Predictors of liver disease outcomes in individuals with hemophilia and HCV infection.

Hemophilia is an X-linked congenital bleeding disorder for which factor replacement is life-saving but complicated by the sequelae of chronic hepatitis C virus (HCV) infection acquired decades ago. Although antiviral therapy clears HCV and reduces end-stage liver disease (ESLD), it may not reverse cirrhosis or prevent hepatocellular cancer (HCC). This was a retrospective cohort study of 121 men with hemophilia and HCV infection cared for at the Hemophilia Center of Western Pennsylvania to determine the incidence and predictors of ESLD and HCC. ESLD and HCC predictors were analyzed using Fisher exact test, and HCV-associated outcomes by Kaplan-Meier time-to-event and Cox proportional hazards regression analyses. At a mean 54 years (36-80) duration of HCV, ESLD occurred in 24 (19.8%), 0.365 per 100 person-years (py); and HCC in 7 (5.8%), 0.106 per 100 py. All 46 (38.0%) alive when HCV antiviral therapy became available, received it. Overall, 31 (25.6%) were HIV+. The leading causes of death were ESLD in 11 (32.3%), bleeding in 9 (26.5%), and HCC in 6 (17.6%). Major risk factors for ESLD included platelets <100 × 103/μL (odds ratio [OR], 6.009; P = .012) and HIV infection (OR, 3.883; P = .001). The major predictors of HCC were ESLD (OR, 11.476; P = .003) and platelets <100 000/μL (OR, 6.159; P = .014). No antiviral-treated patient developed ESLD, P = .001. For men with hemophilia, the sequelae of chronic HCV infection were significant. The major risk factors for ESLD were platelets <100 000/μL and HIV infection. Despite antiviral therapy, ESLD is the most significant predictor of HCC, and ESLD is the leading cause of death.