Molecular therapy for acute myeloid leukaemia Journal Article


Authors: Coombs, C. C.; Tallman, M. S.; Levine, R. L.
Article Title: Molecular therapy for acute myeloid leukaemia
Abstract: Acute myeloid leukaemia (AML) is a heterogeneous disease that is, in general, associated with a very poor prognosis. Multiple cytogenetic and molecular abnormalities that characterize different forms of AML have been used to better prognosticate patients and inform treatment decisions. Indeed, risk status in patients with this disease has classically been based on cytogenetic findings; however, additional molecular characteristics have been shown to inform risk assessment, including FLT3, NPM1, KIT, and CEBPA mutation status. Advances in sequencing technology have led to the discovery of novel somatic mutations in tissue samples from patients with AML, providing deeper insight into the mutational landscape of the disease. The majority of patients with AML (>97%) are found to have a clonal somatic abnormality on mutational profiling. Nevertheless, our understanding of the utility of mutation profiling in clinical practice remains incomplete and is continually evolving, and evidence-based approaches to application of these data are needed. In this Review, we discuss the evidence-base for integrating mutational data into treatment decisions for patients with AML, and propose novel therapeutic algorithms in the era of molecular medicine.
Keywords: gastrointestinal stromal tumors; patients; stem-cell transplantation; phase-ii trial; acute myelogenous leukemia; trans-retinoic acid; 1st complete remission; risk myelodysplastic syndrome; internal; tandem duplication; farnesyltransferase inhibitor tipifarnib; younger adult
Journal Title: Nature Reviews Clinical Oncology
Volume: 13
Issue: 5
ISSN: 1759-4774
Publisher: Nature Publishing Group  
Date Published: 2016-05-01
Start Page: 305
End Page: 318
Language: English
ACCESSION: WOS:000374452300006
DOI: 10.1038/nrclinonc.2015.210
PROVIDER: wos
PUBMED: 26620272
PMCID: PMC5525060
Notes: Review -- Source: Wos
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  1. Martin Stuart Tallman
    649 Tallman
  2. Ross Levine
    775 Levine
  3. Catherine C Coombs
    18 Coombs