Patient characteristics
| Characteristic . | Overall, N = 57 . | VEN dosage . | |
|---|---|---|---|
| VEN 400 mg, n = 28 . | VEN 600 mg, n = 29 . | ||
| Age, median (range), y | 54 (18-66) | 55 (30-66) | 53 (18-66) |
| Male, n (%) | 37 (65) | 17 (61) | 20 (69) |
| ELN 2017 intermediate risk, n (%)∗ | 32 (56) | 15 (54) | 17 (59) |
| ELN 2017 high risk, n (%)∗ | 25 (44) | 13 (46) | 12 (41) |
| White blood cells, median (range), ×109/L | 3 (0-274) | 3 (0-120) | 3 (1-274) |
| FLT3 mutation, n (%) | |||
| ITD | 4 (7.7) | 1 (3.9) | 3 (11.5) |
| TKD | 1 (1.9) | 0 (0) | 1 (3.8) |
| ITD and TKD | 2 (3.9) | 0 (0) | 2 (7.8) |
| WT | 45 (86.5) | 26 (96.1) | 20 (76.9) |
| Unknown† | 5 | 2 | 3 |
| NPM1, n (%) | |||
| WT | 46 (100) | 24 (100) | 22 (100) |
| Unknown† | 11 | 4 | 7 |
| Complex/high-risk cytogenetic, n (%) | 25 (44) | 13 (46) | 12 (41) |
| Secondary AML, n (%) | 12 (21.1) | 8 (28.6) | 4 (13.8) |
| Therapy-related AML, n (%) | 5 (9) | 3 (10.7) | 2 (6.9) |
| Characteristic . | Overall, N = 57 . | VEN dosage . | |
|---|---|---|---|
| VEN 400 mg, n = 28 . | VEN 600 mg, n = 29 . | ||
| Age, median (range), y | 54 (18-66) | 55 (30-66) | 53 (18-66) |
| Male, n (%) | 37 (65) | 17 (61) | 20 (69) |
| ELN 2017 intermediate risk, n (%)∗ | 32 (56) | 15 (54) | 17 (59) |
| ELN 2017 high risk, n (%)∗ | 25 (44) | 13 (46) | 12 (41) |
| White blood cells, median (range), ×109/L | 3 (0-274) | 3 (0-120) | 3 (1-274) |
| FLT3 mutation, n (%) | |||
| ITD | 4 (7.7) | 1 (3.9) | 3 (11.5) |
| TKD | 1 (1.9) | 0 (0) | 1 (3.8) |
| ITD and TKD | 2 (3.9) | 0 (0) | 2 (7.8) |
| WT | 45 (86.5) | 26 (96.1) | 20 (76.9) |
| Unknown† | 5 | 2 | 3 |
| NPM1, n (%) | |||
| WT | 46 (100) | 24 (100) | 22 (100) |
| Unknown† | 11 | 4 | 7 |
| Complex/high-risk cytogenetic, n (%) | 25 (44) | 13 (46) | 12 (41) |
| Secondary AML, n (%) | 12 (21.1) | 8 (28.6) | 4 (13.8) |
| Therapy-related AML, n (%) | 5 (9) | 3 (10.7) | 2 (6.9) |
ITD, internal tandem duplication; TKD, tyrosine kinase domain; WT, wild type.
Karyotypes are reported in supplemental Table 2.
Extended next-generation sequencing characterization was performed as a post-hoc analysis on centralized and biobanked samples (54/57 analyzable; 3/57 insufficient material). We confirmed that no NPM1 hot spot mutation was present in NPM1, and no new FLT3 mutation was detected. IDH1 was mutated in 4 of 54 (7.4%), IDH2 in 9 of 54 (16.6%), TP53 in 8 of 54 (14.8%) patients. Accounting for genetic abnormalities defined post hoc, ELN 2022 risk stratification is fully applicable in 55 patients, and 42 of 55 patients (76.3%) are classified as high risk.