22 (21) | 8 (29) | 8 (20) | |
| WT1, n (%) | 0.53/0.53 | |||||
| WT (VAF < 20%) | 383 (97) | 217 (97) | 98 (95) | 28 (100) | 40 (100) | |
| Mutated (VAF ≥ 20%) | 12 (3) | 7 (3) | 5 (5) | 0 (0) | 0 (0) |
Fisher’s exact and Kruskal–Wallis tests were used to test for associations between elected treatment groups and categorical or continuous baseline characteristics, respectively. P values from global tests, including the four elected treatment groups, are represented by P1; P values from tests including two elected treatment groups (Beat AML sub-study versus SOC therapy) are represented by P2. All tests were two-sided and P values were not adjusted for multiple testing. CBF, core binding factor; TKD, tyrosine kinase domain; WT, wild type.