Table 2.
Response Rates by Treatment Arm: Primary and Final Analyses
| Primary Analysis | Final Analysis | |||
|---|---|---|---|---|
| P+LDAC (n = 125) | V+LDAC (n = 246) | P+LDAC (n = 222) | V+LDAC (n = 444) | |
| Patients who achieved CR, n (%) | 12 (9.6) | 23 (9.3) | 27 (12.2) | 67 (15.1) |
| Patients who achieved CRi, n (%) | 9 (7.2) | 39 (15.9) | 11 (5.0) | 56 (12.6) |
| Patients who achieved CR or CRi, n (%) | 21 (16.8) | 62 (25.2) | 38 (17.1) | 123 (27.7) |
| 95% CIa | 11.26–24.32 | 20.19–30.98 | 12.73–22.62 | 23.74–32.04 |
| OR V+LDAC vs P+LDACb | 1.66 | 1.88 | ||
| 95% CI | 0.95–2.89 | 1.24–2.83 | ||
| P | 0.071 | 0.002 | ||
| No response assessment/not evaluable for response, n (%) | 16 (12.8) | 95 (38.6) | 39 (17.6) | 158 (35.6) |
| Death ≤28 d after randomization, n (%) | 4 (3.2) | 27 (11.0) | 8 (3.6) | 52 (11.7) |
| Death >28 and ≤56 d after randomization, n (%) | 7 (5.6) | 30 (12.2) | 16 (7.2) | 50 (11.3) |
| Death >56 and ≤84 d after randomization, n (%) | 0 | 8 (3.3) | 2 (0.9) | 18 (4.1) |
| Median OS, mo (95% CI) | 6.5 (5.1–8.1) | 4.8 (3.8–6.4) | 6.5 (4.9–8.0) | 5.6 (4.5–6.8) |
| HR V+LDAC vs P+LDAC | 1.26 | 0.97 | ||
| 95% CI | (0.9–1.7) | (0.8–1.2) | ||
| P | 0.11 | 0.76 | ||
| Median EFS, mo (95% CI) | 3.1 (2.1–5.8) | 2.8 (2.3–3.8) | 2.8 (2.1–4.9) | 3.3 (2.6–4.2) |
| HR V+LDAC vs P+LDAC | 1.18 | 0.96 | ||
| 95% CI | (0.9, 1.6) | (0.8, 1.2) | ||
| P | 0.26 | 0.67 | ||
| Median RFS, mo (95% CI) | NE (3.7–NE) | 4.9 (3.6–13.4) | 18.7 (11.3–NE) | 13.1 (6.2–NE) |
| HR V+LDAC vs P+LDAC | 1.26 | 1.37 | ||
| 95% CI | (0.4–4.1) | (0.7–2.7) | ||
aWilson’s CI.
bOdds ratio derived from a Cochran–Mantel–Haenszel test stratified by baseline ECOG PS and type of AML. OR > 1 favors V+LDAC.
CI = confidence interval; CR = complete remission; CRi = complete remission with incomplete blood count recovery; EFS = event-free survival; HR = hazard ratio; NE = nonevaluable; OR = odds ratio; OS = overall survival; P+LDAC = placebo plus low-dose cytarabine; RFS = relapse-free survival; V+LDAC = volasertib plus low-dose cytarabine.