title: Current and emerging treatment options for Wiskott-Aldrich syndrome.
abstract: Wiskott-Aldrich syndrome is a life-threatening primary immunodeficiency associated with a bleeding tendency, eczema and a high incidence of autoimmunity and malignancy. Stem cell transplantation offers the opportunity of cure for all these complications, and over the past 35 years there has been a remarkable improvement in survival following this treatment. Here, we review advances in management of clinical complications pre- and post-transplant, as well as discuss the morbidity Wiskott-Aldrich syndrome patients experience following treatment. For patients with a poorly matched stem cell donor, recent gene therapy trials demonstrate encouraging results and the potential of low-toxicity therapy for all patients.
authors.affiliation: Department of Clinical Immunology and Bone Marrow Transplantation, Great Ormond Street Hospital National Health Service Trust, London WC1N 1JH, UK.
authors.forename: Austen J J
authors.initials: AJ
authors.lastname: Worth
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
mesh_terms: D002675:Child, Preschool; D020022:Genetic Predisposition to Disease / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D009154:Mutation; D016019:Survival Analysis; D014923:Wiskott-Aldrich Syndrome / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D051316:Wiskott-Aldrich Syndrome Protein / Q000235:genetics*
chemical_list: C495767:WAS protein, human; D051316:Wiskott-Aldrich Syndrome Protein
keywords: Wiskott Aldrich syndrome; Wiskott Aldrich syndrome protein; X-linked thrombocytopenia; allogeneic hematopoietic stem cell transplantation; autoimmunity; gene therapy; primary immunodeficiency; thrombocytopenia