This single-centre phase 2 study demonstrates that oral ASTX727 plus venetoclax is effective and tolerable in older or unfit patients with acute myeloid leukemia, inducing rapid remissions and comparable response rates to standard venetoclax plus hypomethylating agent regimens. Myelosuppression and infectious complications were common but manageable, with overall survival outcomes aligned with adverse risk patient characteristics.
Study
|
Single-centre, phase 2 study [NCT04746235] |
| Newly diagnosed AML ineligible for intensive chemotherapy (n=49) or relapsed/refractory AML (n=13) |
| Oral decitabine/cedazuridine plus venetoclax
|
Efficacy
|
ORR: 64% frontline (95% CI 49-77) vs 46% relapsed/refractory (19-75) |
| CR+CRi rate: 57% frontline vs 46% relapsed/refractory |
| mOS: 11.5 mos frontline (95% CI 9.1-16.6) vs 7.2 mos relapsed/refractory (6.3-NA) |
| 1-yr OS: 49% frontline (36-68) vs 18% relapsed/refractory (5-64) |
| 2-yr OS: 18% frontline (6-84) vs 18% relapsed/refractory (5-64)
|
Safety
|
Grade >=3 AE: febrile neutropenia (18%), pneumonia (13%), respiratory failure (8%), bacteraemia (6%), sepsis (6%) |
| 3 treatment-related deaths in remission: sepsis, gastrointestinal hemorrhage, respiratory failure
|
Lancet Haematol. 2024;11(4):e276.
http://doi.org/10.1016/s2352-3026(24)00033-4
Reviewed by Ulas D. Bayraktar, MD on May 15, 2026
