Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents.

  title={Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents.},
  author={M. Wlodarski and Shinsuke Hirabayashi and V. Pastor and J. Star{\'y} and H. Hasle and R. Masetti and M. Dworzak and M. Schmugge and M. M. van den Heuvel-Eibrink and M. Ussowicz and B. De Moerloose and A. Catal{\`a} and O. Smith and P. Sedl{\'a}cek and A. Lankester and M. Zecca and V. Bordon and S. Matthes-Martin and J. Abrahamsson and J. K{\"u}hl and K. Sykora and M. Albert and Bartlomiej Przychodzien and J. Maciejewski and S. Schwarz and G. G{\"o}hring and B. Schlegelberger and A. Cseh and P. N{\"o}llke and A. Yoshimi and F. Locatelli and I. Baumann and B. Strahm and C. Niemeyer},
  volume={127 11},
          1387-97; quiz 1518
  • M. Wlodarski, Shinsuke Hirabayashi, +31 authors C. Niemeyer
  • Published 2016
  • Medicine
  • Blood
  • Germline GATA2 mutations cause cellular deficiencies with high propensity for myeloid disease. [...] Key Method 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 advanced and 7% of all primary MDS cases, but were absent in children with MDS…Expand Abstract
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