title: 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: BACKGROUND
  Off-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.
  
  
  METHODS
  A 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.
  
  
  CONCLUSIONS
  An 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.
authors.affiliation: Hematology Department, Amiri Hospital, Gulf Street, Kuwait City, Kuwait. Cbc9@hotmail.com.
authors.forename: Hassan A
authors.initials: HA
authors.lastname: Al-Jafar
authors.forename: Jameela
authors.initials: J
authors.lastname: Al-Khaldi
authors.forename: Ahmad
authors.initials: A
authors.lastname: Alduaij
authors.forename: Khalifa
authors.initials: K
authors.lastname: Al-Banwan
mesh_terms: D001565:Benzoates / Q000627:therapeutic use*; D005260:Female; D006526:Hepatitis C / Q000150:complications*; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D008875:Middle Aged; D056687:Off-Label Use; D011720:Pyrazoles / Q000627:therapeutic use*; D012720:Severity of Illness Index; D013921:Thrombocytopenia / Q000150:complications* / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; C520809:eltrombopag