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J Clin Med. 2015 Jan 9;4(1):127-49. doi: 10.3390/jcm4010127.

Pediatric AML: From Biology to Clinical Management.

Author information

1
Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands. j.d.e.derooij@erasmusmc.nl.
2
Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands. c.m.zwaan@erasmusmc.nl.
3
Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands. m.vandenheuvel@erasmusmc.nl.

Abstract

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.

KEYWORDS:

clinical management; cytogenetics; molecular aberrations; pediatric AML

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