Abstract / Summary
Background and Clinical Significance: Before effective viral inactivation of plasma-derived factor concentrates, many patients with hemophilia acquired hepatitis C virus infection and later developed cirrhosis or hepatocellular carcinoma. Liver transplantation treats end-stage liver disease and may also restore factor VIII production; however, long-term oncologic outcomes in this setting are rarely reported. Case Presentation: Two men with severe hemophilia A (baseline factor VIII activity < 1%) and hepatitis C-related cirrhosis underwent deceased-donor orthotopic liver transplantation. Both received an extended-half-life recombinant factor VIII (efmoroctocog alfa; 33,000 and 35,000 IU in total) tapered until postoperative days 13–14, and intraoperative hemostasis was guided by conventional coagulation tests and thromboelastography. Neither patient had major bleeding or thrombotic events, and replacement therapy was not required thereafter. In Patient 1, graft function remained satisfactory 45 months after transplantation, with factor VIII activity of 62.5%. Patient 2 was transplanted for hepatocellular carcinoma outside the Milan criteria (two lesions of 6.8 and 2.7 cm; alpha-fetoprotein 110–150 ng/mL) under center-specific expanded criteria, without bridging therapy. Factor VIII activity remained clinically sufficient, but pulmonary nodules suspected 20 months after transplantation progressed; atypical pulmonary resections 45 months after transplantation confirmed metastatic hepatocellular carcinoma, and lenvatinib was initiated. Conclusions: Liver transplantation can provide durable correction of severe hemophilia A without long-term replacement therapy. However, correction of the coagulation disorder does not eliminate risks related to the original liver disease; in patients transplanted for hepatocellular carcinoma, particularly beyond conventional criteria, long-term outcome remains dependent on tumor burden, tumor biology, candidate selection, and sustained oncologic surveillance.