ATHN 11: Liver Transplantation Outcomes Study

NCT04055051 | Completed

• 1 other identifier: ATHN 11: Liver Transplantation
• Study Type: observational
• Target: 86
• Locations: 1 country
• Sites: 15

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.

Conditions

Hemophilia A and B; Liver Transplantation

Keywords

Hemophilia A; Hemophilia B; Liver Transplantation; Quality of Life; Hemophilia Treatment Centers (HTCs)

Outcome Measures

Primary Outcomes (3)

• 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.

  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.

  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.

  1 year

Secondary Outcomes (1)

• Clotting factor protein

  1year

Study Arms (2)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

• \. Age \<18.

Sponsors & Collaborators

• American Thrombosis and Hemostasis Network: lead
• BioMarin Pharmaceutical: collaborator

Study Sites (15)

Orthopaedic Institute for Children Hemophilia Program

Los Angeles, California, 90007, United States

Location

University of California, San Francisco

San Francisco, California, 94117, United States

Location

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614, United States

Location

Indiana Hemophilia and Thrombosis Center (IHTC)

Indianapolis, Indiana, 46260, United States

Location

Maine Hemophilia and Thrombosis Center

Scarborough, Maine, 04074, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Mayo Comprehensive Hemophilia Center

Rochester, Minnesota, 55905, United States

Location

Mary M. Gooley Hemophilia Center, Inc.

Rochester, New York, 14621, United States

Location

Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27517, United States

Location

Ohio State University Medical Center Hemophilia Treatment Center

Columbus, Ohio, 43210, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, 77030, United States

Location

Utah Center for Bleeding and Clotting Disorders

Salt Lake City, Utah, 84113, United States

Location

Hemophilia Outreach Center Green Bay

Green Bay, Wisconsin, 54311, United States

Location

Comprehensive Center for Bleeding Disorders, Milwaukee

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (9)

• Madeira CL, Layman ME, de Vera RE, Fontes PA, Ragni MV. Extrahepatic factor VIII production in transplant recipient of hemophilia donor liver. Blood. 2009 May 21;113(21):5364-5. doi: 10.1182/blood-2009-02-206979. No abstract available.

  PMID: 19470440 BACKGROUND

• Subramanian A, Sulkowski M, Barin B, Stablein D, Curry M, Nissen N, Dove L, Roland M, Florman S, Blumberg E, Stosor V, Jayaweera DT, Huprikar S, Fung J, Pruett T, Stock P, Ragni M. MELD score is an important predictor of pretransplantation mortality in HIV-infected liver transplant candidates. Gastroenterology. 2010 Jan;138(1):159-64. doi: 10.1053/j.gastro.2009.09.053. Epub 2009 Sep 30.

  PMID: 19800334 BACKGROUND

• Ragni MV, Moore CG, Soadwa K, Nalesnik MA, Zajko AB, Cortese-Hassett A, Whiteside TL, Hart S, Zeevi A, Li J, Shaikh OS; HHH Study Group. Impact of HIV on liver fibrosis in men with hepatitis C infection and haemophilia. Haemophilia. 2011 Jan;17(1):103-11. doi: 10.1111/j.1365-2516.2010.02366.x. Epub 2010 Aug 16.

  PMID: 20722744 BACKGROUND

• Ragni MV, Devera ME, Roland ME, Wong M, Stosor V, Sherman KE, Hardy D, Blumberg E, Fung J, Barin B, Stablein D, Stock PG. Liver transplant outcomes in HIV+ haemophilic men. Haemophilia. 2013 Jan;19(1):134-40. doi: 10.1111/j.1365-2516.2012.02905.x. Epub 2012 Jul 5.

  PMID: 22762561 BACKGROUND

• Terrault NA, Roland ME, Schiano T, Dove L, Wong MT, Poordad F, Ragni MV, Barin B, Simon D, Olthoff KM, Johnson L, Stosor V, Jayaweera D, Fung J, Sherman KE, Subramanian A, Millis JM, Slakey D, Berg CL, Carlson L, Ferrell L, Stablein DM, Odim J, Fox L, Stock PG; Solid Organ Transplantation in HIV: Multi-Site Study Investigators. Outcomes of liver transplant recipients with hepatitis C and human immunodeficiency virus coinfection. Liver Transpl. 2012 Jun;18(6):716-26. doi: 10.1002/lt.23411.

  PMID: 22328294 BACKGROUND

• Aguero F, Rimola A, Stock P, Grossi P, Rockstroh JK, Agarwal K, Garzoni C, Barcan LA, Maltez F, Manzardo C, Mari M, Ragni MV, Anadol E, Di Benedetto F, Nishida S, Gastaca M, Miro JM; FIPSE/NIH HIVTR/NEAT023 Investigators. Liver Retransplantation in Patients With HIV-1 Infection: An International Multicenter Cohort Study. Am J Transplant. 2016 Feb;16(2):679-87. doi: 10.1111/ajt.13461. Epub 2015 Sep 28.

  PMID: 26415077 BACKGROUND

• Mehta KD, Ragni MV. Bleeding and liver transplantation outcomes in haemophilia. Haemophilia. 2017 Mar;23(2):230-237. doi: 10.1111/hae.13104. Epub 2016 Nov 4.

  PMID: 27813318 BACKGROUND

• Ragni MV, Humar A, Stock PG, Blumberg EA, Eghtesad B, Fung JJ, Stosor V, Nissen N, Wong MT, Sherman KE, Stablein DM, Barin B. Hemophilia Liver Transplantation Observational Study. Liver Transpl. 2017 Jun;23(6):762-768. doi: 10.1002/lt.24688.

  PMID: 27935212 BACKGROUND

• Ragni MV, Nalesnik MA, Schillo R, Dang Q. Highly active antiretroviral therapy improves ESLD-free survival in HIV-HCV co-infection. Haemophilia. 2009 Mar;15(2):552-8. doi: 10.1111/j.1365-2516.2008.01935.x.

  PMID: 19347994 BACKGROUND

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Margaret Ragni, MD, MPH

  University of Pittsburgh Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 8, 2019
• First Posted: August 13, 2019
• Study Start: June 18, 2020
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: May 10, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (15)