Patient and disease characteristics and response to therapy by 2017 ELN risk groups
| . | Favorable risk* . | Intermediate risk† . | Adverse risk‡ . | P . |
|---|---|---|---|---|
| Patients, n (%) | 85 (26.7) | 111 (34.9) | 122 (38.4) | |
| Age, median, y | 50.6 | 47.9 | 47.0 | .07 |
| Female, % | 64.7 | 64.9 | 49.2 | .04 |
| WBC count, median (range), ×109/L | 23.6 (1.4-253.2) | 42.6 (0.6-329.8) | 38.0 (0.8-236.0) | .0019 |
| Median BM blasts, % | 72 | 78 | 76 | .33 |
| Treatment, n (%) | ||||
| Placebo | 47 (55.3) | 57 (51.4) | 54 (44.3) | .27 |
| Midostaurin | 38 (44.7) | 54 (48.6) | 68 (55.7) | |
| Allogeneic HCT in CR1 | 24 (28.2) | 31 (27.9) | 36 (29.5) | .95 |
| Concurrent gene mutations, n (%) | ||||
| RUNX1 | 1/73 (1.4) | 0/110 (0) | 31/106 (29.2) | .0005 |
| ASXL1 | 4/73 (5.5) | 0/110 (0) | 21/106 (19.8) | .0005 |
| TP53 | 0/73 (0) | 0/110 (0) | 2/106 (1.9) | .19 |
| Karyotype, n (%) | ||||
| Normal | 52 (89.7) | 98 (88.3) | 68 (57.1) | .0005 |
| Abnormal | 6 (10.3) | 13 (11.7) | 51 (42.9) | |
| Missing | 27 | 1 | 3 |
| . | Favorable risk* . | Intermediate risk† . | Adverse risk‡ . | P . |
|---|---|---|---|---|
| Patients, n (%) | 85 (26.7) | 111 (34.9) | 122 (38.4) | |
| Age, median, y | 50.6 | 47.9 | 47.0 | .07 |
| Female, % | 64.7 | 64.9 | 49.2 | .04 |
| WBC count, median (range), ×109/L | 23.6 (1.4-253.2) | 42.6 (0.6-329.8) | 38.0 (0.8-236.0) | .0019 |
| Median BM blasts, % | 72 | 78 | 76 | .33 |
| Treatment, n (%) | ||||
| Placebo | 47 (55.3) | 57 (51.4) | 54 (44.3) | .27 |
| Midostaurin | 38 (44.7) | 54 (48.6) | 68 (55.7) | |
| Allogeneic HCT in CR1 | 24 (28.2) | 31 (27.9) | 36 (29.5) | .95 |
| Concurrent gene mutations, n (%) | ||||
| RUNX1 | 1/73 (1.4) | 0/110 (0) | 31/106 (29.2) | .0005 |
| ASXL1 | 4/73 (5.5) | 0/110 (0) | 21/106 (19.8) | .0005 |
| TP53 | 0/73 (0) | 0/110 (0) | 2/106 (1.9) | .19 |
| Karyotype, n (%) | ||||
| Normal | 52 (89.7) | 98 (88.3) | 68 (57.1) | .0005 |
| Abnormal | 6 (10.3) | 13 (11.7) | 51 (42.9) | |
| Missing | 27 | 1 | 3 |
| . | All patients . | Midostaurin . | Placebo . | . |
|---|---|---|---|---|
| Response to induction therapy, %§ | ||||
| Favorable | 69.4 | 71.1 | 68.1 | .82 |
| Intermediate | 63.1 | 66.7 | 59.6 | .56 |
| Adverse | 51.6 | 57.4 | 44.4 | .20 |
| . | All patients . | Midostaurin . | Placebo . | . |
|---|---|---|---|---|
| Response to induction therapy, %§ | ||||
| Favorable | 69.4 | 71.1 | 68.1 | .82 |
| Intermediate | 63.1 | 66.7 | 59.6 | .56 |
| Adverse | 51.6 | 57.4 | 44.4 | .20 |
For definition of risk groups, see also supplemental Table 2.
NPM1mut/FLT3-ITDlow AML cases, irrespective of additional high-risk gene mutation or additional chromosomal abnormalities.
NPM1mut/FLT3-ITDhigh AML (n = 93) and NPM1wt/FLT3-ITDlow AML (n = 18), both subgroups without the concurrent presence of the high-risk molecular markers RUNX1, ASXL1, and TP53. Also, NPM1wt/FLT3-ITDlow AML without adverse-risk cytogenetics.
NPM1wt/FLT3-ITDhigh AML (n = 92), NPM1mut/FLT3-ITDhigh AML exhibiting high-risk molecular markers (n = 8), and NPM1wt/FLT3-ITDlow AML with high-risk molecular markers and/or adverse-risk cytogenetics (n = 22).
CRs achieved during induction therapy (cycles 1 and 2).