Table 1 Summary of efficacy and durability outcomes in all phase 2 treated patients (N = 55) and pooled phase 1 and 2 treated patients (N = 78) by age and baseline bone marrow blast percentage
N | Overall CR rate, n (%) | CR, n (%) | CRi, n (%) | BFBM, n (%) | No response, n (%) | Median DOR, mo. (95% CI) | Median RFS, mo. (95% CI) | Median OS, mo. (95% CI) | |
|---|---|---|---|---|---|---|---|---|---|
Phase 2 treated | 55 | 39 (70.9) | 31 (56.4) | 8 (14.5) | 4 (7.3) | 9 (16.4) | 14.6 (9.4–NE) | 11.6 (2.7–20.5) | 25.4 (16.2–NE) |
Age, years | |||||||||
18–25 | 12 | 8 (66.7) | 7 (58.3) | 1 (8.3) | 1 (8.3) | 1 (8.3) | 16.6 (14.6–NE) | 15.5 (0.0–NE) | NR (0.6–NE) |
18–39 | 26 | 16 (61.5) | 14 (53.8) | 2 (7.7) | 2 (7.7) | 5 (19.2) | 18.6 (12.8–NE) | 14.2 (0.0–20.5) | 25.4 (9.5–NE) |
40–59 | 20 | 14 (70.0) | 11 (55.0) | 3 (15.0) | 2 (10.0) | 4 (20.0) | 10.3 (1.3–NE) | 11.6 (0.0–NE) | 26.0 (7.6–NE) |
≥ 60 | 9 | 9 (100) | 6 (66.7) | 3 (33.3) | 0 | 0 | 9.4 (1.8–NE) | 11.7 (2.8–NE) | NR (12.2–NE) |
Baseline BM blasts* | |||||||||
≤ 5% | 5 | 4 (80) | 4 (80) | 0 | 1 (20) | 0 | 14.4 (1.3–NE) | 5.6 (0.0–NE) | NR (8.8–NE) |
> 5%–25% | 10 | 9 (90) | 7 (70) | 2 (20) | 0 | 1 (10) | 23.6 (1.8–NE) | 25.4 (0.0–NE) | 25.4 (8.3–NE) |
> 25%–50% | 11 | 10 (90.9) | 9 (81.8) | 1 (9.1) | 0 | 0 | 18.6 (9.4–NE) | 20.5 (10.3–NE) | NR (9.0–NE) |
> 50%–75% | 10 | 8 (80) | 5 (50) | 3 (30) | 1 (10) | 1 (10) | 20.0 (1.0–NE) | 6.1 (0.0–NE) | NR (2.1–NE) |
> 75%–100% | 19 | 8 (42.1) | 6 (31.6) | 2 (10.5) | 2 (10.5) | 7 (36.8) | 9.6 (0.8–12.8) | 0.0 (0.0–11.6) | 14.2 (2.2–NE) |
Phase 1 and 2 | 78 | 57 (73.1) | 47 (60.3) | 10 (12.8) | 6 (7.7) | 12 (15.4) | 18.6 (9.6–NE) | 11.7 (6.1–20.5) | 25.4 (16.2–NE) |
Age, years | |||||||||
18–25 | 15 | 11 (73.3) | 9 (60.0) | 2 (13.3) | 1 (6.7) | 1 (6.7) | 14.6 (0.7–NE) | 15.5 (0.0–NE) | 23.2 (9.0–NE) |
18–39 | 36 | 25 (69.4) | 21 (58.3) | 4 (11.1) | 2 (5.6) | 6 (16.7) | 18.6 (12.8–NE) | 14.2 (2.3–NE) | 23.2 (14.2–NE) |
40–59 | 27 | 19 (70.4) | 16 (59.3) | 3 (11.1) | 3 (11.1) | 5 (18.5) | 20.0 (4.7–NE) | 7.7 (0.0–22.1) | 26.0 (8.3–NE) |
≥ 60 | 15 | 13 (86.7) | 10 (66.7) | 3 (20.0) | 1 (6.7) | 1 (6.7) | NR (1.8–NE) | 14.4 (2.8–NE) | 47.0 (12.2–NE) |
Baseline BM blasts* | |||||||||
0–5% | 8 | 6 (75.0) | 6 (75.0) | 0 | 1 (12.5) | 1 (12.5) | 4.9 (1.3–NE) | 5.6 (0.0–NE) | 26.0 (2.2–NE) |
> 5%–25% | 14 | 12 (85.7) | 10 (71.4) | 2 (14.3) | 1 (7.1) | 1 (7.1) | 23.6 (1.8–NE) | 25.4 (2.8–NE) | 25.4 (21.9–NE) |
> 25%–50% | 12 | 10 (83.3) | 9 (75.0) | 1 (8.3) | 1 (8.3) | 0 | 18.6 (9.4–NE) | 20.5 (0.0–NE) | NR (1.7–NE) |
> 50%–75% | 14 | 12 (85.7) | 8 (57.1) | 4 (28.6) | 1 (7.1) | 1 (7.1) | 20.0 (5.2–NE) | 22.1 (1.8–NE) | NR (3.2–NE) |
> 75%–100% | 30 | 17 (56.7) | 14 (46.7) | 3 (10.0) | 2 (6.7) | 9 (30.0) | 10.3 (1.3–NE) | 2.7 (0.0–11.7) | 16.1 (9.5–NE) |