Eltrombopag is an effective drug that can help patients suffering from thrombocytopenia. This disease occurs when the percentage of platelets in the patient’s blood decreases to abnormally low levels. The molecule of Eltrombopag acts as an agonist for TpoR receptor and it stimulates the thrombopoietin hormone which helps to increase the presence of platelets in the blood. In 2008, the US Food and Drug Administration approved Eltrombopag tablets for treating adult patients suffering from chronic immune thrombocytopenic purpura (ITP).
Safety and efficacy of Eltrombopag
In order to confirm the effectiveness of Eltrombopag in helping patients suffering from chronic IPT, two double-blind, placebo-controlled clinical studies were performed. 231 patients took part in one of these studies. All of them were suffering from ITP with a platelet count of less than 30,000 per micro Liter of blood. Eltrombopag and placebo tablets were administered for patients, regularly, for six weeks. The results showed that the platelet count increased to 50,000/micro Liter or more in the patients who were administered with Eltrombopag.
In the other experiment, 109 patients were administered with Eltrombopag. The platelet count of each of the patients were equal to or less than 18,000/micro liter. After studying the results, it was found that Eltrombopag helped to increase the platelet count up to 74,000/mcL in three months and 67,000/mcL in six months. Discontinuation of Eltrombopag increased the risks of hepatotoxicty, hemorrhage and serious thrombocytopenia for some patients.
Developmental experiments with Eltrombopag
During the preclinical studies, researchers confirmed that Eltrombopag can interact with the hormone thrombopoietin to activate the JAK-STAT signaling pathway in a selective manner that results in differentiation of megakaryocytes. Certain megakaryocytes can help in producing blood thrombocytes. Several medical experiments on animals confirmed that controlled doses of Eltrombopag can increase platelet counts to satisfactory levels. During a medical experiment, the drug was administered to 73 healthy people with no signs of ITP. Heavy doses of Eltrombopag raised levels of blood thrombocytes in all volunteers and none of them complained about any tolerability issue.
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