VIALE-C compared the safety and efficacy of venetoclax or placebo plus low-dose cytarabine (+LDAC) in patients with untreated AML ineligible for intensive chemotherapy. Overall, 211 patients were enrolled (n = 143, venetoclax; n = 68, placebo). At the primary analysis, the study did not meet its primary endpoint of a statistically significant improvement in overall survival (OS), however, ~60% of patients had been on study for ≤6-months. Here, we present an additional 6-months of follow-up of VIALE-C (median follow-up 17.5 months; range 0.1-23.5). Median OS was (venetoclax +LDAC vs. placebo +LDAC) 8.4 vs. 4.1 months (HR = 0.70, 95% CI 0.50,0.99; P = 0.040); a 30% reduction in the risk of death with venetoclax. Complete response (CR)/CR with incomplete hematologic recovery (CRi) rates were 48.3% vs. 13.2%. Transfusion independence rates (RBC) were 43% vs.19% and median event-free survival was 4.9 vs. 2.1 months (HR = 0.61; 95% CI 0.44,0.84; P = 0.002). These results represent improved efficacy over the primary analysis. Incidence of grade ≥3 adverse events were similar between study arms and overall safety profiles were comparable to the primary analysis. These data support venetoclax +LDAC as a frontline treatment option for patients with AML ineligible for intensive chemotherapy.This trial was registered at www.clinicaltrials.gov as #NCT03069352.

AbbVie Inc North Chicago IL USA

Almazov National Medical Research Center Saint Petersburg Russia

Centre Hospitalier Le Mans Le Mans France

Chinese Academy of Medical Sciences Tianjin China

CIBERONC Instituto Carlos 3 Madrid Spain

CIUSSS EMTL Installation Maisonneuve Rosemont Montreal QC Canada

Eastern Health and Monash University Melbourne VIC Australia

Evaggelismos General Hospital Athens Greece

George Papanicolaou General Hospital Thessalonica Greece

Heartlands Hospital Birmingham UK

Hospital Universitario y Politécnico La Fe Valencia Spain

Hubertus Wald University Cancer Center University Medical Center Hamburg Eppendorf Hamburg Germany

National and Kapodistrian University of Athens Medical School Laiko General Hospital Athens Greece

Netcare Pretoria East Hospital Moreletapark Pretoria South Africa

Norton Cancer Institute Louisville KY USA

Seoul National University Hospital Seoul South Korea

The Alfred Hospital and Monash University Melbourne VIC Australia

The University of Texas MD Anderson Cancer Center Houston TX USA

University Hospital Královské Vinohrady and 3rd Faculty of Medicine Charles University Prague Czech Republic

University of Fukui Hospital Fukui Japan

University of Pittsburgh Medical Center Cancer Center Pittsburgh PA USA

Blood Cancer J. 2021 Oct 26;11(10):171. doi: 10.1038/s41408-021-00565-6. PubMed

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ClinicalTrials.gov
NCT03069352