X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options.

@article{Albert2010XlinkedT,
  title={X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options.},
  author={Michael H. Albert and Tanja C. Bittner and Shigeaki Nonoyama and Lucia Dora Notarangelo and Siobhan Burns and Kohsuke Imai and Teresa Espa{\~n}ol and Anders L Fasth and Isabelle Pellier and Gabriele Strauss and Tomohiro Morio and B. Gathmann and Jeroen G. Noordzij and Cristina Fillat and Manfred Hoenig and Michaela Nathrath and Alfons Meindl and Philipp Pagel and Uwe Wintergerst and Alain Fischer and Adrian J. Thrasher and Bernd H. Belohradsky and Hans D. Ochs},
  journal={Blood},
  year={2010},
  volume={115 16},
  pages={3231-8}
}
A large proportion of patients with mutations in the Wiskott-Aldrich syndrome (WAS) protein gene exhibit the milder phenotype termed X-linked thrombocytopenia (XLT). Whereas stem cell transplantation at an early age is the treatment of choice for patients with WAS, therapeutic options for patients with XLT are controversial. In a retrospective multicenter study we defined the clinical phenotype of XLT and determined the probability of severe disease-related complications in patients older than… CONTINUE READING
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