title: Pediatric AML: From Biology to Clinical Management.
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.
authors.affiliation: Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands. j.d.e.derooij@erasmusmc.nl.
authors.forename: Jasmijn D E
authors.initials: JD
authors.lastname: de Rooij
authors.affiliation: Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands. c.m.zwaan@erasmusmc.nl.
authors.forename: C Michel
authors.initials: CM
authors.lastname: Zwaan
authors.affiliation: Department of Pediatric Oncology, Erasmus MC-Sophia Children's Hospital, 3015CN Rotterdam, The Netherlands. m.vandenheuvel@erasmusmc.nl.
authors.forename: Marry
authors.initials: M
authors.lastname: van den Heuvel-Eibrink
keywords: clinical management; cytogenetics; molecular aberrations; pediatric AML