Table 1.
Demographics and Disease Characteristics of Patients with (a) ND and (b) R/R AML in the NICHE-AML Registry
| (a) | ||||
| Characteristic | All ND AML Patients N = 853 | FLT3WT N = 432 | FLT3MUT N = 109 | Unknown FLT3 Mutation Statusa N = 312 |
| Follow-up duration, days | 573.4 (475.9) | 494.8 (364.2) | 399.8 (328.1) | 742.9 (592.6) |
| Age at diagnosis, y | 42.5 (12.8) | 43.1 (12.8) | 44.0 (12.5) | 41.3 (12.8) |
| Male, n (%) | 448 (52.5) | 244 (56.5) | 51 (46.8) | 153 (49.0) |
| Known mutation status at diagnosis, n (%) | 541 (63.4) | 432 (100) | 109 (100) | - |
| FLT3 | 109 (20.1) | 0 | 109 (100) | - |
| NPM1 | 109 (20.1) | 66 (15.3) | 43 (39.4) | - |
| CEBPA | 103 (19.0) | 89 (20.6) | 14 (12.8) | - |
| IDH1 | 38 (7.0) | 32 (7.4) | 6 (5.5) | - |
| Other acquired mutation | 533 (98.5) | 428 (99.1) | 105 (96.3) | - |
| Cytogenetic status, n (%) | 832 (97.5) | 421 (97.5) | 105 (96.3) | 306 (98.1) |
| Abnormal karyotype | 416 (50.0) | 235 (55.8) | 42 (40.0) | 139 (45.4) |
| FAB classification,23 n (%) | 650 (76.2) | 345 (79.9) | 77 (70.6) | 228 (73.1) |
| M0 | 3 (0.5) | 2 (0.6) | 0 | 1 (0.4) |
| M1 | 6 (0.9) | 2 (0.6) | 1 (1.3) | 3 (1.3) |
| M2 | 253 (38.9) | 158 (45.8) | 20 (26.0) | 75 (32.9) |
| M4 | 113 (17.4) | 54 (15.7) | 12 (15.6) | 47 (20.6) |
| M5 | 265 (40.8) | 126 (36.5) | 44 (57.1) | 95 (41.7) |
| M6 | 6 (0.9) | 1 (0.3) | 0 | 5 (2.2) |
| M7 | 1 (0.2) | 0 | 0 | 1 (0.4) |
| Type of AML | ||||
| De novo | 849 (99.5) | 428 (99.1) | 109 (100) | 312 (100) |
| Secondary | 4 (0.5) | 4 (0.9) | 0 | 0 |
| Risk stratification7 | 746 (87.5) | 427 (98.8) | 108 (99.1) | 211 (67.6) |
| Favorable | 283 (37.9) | 200 (46.8) | 82 (75.9) | 1 (0.5) |
| Intermediate | 423 (56.7) | 227 (53.2) | 26 (24.1) | 170 (80.6) |
| Adverse | 40 (5.4) | 0 | 0 | 40 (19.0) |
| (b) | ||||
| Characteristic | All R/R AML Patients N = 289 | FLT3WT N = 58 | FLT3MUT N = 14 | Unknown FLT3 Mutation Statusb N = 217 |
| Follow-up duration, days | 562.7 (438.3) | 606.2 (371.7) | 479.2 (261.3) | 556.4 (463.2) |
| Age at diagnosis, y | 43.6 (13.5) | 43.5 (13.1) | 44.4 (15.4) | 43.6 (13.6) |
| Male, n (%) | 154 (53.3) | 32 (55.2) | 9 (64.3) | 113 (52.1) |
| Known mutation status at diagnosis, n (%) | 183 (63.3) | 45 (77.6) | 9 (64.3) | 129 (59.4) |
| FLT3 | 29 (15.8) | 3 (6.7) | 4 (44.4) | 22 (17.1) |
| NPM1 | 30 (16.4) | 8 (17.8) | 3 (33.3) | 19 (14.7) |
| CEBPA | 45 (24.6) | 20 (44.4) | 1 (11.1) | 24 (18.6) |
| IDH1 | 15 (8.2) | 2 (4.4) | 2 (22.2) | 11 (8.5) |
| Other acquired mutation | 182 (99.5) | 44 (97.8) | 9 (100) | 129 (100) |
| Cytogenetic status, n (%) | 285 (98.6) | 56 (96.6) | 14 (100) | 215 (99.1) |
| Abnormal karyotype | 135 (47.4) | 21 (37.5) | 2 (14.3) | 112 (52.1) |
| FAB classification,23 n (%) | 229 (79.2) | 56 (96.6) | 11 (78.6) | 162 (74.7) |
| M0 | 0 | 0 | 0 | 0 |
| M1 | 2 (0.9) | 2 (3.6) | 0 | 0 |
| M2 | 87 (38.0) | 30 (53.6) | 1 (9.1) | 56 (34.6) |
| M4 | 34 (14.8) | 5 (8.9) | 2 (18.2) | 27 (16.7) |
| M5 | 103 (45.0) | 19 (33.9) | 7 (63.6) | 77 (47.5) |
| M6 | 2 (0.9) | 0 | 1 (9.1) | 1 (0.6) |
| M7 | 0 | 0 | 0 | 0 |
| Type of AML | ||||
| De novo | 287 (99.3) | 58 (100) | 14 (100) | 215 (99.1) |
| Secondary | 2 (0.7) | 0 | 0 | 2 (0.9) |
| Risk stratification7c | 254 (87.9) | 55 (94.8) | 14 (100) | 185 (85.3) |
| Favorable | 88 (34.6) | 31 (56.4) | 5 (35.7) | 52 (28.1) |
| Intermediate | 152 (59.8) | 23 (41.8) | 9 (64.3) | 120 (64.9) |
| Adverse | 14 (5.5) | 1 (1.8) | 0 | 13 (7.0) |
Notes: Data are mean (SD), unless otherwise stated. aGenetic testing results were not available for patients with unknown FLT3 mutation status at initial AML diagnosis; bGenetic testing results at initial diagnosis were summarized among patients with relevant information at initial diagnosis; cEach patient was classified into only one risk category.
Abbreviations: AML, acute myeloid leukemia; CEBPA, CCAAT enhancer binding protein alpha; FAB, French-American-British; FLT3, FMS-like tyrosine kinase-3; IDH, isocitrate dehydrogenase; MUT, mutated; ND, newly diagnosed; NPM1, nucleophosmin 1; R/R, relapsed or refractory; SD standard deviation, WT, wild-type, y, years.