Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia

Gerrits AJ, 2015 Blood                                                   

Link to abstract: http://www.bloodjournal.org/content/126/11/1367

Platelet activation measured by flow cytometry in 9 Wiskott-Aldrich Syndrome/ X-linked Thrombocytopenia (WAS/XLT) patients and 8 age-matched healthy controls, showed that reduced platelet activation found in WAS/ XLT was directly due to microthrombocytopenia.  GP IIb-IIIa and P-selectin were less expressed due to smaller platelet size. Eltrombopag treatment resulted in an increased platelet count in 5 out of 8 patients with WAS/ XLT and 6/8 had reduced clinical bleeding severity. Although the eltrombopag-induced increase in platelet production in WAS/XLT is less than in ITP (sample cohort compared to 7 age-matched ITP patients), it has beneficial effects on platelet counts but not platelet activation in the majority of WAS/XLT patients.