All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia
Fully oral leukemia treatment eliminates hospital visits, allowing elderly patients ineligible for chemotherapy to receive effective cancer therapy at home.
This NEJM phase 1-2 trial (n=189) demonstrates that fully oral decitabine-cedazuridine combined with oral venetoclax achieves a 47% complete response and 63% composite CR rate in newly diagnosed AML patients ineligible for intensive chemotherapy, with no pharmacokinetic drug-drug interactions. The all-oral formulation eliminates parenteral azacitidine or IV decitabine administration, enabling outpatient treatment for this elderly/unfit population with high unmet need.
What the study was
- Study design
- Phase 1-2 open-label multicenter nonrandomized trial
- Population
- Newly diagnosed AML patients ≥75 years or ineligible for intensive chemotherapy
- Sample size
- 189
- Category
- Treatment Innovation
- Maturity
- Potentially Practice-Changing
- Journal
- N Engl J Med
Why it surfaced
NEJM publication of phase 1-2 multicenter trial; all-oral regimen for AML in ineligible/elderly patients eliminates parenteral burden. CR 47%, CRc 63%, mOS 15.5 months. Trial funded by Taiho Oncology (ASCERTAIN-V, NCT04657081); decitabine-cedazuridine already approved in Europe. Near-term implementation pathway exists.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.