Severe thrombocytopenia in a patient with hepatitis C treated with eltrombopag from off-label drug use to on-label drug use: a case report

Abstract

IntroductionOff-label drug use refers to drug use beyond the specifications authorized for marketing. Eltrombopag is a new thrombopoietin receptor agonist which was used successfully in this critical case of thrombocytopenia associated with hepatitis C infection before it became an approved drug for such cases.Case presentationA 56-year-old Kuwaiti woman with hepatitis C virus infection was treated with pegylated interferon α-2a and ribavirin, laboratory test results prior to therapy were within normal values. After 4 weeks of that treatment, she developed neutropenia and severe thrombocytopenia. Her hepatitis C virus treatment was stopped for many years until eltrombopag was used as an off-label drug with an episode of severe thrombocytopenia. Her platelets count returned to normal level when triple therapy for hepatitis C virus was used successfully.ConclusionsAn off-label drug should be used only when it is the best available drug, based on evidence from on-going multicenter trials. It could be life saving for some patients in critical situations. However, clinical use of eltrombopag later confirmed that it is a safe and effective drug for immune thrombocytopenic purpura or thrombocytopenia associated with hepatitis C virus infection.

Eltrombopag Use for Treatment of Thrombocytopenia in a Patient with Chronic Liver Disease and Portal Vein Thrombosis: Case Report

“…Although eltrombopag has gained approval from the FDA for chronic immune thrombocytopenia, its use is not recommended in patients with CLD due to the increased risk of venous thromboembolism. However, it has been used in several studies as an off-label medication for treatment of thrombocytopenia in CLD [ 2 , 3 , 4 , 5 , 6 ].…”