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Harnessing New Developments in Genomics to Improve Outcome for Children with Poor Prognosis Leukemia
The dramatic improvement in outcome for children with acute lymphoblastic leukemia is one of the major achievements in hematology in the last 40 years. However, while 90% of children with this disease can now be cured, the outlook has not been so good for the less common type of childhood leukemia, acute myeloid leukemia (AML).
Although only 1 in 7 children with leukemia have AML, half of these children will die from their disease. The challenge now is to harness the new developments in genomics to improve the outcome for these children.
At the 19th Congress of the European Hematology Association (EHA), we will learn about the state-of-the-art in management of childhood AML. New concepts in the way AML develops and the way AML cells 'hijack' the main sites of normal blood production within the body will be presented. A whole host of genes has recently been identified as causes of childhood AML. How information about these genes can be used to refine the diagnosis and monitoring of childhood AML will be discussed. In particular, studies are beginning to show that comprehensive analysis of 'leukemia' genes in childhood AML allows treatment to be 'risk adapted'. This new approach aims to adapt treatment in order to minimise the risks of side effects. In this way the use of prolonged treatment with high doses of powerful drugs will be restricted to the small group of children with AML where their genetic profile indicates that these drugs are likely to be the best way to cure the disease.
Efforts are also underway to tackle childhood AML using novel therapies. The relative rarity of childhood AML (5-6 cases/million children per year) means that clinical trials of new drugs are only possible when pediatric hematologists and oncologists from many different countries in Europe work together in close collaboration. We will hear about the successes and future plans of some of these collaborative studies in childhood AML at the 19th Congress of EHA.