Table 1.
Summary of the efficacy results in the DACO-016 study (intention-to-treat population) [17]
| TC (n = 243) | Decitabine (n = 242) | HR (95% CI) | p-value | |
|---|---|---|---|---|
| Overall survival, median (95% CI), months | ||||
| Primary analysis | 5.0 (4.3–6.3) | 7.7 (6.2–9.2) | 0.85 (0.69–1.04) | 0.1079 |
| Censored for the use of DMT | 5.3 (4.3–6.7) | 8.5 (6.5–9.5) | 0.80 (0.64–0.99) | 0.0437 |
| Excluding subjects who received HMA | 4.5 (3.8–5.5) | 7.9 (6.0–9.3) | 0.77 (0.62–0.94) | 0.0109 |
| Analysis of mature data | 5.0 (4.3–6.3) | 7.7 (6.2–9.2) | 0.82 (0.68–0.99) | 0.0373 |
| Censored for the use of DMT | 5.3 (4.3–6.7) | 8.6 (6.5–9.5) | 0.79 (0.65–0.98) | 0.0295 |
| Excluding subjects who received HMA | 4.4 (3.7–5.5) | 7.9 (6.0–9.3) | 0.73 (0.60–0.88) | 0.0014 |
| EFS, PFS, RFS, median (95% CI), months | ||||
| EFS | 2.1 (1.9–2.8) | 3.5 (2.5–4.1) | 0.75 (0.62–0.91) | 0.0029 |
| PFS | 2.1 (1.9–3.1) | 3.7 (2.7–4.6) | 0.75 (0.62–0.91) | 0.0036 |
| RFS | ||||
| In patients with CR | 6.7 (2.9–13.4) | 8.3 (4.6–11.4) | – | |
| In patients with cytogenetic CR | – | – | – | |
| Clinical response, n (%) | ||||
| CR + CRp | 19 (7.8) | 43 (17.8) | 2.5 (1.40–4.78)a | 0.0011 |
| Cytogenetic CR | 3/41 (7.3) | 4/40 (10.0) | 1.4 (0.22–10.24)a | 0.712 |
| Time to and duration of response, median (95% CI), months | ||||
| Time to best response (CR or CRp) | 3.7 (2.8–4.6) | 4.3 (3.8–5.1) | ||
| Duration of response (CR or CRp) | 12.9 (4.2–NE) | 8.3 (6.2–11.4) | ||
CI confidence interval, CR complete remission, CRp CR with incomplete platelet recovery, DMT disease-modifying therapy, EFS event-free survival, HMA hypomethylating agent, HR hazard ratio, NE not estimable, OR odds ratio, PFS progression-free survival, RFS relapse-free survival, TC patient’s choice of treatment with physician’s advice
aValues are odds ratios (95% CI)