Acute myeloid leukaemia (AML) is primarily a disease of older adults.
Standard commonly utilised lower-intensity therapies, such as azacitidine or decitabine, provide only limited responses with expected median survival of 9-10 months and complete remission (CR) / CR with incomplete count recovery (CRi) rates <40%.
We evaluated the efficacy of a combination regimen of azacitidine and venetoclax in treatment-naïve AML patients that were ineligible for intensive therapy.
In a Phase III randomised double-blinded multi-centre trial called “VIALE-A”, the efficacy of the combination regimen was compared to treatment with azacytidine plus a placebo.
In 431 internationally enrolled patients in the VIALE-A trial, the combination of azacitidine and venetoclax led to improved overall survival (14.7 vs 9.6 months), and improved response rates CR/CRi (66% vs 28%), compared to azacitidine alone.
In addition, the combination was associated with responses that occurred more quickly (median time to CR/CRi was only 1.3 months) and were more durable (lasting 1.5 years), and with increased incidence of transfusion independence (58% vs 34%).
In conclusion, this practice-changing multi-centre randomised Phase III trial establishes venetoclax and azacitidine as a new standard of care for older patients with AML.