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Rebecca Olin, MD, MS


Venetoclax-Based Regimen for Pediatric Patients With Resistant AML

By: Joshua Swore
Posted: Tuesday, March 31, 2020

A phase I dose-escalation study published in The Lancet Oncology reported on the overall safe and effective use of venetoclax plus chemotherapy in children with acute myeloid leukemia (AML). According to Seth E. Karol, MD, of St. Jude Children’s Research Hospital, Memphis, and colleagues, “this combination should be tested in newly diagnosed pediatric patients with high-risk acute myeloid leukemia.”

A total of 38 patients, between the ages of 3 and 22, with relapsed or refractory AML were enrolled in the study. Venetoclax was given to patients orally once daily in a 28-day cycle at 240 mg/m2 or 360 mg/m2 in combination with cytarabine intravenously every 12 hours at 100 mg/m2 for 20 doses or 1,000 mg/m2 for eight doses. Treatments were given with or without a single dose of idarubicin at 12 mg/m2.

The overall response rate to treatment was 69%. No patients required dose adjustment or treatment discontinuation due to toxicity, although one patient died during treatment. As a result, the recommended phase II trial dose is 360 mg/m2 in combination with cytarabine at 1,000 mg/m2. A complete response occurred in 14 of 20 patients treated at the phase II dose.

Grade 3 or 4 infections were reported in 29% of patients, with two patients having serious adverse events. The most common grade 3 or 4 adverse events were neutropenia, bloodstream infection, and fungal infection.

The results of the study led to further commentary in The Lancet Oncology from Richard Aplenc, MD PhD, of the Children’s Hospital of Philadelphia, University of Pennsylvania: “Not only is it the first peer-reviewed publication evaluating the efficacy of venetoclax in a pediatric population, but it is also the first evaluation of the combination of venetoclax with intensive acute myeloid leukemia chemotherapy.” 

Disclosure: For full disclosures of the study authors, visit

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