Statistical analysis
toxicity, toxicities, AML, MDS/CMML
AML
The AML arm consisted of both phase 1 and phase 2 portions. The phase 1 portion was conducted using a standard “3 + 3” design and evaluated venetoclax at a dose a maximum dose of 200 mg (dose level 0) and 400 mg (dose level + 1). After completion of phase 1, up to 22 additional patients were to be enrolled in the phase 2 portion of the study (for a total of evaluable 28 patients treated at the recommended phase 2 dose). Interim monitoring rules for efficacy and toxicity were used throughout the phase 2 portion. The study was continuously monitored for efficacy and treatment-related toxicities using a Bayesian design [The MDS/CMML arm was opened after safety of the triplet regimen had been established in AML, and it consisted of a phase 2 portion only. Continuous monitoring for efficacy and treatment-related toxicities was performed as described above [Patient characteristics were summarized using the median (range) for continuous variables and the frequencies (percentages) for categorical variables. Remission duration, RFS and OS were calculated with Kaplan–Meier estimates, and survival estimates were compared with the log-rank test. The data cutoff for this analysis was December 1, 2022. The data analyses were carried out using GraphPad Prism 9. This study was registered at ClinicalTrials.gov (NCT03862157).