Study Design and Objectives

This was a single center; open-label, non-randomized phase II study. The study recruited patients between January 2015 and June 2017. The data cut-off was March 1, 2018. Primary study endpoints were safety and overall response rate (ORR) [ORR = CR, CR with incomplete recovery of peripheral counts (CRi), partial remission (PR), morphologic leukemia-free state (MLFS)(13), durable hematologic improvement (HI) (defined as improvement in one or more parameter of hemoglobin, platelets, neutrophils maintained ≥6 months)(14), captured as the best response achieved on study. Patients who achieved any of these responses were considered responders. Stable disease (SD) was defined as the absence of CR_, CRi, PR, MLFS, HI, after exposure to treatment for a duration considered sufficiently suitable to achieve a response to therapy (≥6 months), but with no evidence of progressive BM disease (defined as more than 50% increase in BM blast or ≥15% in blasts when blast at baseline <30%), no increase in transfusion requirements and/or hospital admissions, the absence of new or progressive extramedullary or CNS disease, and no clinical deterioration in terms of functional status, weight/appetite, level of energy or limiting side effects. Patients who did not achieve CR, CRi, PR, HI, SD were considered non-responders (NRs). Secondary endpoints included overall survival (OS), event free survival (EFS), and the duration of response (DOR).