Key efficacy outcomes (ITT population)
| . | Quizartinib 30-mg group* (n = 38) . | Quizartinib 60-mg group† (n = 38) . | Total (N = 76) . |
|---|---|---|---|
| Overall response (CRc+PR), n (%) | 23 (60.5) | 27 (71.1) | 50 (65.8) |
| CRc (CR+CRp+CRi), n (%) | 18 (47.4) | 18 (47.4) | 36 (47.4) |
| CR, n (%; 95% CI) | 2 (5.3; 0.6-17.7) | 1 (2.6; 0.1-13.8) | 3 (3.9; 0.8-11.1) |
| CRp, n (%; 95% CI) | 0 | 2 (5.3; 0.6-17.7) | 2 (2.6; 0.3-9.2) |
| CRi, n (%; 95% CI) | 16 (42.1; 26.3-59.2) | 15 (39.5; 24.0-56.6) | 31 (40.8; 29.6-52.7) |
| PR, n (%; 95% CI) | 5 (13.2; 4.4-28.1) | 9 (23.7; 11.4-40.2) | 14 (18.4; 10.5-29.0) |
| Median duration of CRc (95% CI), wk‡ | 4.2 (2.1-9.7) | 9.1 (4.1-22.3) | 5.4 (4.1-11.9) |
| Median time to CRc (95% CI), wk | 4.4 (4.1-7.7) | 4.6 (4.1-8.0) | 4.5 (4.3-6.6) |
| Bridge to HSCT transplant rate, n (%) | 12 (31.6) | 16 (42.1) | 28 (36.8) |
| Median OS (95% CI), wk‡,§ | 20.9 (17.7-25.3) | 27.3 (17.3-34.9) | 22.6 (19.9-28.3) |
| Deaths, n (%) | 36 (94.7) | 30 (78.9) | 66 (86.8) |
| Censored, n (%) | 2 (5.3) | 8 (21.1) | 10 (13.2) |
| Median EFS (95% CI), wk | 12.0 (8.3-16.1) | 13.7 (9.7-26.1) | 12.3 (9.7-16.1) |
| Median LFS (95% CI), wk | 4.1 (2.1-9.7) | 9.1 (4.0-22.3) | 5.3 (4.1-11.9) |
| . | Quizartinib 30-mg group* (n = 38) . | Quizartinib 60-mg group† (n = 38) . | Total (N = 76) . |
|---|---|---|---|
| Overall response (CRc+PR), n (%) | 23 (60.5) | 27 (71.1) | 50 (65.8) |
| CRc (CR+CRp+CRi), n (%) | 18 (47.4) | 18 (47.4) | 36 (47.4) |
| CR, n (%; 95% CI) | 2 (5.3; 0.6-17.7) | 1 (2.6; 0.1-13.8) | 3 (3.9; 0.8-11.1) |
| CRp, n (%; 95% CI) | 0 | 2 (5.3; 0.6-17.7) | 2 (2.6; 0.3-9.2) |
| CRi, n (%; 95% CI) | 16 (42.1; 26.3-59.2) | 15 (39.5; 24.0-56.6) | 31 (40.8; 29.6-52.7) |
| PR, n (%; 95% CI) | 5 (13.2; 4.4-28.1) | 9 (23.7; 11.4-40.2) | 14 (18.4; 10.5-29.0) |
| Median duration of CRc (95% CI), wk‡ | 4.2 (2.1-9.7) | 9.1 (4.1-22.3) | 5.4 (4.1-11.9) |
| Median time to CRc (95% CI), wk | 4.4 (4.1-7.7) | 4.6 (4.1-8.0) | 4.5 (4.3-6.6) |
| Bridge to HSCT transplant rate, n (%) | 12 (31.6) | 16 (42.1) | 28 (36.8) |
| Median OS (95% CI), wk‡,§ | 20.9 (17.7-25.3) | 27.3 (17.3-34.9) | 22.6 (19.9-28.3) |
| Deaths, n (%) | 36 (94.7) | 30 (78.9) | 66 (86.8) |
| Censored, n (%) | 2 (5.3) | 8 (21.1) | 10 (13.2) |
| Median EFS (95% CI), wk | 12.0 (8.3-16.1) | 13.7 (9.7-26.1) | 12.3 (9.7-16.1) |
| Median LFS (95% CI), wk | 4.1 (2.1-9.7) | 9.1 (4.0-22.3) | 5.3 (4.1-11.9) |
Quizartinib 30 mg and 60 mg are equivalent to 26.5 mg and 53 mg free base, respectively. Responses were assessed using modified Cheson criteria as described in supplemental Appendix 1.
EFS, event-free survival; and LFS, leukemia-free survival.
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.
From Kaplan-Meier analysis.
Reflects median OS until the time of database lock (study termination).