Table 2.
Patient and disease characteristics as well as molecular characterization, 2017 ELN risk stratification, and treatment by ITD insertion site.
| JMDsole (n = 251) | JMD/TKD1 (n = 117) | TKD1sole (n = 84) | P value | |
|---|---|---|---|---|
| Age, median (years) | 48.3 | 48.1 | 46.3 | 0.171 |
| Gender, male (%) | 41.0 | 48.7 | 54.8 | 0.067 |
| WBC, median (range), ×109/l | 40.0 (0.8–329.8) | 39.9 (0.9–205.4) | 53.9 (1.2–144.8) | 0.254 |
| BM blasts, median (%) | 79.5 | 75.5 | 80 | 0.489 |
| NPM1mut, n/total n (%) | 123/195 (63.1) | 46/92 (50) | 34/71 (47.9) | 0.028 |
| FLT3-ITD AR risk stratification | ||||
| FLT3-ITDhigh (AR > 0.5)a, n/total n (%) | 178/251 (70.9) | 56/117 (47.9) | 56/84 (66.7) | <0.001 |
| NPM1/FLT3-ITD genotypes | 0.003 | |||
| NPM1mut/FLT3-ITDlow, n/total n (%) | 39/195 (20.0) | 19/91 (20.9) | 10/71 (14.1) | 0.494 |
| NPM1mut/FLT3-ITDhigh, n/total n (%) | 84/195 (43.1) | 27/91 (29.7) | 24/71 (33.8) | 0.069 |
| NPM1wt/FLT3-ITDlow, n/total n (%) | 18/195 (9.2) | 26/91 (28.6) | 13/71 (18.3) | <0.001 |
| NPM1wt/FLT3-ITDhigh, n/total n (%) | 54/195 (27.7) | 19/91 (20.9) | 24/71 (33.8) | 0.190 |
| 2017 ELN risk groups | 0.473 | |||
| Favorable, n/total n (%) | 41/149 (27.5) | 24/67 (35.8) | 12/52 (23.1) | 0.277 |
| Intermediate, n/total n (%) | 57/149 (38.3) | 19/67 (28.4) | 19/52 (36.5) | 0.366 |
| Adverse, n/total n (%) | 51/149 (34.2) | 24/67 (35.8) | 21/52 (40.4) | 0.709 |
| No of ITDs | ||||
| Median | 1 | 3 | 1 | <0.001 |
| Range | 1–5 | 2–9 | 1–3 | |
| NGS-based FLT3-ITD cAR | ||||
| Median | 0.41 | 0.19 | 0.14 | <0.001 |
| Range | 0.001–10.76 | 0.003–4.62 | 0.001–2.80 | |
| Treatment | ||||
| Placebo, n (%) | 132 (52.6) | 61 (52.1) | 29 (34.5) | 0.012 |
| Midostaurin, n (%) | 119 (47.4) | 56 (47.9) | 55 (65.5) | |
| Allogeneic HCT in CR1, n (%) | 56 (22.3) | 22 (18.8) | 21 (25.0) | 0.562 |
WBC white blood cell, BM bone marrow, AR allelic ratio, ELN European LeukemiaNet.
aAssessed using Genescan analysis.