What is the treatment protocol for Acute Myeloid Leukemia (AML) using Vidaza (Azacitidine) and Venetoclax?

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Last updated: March 12, 2025View editorial policy

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From the Guidelines

The standard treatment protocol for Acute Myeloid Leukemia (AML) using Vidaza (azacitidine) and Venetoclax combines a hypomethylating agent with a BCL-2 inhibitor, with azacitidine administered at 75 mg/m² subcutaneously or intravenously for 7 days in a 28-day cycle, and Venetoclax given orally with a 3-day ramp-up schedule (100 mg day 1,200 mg day 2,400 mg day 3 and beyond) to reduce tumor lysis syndrome risk, as recommended by the most recent guidelines 1.

Treatment Protocol

The treatment protocol typically involves:

  • Azacitidine administration at 75 mg/m² subcutaneously or intravenously for 7 days in a 28-day cycle
  • Venetoclax given orally with a 3-day ramp-up schedule (100 mg day 1,200 mg day 2,400 mg day 3 and beyond)
  • The Venetoclax dose is usually maintained at 400 mg daily for days 1-28 in the first cycle, then days 1-21 or 1-28 in subsequent cycles depending on response and toxicity
  • Treatment generally continues until disease progression or unacceptable toxicity

Monitoring and Adjustments

Patients require close monitoring for:

  • Neutropenia
  • Infections
  • Tumor lysis syndrome
  • Prophylactic antimicrobials are often recommended
  • Dose adjustments may be necessary based on cytopenias, with possible Venetoclax interruption if severe neutropenia occurs, as suggested by recent studies 1

Rationale

This regimen has become preferred for older AML patients or those unfit for intensive chemotherapy because it targets both epigenetic dysregulation through azacitidine and blocks the anti-apoptotic BCL-2 protein with Venetoclax, effectively inducing leukemic cell death while being better tolerated than traditional intensive chemotherapy, as supported by recent guidelines and studies 1.

Key Considerations

  • The combination of azacitidine and Venetoclax has been shown to be effective in achieving complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) in older patients with AML, with a CR/CRi rate of 61% (95% CI, 47.6–74.0) 1
  • The treatment protocol should be individualized based on patient factors, such as age, comorbidities, and performance status
  • Close monitoring and adjustments to the treatment protocol are necessary to minimize the risk of adverse events and optimize treatment outcomes.

From the FDA Drug Label

Azacitidine was administered subcutaneously at a dose of 75 mg/m 2 daily for 7 consecutive days every 28 days (which constituted one cycle of therapy). The FDA drug label does not answer the question about the treatment protocol for Acute Myeloid Leukemia (AML) using Vidaza (Azacitidine) and Venetoclax, as it only discusses the use of azacitidine alone.

From the Research

Treatment Protocol for Acute Myeloid Leukemia (AML) using Vidaza (Azacitidine) and Venetoclax

  • The treatment of acute myeloid leukemia (AML) in older or unfit patients typically involves a regimen of venetoclax plus azacitidine (ven/aza) 2.
  • Venetoclax, a highly selective BCL-2 inhibitor, combined with hypomethylating agents (HMAs) azacitidine or decitabine, is approved for the treatment of newly diagnosed acute myeloid leukemia (ND AML) in patients who are ineligible to receive intensive chemotherapy 3.
  • The combination of venetoclax and azacitidine has changed the paradigm of treatment of newly diagnosed (ND) acute myeloid leukemia (AML) patients ineligible for intensive chemotherapy 4.

Dosage and Administration

  • Patients received venetoclax in combination with azacitidine (75 mg/m2) or decitabine (20 mg/m2) for up to 6 cycles during the study 3.
  • Adults with newly diagnosed AML ineligible for intensive chemotherapy were enrolled in an open-label, non-randomized, multicenter phase 1b trial of venetoclax with azacitidine (AZA; 75 mg/m2; days 1-7) or decitabine (DEC; 20 mg/m2; days 1-5) 5.

Efficacy and Safety

  • The best response rate of composite complete remission was 66.7%, and the overall post-baseline red blood cell (RBC) and platelet transfusion independence rate was 55.0% 3.
  • Key adverse events included nausea, anemia, thrombocytopenia, neutropenia, and white blood cell count decrease of any grade (≥50% of patients) 3.
  • The CR/CRi rate was 71% for venetoclax plus AZA and 74% for venetoclax plus DEC 5.
  • The median duration of CR/CRi was 21.9 months and 15.0 months, and the median OS was 16.4 months and 16.2 months, for venetoclax plus AZA and DEC, respectively 5.

Patient Selection

  • Variables that favored response to ven/aza over IC included older age, secondary AML, and RUNX1 mutations 6.
  • AML M5 favored response to IC over ven/aza 6.
  • Adverse cytogenetics was associated with treatment failure in the R/R group (Relative Risk = 0.13, p = 0.005) 4.

Related Questions

Is there harm in giving steroids for persistent fever in new acute myeloid leukemia (AML) patients prior to initiating treatment with Vidaza (azacitidine) and Venetoclax (venetoclax)?
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Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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