Portal hypertension
is common; careful
follow-up is essential
Children with liver disease due to Alpha-1 Antitrypsin Deficiency have a significant burden of portal hypertension (30% in those with their native liver), but most of these children have few symptoms and normal growth, according to a recent study report.
The Childhood Liver Disease Research and Education Network (ChiLDREN) is a consortium sponsored by the National Institutes for Health (NIH) and focused on the study of rare pediatric liver diseases. Within this consortium, the Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) study enrolls children and young adults with Alpha-1 Antitrypsin Deficiency (Alpha-1) from 16 centers in North America.
Jeffrey Teckman, MD, reported the results of the NIH ChiLDREN study at the annual Digestive Disease Week meeting in San Diego in May. This is the first Alpha-1 related data to come out of that study, which is partly funded by the Alpha-1 Foundation.
“I must emphasize that this study includes only Alpha-1 children who have liver disease,” Teckman said. “Most Alpha-1 children do not have liver disease, and these healthy Alpha-1 children are not included in this study. The 30% we found with portal hypertension only applies to the children with liver disease in this group. It does NOT apply to other Alpha-1 kids.”
The aim of the LOGIC study is to characterize the baseline clinical, laboratory and growth parameters of Alpha-1 children with liver disease and to evaluate the role of portal hypertension – high blood pressure in the liver’s portal vein, often caused by cirrhosis (scarring) of the liver.
Alpha-1 patients from birth to 25 years old with liver disease were eligible for the LOGIC study if they were diagnosed with liver disease associated with Alpha-1.
Of the 244 eligible subjects (91% ZZ and 9% SZ) with Alpha-1 liver disease enrolled between November 2007 and October 2011, a total of 181 still had their native liver and 63 (26%) had received liver transplants.
Among subjects with their native liver, 31% had portal hypertension.
Subjects with their native liver and portal hypertension were more likely to have jaundice and elevated total bilirubin. Jaundice was relatively rare in the study group (just 3%), but statistically greater in those with portal hypertension.
The overall conclusion of the study was that patients with Alpha-1 liver disease seen at North American care centers have a significant burden of portal hypertension (30% in those with their native liver), but generally have few symptoms and normal growth.
“This suggests that successful management of the health of children with Alpha-1 liver disease is likely to require regular physical examination and laboratory testing as part of follow-up care,” Teckman said. “Note that these findings are preliminary and could change as we get more results. Enrollment in the study and longitudinal assessments are ongoing.”
Digestive Disease Week is the largest gastroenterology meeting in the world. It is jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA) Institute, the American Society for Gastrointestinal Endoscopy and the Society for Surgery of the Alimentary Tract.
For more information about Childhood Liver Disease Research, please visit, www.childrennetwork.org
