Texas Children's Cancer and Hematology Center UPDATE AML: A New Chemotherapy Approach for Pediatric Acute Myeloid Leukemia

A pilot feasibility study testing new chemotherapy combinations for children and young adults with intermediate- or high-risk acute myeloid leukemia (AML)

Description

Acute myeloid leukemia (AML) is a fast-growing cancer of the blood and bone marrow. It affects how blood cells develop and can be very aggressive in children and young adults.
This study, called UPDATE AML, is testing whether two new chemotherapy combinations can safely replace parts of the standard treatment for pediatric AML. The goal is to maintain or improve survival while reducing long-term side effects.

The two new combinations being tested are:

• Ida-FLA: idarubicin, fludarabine, and cytarabine
• VIA: venetoclax, idarubicin, and cytarabine

These combinations have shown promise in treating relapsed AML but haven’t been widely studied in newly diagnosed patients. Venetoclax is FDA-approved for adults with AML but not yet for children.

Another key part of this study is improving how doctors detect tiny amounts of leftover leukemia (called residual disease) after treatment. Researchers will develop personalized tests based on each patient’s genetic profile to better identify hidden leukemia cells. This includes blood and bone marrow sample testing and may involve biomarker analysis.

This study is important because it could lead to safer, more effective treatments and better ways to monitor the disease.

Eligibility

• Ages 1 month to 30 years
• Diagnosed with AML or myeloid sarcoma
• Must have completed Induction 1 chemotherapy (DA10+GO)
• No FLT3-ITD mutation
• Performance status score above 40 (Karnofsky/Lansky)
• Adequate kidney, liver, heart, and blood clotting function
• Specific genetic markers may be required (e.g., RUNX1::RUNX1T1, CBFB::MYH11, KMT2A rearrangement, etc.)

Detailed inclusion and exclusion criteria are listed on clinicaltrials.gov