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J. Clin. Med. 2015, 4(1), 127-149;

Pediatric AML: From Biology to Clinical Management

Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands
Author to whom correspondence should be addressed.
Academic Editor: Celalettin Ustun
Received: 17 October 2014 / Accepted: 28 November 2014 / Published: 9 January 2015
(This article belongs to the Special Issue AML in the Molecular Age: From Biology to Clinical Management)
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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. View Full-Text
Keywords: pediatric AML; clinical management; cytogenetics; molecular aberrations pediatric AML; clinical management; cytogenetics; molecular aberrations

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de Rooij, J.D.E.; Zwaan, C.M.; van den Heuvel-Eibrink, M. Pediatric AML: From Biology to Clinical Management. J. Clin. Med. 2015, 4, 127-149.

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