Related Experiment Video
Updated: Feb 23, 2026
10:49
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
18.7K
Pediatric acute myeloid leukemia with genetic alterations
1Department of Pediatric Hematology/Oncology, Okayama University Hospital.
[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology
|September 9, 2017
Summary
Pediatric acute myeloid leukemia (AML) outcomes have improved with risk-stratified chemotherapy and stem cell transplant. Future personalized therapies may emerge with next-generation sequencing for better pediatric AML treatment.
Area of Science:
- Pediatric Oncology
- Hematology
- Molecular Genetics
Background:
- Acute myeloid leukemia (AML) affects 150-200 children annually in Japan.
- Historical clinical studies (ANLL91, AML99, CCLSG-AML9805, JPLSG-AML05) improved outcomes through risk stratification.
- Pediatric AML molecular landscape differs from adult AML, with specific mutations impacting prognosis.
Purpose of the Study:
- To review historical clinical studies and molecular findings in pediatric acute myeloid leukemia (AML).
- To explore current challenges and future directions for improving pediatric AML treatment outcomes.
- To highlight the potential of next-generation sequencing for personalized pediatric AML therapy.
Main Methods:
- Review of historical Japanese clinical trial data for pediatric AML.
- Analysis of molecular mutation profiles in pediatric AML cases.
- Discussion of genetic syndromes associated with pediatric AML.
Main Results:
- Risk stratification combining chemotherapy and hematopoietic stem cell transplantation has improved pediatric AML outcomes.
- FLT3-ITD or KIT mutations (Class I) are associated with poor prognosis in pediatric AML.
- Pediatric AML can be complicated by chromosome fragility or bone marrow failure syndromes.
Conclusions:
- While significant progress has been made, further improvements in pediatric AML treatment are needed.
- Next-generation sequencing holds promise for enabling personalized therapeutic strategies in pediatric AML.
- Understanding the unique molecular and genetic factors in pediatric AML is crucial for advancing patient care.
Related Concept Videos
01:24Pharmacokinetics in Pediatric Patients: Drug Metabolism
276
In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
276
01:11Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase
30
Genetic polymorphisms in drug targets have emerged as critical determinants of interindividual variability in drug response and toxicity. Pharmacogenomic investigations increasingly focus on identifying these variations to personalize and optimize therapeutic interventions. A drug target may be a receptor, enzyme, or signaling protein involved in pharmacologic responses or disease-related pathways. While early pharmacogenetic studies focused primarily on drug metabolism, current research...
30
01:26Pharmacokinetics in Pediatric Patients: Drug Excretion
302
In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
302
02:50Combination Therapies and Personalized Medicine
6.2K
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
6.2K