Statistical analysis
MINOR, DISEASE
For the primary endpoint, a sample size of 40 evaluable patients would provide at least 80% power to reject the null hypothesis of an ORR of <10% if the true ORR observed was 25%, which is ~20% higher than the estimated historical response rate [The primary endpoint was analyzed using the exact binomial test (one-sided) using SAS Version 9.4, with an overall significance level of 0.05, corresponding to an alpha of 0.1 for a two-sided test. Post-hoc analyses of the primary endpoint were also undertaken: (1) in patients with bone marrow involvement, (2) in patients with relapsed versus refractory disease, and (3) based on an updated INRC definition of ORR that includes minor response [Survival probabilities were estimated using Kaplan–Meier methods, and statistical comparisons were performed using the log-rank test (assuming proportional hazard). Three-year survival rates are shown as means and 95% confidence interval (CI). A post-hoc analysis was conducted to evaluate PFS and OS in patients stratified by disease status at study entry (relapsed versus refractory disease).Pharmacokinetic, pharmacodynamic and immunogenicity data are presented as mean and standard deviation (SD). Differences between groups were calculated using a Mann–Whitney rank sum test [