Multi-million dollar study will include 19 centers in three countries
A renowned liver expert from Children's Hospital of Pittsburgh is leading an international study examining the causes and potential treatments of acute liver failure in children.
Robert H. Squires Jr., MD, director of hepatology and clinical director of the Division of Gastroenterology at Children's, is the principal investigator of a five-year, $5.8 million study that was recently funded by the National Institute of Diabetes and Digestive and Kidney Diseases, one of the National Institutes of Health (NIH).
Children's and the University of Pittsburgh will serve as the data coordinating center and one of approximately 19 sites that will study acute liver failure in children. Acute liver failure is a rapidly developing clinical condition that results in either death or liver transplantation in 50 percent of the cases. It accounts for 13 percent of all liver transplants in children, according to the NIH-funded Studies of Pediatric Liver Transplantation.
"Unfortunately, physicians are often not able to determine a cause for acute liver failure," said Dr. Squires, who also is a professor of Pediatrics at the University of Pittsburgh School of Medicine. "However, known causes in children include defects in liver cell metabolism, autoimmune liver disease, viral infections, reactions to medications and acetaminophen (Tylenol®) poisoning. A better understanding of the causes of acute liver failure will help us develop effective treatments."
The pediatric acute liver study, directed from Children's, will be divided into three aspects:
- Developing a registry of patients admitted to hospitals for acute liver failure. This registry will provide important epidemiological data vital to understanding the causes of acute liver failure.
- A therapeutic clinical trial to study the use of N-acetylcysteine (NAC) as a treatment for acute liver failure that is caused by factors other than acetaminophen overdose. NAC is approved only as treatment for acetaminophen overdose.
- Basic research into genetic causes of acute liver failure such as immune disregulation and fatty acid oxidation defects. This project also includes the assessment of a technique to identify proteins connected to acetaminophen with the idea that some people have a genetic tendency to not metabolize acetaminophen properly.
Source: Eurekalert & othersLast reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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