ATHN 11: Liver Transplantation Outcomes Study
- Conditions
- Hemophilia A and BLiver Transplantation
- Registration Number
- NCT04055051
- Lead Sponsor
- American Thrombosis and Hemostasis Network
- Brief Summary
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.
- Detailed Description
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:
1. Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL): A quality of life questionnaire designed to asses health-related quality of life in adult patients with hemophilia. 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.
2. Patient Reported Outcomes Measurement Information System (PROMIS-29): A quality of life questionnaire designed to assess physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles and activities, pain interference, and pain intensity.
3. Hemophilia Orthotopic Liver Transplantation QoL Questionnaire (HOT): A quality of life questionnaire, adapted from a cancer survivorship tool, designed to assess adults with hemophilia who have received a liver transplant. Domains include physical well-being, psychological well-being, stress, anxiety, fear, work, family, relationships, and spiritual well-being.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 86
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Quality of life (Haem-A-QoL) 1 year 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.Quality of Life (HOT) 1 year 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.
Quality of life (PROMIS-29) 1 year 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.
- Secondary Outcome Measures
Name Time Method Clotting factor protein 1year 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.
Trial Locations
- Locations (15)
Bleeding and Clotting Disorders Institute
🇺🇸Peoria, Illinois, United States
Maine Hemophilia and Thrombosis Center
🇺🇸Scarborough, Maine, United States
Orthopaedic Institute for Children Hemophilia Program
🇺🇸Los Angeles, California, United States
Indiana Hemophilia and Thrombosis Center (IHTC)
🇺🇸Indianapolis, Indiana, United States
Mayo Comprehensive Hemophilia Center
🇺🇸Rochester, Minnesota, United States
Henry Ford Health System
🇺🇸Detroit, Michigan, United States
Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Ohio State University Medical Center Hemophilia Treatment Center
🇺🇸Columbus, Ohio, United States
Mary M. Gooley Hemophilia Center, Inc.
🇺🇸Rochester, New York, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Gulf States Hemophilia and Thrombophilia Center
🇺🇸Houston, Texas, United States
Utah Center for Bleeding and Clotting Disorders
🇺🇸Salt Lake City, Utah, United States
Hemophilia Outreach Center Green Bay
🇺🇸Green Bay, Wisconsin, United States
Comprehensive Center for Bleeding Disorders, Milwaukee
🇺🇸Milwaukee, Wisconsin, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States