Article
Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.
Blood - 4 Jun 2020
Burroughs Lauri M, Petrovic Aleksandra, Brazauskas Ruta, Liu Xuerong, Griffith Linda M, Ochs Hans D, Bleesing Jack J, Edwards Stephanie, Dvorak Christopher C, Chaudhury Sonali, Prockop Susan E, Quinones Ralph, Goldman Frederick D, Quigg Troy C, Chandrakasan Shanmuganathan, Smith Angela R, Parikh Suhag, Dávila Saldaña Blachy J, Thakar Monica S, Phelan Rachel, Shenoy Shalini, Forbes Lisa R, Martinez Caridad, Chellapandian Deepak, Shereck Evan, Miller Holly K, Kapoor Neena, Barnum Jessie L, Chong Hey, Shyr David C, Chen Karin, Abu-Arja Rolla, Shah Ami J, Weinacht Katja G, Moore Theodore B, Joshi Avni, DeSantes Kenneth B, Gillio Alfred P, Cuvelier Geoffrey D E, Keller Michael D, Rozmus Jacob, Torgerson Troy, Pulsipher Michael A, Haddad Elie, Sullivan Kathleen E, Logan Brent R, Kohn Donald B, Puck Jennifer M, Notarangelo Luigi D, Pai Sung-Yun, Rawlings David J, Cowan Morton J
Abstract excerpt
Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outcomes have improved over time, particularly for patients with...
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