Patient demographics and disease characteristics
| Characteristic . | Venetoclax 400 mg (14 d/cycle) + azacitidine 75 mg/m2 (7 d/cycle) N = 107 . |
|---|---|
| Age, median (range), y | 68 (26-87) |
| <65, n (%) | 35 (32.7) |
| ≥65, n (%) | 72 (67.3) |
| ≥75, n (%) | 27 (25.2) |
| Male, n (%) | 74 (69.2) |
| Race, n (%) | |
| White | 98 (92.5) |
| Black or African American | 1 (0.9) |
| Asian | 7 (6.6) |
| Missing | 1 (0.9) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 4 (3.8) |
| Not Hispanic or Latino | 102 (96.2) |
| Missing | 1 (0.9) |
| ECOG PS, n (%) | |
| 0 | 56 (52.8) |
| 1 | 43 (40.6) |
| 2 | 7 (6.6) |
| Missing | 1 (0.9) |
| Time to treatment from MDS diagnosis, median (range), d | 61 (−3 to 2682)∗ |
| IPSS-R prognostic score, n (%)† | |
| Low | 1 (0.9) |
| Intermediate | 14 (13.1) |
| High | 40 (37.4) |
| Very high | 52 (48.6) |
| BM blast category, n (%) | |
| ≤5% | 11 (10.3) |
| >5 to ≤10% | 32 (29.9) |
| >10% | 64 (59.8) |
| BM blast count, median (range), % | 11.0 (1-19.5) |
| Baseline transfusion dependence, n (%) | |
| RBC | 56 (52.3) |
| Platelet | 18 (16.8) |
| RBC or platelet | 59 (55.1) |
| Eligible for SCT at study entry according to treating investigator, n (%) | 34 (31.8) |
| IPSS-R cytogenetic risk, n (%) | |
| Very good | 1 (0.9) |
| Good | 38 (35.5) |
| Intermediate | 35 (32.7) |
| Poor | 7 (6.5) |
| Very poor | 26 (24.3) |
| Baseline mutations, n (%)‡ | |
| No mutations detected | 21 (25.3) |
| ASXL1 | 29 (34.5) |
| TP53 | 20 (23.8) |
| SRSF2 | 19 (22.6) |
| RUNX1 | 18 (21.4) |
| TET2 | 15 (17.9) |
| Characteristic . | Venetoclax 400 mg (14 d/cycle) + azacitidine 75 mg/m2 (7 d/cycle) N = 107 . |
|---|---|
| Age, median (range), y | 68 (26-87) |
| <65, n (%) | 35 (32.7) |
| ≥65, n (%) | 72 (67.3) |
| ≥75, n (%) | 27 (25.2) |
| Male, n (%) | 74 (69.2) |
| Race, n (%) | |
| White | 98 (92.5) |
| Black or African American | 1 (0.9) |
| Asian | 7 (6.6) |
| Missing | 1 (0.9) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 4 (3.8) |
| Not Hispanic or Latino | 102 (96.2) |
| Missing | 1 (0.9) |
| ECOG PS, n (%) | |
| 0 | 56 (52.8) |
| 1 | 43 (40.6) |
| 2 | 7 (6.6) |
| Missing | 1 (0.9) |
| Time to treatment from MDS diagnosis, median (range), d | 61 (−3 to 2682)∗ |
| IPSS-R prognostic score, n (%)† | |
| Low | 1 (0.9) |
| Intermediate | 14 (13.1) |
| High | 40 (37.4) |
| Very high | 52 (48.6) |
| BM blast category, n (%) | |
| ≤5% | 11 (10.3) |
| >5 to ≤10% | 32 (29.9) |
| >10% | 64 (59.8) |
| BM blast count, median (range), % | 11.0 (1-19.5) |
| Baseline transfusion dependence, n (%) | |
| RBC | 56 (52.3) |
| Platelet | 18 (16.8) |
| RBC or platelet | 59 (55.1) |
| Eligible for SCT at study entry according to treating investigator, n (%) | 34 (31.8) |
| IPSS-R cytogenetic risk, n (%) | |
| Very good | 1 (0.9) |
| Good | 38 (35.5) |
| Intermediate | 35 (32.7) |
| Poor | 7 (6.5) |
| Very poor | 26 (24.3) |
| Baseline mutations, n (%)‡ | |
| No mutations detected | 21 (25.3) |
| ASXL1 | 29 (34.5) |
| TP53 | 20 (23.8) |
| SRSF2 | 19 (22.6) |
| RUNX1 | 18 (21.4) |
| TET2 | 15 (17.9) |
A protocol deviation resulted in enrollment of 1 patient without a documented diagnosis of MDS. A diagnosis was documented 3 days after treatment initiation.
IPSS-R risk groups overall score is calculated as the blast score + cytogenetics score + hemoglobin score + platelet score + absolute neutrophil count score. Overall risk score for low is >1.5 to 3.0, intermediate is >3.0 to 4.5, high is >4.5 to 6.0, and very high is >6.0.
Samples with sufficient material to be analyzed for mutations were available from 84 patients and were tested using a panel of 37 genes. Additional detected mutations included SF3B1 (9.5%), DNMT3A (8.3%), EZH2 (7.1%), IDH2 (6.0%), IDH1 (3.6%), and BCORL1 (1.2%).