Liver Transplantation in Severe Hemophilia A: A Two-Patient Case Series with Divergent Long-Term Outcomes.
- 1Botkin Hospital, Moscow Healthcare Department, 5, 2nd Botkinsky Proezd, Moscow 125284, Russia.
- 2Russian Medical Academy of Continuous Professional Education, Ministry of Health of the Russian Federation, 2/1 Barrikadnaya Street, Moscow 125993, Russia.
Abstract
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.
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BibTeX
@article{shabunin2026liver,
title = {{Liver Transplantation in Severe Hemophilia A: A Two-Patient Case Series with Divergent Long-Term Outcomes.}},
author = {Alexey Vasilyevich Shabunin and Pavel Alexeevich Drozdov and Oksana Nikolaevna Levina and Sergey Andreevich Astapovich and Daria Andreevna Teplyuk and Dzhennet Agamovna Beibalaeva},
journal = {Reports (MDPI)},
year = {2026},
month = oct,
doi = {10.3390/reports9040342},
url = {https://europepmc.org/article/MED/42924794},
note = {CC BY},
}
RIS
TY - JOUR
TI - Liver Transplantation in Severe Hemophilia A: A Two-Patient Case Series with Divergent Long-Term Outcomes.
AU - Alexey Vasilyevich Shabunin
AU - Pavel Alexeevich Drozdov
AU - Oksana Nikolaevna Levina
AU - Sergey Andreevich Astapovich
AU - Daria Andreevna Teplyuk
AU - Dzhennet Agamovna Beibalaeva
PY - 2026
DA - 2026/10/10
JO - Reports (MDPI)
DO - 10.3390/reports9040342
UR - https://europepmc.org/article/MED/42924794
AB - 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.
SN - 2571-841X
ER -