Clinical Medicine Insights: Therapeutics

Thrombocytopenia is the most common hematological abnormality in patients with chronic, advanced liver disease. In these patients, the presence of severe thrombocytopenia is an obstacle to the performance of invasive diagnostic and therapeutic procedures, and the current standard treatment for these patients is platelet transfusions, a remedy whose characteristics are far from being ideal. Furthermore, thrombocytopenia in patients with chronic hepatitis C virus infection may render the patients ineligible to antiviral treatment or may limit its efficacy because of premature discontinuation. Although the cause of thrombocytopenia in patients with chronic liver disease is likely multi-factorial, decreased thrombopoietin production by the liver undoubtedly plays a significant role. In this regard, eltrombopag, a non-peptide, orally bioavailable thrombopoietin receptor agonist has been shown to safely increase platelet count in a dose-dependent fashion in both healthy subjects and thrombocytopenic patients with chronic hepatitis C. Furthermore, in this latter group of patients, it has been shown to be superior to placebo in counteracting the myelosuppressive effect of short-term pegylated interferon treatment.