Patient characteristics and treatment history (ITT population)
| . | Quizartinib 30-mg group* (n = 38) . | Quizartinib 60-mg group† (n = 38) . | Total (N = 76) . |
|---|---|---|---|
| Secondary AML, n (%) | 3 (8) | 7 (18) | 10 (13) |
| Median age (range), y | 57 (19-77) | 53 (20-74) | 55 (19-77) |
| Male, n (%) | 22 (58) | 22 (58) | 44 (58) |
| Race, n (%) | |||
| White | 29 (76) | 30 (79) | 59 (78) |
| Black or African American | 1 (3) | 2 (5) | 3 (4) |
| Other or missing‡ | 8 (21) | 6 (16) | 14 (18) |
| Median weight (range), kg | 76.8 (40-116) | 75.1 (47-101) | 75.9 (40-116) |
| ECOG PS, n (%)§ | |||
| Grade 0 | 8 (21.1) | 7 (18.4) | 15 (19.7) |
| Grade 1 | 23 (60.5) | 24 (63.2) | 47 (61.8) |
| Grade 2 | 7 (18.4) | 4 (10.5) | 11 (14.5) |
| FLT3-ITD–mutated allelic ratio, n (%)|| | 37 (97) | 36 (95) | 73 (96) |
| >0 to < 10% | 2 (5) | 2 (5) | 4 (5) |
| ≥10% and ≤ 25% | 8 (21) | 4 (11) | 12 (16) |
| ≥25% and ≤ 50% | 20 (53) | 13 (34) | 33 (43) |
| >50% | 7 (18) | 17 (45) | 24 (32) |
| FLT3-ITD size, median (range) base pairs¶ | 51.0 (21-201) | 54.2 (18-114) | 54.0 (18-201) |
| Risk status with specific cytogenetic patterns, n (%)# | |||
| Favorable | 0 | 2 (5) | 2 (3) |
| Intermediate | 26 (68) | 25 (66) | 51 (67) |
| Unfavorable | 4 (11) | 3 (8) | 7 (9) |
| Unknown | 8 (21) | 7 (18) | 15 (20) |
| AML with recurrent genetic abnormalities | |||
| AML with mutated NPM1 | 8 (21) | 11 (29) | 19 (25) |
| AML with mutated CEBPA | 0 | 0 | 0 |
| Previous HSCT, n (%) | 9 (24) | 12 (32) | 21 (28) |
| Prior AML chemotherapy regimens, median (range)** | 3 (1-6) | 3 (1-9) | 3 (1-9) |
| Prior anthracycline treatment, n (%)** | 35 (92) | 33 (92) | 68 (92) |
| Refractory, n (%)** | 26 (68) | 26 (72) | 52 (70) |
| Relapsed, n (%)** | 12 (32) | 10 (28) | 22 (30) |
| Duration of best response (CR or PR) to last AML therapy, months, median (range)** | 5 (0.4-12) | 8 (1-18) | 6.5 (0.4-18) |
| Prior FLT3 therapy, n (%)**†† | 5 (13) | 6 (17) | 11 (15) |
| . | Quizartinib 30-mg group* (n = 38) . | Quizartinib 60-mg group† (n = 38) . | Total (N = 76) . |
|---|---|---|---|
| Secondary AML, n (%) | 3 (8) | 7 (18) | 10 (13) |
| Median age (range), y | 57 (19-77) | 53 (20-74) | 55 (19-77) |
| Male, n (%) | 22 (58) | 22 (58) | 44 (58) |
| Race, n (%) | |||
| White | 29 (76) | 30 (79) | 59 (78) |
| Black or African American | 1 (3) | 2 (5) | 3 (4) |
| Other or missing‡ | 8 (21) | 6 (16) | 14 (18) |
| Median weight (range), kg | 76.8 (40-116) | 75.1 (47-101) | 75.9 (40-116) |
| ECOG PS, n (%)§ | |||
| Grade 0 | 8 (21.1) | 7 (18.4) | 15 (19.7) |
| Grade 1 | 23 (60.5) | 24 (63.2) | 47 (61.8) |
| Grade 2 | 7 (18.4) | 4 (10.5) | 11 (14.5) |
| FLT3-ITD–mutated allelic ratio, n (%)|| | 37 (97) | 36 (95) | 73 (96) |
| >0 to < 10% | 2 (5) | 2 (5) | 4 (5) |
| ≥10% and ≤ 25% | 8 (21) | 4 (11) | 12 (16) |
| ≥25% and ≤ 50% | 20 (53) | 13 (34) | 33 (43) |
| >50% | 7 (18) | 17 (45) | 24 (32) |
| FLT3-ITD size, median (range) base pairs¶ | 51.0 (21-201) | 54.2 (18-114) | 54.0 (18-201) |
| Risk status with specific cytogenetic patterns, n (%)# | |||
| Favorable | 0 | 2 (5) | 2 (3) |
| Intermediate | 26 (68) | 25 (66) | 51 (67) |
| Unfavorable | 4 (11) | 3 (8) | 7 (9) |
| Unknown | 8 (21) | 7 (18) | 15 (20) |
| AML with recurrent genetic abnormalities | |||
| AML with mutated NPM1 | 8 (21) | 11 (29) | 19 (25) |
| AML with mutated CEBPA | 0 | 0 | 0 |
| Previous HSCT, n (%) | 9 (24) | 12 (32) | 21 (28) |
| Prior AML chemotherapy regimens, median (range)** | 3 (1-6) | 3 (1-9) | 3 (1-9) |
| Prior anthracycline treatment, n (%)** | 35 (92) | 33 (92) | 68 (92) |
| Refractory, n (%)** | 26 (68) | 26 (72) | 52 (70) |
| Relapsed, n (%)** | 12 (32) | 10 (28) | 22 (30) |
| Duration of best response (CR or PR) to last AML therapy, months, median (range)** | 5 (0.4-12) | 8 (1-18) | 6.5 (0.4-18) |
| Prior FLT3 therapy, n (%)**†† | 5 (13) | 6 (17) | 11 (15) |
Quizartinib 30 mg and 60 mg are equivalent to 26.5 mg and 53 mg free base, respectively.
ECOG PS, Eastern Cooperative Oncology Group performance status.
30-mg starting dose with permitted escalation to 60 mg for lack of or loss of initial response.
60-mg starting dose with permitted escalation to 90 mg for lack of or loss of initial response.
Ethnicity was not collected from patients in France per local regulations.
At screening, all patients had ECOG performance status ≤2 per eligibility criteria. The baseline ECOG scores reflects data collected immediately before first on-study treatment dose, and could have changed from the screening ECOG scores.
FLT3-ITD-mutated allele was not detectable (below the assay’s limit of detection) in 1 patient each in the 30-mg group and the 60-mg group. In addition, value is missing for 1 patient in the 60-mg group, who was randomized, specimen was not received at central laboratory, and patient did not receive quizartinib.
Total number of patients, N (%) = 30-mg group, 37 (97); 60-mg group, 36 (95); total, 73 (96).
Cytogenetic information based on available data.
Total number of patients (Safety analysis set), N = 30-mg group, 38; 60-mg group, 36; total, 74.
10 patients received sorafenib and 1 patient received both sorafenib and midostaurin.