Venetoclax plus LDAC for patients with untreated AML ineligible for intensive chemotherapy: phase 3 randomized placebo-controlled trial.

Wei, Andrew Henry, Montesinos, Pau, Ivanov, Vladimir, DiNardo, Courtney D, Novak, Jan, Laribi, Kamel, Kim, Inho, Stevens, Don, Fiedler, Walter, Pagoni, Maria, Samoilova, Olga, Hu, Yu, Anagnostopoulos, Achilles, Bergeron, Julie, Hou, Jing-Zhou, Murthy, Vidhya, Yamauchi, Takahiro, McDonald, Andrew Bruce, Chyla, Brenda, Gopalakrishnan, Sathej, Jiang, Qi, Mendes, Wellington L, Hayslip, John and Panayiotidis, Panayiotis (2020) Venetoclax plus LDAC for patients with untreated AML ineligible for intensive chemotherapy: phase 3 randomized placebo-controlled trial. Blood. ISSN 1528-0020.

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Effective treatment options are limited for patients with acute myeloid leukemia (AML) who cannot tolerate intensive chemotherapy.


Adults ≥18 years with newly diagnosed AML ineligible for intensive chemotherapy were enrolled in this international Phase 3 randomized, double-blind, placebo-controlled trial. Patients (N=211) were randomized 2:1 to either: venetoclax (N=143) or placebo (N=68) in 28-day cycles, plus low-dose cytarabine (LDAC) on days 1-10. The primary endpoint was overall survival (OS); secondary endpoints included response rates, transfusion independence, and event-free survival.


Median age was 76 years (range 36-93), 38% had secondary AML, and 20% had prior hypomethylating agent (HMA) treatment. The planned primary analysis showed that the venetoclax arm provided a benefit of 25% reduction in the risk-of-death over the LDAC-alone arm (hazard ratio [HR] 0.75 [95% CI 0.52-1.07], p=0.11), although it was not statistically significant; with median OS of 7.2 months and 4.1 months, respectively. An unplanned analysis with an additional 6 months of follow up demonstrated a median OS of 8.4 months for the venetoclax arm (HR 0.70; 95% CI 0.50-0.98; p=0.04). The CR/CRi rates were 48% and 13% for the Venetoclax plus LDAC arm and LDAC-alone arm, respectively. Key grade ≥3 adverse events (Ven vs. LDAC-alone) were febrile neutropenia (32% vs 29%), neutropenia (47% vs. 16%), and thrombocytopenia (45% vs 37%).


Venetoclax plus LDAC demonstrates a clinically meaningful improvement in remission rates and OS compared to LDAC alone, in the context of a manageable safety profile. These results confirm venetoclax plus LDAC is an important frontline AML treatment option for patients unfit for intensive chemotherapy.

Item Type: Article
Subjects: QV Pharmacology
QW Microbiology. Immunology
QY Clinical pathology
WH Haemic and lymphatic systems. Haematology
Divisions: Planned IP Care > Oncology and Clinical Haematology
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Depositing User: Jamie Edgar
Date Deposited: 03 Apr 2020 13:32
Last Modified: 03 Apr 2020 13:32

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