Disease characteristics and outcomes of acute myeloid leukemia in germline RUNX1 deficiency (Familial Platelet Disorder with associated Myeloid Malignancy)

Disease characteristics and outcomes of acute myeloid leukemia in germline RUNX1 deficiency (Familial Platelet Disorder with associated Myeloid Malignancy)

Abstract Familial Platelet Disorder with associated Myeloid Malignancy (FPDMM, FPD/AML, RUNX1‐FPD), caused by monoallelic deleterious germline RUNX1 variants, is characterized by bleeding diathesis and predisposition for hematologic malignancies, particularly myelodysplastic syndrome (MDS) and acute...

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Main Authors: Martijn P. T. Ernst, Jurjen Versluis, Peter J. M. Valk, Marc Bierings, Rienk Y. J. Tamminga, Louise H. Hooimeijer, Konstanze Döhner, Paolo Gresele, Kiran Tawana, Saskia M. C. Langemeijer, Bert A. Van der Reijden, Helena Podgornik, Matjaz Sever, Tor H. A. Tvedt, Tom Vulliamy, Jude Fitzgibbon, Inderjeet Dokal, Panagiotis Baliakas, José M. Bastida, Christian Pohlkamp, Torsten Haferlach, Lise Larcher, Jean Soulier, Roger E. G. Schutgens, Kathleen Freson, Nicolas Duployez, Bob Löwenberg, Katrin Ericson, Jörg Cammenga, Tim Ripperger, Marc H. G. P. Raaijmakers
Format: Article
Language:English
Published: Wiley 2025-01-01
Series:HemaSphere
Online Access:https://doi.org/10.1002/hem3.70057
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