What is the role of eltrombopag in the treatment of immune thrombocytopenia (ITP)?

Updated: Jan 11, 2020
  • Author: Craig M Kessler, MD, MACP; Chief Editor: Srikanth Nagalla, MBBS, MS, FACP  more...
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Like romiplostim, eltrombopag also became available in 2008. In August 2015, the US Food and Drug Administration expanded the indication for eltrombopag to include treatment of chronic ITP in patients 1 year of age and older who have not achieved an appropriate response with other medical therapy or splenectomy. [67]  An oral TPO receptor agonist, eltrombopag increases platelet counts after 8 days of daily dosing.

Eltrombopag was studied in a phase III double-blind trial in adults with previously treated ITP lasting more than 6 months and with platelet counts lower than 30,000/µL. Patients received treatment with local standard care plus eltrombopag (50 mg) or placebo for 6 months. Of 196 patients in the study, 106 (79%) patients in the eltrombopag group responded to treatment at least once, compared with 17 (28%) in the placebo group. Toxic reactions in the eltrombopag group included thromboembolic events (2%), mild increases in alanine aminotransferase levels (3%), and increased total bilirubin levels (4%). [68]

Eltrombopag has a boxed warning for the risk of severe and potentially life‐threatening hepatotoxicity. Dose reductions are needed for patients with hepatic impairment and some patients of East Asian ancestry. Eltrombopag must be taken on an empty stomach (1 hr before or 2 hrs after meals).  

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