Eltrombopag in Children With Idiopathic Aplastic Anemia
Status:
Completed
Trial end date:
2020-01-05
Target enrollment:
Participant gender:
Summary
Aplastic anemia is a rare disorder characterized by pancytopenia and a hypo cellular bone
marrow.but,It is very serious disease causing morbidity and mortality.
Aplastic anemia can be treated effectively with haematopoietic stem cell transplantation and
immunosuppressive drug regimens but haematopoietic stem cell transplantation has limitations
due to its cost and many patient are unsuitable. Immunosuppressive drug has a significant
number of patients have persistent cytopenias. Currently, the treatment of these patients is
regular transfusion, which are expensive, inconvenient, and associated with serious side
effects related to iron overload and transfusion.
Eltrombopag is an oral thrombopoietin mimetic that selectively binds at the transmembrane and
juxtamembrane domains of the thrombopoietin receptor, at sites distinct from the binding site
of thrombopoietin therefore it does not compete for binding with the native molecule. It
promoting thrombopoiesis and release of platelets from mature megakaryocytes. Also, promote
other hematopoietic stem cell as well as in thrombopoiesis .