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Expert Rev Hematol. 2014 Apr;7(2):301-15. doi: 10.1586/17474086.2014.857596. Epub 2013 Dec 6.

Allogeneic stem cell transplantation and targeted therapy for FLT3/ITD+ acute myeloid leukemia: an update.

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  • 1Department of Medicine, Vanderbilt University Medical Center, Nashville, TN, USA.


Survival of patients with acute myelogenous leukemia (AML), particularly in younger patients, has improved in recent years due to improved understanding of disease biology, post remission therapies and supportive care. AML, however, remains difficult to treat as many patients will still ultimately relapse and die of their disease. This is particularly true in AML patients with identified FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) molecular mutations, which typically confers a poor prognosis. The FLT3-ITD mutation occurs in about one-quarter of patients diagnosed with AML. Oftentimes, these patients are referred for early allogeneic hematopoietic stem cell transplantation (HSCT) in hopes of overcoming this poor prognostic factor. Several studies have demonstrated some benefit with HSCT in patients with FLT3-ITD mutation. However, recent data suggested that FLT3-ITD mutation remains a poor prognostic factor even after early HSCT; these patients remain at risk for early relapse after transplantation, emphasizing ongoing efforts to explore maintenance therapy with FLT3-ITD inhibitors in the post-transplant setting.

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