Baseline characteristics
| Characteristic . | All patients, n = 654 . | Azacitidine, n = 587 . | LDAC, n = 67 . |
|---|---|---|---|
| Median age (IQR) | 73 (68-76) | 73 (68-76) | 73 (68-77) |
| Female | 261 (40%) | 230 (39%) | 31 (46%) |
| Clinical disease type | |||
| De novo | 387 (59%) | 341 (58%) | 46 (69%) |
| Secondary | 202 (31%) | 186 (32%) | 16 (24%) |
| MDS | 107 (53%) | 98 (53%) | 9 (56%) |
| MDS/MPN | 40 (20%) | 35 (19%) | 5 (31%) |
| MPN | 44 (22%) | 42 (23%) | 2 (13%) |
| Other or unknown | 11 (5.5%) | 11 (5.9%) | 0 (0%) |
| Therapy related | 65 (9.9%) | 60 (10%) | 5 (7.5%) |
| Baseline blood counts | |||
| WCC, median (IQR) | 5 (2-22) | 5 (2-19) | 14 (2-62) |
| Hemoglobin, median (IQR) | 87 (75-101) | 87 (75-101) | 88 (78-100) |
| Platelet count, median (IQR) | 71 (35-123) | 74 (36-124) | 55 (32-93) |
| Missing | 289 | 264 | 25 |
| Bone marrow blast, median (IQR), % | 42 (25-70) | 41 (25-70) | 52 (30-79) |
| Missing | 286 | 257 | 29 |
| Cytogenetic/FISH abnormalities | |||
| Core binding factor fusions | 5 (0.8%) | 5 (0.9%) | 0 (0%) |
| +4 | 16 (2.6%) | 16 (2.9%) | 0 (0%) |
| +8 | 66 (11%) | 61 (11%) | 5 (7.8%) |
| +11 | 15 (2.5%) | 14 (2.6%) | 1 (1.6%) |
| +13 | 19 (3.1%) | 18 (3.3%) | 1 (1.6%) |
| +19 | 11 (1.8%) | 11 (2.0%) | 0 (0%) |
| +21 | 16 (2.6%) | 15 (2.7%) | 1 (1.6%) |
| del9q | 4 (0.7%) | 3 (0.5%) | 1 (1.6%) |
| KMT2A rearrangement | 8 (1.3%) | 8 (1.5%) | 0 (0%) |
| t(9;22) | 2 (0.3%) | 1 (0.2%) | 1 (1.6%) |
| MECOM rearrangement | 12 (2.0%) | 12 (2.2%) | 0 (0%) |
| −5/del5q | 59 (10%) | 57 (11%) | 2 (3.2%) |
| −7/abn7q | 83 (14%) | 81 (15%) | 2 (3.2%) |
| −17/abn17p | 40 (6.8%) | 40 (7.7%) | 0 (0%) |
| Complex karyotype | 91 (15%) | 89 (16%) | 2 (3.1%) |
| Hyperdiploid karyotype | 14 (2.3%) | 13 (2.4%) | 1 (1.6%) |
| Normal karyotype | 313 (51%) | 267 (49%) | 46 (72%) |
| Missing | 43 | 40 | 3 |
| FLT3-ITD | 68 (11%) | 58 (10%) | 10 (15%) |
| Missing | 17 | 16 | 1 |
| FLT3 TKD | 41 (6.6%) | 36 (6.5%) | 5 (7.9%) |
| Missing | 33 | 29 | 4 |
| NPM1 | 149 (24%) | 112 (20%) | 37 (56%) |
| Missing | 22 | 21 | 1 |
| Mutations on NGS panel | |||
| CEBPA | 33 (6.4%) | 28 (6.0%) | 5 (10%) |
| ASXL1 | 119 (23%) | 108 (23%) | 11 (22%) |
| BCOR | 34 (6.6%) | 34 (7.3%) | 0 (0%) |
| EZH2 | 20 (3.9%) | 18 (3.9%) | 2 (4.0%) |
| RUNX1 | 107 (21%) | 102 (22%) | 5 (10%) |
| SF3B1 | 16 (3.1%) | 15 (3.2%) | 1 (2.0%) |
| SRSF2 | 124 (24%) | 113 (24%) | 11 (22%) |
| STAG2 | 44 (8.5%) | 40 (8.6%) | 4 (8.0%) |
| U2AF1 | 35 (6.8%) | 33 (7.1%) | 2 (4.0%) |
| ZRSR2 | 13 (2.5%) | 11 (2.4%) | 2 (4.0%) |
| TP53 | 67 (13%) | 66 (14%) | 1 (2.0%) |
| IDH1 | 46 (8.9%) | 45 (9.7%) | 1 (2.0%) |
| IDH2 | 88 (17%) | 74 (16%) | 14 (28%) |
| NRAS | 47 (9.1%) | 45 (9.7%) | 2 (4.0%) |
| KRAS | 22 (4.3%) | 19 (4.1%) | 3 (6.0%) |
| DNMT3A | 91 (18%) | 78 (17%) | 13 (26%) |
| TET2 | 112 (22%) | 97 (21%) | 15 (30%) |
| KIT | 6 (1.2%) | 5 (1.1%) | 1 (2.0%) |
| JAK2 | 33 (6.4%) | 32 (6.9%) | 1 (2.0%) |
| Missing | 138 | 121 | 17 |
| Characteristic . | All patients, n = 654 . | Azacitidine, n = 587 . | LDAC, n = 67 . |
|---|---|---|---|
| Median age (IQR) | 73 (68-76) | 73 (68-76) | 73 (68-77) |
| Female | 261 (40%) | 230 (39%) | 31 (46%) |
| Clinical disease type | |||
| De novo | 387 (59%) | 341 (58%) | 46 (69%) |
| Secondary | 202 (31%) | 186 (32%) | 16 (24%) |
| MDS | 107 (53%) | 98 (53%) | 9 (56%) |
| MDS/MPN | 40 (20%) | 35 (19%) | 5 (31%) |
| MPN | 44 (22%) | 42 (23%) | 2 (13%) |
| Other or unknown | 11 (5.5%) | 11 (5.9%) | 0 (0%) |
| Therapy related | 65 (9.9%) | 60 (10%) | 5 (7.5%) |
| Baseline blood counts | |||
| WCC, median (IQR) | 5 (2-22) | 5 (2-19) | 14 (2-62) |
| Hemoglobin, median (IQR) | 87 (75-101) | 87 (75-101) | 88 (78-100) |
| Platelet count, median (IQR) | 71 (35-123) | 74 (36-124) | 55 (32-93) |
| Missing | 289 | 264 | 25 |
| Bone marrow blast, median (IQR), % | 42 (25-70) | 41 (25-70) | 52 (30-79) |
| Missing | 286 | 257 | 29 |
| Cytogenetic/FISH abnormalities | |||
| Core binding factor fusions | 5 (0.8%) | 5 (0.9%) | 0 (0%) |
| +4 | 16 (2.6%) | 16 (2.9%) | 0 (0%) |
| +8 | 66 (11%) | 61 (11%) | 5 (7.8%) |
| +11 | 15 (2.5%) | 14 (2.6%) | 1 (1.6%) |
| +13 | 19 (3.1%) | 18 (3.3%) | 1 (1.6%) |
| +19 | 11 (1.8%) | 11 (2.0%) | 0 (0%) |
| +21 | 16 (2.6%) | 15 (2.7%) | 1 (1.6%) |
| del9q | 4 (0.7%) | 3 (0.5%) | 1 (1.6%) |
| KMT2A rearrangement | 8 (1.3%) | 8 (1.5%) | 0 (0%) |
| t(9;22) | 2 (0.3%) | 1 (0.2%) | 1 (1.6%) |
| MECOM rearrangement | 12 (2.0%) | 12 (2.2%) | 0 (0%) |
| −5/del5q | 59 (10%) | 57 (11%) | 2 (3.2%) |
| −7/abn7q | 83 (14%) | 81 (15%) | 2 (3.2%) |
| −17/abn17p | 40 (6.8%) | 40 (7.7%) | 0 (0%) |
| Complex karyotype | 91 (15%) | 89 (16%) | 2 (3.1%) |
| Hyperdiploid karyotype | 14 (2.3%) | 13 (2.4%) | 1 (1.6%) |
| Normal karyotype | 313 (51%) | 267 (49%) | 46 (72%) |
| Missing | 43 | 40 | 3 |
| FLT3-ITD | 68 (11%) | 58 (10%) | 10 (15%) |
| Missing | 17 | 16 | 1 |
| FLT3 TKD | 41 (6.6%) | 36 (6.5%) | 5 (7.9%) |
| Missing | 33 | 29 | 4 |
| NPM1 | 149 (24%) | 112 (20%) | 37 (56%) |
| Missing | 22 | 21 | 1 |
| Mutations on NGS panel | |||
| CEBPA | 33 (6.4%) | 28 (6.0%) | 5 (10%) |
| ASXL1 | 119 (23%) | 108 (23%) | 11 (22%) |
| BCOR | 34 (6.6%) | 34 (7.3%) | 0 (0%) |
| EZH2 | 20 (3.9%) | 18 (3.9%) | 2 (4.0%) |
| RUNX1 | 107 (21%) | 102 (22%) | 5 (10%) |
| SF3B1 | 16 (3.1%) | 15 (3.2%) | 1 (2.0%) |
| SRSF2 | 124 (24%) | 113 (24%) | 11 (22%) |
| STAG2 | 44 (8.5%) | 40 (8.6%) | 4 (8.0%) |
| U2AF1 | 35 (6.8%) | 33 (7.1%) | 2 (4.0%) |
| ZRSR2 | 13 (2.5%) | 11 (2.4%) | 2 (4.0%) |
| TP53 | 67 (13%) | 66 (14%) | 1 (2.0%) |
| IDH1 | 46 (8.9%) | 45 (9.7%) | 1 (2.0%) |
| IDH2 | 88 (17%) | 74 (16%) | 14 (28%) |
| NRAS | 47 (9.1%) | 45 (9.7%) | 2 (4.0%) |
| KRAS | 22 (4.3%) | 19 (4.1%) | 3 (6.0%) |
| DNMT3A | 91 (18%) | 78 (17%) | 13 (26%) |
| TET2 | 112 (22%) | 97 (21%) | 15 (30%) |
| KIT | 6 (1.2%) | 5 (1.1%) | 1 (2.0%) |
| JAK2 | 33 (6.4%) | 32 (6.9%) | 1 (2.0%) |
| Missing | 138 | 121 | 17 |
FISH, fluorescence in situ hybridization; MPN, myeloproliferative neoplasm.