In this phase 1-2 trial, an all-oral regimen of decitabine-cedazuridine plus venetoclax was evaluated for newly diagnosed acute myeloid leukemia in patients ineligible for intensive chemotherapy. The study found no drug-drug interactions and achieved a complete response in nearly half of the patients, with a median overall survival of 15.5 months in the pivotal phase 2b. Myelosuppressive effects were observed but managed with schedule adjustments.
Study
|
Phase 1-2, open-label, multicenter, nonrandomized trial [ASCERTAIN-V, NCT04657081] |
| Newly diagnosed AML patients >=75 years or ineligible for intensive induction chemotherapy |
| Oral decitabine-cedazuridine plus venetoclax (n=189 overall; n=101 in pivotal phase 2b)
|
Efficacy
|
CR: 47% [36-57] |
| CR+CR with incomplete hematologic recovery: 63% [53-73] |
| mOS: 15.5 mos [7.6-Not estimated]
|
Safety
|
Grade >=3 AEs: anemia (30%), neutropenia (26%), febrile neutropenia (25%), platelet count decreased (25%), thrombocytopenia (20%), neutrophil count decreased (20%), white-cell count decreased (15%) |
| AEs leading to treatment discontinuation: 9% |
| AEs leading to death: 16%
|
N Engl J Med 2026;394:2107-16
Roboz GJ, Zeidan AM, Mannis GN All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia
http://doi.org/10.1056/NEJMoa2510223
Reviewed by Ulas D. Bayraktar, MD on Aug 13, 2026
