ATHN 11: Liver Transplantation Outcomes Study (HOT)

May 9, 2023 updated by: American Thrombosis and Hemostasis Network

An Observational Cohort Study of Long-Term Outcomes of Orthotopic Liver Transplantation in People With Hemophilia

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.

Study Overview

Status

Completed

Conditions

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.

Study Type

Observational

Enrollment (Actual)

86

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Carol Fedor, ND, RN, CCRC
  • Phone Number: 800-360-2846
  • Email: cfedor@athn.org

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Orthopaedic Institute for Children Hemophilia Program
      • San Francisco, California, United States, 94117
        • University of California, San Francisco
    • Illinois
      • Peoria, Illinois, United States, 61614
        • Bleeding and Clotting Disorders Institute
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia and Thrombosis Center (IHTC)
    • Maine
      • Scarborough, Maine, United States, 04074
        • Maine Hemophilia and Thrombosis Center
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Comprehensive Hemophilia Center
    • New York
      • Rochester, New York, United States, 14621
        • Mary M. Gooley Hemophilia Center, Inc.
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
        • Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University Medical Center Hemophilia Treatment Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh
    • Texas
      • Houston, Texas, United States, 77030
        • Gulf States Hemophilia and Thrombophilia Center
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Utah Center for Bleeding and Clotting Disorders
    • Wisconsin
      • Green Bay, Wisconsin, United States, 54311
        • Hemophilia Outreach Center Green Bay
      • Milwaukee, Wisconsin, United States, 53226
        • Comprehensive Center for Bleeding Disorders, Milwaukee

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Hemophilia A and B Cases
No intervention. Only patients that have undergone a liver transplant per study eligibility are in this cohort.
Hemophilia A and B Controls
No intervention. Comparable patients to those in Case cohort will be put in this cohort.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quality of life (Haem-A-QoL)
Time Frame: 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.
1 year
Quality of life (PROMIS-29)
Time Frame: 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.
1 year
Quality of Life (HOT)
Time Frame: 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.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clotting factor protein
Time Frame: 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.
1year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

American Thrombosis and Hemostasis Network

Collaborators

BioMarin Pharmaceutical

Investigators

  • Principal Investigator: Margaret Ragni, MD, MPH, University of Pittsburgh Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 18, 2020

Primary Completion (Actual)

December 1, 2022

Study Completion (Actual)

December 1, 2022

Study Registration Dates

First Submitted

August 8, 2019

First Submitted That Met QC Criteria

August 12, 2019

First Posted (Actual)

August 13, 2019

Study Record Updates

Last Update Posted (Actual)

May 10, 2023

Last Update Submitted That Met QC Criteria

May 9, 2023

Last Verified

May 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATHN 11: Liver Transplantation

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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