Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia
| Autor: | James B. Bussel, Michelle A. Berny-Lang, Emily Leven, Marc R. Barnard, W. Beau Mitchell, Alan D. Michelson, Andrew L. Frelinger, Hannah Tamary, Anja J. Gerrits, Sophie L. Brigstocke, Shoshana Revel-Vilk, Sabrina L. Carmichael |
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| Rok vydání: | 2015 |
| Předmět: |
Adult
Male Adolescent Wiskott–Aldrich syndrome Immunology Eltrombopag Platelet Glycoprotein GPIIb-IIIa Complex Benzoates Biochemistry X linked thrombocytopenia Inosine triphosphate Young Adult chemistry.chemical_compound medicine Platelet Count measurement Humans Platelet Platelet activation Mean platelet volume Child Platelet Count business.industry Infant Genetic Diseases X-Linked Cell Biology Hematology Platelet Activation Platelets and Thrombopoiesis medicine.disease Thrombocytopenia Wiskott-Aldrich Syndrome P-Selectin Hydrazines chemistry Case-Control Studies Child Preschool Pyrazoles business Mean Platelet Volume Receptors Thrombopoietin |
| Zdroj: | Blood. 126:1367-1378 |
| ISSN: | 1528-0020 0006-4971 |
| Popis: | Because Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) patients have microthrombocytopenia, hemorrhage is a major problem. We asked whether eltrombopag, a thrombopoietic agent, would increase platelet counts, improve platelet activation, and/or reduce bleeding in WAS/XLT patients. In 9 WAS/XLT patients and 8 age-matched healthy controls, platelet activation was assessed by whole blood flow cytometry. Agonist-induced platelet surface activated glycoprotein (GP) IIb-IIIa and P-selectin in WAS/XLT patients were proportional to platelet size and therefore decreased compared with controls. In contrast, annexin V binding showed no differences between WAS/XLT and controls. Eltrombopag treatment resulted in an increased platelet count in 5 out of 8 patients. Among responders to eltrombopag, immature platelet fraction in 3 WAS/XLT patients was significantly less increased compared with 7 pediatric chronic immune thrombocytopenia (ITP) patients. Platelet activation did not improve in 3 WAS/XLT patients whose platelet count improved on eltrombopag.(1) the reduced platelet activation observed in WAS/XLT is primarily due to the microthrombocytopenia; and (2) although the eltrombopag-induced increase in platelet production in WAS/XLT is less than in ITP, eltrombopag has beneficial effects on platelet count but not platelet activation in the majority of WAS/XLT patients. This trial was registered at www.clinicaltrials.gov as #NCT00909363. |
| Databáze: | OpenAIRE |
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