title: Once daily ledipasvir/sofosbuvir fixed-dose combination with ribavirin in patients with inherited bleeding disorders and hepatitis C genotype 1 infection.
abstract: OBJECTIVE
  People with inherited bleeding disorders have been disproportionally affected by HCV. We assessed the fixed-dose combination of the NS5A inhibitor ledipasvir (LDV) with the NS5B polymerase inhibitor sofosbuvir (SOF) with ribavirin (RBV) in patients with genotype 1 HCV and inherited bleeding disorders.
  
  
  METHODS
  To be eligible, patients had to be over 18 years of age and have an inherited bleeding disorder. HCV treatment-naïve and -experienced patients could enrol. All patients received LDV 90 mg per SOF 400 mg once daily and weight-based RBV in a divided dose for 12 weeks. The primary efficacy endpoint was sustained virologic response (SVR), defined as HCV RNA below the limit of detection (15 IU mL-1) 12 weeks after the end of treatment (SVR12).
  
  
  RESULTS
  Of the 14 patients enrolled, 8 (57%) had haemophilia A, 3 (21%) had haemophilia B and 2 (14%) had von Willebrand disease, and 1 (7%) had factor XIII deficiency. All 14 patients (100%, 95% CI: 77-100%) achieved SVR12. Treatment was well tolerated: all patients completed therapy, with mostly mild adverse events. No specific safety concerns associated with the patient's underlying bleeding disorders were noted.
  
  
  CONCLUSIONS
  These results appear to suggest that people with HCV and inherited bleeding disorders can be safely and effectively treated with 12 weeks of LDV/SOF plus RBV.
authors: Gastroenterology Department, Christchurch Hospital and University of Otago, Christchurch, New Zealand.
authors: C A M
authors: CAM
authors: Stedman
authors: Gilead Sciences Inc., Foster City, CA, United States.
authors: R H
authors: RH
authors: Hyland
authors: Gilead Sciences Inc., Foster City, CA, United States.
authors: X
authors: X
authors: Ding
authors: Gilead Sciences Inc., Foster City, CA, United States.
authors: P S
authors: PS
authors: Pang
authors: Gilead Sciences Inc., Foster City, CA, United States.
authors: J G
authors: JG
authors: McHutchison
authors: New Zealand Liver Transplant Unit, Auckland City Hospital, Auckland, New Zealand.
authors: E J
authors: EJ
authors: Gane
keywords: antiviral; haemophilia; hepatitis C virus; ledipasvir; sofosbuvir