Outcomes
toxicity, AML, AML transformation, MDS/CMML
RESIDUAL DISEASE, REMISSION, AML
The primary endpoint of the phase 1 portion of the study (AML only) was the dose-limiting toxicity (DLT) of the combination regimen. The primary endpoint for the phase 2 portion of the AML cohort was the complete remission (CR) or CR with incomplete hematological recovery (CRi) rate, and the primary endpoint for the phase 2 portion of the MDS/CMML cohort was the composite rate of CR, marrow CR (mCR), partial remission (PR) and hematologic improvement (HI). Secondary endpoints included the CR rate, composite rate of CR, CRi, PR, and morphologic leukemia-free state (MLFS) (AML cohort only), measurable residual disease (MRD) negativity rate by flow cytometry (AML cohort only), time to AML transformation (MDS/CMML cohort only), relapse-free survival (RFS), overall survival (OS) and safety of the regimen.AML responses were defined according to the European LeukemiaNet 2017 guidelines [MRD assessment was performed on fresh bone marrow aspiration samples using 8-color multiparameter flow cytometry as described previously [