Table. Patient Clinical Characteristics.
| Characteristic | No. (%) of patients | |
|---|---|---|
| Discovery (2013-2017) | Validation (2018-2019) | |
| No. of patients | 454 | 621 |
| Sites | 81 | 101 |
| Age, y | ||
| Median (range) | 56.8 (18.4-75.6) | 58.2 (18-80.5) |
| <60 | 275 (61) | 346 (56) |
| ≥60 | 179 (39) | 275 (44) |
| Sex | ||
| Female | 237 (52) | 331 (53) |
| Male | 217 (48) | 290 (47) |
| Race and ethnicity | ||
| No. | 443 | 603 |
| American Indian or Alaska Native | 2 (<1) | 3 (<1) |
| Asian | 32 (7) | 31 (5) |
| Black or African American | 32 (7) | 30 (5) |
| Multiracial | 2 (<1) | 2 (<1) |
| Native Hawaiian or Pacific Islander | 1 (<1) | 3 (<1) |
| White (Hispanic or Latino) | 18 (4) | 34 (6) |
| White (non-Hispanic, non-Latino) | 356 (80) | 500 (83) |
| HCT-comorbidity indexa | ||
| No. | 444 | 617 |
| 0 (lowest comorbidity) | 75 (17) | 132 (21) |
| 1-2 (intermediate comorbidity) | 160 (36) | 175 (28) |
| >2 (highest comorbidity) | 209 (47) | 310 (50) |
| Karnofsky performance scoreb | ||
| No. | 449 | |
| <90 (less able to carry out daily activities) | 186 (41) | 249 (40) |
| ≥90 (normal activity without extra effort) | 263 (59) | 372 (60) |
| AML type | ||
| De novo | 390 (86) | 556 (90) |
| Secondary | ||
| Therapy-related | 17 (4) | 31 (5) |
| Transformed MDS/MPN | 47 (10) | 34 (6) |
| ELN risk groupc | ||
| Favorable | 86 (19) | 155 (25) |
| Intermediate | 220 (49) | 255 (41) |
| Adverse | 146 (32) | 211 (34) |
| Reported variants from AML diagnosis | ||
| FLT3-TKD | 63 | 80 |
| FLT3-ITD | 276 | 332 |
| IDH1 | 27 | 79 |
| IDH2 | 51 | 149 |
| KIT | 26 | 34 |
| NPM1 | 239 | 292 |
| Baseline FLT3-ITD and NPM1 co-occurrence | ||
| FLT3-ITD plus NPM1 mutated | 144 | 173 |
| FLT3-ITD plus NPM1 wild type | 98 | 128 |
| FLT3-ITD plus unknown | 34 | 31 |
| Conditioning regimen | ||
| Myeloablative | 254 (56) | 351 (57) |
| Reduced intensity/nonmyeloablative | 200 (44) | 270 (43) |
| Graft typed | ||
| Peripheral blood | 287 (63) | 490 (79) |
| Bone marrow | 64 (14) | 96 (15) |
| Cord blood | 103 (23) | 35 (6) |
| Donor type | ||
| Matched unrelated | 231 (51) | 404 (65) |
| Cord blood | 103 (23) | 32 (5) |
| HLA-identical sibling | 68 (15) | 69 (11) |
| Haploidentical related | 44 (10) | 55 (9) |
| Mismatched | 8 (2) | 61 (10) |
| Antithymocyte globulin used | 123/451 (27) | 142/621 (23) |
Abbreviations: AML, acute myeloid leukemia; ELN, European LeukemiaNet; HCT, hematopoietic cell transplantation; HLA, human leukocyte antigen; ITD, internal tandem duplication; MDS, myelodysplastic syndrome; MPN, myeloproliferative neoplasm; TKD, tyrosine kinase domain.
The HCT comorbidity index stratifies patients based on the number and severity of pretransplant comorbidities to predict nonrelapse mortality and survival.50
The Center for International Blood and Marrow Transplant Research uses Karnofsky performance status to determine the functional status of transplant recipients aged 16 or older.
Standardized prognostic system distinguishes distinct risk groups of patients with newly diagnosed AML.36,37
Three recipients in the validation cohort listed as graft type cord blood received haploidentical and cord blood transplants.