Article
Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents.
Blood - 17 Mar 2016
Wlodarski Marcin W, Hirabayashi Shinsuke, Pastor Victor, Starý Jan, Hasle Henrik, Masetti Riccardo, Dworzak Michael, Schmugge Markus, van den Heuvel-Eibrink Marry, Ussowicz Marek, De Moerloose Barbara, Catala Albert, Smith Owen P, Sedlacek Petr, Lankester Arjan C, Zecca Marco, Bordon Victoria, Matthes-Martin Susanne, Abrahamsson Jonas, Kühl Jörn Sven, Sykora Karl-Walter, Albert Michael H, Przychodzien Bartlomiej, Maciejewski Jaroslaw P, Schwarz Stephan, Göhring Gudrun, Schlegelberger Brigitte, Cseh Annámaria, Noellke Peter, Yoshimi Ayami, Locatelli Franco, Baumann Irith, Strahm Brigitte, Niemeyer Charlotte M
Abstract excerpt
Germline GATA2 mutations cause cellular deficiencies with high propensity for myeloid disease. We investigated 426 children and adolescents with primary myelodysplastic syndrome (MDS) and 82 cases with secondary MDS enrolled in 2 consecutive prospective studies of the European Working Group of MDS in Childhood (EWOG-MDS) conducted in Germany over a period of 15 years. Germline GATA2 mutations accounted for 15% of...
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