Article
X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options.
Blood - 22 Apr 2010
Albert Michael H, Bittner Tanja C, Nonoyama Shigeaki, Notarangelo Lucia Dora, Burns Siobhan, Imai Kohsuke, Espanol Teresa, Fasth Anders, Pellier Isabelle, Strauss Gabriele, Morio Tomohiro, Gathmann Benjamin, Noordzij Jeroen G, Fillat Cristina, Hoenig Manfred, Nathrath Michaela, Meindl Alfons, Pagel Philipp, Wintergerst Uwe, Fischer Alain, Thrasher Adrian J, Belohradsky Bernd H, Ochs Hans D
Abstract excerpt
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...
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