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Rebecca Olin, MD, MS


Pediatric AML: Combination Therapy With Venetoclax and Azacitidine

By: Sarah Campen, PharmD
Posted: Friday, October 9, 2020

The combination of venetoclax and azacitidine as a treatment for pediatric acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) appears to be a safe and effective regimen, according to a small single-center case series published in Pediatric Blood & Cancer. A morphologic response was achieved in 75% of patients treated with the doublet. “The regimen had acceptable toxicity in our patients and showed promising rates of remission induction, including patients refractory to cytotoxic chemotherapy,” stated Anna K. Franklin, MD, of the Children’s Hospital Colorado, Aurora, and colleagues.

In this retrospective chart review, the authors identified eight patients between the ages of 2 and 20 years—six with AML and two with high-grade MDS—who were treated with venetoclax plus azacitidine. Patients with AML had relapsed or primary refractory disease or were deemed poor candidates for standard chemotherapy.

Morphologic responses were seen in six patients—including both patients with MDS—and all four responders with AML became minimal residual disease–negative. Thus far, three responders have proceeded to allogeneic hematopoietic stem cell transplantation following the combination therapy.

Overall, the regimen was reported to be well tolerated. The most common grade 4 adverse events included neutropenia and thrombocytopenia; other common adverse events included anemia, nausea, and fatigue. “We would predict the organ toxicities related to venetoclax plus azacitidine would be less than those associated with standard chemotherapeutic regimens,” concluded the authors. However, “larger patient numbers are needed to confirm the safety and efficacy of venetoclax-based regimens for pediatric myeloid malignancies.”

Disclosure: For full disclosures of the study authors, visit

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