Characteristics of patients with a FLT3-TKDmut AML included in the RATIFY trial (n = 163)
| . | Total patient population (N = 163) . | Midostaurin (n = 83) . | Placebo (n = 80) . | P, mido vs placebo . |
|---|---|---|---|---|
| Sex, males/females, n | 79/84 | 45/38 | 34/46 | .180 |
| Age, median (range), y | 48.8 (19.3-59.9) | 48.0 (19.8-59.8) | 49.4 (19.3-59.9) | .827 |
| ECOG PS 2, % | 13 (8) | 6 (7.2) | 7 (8.8) | .945 |
| WBC count, median (range), × 109/L | 28.80 (1.20-300.00) | 29.70 (1.20-298.40) | 28.15 (1.30-300.00) | .480 |
| Platelet count, median (range), × 109/L | 49.00 (10.00-328.00) | 53.00 (11.00-328.00) | 46.50 (10.00-237.00) | .430 |
| Bone marrow blasts, median (range), % | 75.00 (0.00-100.00) | 75.00 (0.00-100.00) | 77.00 (0.00-100.00 | .817 |
| Peripheral blood blasts, median (range), % | 55.00 (0.00-99.00) | 56.50 (0.00-99.00) | 55.00 (0.00-98.00) | .594 |
| Prior myelodysplastic syndrome, n (%) | 4 (2.5) | 2 (2.4) | 2 (2.5) | >.99 |
| Extramedullary disease, n (%) | 31 (19.0) | 13 (15.7) | 18 (22.5) | .362 |
| NPM1, n (%) (n = 134) | ||||
| Mutated | 79 (59.0) | 45 (64.3) | 34 (53.1) | .256 |
| WT | 55 (41) | 25 (35.7) | 30 (46.9) | |
| Karyotype, n (%) (n = 135) | ||||
| CBF* | 20 (14.8) | 10 (15.6) | 10 (14.1) | .140 |
| Normal | 74 (54.8) | 39 (60.9) | 35 (49.3) | |
| Others† | 41 (30.4) | 15 (23.4) | 26 (36.6) | |
| CR after induction, n (%) | 105 (64.4) | 57 (68.6) | 48 (60) | .321 |
| Allogeneic HCT in first CR, n (%) | 62 (38.0) | 32 (38.6) | 30 (37.5) | >.99 |
| Midostaurin maintenance, n (%) | 58 (35.6) | 32 (38.6) | 26 (32.5) | .52 |
| . | Total patient population (N = 163) . | Midostaurin (n = 83) . | Placebo (n = 80) . | P, mido vs placebo . |
|---|---|---|---|---|
| Sex, males/females, n | 79/84 | 45/38 | 34/46 | .180 |
| Age, median (range), y | 48.8 (19.3-59.9) | 48.0 (19.8-59.8) | 49.4 (19.3-59.9) | .827 |
| ECOG PS 2, % | 13 (8) | 6 (7.2) | 7 (8.8) | .945 |
| WBC count, median (range), × 109/L | 28.80 (1.20-300.00) | 29.70 (1.20-298.40) | 28.15 (1.30-300.00) | .480 |
| Platelet count, median (range), × 109/L | 49.00 (10.00-328.00) | 53.00 (11.00-328.00) | 46.50 (10.00-237.00) | .430 |
| Bone marrow blasts, median (range), % | 75.00 (0.00-100.00) | 75.00 (0.00-100.00) | 77.00 (0.00-100.00 | .817 |
| Peripheral blood blasts, median (range), % | 55.00 (0.00-99.00) | 56.50 (0.00-99.00) | 55.00 (0.00-98.00) | .594 |
| Prior myelodysplastic syndrome, n (%) | 4 (2.5) | 2 (2.4) | 2 (2.5) | >.99 |
| Extramedullary disease, n (%) | 31 (19.0) | 13 (15.7) | 18 (22.5) | .362 |
| NPM1, n (%) (n = 134) | ||||
| Mutated | 79 (59.0) | 45 (64.3) | 34 (53.1) | .256 |
| WT | 55 (41) | 25 (35.7) | 30 (46.9) | |
| Karyotype, n (%) (n = 135) | ||||
| CBF* | 20 (14.8) | 10 (15.6) | 10 (14.1) | .140 |
| Normal | 74 (54.8) | 39 (60.9) | 35 (49.3) | |
| Others† | 41 (30.4) | 15 (23.4) | 26 (36.6) | |
| CR after induction, n (%) | 105 (64.4) | 57 (68.6) | 48 (60) | .321 |
| Allogeneic HCT in first CR, n (%) | 62 (38.0) | 32 (38.6) | 30 (37.5) | >.99 |
| Midostaurin maintenance, n (%) | 58 (35.6) | 32 (38.6) | 26 (32.5) | .52 |
Mido, midostaurin; ECOG-PS, Eastern Cooperative Oncology Group performance status; WBC, white blood cell.
CBF rearrangements: inv(16), n = 11, and t(8;21), n = 9
Includes 8 patients with adverse and 7 with intermediate karyotype in the midostaurin arm, vs 7 and 19 patients, respectively, in the placebo arm.