Efficacy of outcomes by assigned treatment
| Endpoint . | All patients . | Randomized . | P* . | |
|---|---|---|---|---|
| Placebo . | Zosuquidar . | |||
| No. of patients | 433 | 221 | 212 | — |
| Overall survival | ||||
| Median, mo | 8.3 | 9.4 | 7.2 | .281 |
| % at 2 y | 21 | 23 | 20 | |
| PFS | ||||
| Median, mo | 2.7 | 2.0 | 3.0 | .165 |
| Remissions (CR + CRi) | ||||
| No. (%) | 218 (50.3) | 108 (48.9) | 110 (51.9) | .583 |
| Treatment-related mortality | ||||
| % | 19.2 | 16.3 | 22.2 | .158 |
| Refractory disease | ||||
| % | 36.3 | 40.7 | 31.6 | .057 |
| Endpoint . | All patients . | Randomized . | P* . | |
|---|---|---|---|---|
| Placebo . | Zosuquidar . | |||
| No. of patients | 433 | 221 | 212 | — |
| Overall survival | ||||
| Median, mo | 8.3 | 9.4 | 7.2 | .281 |
| % at 2 y | 21 | 23 | 20 | |
| PFS | ||||
| Median, mo | 2.7 | 2.0 | 3.0 | .165 |
| Remissions (CR + CRi) | ||||
| No. (%) | 218 (50.3) | 108 (48.9) | 110 (51.9) | .583 |
| Treatment-related mortality | ||||
| % | 19.2 | 16.3 | 22.2 | .158 |
| Refractory disease | ||||
| % | 36.3 | 40.7 | 31.6 | .057 |
The total of CR, refractory disease, and treatment-related mortality exceeds 100% because 54 of the 157 patients with documented refractory disease died in the first 42 days.
— indicates not applicable; CRi, complete remission with incomplete platelet recovery; Treatment-related mortality, death from any cause within the first 42 days after enrollment.
Stratified log-rank test was used for OS and PFS comparisons, and Mantel-Haenszel test was used for other comparisons.