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Title: Treatment practice and outcomes in FLT3-mutant acute myeloid leukemia in the pre-midostaurin era: a real-world experience from Australian tertiary hospitals.
Austin Authors: Chua, Chong C;Grigg, Andrew P ;Singh, Jasmine;Droogleever, Mark P;Zhang, Lan;Lim, Andrew;Fong, Chun Y;Ting, Stephen B;Schwarer, Anthony;Tiong, Ing S ;Wei, Andrew H
Affiliation: Department of Haematology, The Alfred Hospital and Monash University, Melbourne, Australia
Department of Clinical Haematology, Austin Health, Heidelberg, Victoria, Australia
Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands
Department of Haematology, Eastern Health and Monash University, Box Hill, Australia
Issue Date: Apr-2020 2019-11-21
Publication information: Leukemia & lymphoma 2020; 61(4): 848-854
Abstract: Recent regulatory approval of midostaurin, a FLT3 targeting small molecular inhibitor, will likely lead to increased use of midostaurin in combination with intensive chemotherapy for patients with FLT3-mutant AML. Translation of clinical trial results into everyday practice has its challenges. This study compared the relevance of the trial population and practices studied in the midostaurin registration study (RATIFY) with real-world practice in terms of patient factors, chemotherapy, mutation-specific frequencies and clinical outcomes among patients with FLT3-mutant AML in the pre-midostaurin era (2010-2015) in Australia. We observed substantial diversity of chemotherapy regimens used in the community and limitations of the generalizability of eligibility criteria used in RATIFY (such as age and hyperleukocytosis). This study provides real-world historical data that may be used for comparison with future trial cohorts incorporating FLT3 inhibitors into the management of FLT3-mutant AML and highlights the inherent difficulties in translating clinical trial data into routine practice.
DOI: 10.1080/10428194.2019.1691192
ORCID: 0000-0002-2162-3288
PubMed URL: 31752581
Type: Journal Article
Subjects: Acute myeloid leukemia
FLT3 inhibitor
FLT3 mutation
Appears in Collections:Journal articles

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