An X-linked primary immunodeficiency caused by a mutation in the Wiskott-Aldrich syndrome (WAS) gene
Originally described as clinical triad of thrombocytopenia with small platelets, e...
Diagnosis should be considered in any boy who has congenital or early-onset thrombocytopenia with small platelets.
Definitive diagnosis
Male patient
Congenital thrombocytopenia (<70,000/mm3)
...
Antibiotics for acute infections and prophylactically in postsplenectomy patients
Splenectomy
May be helpful for persistent severe thrombocytopenia in select patients. However,...
Signs and symptoms of malignancy should be evaluated expeditiously.
As patients age, a progressive increase in infectious and autoimmune complica...
Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science. 2013;341(6148):1233151. [View Abstr...
Q: What is the life expectancy for patients with WAS?
A: Before currently available therapies, most affected patients died in childhood. Currently, many patients live into their 3rd and 4th decades...
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