ATHN 11: An Observational Cohort Study of Long-Term Outcomes of Orthotopic Liver Transplantation in People with Hemophilia
The primary objective of ATHN 11: Liver Transplantation Outcomes Study is to examine the impact of sustained increase in Factor VIII (FVIII) and Factor IX (FIX) levels after liver transplantation on quality of life as measured by pain, health outcomes, work, activity, and mental and physical health in adults with hemophilia A and B. Researchers will also be looking to determine predictors, such as factor level and other covariates, of health outcomes and quality of life.
ATHN 11: Liver Transplantation Outcomes Study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have undergone liver transplantation compared with those who have not undergone liver transplantation. The study will enroll approximately 112 participants (28 cases and 84 controls) born with hemophilia who meet the eligibility criteria and are receiving care from one of the participating hemophilia treatment centers (HTCs). Participants who meet the following inclusion criteria are eligible for enrollment into the study:
- Congenital hemophilia A or B of any severity who have and have not undergone a liver transplant
- 18 years of age or older
- Sex assigned at birth was male
The approximate duration of the study is 1.5 years. HTC staff will conduct the informed consent process and administer questionnaires to participants by telephone. This cohort study will compare the quality of life outcomes between participants who have received a liver transplant (cases) and patients who have not received a liver transplant (controls).