Statistics
toxicity
ADVERSE EVENTS, ADVERSE EVENT
This was a phase II trial designed using a Simon design with an interim analysis to evaluate efficacy. Efficacy (success) was defined as a complete response (CR or CR incomplete (i)). All patients meeting the eligibility criteria who signed a consent form and began treatment were deemed evaluable for toxicity. Similar studies showed a CR rate ranging from 20 to 47% in this patient population.The primary endpoint was the proportion of successes, which was estimated by the number of successes divided by the total number of evaluable patients. Secondary endpoints of duration of response (CR or CRi) and overall survival (OS) were evaluated using Kaplan–Meier techniques; the frequency of proceeding to allogeneic stem cell transplant after achieving response and adverse events (AEs), as evaluated using reported the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0, were presented using descriptive statistics.