ATHN 11: Liver Transplantation Outcomes Study (HOT)

American Thrombosis and Hemostasis Network (ATHN)

Status: Completed

Conditions: Liver Transplantation; Hemophilia A and B

Study type: Observational

Funder types: NETWORK; Industry

Identifiers

NCT04055051

ATHN 11: Liver Transplantation

Details and patient eligibility

About

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.

Full 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.

Enrollment: 86 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

Participants who meet the following inclusion criteria are eligible for enrollment into the study:

1. Congenital hemophilia A or B of any severity, who have and have not undergone a liver transplant;
2. Age > 18; and
3. Sex assigned at birth was male

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:

1. Age <18.

Trial design

86 participants in 2 patient groups

Hemophilia A and B Cases

Description:

No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort.

Hemophilia A and B Controls

Description:

No intervention. Comparable patients to those in Case cohort will be put in this cohort.