Efficacy assessment
extramedullary leukemia, AML, SD
DISEASE, REMISSION, AML
Bone marrow and peripheral blood results were used to assess the antitumor effect of milademetan. AML response criteria were defined as shown in Supplementary Table 1. A morphologic leukemia-free state (MLFS) was defined as bone marrow blasts < 5% in the absence of blasts with Auer rods and the absence of extramedullary leukemia. The presence/absence of hematological recovery or blood transfusion was not considered. Stable disease (SD) was defined as an absence of CR, CR with incomplete hematologic recovery (CRi) or partial hematologic recovery (CRh), partial remission (PR), or MLFS, provided that the criteria for the progressive disease were not met. SD had to last at least 3 months (assessed as SD more than thrice in a row) after initiating milademetan treatment. SD < 3 months was deemed not applicable. The best-measured response was defined as the best overall response (all time points after the start of study treatment until the end of treatment). The composite CR (CRc) duration was defined as the time between when the criteria for CRc (CR + CRi + CRh) were first met and when a relapse was first confirmed.