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.

Keywords: Wiskott Aldrich syndrome; Wiskott Aldrich syndrome protein; X-linked thrombocytopenia; allogeneic hematopoietic stem cell transplantation; autoimmunity; gene therapy; primary immunodeficiency; thrombocytopenia.