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Updated: Jul 16, 2026

Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants
Published on: June 6, 2025
[Acute myeloid leukemia]
1Division of Hemato-oncology and Regeneration Medicine, Kanagawa Children Medical Center.
Insights
Pediatric acute myeloid leukemia (AML) survival rates improved with risk-adapted therapy. Identifying prognostic factors like chromosomal abnormalities and genetic mutations guides tailored AML treatment strategies for better outcomes in children.
Area of Science:
- Pediatric Hematology/Oncology
- Cancer Genetics and Genomics
- Clinical Trial Design
Context:
- Pediatric acute myeloid leukemia (AML) incidence varies globally, with Japan reporting higher rates than the USA and Europe.
- Significant advancements have improved long-term survival for pediatric AML patients, exceeding 50% in the USA and Europe.
- Understanding prognostic factors is crucial for stratifying risk and personalizing AML treatment in children.
Purpose:
- To analyze prognostic factors in pediatric AML and classify patients accordingly.
- To evaluate the efficacy and safety of risk-stratified treatment strategies in newly diagnosed pediatric AML.
- To assess the impact of genetic mutations (e.g., FLT3/ITD) and chromosomal abnormalities on AML prognosis.
Summary:
- Specific chromosomal translocations (t(15;17), inv(16), t(8;21)) predict a favorable prognosis, while monosomy 7, monosomy 5, and del(5q) indicate a poor prognosis.
- FLT3/ITD mutations are associated with a particularly poor prognosis in pediatric AML.
- Risk-adapted therapy, incorporating quality of life considerations, has led to high survival rates, such as 79% at 3 years in the AML 99 trial.
Impact:
- Establishes the importance of risk stratification based on leukemia cell biology and initial treatment response for optimizing pediatric AML therapy.
- Highlights the success of risk-adapted therapeutic strategies in improving survival rates for childhood de novo AML.
- Informs future clinical trial design and treatment protocols for pediatric AML, aiming for enhanced efficacy and safety.
Abstract:
The annual incident rate of pediatric acute myeloid leukemia (AML) is now 10 per million in Japan, against 5 to 9 per million in the USA and Europe. Overall long-term survival has now been achieved for more than 50% of pediatric patients with AML in the USA and in Europe. The prognostic factors of pediatric AML were analyzed,and patients with AML were classified according to prognostic factors. The t(15;17), inv(16) and t(8;21) have emerged as predictors of good prognosis in children with AML. Monosomy 7, monosomy 5 and del (5 q) abnormalities showed a poor prognosis. In addition to chromosomal deletions, FLT 3/ITD identifies pediatric patients with a particularly poor prognosis. Clinical trials of AML feature intensive chemotherapy with or without subsequent stem cell transplantation. Risk group stratification is becoming increasingly important in planning AML therapy. APL can be distinguished from other subtypes of AML by virtue of its excellent response and overall outcome as a result of differentiation therapy with ATRA. Children with Down syndrome and AML have been shown to have a superior prognosis to AML therapy compared to other children with AML. The results of the Japan Cooperative Study Group protocol ANLL 91 was one of the best previously reported in the literature. With the consideration of quality of life (QOL), risk-adapted therapy was introduced in the AML 99 trial conducted by the Japanese Childhood AML Cooperative Study Group. A high survival rate of 79% at 3 years was achieved for childhood de novo AML in the AML 99 trial. To evaluate the efficacy and safety of the treatment strategy according to risk stratification based on leukemia cell biology and response to the initial induction therapy in children with AML, the Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG) has organized multi-center phase II trials in children with newly diagnosed AML.
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