Prevalence and Development of Liver Dysfunction in Hematopoietic Stem Cell Transplant: A Prospective Natural History Study
Trial Snapshot
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Enrollment
- 500
- Locations
- 1
- Primary Endpoint
- Mortality rate
Study Overview
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.
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Detailed Description
Study Description:
Liver dysfunction is common in patients that have undergone hematopoietic stem cell transplant (HSCT) and is associated with increased mortality. We aim to study the natural history of liver dysfunction in HSCT, what factors contribute to the development of liver dysfunction, and how underlying liver disease affects complications and outcomes of HSCT. We hypothesize that those patients with underlying liver disease or those who develop liver disease have increased morbidity and mortality compared to those without liver disease.
Objectives:
Primary Objective:
To determine whether, at 3 months (Visit 7) after transplant, patients with liver disease at transplant are more likely to have died or have a total bilirubin >=4 mg/dL than those without liver disease at transplant.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 3 Years to 90 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •INCLUSION CRITERIA:
- •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
Exclusion Criteria
- •An individual who meets any of the following criteria will be excluded from participation in this
- •Pregnancy or lactation
- •Unable to comply with study procedures
- •Inability to provide written informed consent
Arms & Interventions
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
Outcomes
Primary Outcomes
Mortality rate
Time Frame: 91 days after 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.
Death or total bilirubin >=4 at 91 days after the transplant
Time Frame: 91 days after 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.
Secondary Outcomes
- 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)