Journal: International journal of hematology
This retrospective study used the Hokkaido Leukemia Net database to examine how the approval of venetoclax changed real-world management and outcomes in Japanese patients aged 65–74 years with acute myeloid leukemia who were unfit for intensive chemotherapy.
Key points:
- Population: 101 patients, divided into a pre‑approval cohort (n=46) and post‑approval cohort (n=55); excluded acute promyelocytic leukemia and those who received intensive chemotherapy.
- Treatment shift: After approval, venetoclax plus azacitidine became the most common frontline regimen, used in 56% of patients.
- Disease risk profile: Patients receiving venetoclax plus azacitidine had a high‑risk profile, with 35.5% harboring TP53 mutations and/or complex karyotypes.
- Efficacy:
- Venetoclax + azacitidine: CR+CRi 64.5%; median overall survival (OS) 11.7 months.
- Reduced‑dose “7+3” (cytarabine + anthracycline): CR+CRi 64.5%; median OS 13.1 months.
- CAG (cytarabine + aclarubicin + G‑CSF): CR+CRi 37.5%; median OS 6.8 months.
- Azacitidine monotherapy: CR+CRi 12.5%; median OS 4.5 months.
- Early mortality (60 days from diagnosis):
- Venetoclax + azacitidine: 3.2%.
- Reduced‑dose 7+3: 12.9%.
- CAG: 26.7%.
- Azacitidine monotherapy: 18.8%.
Overall: The approval of venetoclax led to a major change in first‑line practice for older, unfit AML patients in this region, and venetoclax plus azacitidine provided response rates and survival comparable to reduced‑intensity 7+3, but with lower early mortality and better outcomes than CAG or azacitidine alone, despite a higher burden of adverse genetic features.