Article
Hematopoietic Stem Cell Transplantation for X-Linked Thrombocytopenia With Mutations in the WAS gene.
Journal of clinical immunology - 1 Jan 2015
Oshima Koichi, Imai Kohsuke, Albert Michael H, Bittner Tanja C, Strauss Gabriele, Filipovich Alexandra H, Morio Tomohiro, Kapoor Neena, Dalal Jignesh, Schultz Kirk R, Casper James T, Notarangelo Luigi D, Ochs Hans D, Nonoyama Shigeaki
Abstract excerpt
X-linked thrombocytopenia (XLT) is a mild form of the Wiskott-Aldrich syndrome (WAS) caused by mutations in the WAS gene. A recent retrospective study of the clinical outcome and molecular basis of a large cohort of XLT patients demonstrated that although overall survival is excellent, event free survival is severely affected with conservative treatment. To answer the question whether hematopoietic stem cell...
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