Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant
2 other identifiers
observational
500
1 country
1
Brief Summary
Background: Hematopoietic stem cell transplant (HSCT) is a common treatment for many cancers and other illnesses. But many people who have HSCT go on to develop liver dysfunction. Researchers want to know more about how and why this happens. In this natural history study, they will try to learn what factors lead to liver dysfunction; how underlying liver disease may affect the results of HSCT; and how HSCT may contribute to liver dysfunction. Objective: To understand the links between HSCT and liver dysfunction. Eligibility: Adults aged 18 years or older and children 3 to 17 years who are being evaluated for HSCT. Design: This study involves 11 visits in 4 years. Most visits will be in the first year. Before and after their HSCT, participants will undergo these tests: Physical exam, including blood tests and a test of heart function. Participants will provide stool samples. Liver biopsies. Samples of liver tissue will be removed. This may be done either by inserting a needle through the right side of the chest, or with a thin tube threaded to the liver from a vein in the neck. Adult participants will undergo this procedure 2 times: once before the HSCT and once about a year later. Imaging scans. Participants will lie on a bed that moves into either a cylinder or a donut-shaped machine. Ultrasound. Participants will lie still. A probe that uses sound waves will be slid over their skin to get pictures of the liver. Fibroscan exam. This is like an ultrasound that uses a special probe to measure the toughness of the liver. ...
Trial Health
Trial Health Score
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participants targeted
Target at P75+ for all trials
Started Apr 2026
Longer than P75 for all trials
1 active site
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 9, 2023
CompletedFirst Posted
Study publicly available on registry
February 10, 2023
CompletedStudy Start
First participant enrolled
April 21, 2026
ExpectedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2031
Study Completion
Last participant's last visit for all outcomes
June 30, 2031
April 16, 2026
September 16, 2025
5.2 years
February 9, 2023
April 15, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Death or total bilirubin >=4 at 91 days after the transplant
To determine whether, at 91 days after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin \>=4 than those without liver disease at transplant.
91 days after transplant
Mortality rate
To determine whether, at 91 days after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin \>=4 than those without liver disease at transplant.
91 days after transplant
Secondary Outcomes (4)
Rate of infection (type: bacterial, viral, fungal; location: central line, organ infection, sepsis, etc.)
91 days after transplant
Development of portal hypertension and sequelae (i.e., ascites, variceal bleed, thrombocytopenia, elevated portal pressure)
91 days after transplant
Morbitity/cause of death
91 days after transplant
Development of liver failure
91 days after transplant
Study Arms (2)
Adults >= 18 years of age
undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
Children 3-17 years of age
undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
Eligibility Criteria
male and female subjects: adults 18 years of age and older, children 3-17 years of age undergoing hematopoietic stem cell transplant at the National Institutes of Health.
You may qualify if:
- An individual who meets any of the following criteria will be included in this study:
- Male and female adults \>=18 years of age and children 3-17 years of age
- Undergoing evaluation for hematopoietic stem cell transplant at the NIH Clinical Center
You may not qualify if:
- An individual who meets any of the following criteria will be excluded from participation in this
- study:
- Pregnancy or lactation
- Unable to comply with study procedures
- Inability to provide written informed consent
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Theo Heller, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 9, 2023
First Posted
February 10, 2023
Study Start (Estimated)
April 21, 2026
Primary Completion (Estimated)
June 30, 2031
Study Completion (Estimated)
June 30, 2031
Last Updated
April 16, 2026
Record last verified: 2025-09-16