Bleeding Clinical Trial
Official title:
Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
Wiskott Aldrich Syndrome is an X-linked disease characterized by immunodeficiency, eczema and
thrombocytopenia; a milder form of the disease known as X-Linked thrombocytopenia also
exists. The thrombocytopenia in both WAS and XLT is characterized by: severe thrombocytopenia
with platelet counts frequently less than 10-30,000/ul; small platelets which may be
dysfunctional; and, as a result, a high rate of serious bleeding including intracranial
hemorrhage.
Because eltrombopag has been shown to be remarkably efficacious in substantially increasing
platelet counts in a high percentage of ITP patients, this study seeks to effectively treat
patients who exhibit similar pathologies, as well as evaluate the state of platelets in
patients with WAS and relate it to clinical bleeding. It also aims to demonstrate whether
eltrombopag administered daily will enhance stem cell function, increase platelet production
and platelet count, and reduce bleeding in patients with WAS.
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