Table 1.
Patient demographics and baseline characteristics
| Characteristic | Venetoclax-azacitidine (n = 24) | Placebo-azacitidine (n = 13) |
|---|---|---|
| Age | ||
| Median, years (range) | 77.5 (68–85) | 77.0 (67–86) |
| ≥ 75 years, n (%) | 19 (79.2) | 9 (69.2) |
| Male, n (%) | 14 (58.3) | 9 (69.2) |
| AML type, n (%) | ||
| De novo | 20 (83.3) | 10 (76.9) |
| Secondary | 4 (16.7) | 3 (23.1) |
| Secondary AML type, n/N (%) | ||
| Prior MDS or CMML | 3/4 (75.0) | 3/3 (100) |
| Treatment-related AML | 1/4 (25.0) | 0/3 |
| ECOG performance status, n (%) | ||
| 0 or 1 | 18 (75.0) | 8 (61.5) |
| 2 or 3 | 6 (25.0) | 5 (38.5) |
| Blast count, n (%) | ||
| < 30% | 7 (29.2) | 4 (30.8) |
| ≥ 30% to <50% | 7 (29.2) | 2 (15.4) |
| ≥ 50% | 10 (41.7) | 7 (53.8) |
| AML with myelodysplasia-related changes, n (%) | 9 (37.5) | 5 (38.5) |
| Cytogenetic risk, n (%) | ||
| Intermediate | 18 (75.0) | 9 (69.2) |
| Poor | 6 (25.0) | 4 (30.8) |
| Somatic mutations, n/N (%) | ||
| IDH1 or IDH2 | 6/21 (28.6) | 4/13 (30.8) |
| FLT3 | 2/23 (8.7) | 2/12 (16.7) |
| NPM1 | 0/14 | 6/9 (66.7) |
| TP53 | 2/14 (14.3) | 0/9 |
| Baseline Grade 3 or 4 cytopenias, n (%) | ||
| Neutropenia | 19 (79.2) | 12 (92.3) |
| Grade 3 | 3 (12.5) | 3 (23.1) |
| Grade 4 | 16 (66.7) | 9 (69.2) |
| Anemia | 8 (33.3) | 5 (38.5) |
| Thrombocytopenia | 7 (29.2) | 4 (30.8) |
| Baseline transfusion dependence,an (%) | ||
| RBCs | 2 (8.3) | 2 (15.4) |
| Platelets | 0 | 1 (7.7) |
| ≥2 reasons for ineligibility to receive intensive therapy,bn (%) | 8 (33.3) | 7 (53.8) |
aBaseline transfusion dependence defined as RBC or platelet transfusion within 8 weeks prior to the first dose of study drug or randomization.
bPatients could report more than 1 reason.
Abbreviations: AML, acute myeloid leukemia; CMML, chronic myelomonocytic leukemia; ECOG, Eastern Cooperative Oncology Group; FLT3, fms-like tyrosine kinase 3; IDH, isocitrate dehydrogenase; MDS, myelodysplastic syndrome; NPM1, nucleophosmin; RBC, red blood cell; TP53, tumor protein 53.