Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura

• Published in: The New England journal of medicine Vol. 357; no. 22; pp. 2237 - 2247
• Main Authors: Bussel, James B, Cheng, Gregory, Saleh, Mansoor N, Psaila, Bethan, Kovaleva, Lidia, Meddeb, Balkis, Kloczko, Janusz, Hassani, Habib, Mayer, Bhabita, Stone, Nicole L, Arning, Michael, Provan, Drew, Jenkins, Julian M
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 29.11.2007
• Subjects: Adolescent; Adult; Aged; Aged, 80 and over; Autoimmune diseases; Benzoates - administration & dosage; Benzoates - adverse effects; Benzoates - therapeutic use; Chronic Disease; Dose-Response Relationship, Drug; Double-Blind Method; Drug therapy; Female; Heart attacks; Hemorrhage - chemically induced; Hemorrhage - epidemiology; Hepatitis; Humans; Hydrazines - administration & dosage; Hydrazines - adverse effects; Hydrazines - therapeutic use; Male; Medical research; Middle Aged; Platelet Count; Purpura, Thrombocytopenic, Idiopathic - blood; Purpura, Thrombocytopenic, Idiopathic - drug therapy; Pyrazoles - administration & dosage; Pyrazoles - adverse effects; Pyrazoles - therapeutic use; Quality of Life; Receptors, Thrombopoietin - agonists; Recurrence; Thrombopoietin - analysis
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMoa073275

This trial tested the efficacy of eltrombopag, a small nonpeptide agonist of the thrombopoietin receptor, in patients with immune thrombocytopenia who had not had a response to at least one previous treatment for the disorder. At a dose of 50 or 75 mg, the agonist, which had been shown to increase platelet production in normal volunteers, increased platelet counts to a clinically safe level (≥50,000 per cubic millimeter) in most patients. Eltrombopag, a small nonpeptide agonist of the thrombopoietin receptor, increased platelet counts to a clinically safe level (≥5;50,000 per cubic millimeter) in most patients. Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease in which antiplatelet antibodies accelerate the destruction of platelets. In addition, platelet production can be impaired 1 because the antiplatelet antibodies can also damage megakaryocytes. 2 – 4 Although the thrombocytopenia of ITP can be severe, signs of bleeding are usually only minor. Persistently low platelet counts (<20,000 per cubic millimeter), however, are associated with an increased risk of serious bleeding, such as intracranial hemorrhage. 5 , 6 The goal of managing chronic ITP is to maintain platelet counts, with the least possible intervention, at levels that prevent bleeding, thereby reducing treatment-related toxicity. 7 Glucocorticoids and intravenous immunoglobulins . . .