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[Pediatric acute myeloid leukemia].

Hiroshi Moritake¹

¹Division of Pediatrics, Department of Developmental and Urological Reproductive Medicine, Faculty of Medicine, University of Miyazaki.

[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology

|October 2, 2024

Summary

Pediatric acute myeloid leukemia (AML) in Japan has distinct subtypes. While some AML types are curable, relapsed cases, particularly de novo AML, require novel therapeutic strategies.

Keywords:

Acute promyelocytic leukemia De novo acute myeloid leukemia Down syndrome Pediatric acute myeloid leukemia

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Area of Science:

Hematology
Pediatric Oncology
Clinical Research

Background:

Acute myeloid leukemia (AML) constitutes about 25% of pediatric leukemia diagnoses in Japan, with around 150 new cases annually.
Pediatric AML is categorized into myeloid leukemia associated with Down syndrome (ML-DS), acute promyelocytic leukemia (APL), and de novo AML.
Outcomes vary significantly among subtypes, with ML-DS having high survival rates, APL being curable, and de novo AML presenting challenges in remission and relapse.

Purpose of the Study:

To review the therapeutic approaches for the three main pediatric AML subtypes in Japan.
To provide context from past clinical studies conducted in Japan.
To highlight promising novel therapeutic options for relapsed or refractory de novo AML.

Main Methods:

Literature review of past clinical studies on pediatric AML in Japan.
Analysis of treatment outcomes for ML-DS, APL, and de novo AML.
Identification and discussion of emerging therapies for relapsed/refractory de novo AML.

Main Results:

Patients with ML-DS demonstrate over 80% event-free survival, though relapsed cases have poor prognoses.
APL is highly curable using all-trans retinoic acid and arsenic trioxide.
De novo AML shows a 10% initial remission failure rate and a ~30% relapse rate after achieving remission.

Conclusions:

Current therapeutic strategies offer distinct outcomes for different pediatric AML subtypes.
Relapsed and refractory de novo AML remains a significant clinical challenge.
New therapeutic avenues are crucial for improving outcomes in difficult-to-treat pediatric AML populations.