Precision Medicine Challenge in Acute Myeloid Leukemia

In a comprehensive evaluation of molecular and clinical heterogeneity in 1540 patients with acute myeloid leukemia, Elli Papaemmanuil, PhD, of Wellcome Trust Sanger Institute, Hinxton, Cambridgeshire, United Kingdom, and colleagues found that gene mutations accounted for approximately 70% of variance in overall survival, compared with 5% for treatment, 9% for clinical variables, and 17% for demographics (Abstract 803).

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Based on comprehensive genomic and clinical data, Moritz Gerstung, PhD, European Bioinformatics Institute EMBL-EBI, Hinxton, Cambridgeshire, United Kingdom, and colleagues developed a personally tailored risk prediction of acute myeloid leukemia.

They have implemented their novel prediction framework into a web portal to explore risk prediction, which uses genetic and phenotypic heterogeneity to deliver personalized outcome and treatment predictions, such as benefit of hematopoietic stem cell transplantation in first remission or after relapse for individual patients (Abstract 85).

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Richard M. Stone, MD, Dana-Farber Cancer Institute in Boston, MA, and colleagues found that midostaurin represents a new standard of care in patients newly diagnosed with internal tandem duplication and tyrosine kinase domain FLT3 mutant acute myeloid leukemia. The multi-targeted kinase inhibitor improved overall survival when added to standard chemotherapy compared with placebo (Abstract 6).