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How I manage patients with Wiskott Aldrich syndrome

Rivers, E; Worth, A; Thrasher, AJ; Burns, SO; (2019) How I manage patients with Wiskott Aldrich syndrome. British Journal of Haematology , 185 (4) pp. 647-655. 10.1111/bjh.15831. Green open access

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Abstract

Wiskott Aldrich syndrome (WAS) is a primary immunodeficiency disease resulting in recurrent infections, eczema and microthrombocytopaenia. In its classical form, significant combined immune deficiency, autoimmune complications and risk of haematological malignancy necessitate early correction with stem cell transplantation or gene therapy. A milder form, X‐linked thrombocytopaenia (XLT), shares similar bleeding risk from thrombocytopaenia but is not associated with other significant clinical features and is generally managed conservatively. Here, we detail our approach to the diagnosis and treatment of classical WAS and XLT.

Type: Article
Title: How I manage patients with Wiskott Aldrich syndrome
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/bjh.15831
Publisher version: https://doi.org/10.1111/bjh.15831
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Wiskott Aldrich syndrome, X-linked thrombocytopenia, Immunodeficiency, Haematopoietic stem cell transplant
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Infection and Immunity
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10070700
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