Venetoclax Shows Promise as AML Treatment Option

Key Takeaways

  • Venetoclax treatment combinations show benefit as first-line and follow-up therapy for adults with acute myeloid leukemia (AML), as well as patients who relapse after receiving allogeneic stem cell transplants.

Why This Matters

  • Failure of conventional induction chemotherapy and posttransplant relapse are key challenges in AML, with limited therapeutic options, particularly for the elderly and patients with comorbidities.

  • Venetoclax combinations are currently approved in the US only for first-line treatment of AML in adults aged 75 years or older who are ineligible for intensive induction chemotherapy.

  • The new findings suggest broader benefits, perhaps even an improved chance at a second transplant if the first fails.

Study Design

  • The study was a single-center review of 56 adults (mean age, 66.5 years) who were either treatment naive or had relapsed or recurrent disease. They were treated with venetoclax combined with either hypomethylating agents or low-dose cytarabine in 28-day cycles.

  • Eighteen patients received first-line therapy, 23 were treated after failure with conventional induction chemotherapy, and 15 were treated after relapse from stem cell transplant. Median follow-up was 4.7 months.

Key Results

  • In the first-line setting, median overall survival from the start of venetoclax treatment was 13.3 months, and the overall response rate was 61.1%.

  • Overall survival was 5 months, and overall response rate was 52.2% in subsequent lines.

  • Following transplant relapse, overall survival was 4 months, and overall response rate was 42.8%. Among patients who underwent a subsequent transplant, median overall survival was 11.5 months from the start of venetoclax treatment.

  • Nine patients who carried FLT3-ITD mutations had lower overall survival rates with a median of 3.4 months compared to 10.4 months for patients without FLT3-ITD mutations. Patients with NPM1 or IDH1/2 mutations showed a survival advantage.

  • Although not statistically significant, venetoclax doses higher than 100 mg were associated with a greater likelihood of complete remission.


  • The study was retrospective and was carried out at a single academic center, with only a small number of patients and no comparator group.


  • There was no commercial funding, and the investigators had no conflicts of interest.

This is a summary of preprint research led by Ulf Schnetzke, MD, Jena University Hospital, Germany, and was provided to you by Medscape. The study has not yet been peer reviewed. The full text is published on Research Square.

M. Alexander Otto is a physician assistant with a master’s degree in medical science. He is an award-winning medical journalist who has worked for several major news outlets before joining Medscape. He is an MIT Knight Science Journalism fellow. Email: [email protected]

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