Pediatric AML: From Biology to Clinical Management

• Published in: Journal of clinical medicine Vol. 4; no. 1; pp. 127 - 149
• Main Authors: de Rooij, Jasmijn D E, Zwaan, C Michel, van den Heuvel-Eibrink, Marry
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI 09.01.2015
• Subjects: Review; clinical management; molecular aberrations; cytogenetics; pediatric AML
• ISSN: 2077-0383; 2077-0383
• DOI: 10.3390/jcm4010127

Pediatric acute myeloid leukemia (AML) represents 15%-20% of all pediatric acute leukemias. Survival rates have increased over the past few decades to ~70%, due to improved supportive care, optimized risk stratification and intensified chemotherapy. In most children, AML presents as a de novo entity, but in a minority, it is a secondary malignancy. The diagnostic classification of pediatric AML includes a combination of morphology, cytochemistry, immunophenotyping and molecular genetics. Outcome is mainly dependent on the initial response to treatment and molecular and cytogenetic aberrations. Treatment consists of a combination of intensive anthracycline- and cytarabine-containing chemotherapy and stem cell transplantation in selected genetic high-risk cases or slow responders. In general, ~30% of all pediatric AML patients will suffer from relapse, whereas 5%-10% of the patients will die due to disease complications or the side-effects of the treatment. Targeted therapy may enhance anti-leukemic efficacy and minimize treatment-related morbidity and mortality, but requires detailed knowledge of the genetic abnormalities and aberrant pathways involved in leukemogenesis. These efforts towards future personalized therapy in a rare disease, such as pediatric AML, require intensive international collaboration in order to enhance the survival rates of pediatric AML, while aiming to reduce long-term toxicity.