An observational study in Hemophilia A and B and Liver Transplantation, sponsored by American Thrombosis and Hemostasis Network. Completed at 15 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-05-10.
Sponsored by American Thrombosis and Hemostasis Network · Observational
This study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation. Participants will be asked to complete health related quality of life questionnaires and provide medical history.
At a time when gene therapy is becoming a reality for individuals with hemophilia A and B, little is known about long-term cures. Yet, there are few data to provide real life testing of the impact of long-term cures. Specifically, little is known about the relationship between factor level achieved with gene therapy and clinical outcomes or quality of life. The analogy to liver transplant is clear. There has been no systematic method to collect and analyze the long-term outcomes of liver transplantation in individuals with hemophilia. Previous efforts to access data through the United Network for Organ Sharing national database have been complicated by lack of identification of those with hemophilia. As most of the individuals with hemophilia who undergo liver transplantation receive care at a hemophilia treatment center (HTC), ATHN proposes to leverage its existing relationship with the HTCs to support HTC execution of this study.
ATHN, in collaboration with US Hemophilia Treatment Centers (HTCs), can provide the infrastructure and organization to support a longitudinal cohort study of subjects with congenital hemophilia who have and have not undergone liver transplantation. This cohort study will compare quality of life outcomes between cases that have undergone liver transplantation and controls that have not undergone liver transplantation. The study will determine if factor levels attained post-transplantation correlate with or predict improvement in quality of life measures.
The existence of ATHN and the ATHN System for clinical care and research provides an opportunity to observe a cohort of subjects after liver transplantation with facility. During the study period, it is predicted that sustained normalization in factor VIII or IX levels following liver transplantation improves clinical functioning and quality of life in adults with hemophilia A and B.
All study related contact will be timed to coincide with routine, scheduled care whenever possible. The study schedule will include:
Study enrollment - Study activities will begin after participant consent. Activities include the documentation of relevant medical history and the administration of quality of life (QoL) questionnaires.
Participants will be asked to complete health related (QoL) questionnaires and provide medical history. These questionnaires include:
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 86 is close to the median of 80 across 314 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →American Thrombosis and Hemostasis Network is the lead sponsor of 8 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
The study will enroll a cohort of approximately 112 participants born with hemophilia who meet the eligibility criteria and are receiving care from one of approximately 12 participating HTCs. The participants will be approached for enrollment and matched 1:3 with age-, race-, ethnicity-matched controls (N=84) with hemophilia who have not undergone transplantation, for a total of 112 individuals with hemophilia who will compete quality of life questionnaires.
Participants who meet the following inclusion criteria are eligible for enrollment into the study:
Exclusion Criteria
Participants who fall into any of the following exclusion criteria at the time of screening are not eligible for enrollment into the study:
No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort.
No intervention. Comparable patients to those in Case cohort will be put in this cohort.
Quality of life (Haem-A-QoL)
Health related quality of life questionnaire Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL): Domains include physical health, feelings, view of self, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, and partnership and sexuality. For comparing questionnaire scores between two cohorts, we could use a combined score of PROMIS-29, Haem-A-QoL, and HOT questionnaires rescaled in 0-100 or use each separate survey.
Time frame: 1 year
Quality of life (PROMIS-29)
Health related Quality of Life measured by the Patient Reported Outcome Measurement Information System (PROMIS-29) Profile measure physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles and activities, pain interference, and pain intensity.
Time frame: 1 year
Quality of Life (HOT)
Hemophilia Orthotopic Liver Transplantation QoL Questionnaire (HOT) Domains include physical well-being, psychological well-being, stress, anxiety, fear, work, family, relationships and spiritual well-being.
Time frame: 1 year
Clotting factor protein
impact of sustained increase in clotting factor proteins (FVIII and FIX) levels after liver transplantation. The medical record will be accessed to record baseline hemophilia history including primary factor diagnosis and baseline factor level used to access disease severity, post-transplant factor levels and genotype.
Time frame: 1year
Plan to share: No
This study is completed, as verified in May 2023. You cannot join it, but the record below documents what was studied.
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American Thrombosis and Hemostasis Network