Liver Disease in Children

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Research in the lab and at the bedside offer new hope for young patients with biliary atresia, Alagille syndrome and other chronic liver disorders.


Kathleen M. Loomes, MD Kathleen M. Loomes, MD Physicians and scientists at Children’s Hospital of Philadelphia (CHOP) have been actively researching pediatric liver disease for more than two decades with the goals of better understanding the causes of liver diseases, developing effective treatments, and improving quality of life and long-term outcomes for children with these disorders.

Since most pediatric liver diseases are rare, collaboration among leading pediatric centers is incredibly important to move the field forward. The Fred and Suzanne Biesecker Pediatric Liver Center at CHOP actively supports basic, clinical and translation liver research, with a focus on biliary atresia, and other developmental liver disorders such as Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). Many children with liver disease will require medication or surgery to treat their disorder during childhood; some will also need a liver transplant.

Pediatric liver disease care at CHOP

CHOP has one of the largest and most active pediatric liver programs in the country with more than a dozen world-renowned hepatologists, gastroenterologists, surgeons and researchers. A multidisciplinary team of pediatric liver specialists evaluates and manages the care of children with all forms of liver disease, providing patients with the latest diagnostic tools, evidence-based treatment and optimal longterm outcomes.

The clinical team works closely with researchers at CHOP, the Hospital of the University of Pennsylvania (HUP) and other centers to rapidly move important discoveries from bench to bedside to benefit young patients.

From Kasai to today

CHOP has been caring for children with biliary atresia since the 1970s. Biliary atresia (BA) is a progressive fibro inflammatory disorder of the bile ducts presenting in early infancy. Surgical intervention – specifically the Kasai hepatoportoenterostomy – can successfully restore bile drainage, but in many cases progressive liver fibrosis will occur, resulting in complications of portal hypertension and ultimately, cirrhosis. Biliary atresia is the leading indication for liver transplant in children, with the majority of infants with the disorder requiring a transplant before 18 years of age.

The etiology of BA is unknown, but is hypothesized to be multifactorial, involving genetic susceptibility, an environmental exposure such as toxin or infection, and progressive inflammation and fibrosis. Active research at CHOP and around the world seeks to understand the underlying pathophysiology of BA to improve care for these vulnerable patients.

Pediatric Liver Transplant Program at CHOP

Elizabeth B. Rand, MD Elizabeth B. Rand, MD When liver transplant is needed, CHOP’s Pediatric Liver Transplant Program, led by Elizabeth B. Rand, MD, provides optimal care for children before, during and after the transplant surgery. Our surgeons perform an average of 15 to 20 pediatric liver transplants a year at CHOP, and more than 100 at the adjacent Hospital of the University of Pennsylvania (HUP). (See page 9.)

Since 1995, CHOP and HUP have conducted a joint liver transplant program. This affiliation allows CHOP to draw upon the experience of one of the nation’s largest adult programs and partner to conduct clinical and translational research throughout the lifespan. To date, CHOP’s program has performed more than 350 liver transplants, and our survival rates consistently exceed national averages.

Pediatric Liver Disease Clinical Care Programs at CHOP include:

  • Biliary Atresia Clinical Care Program
  • Alagille Syndrome Clinical Care Program
  • Combined Liver/Kidney Program – This collaborative program is focused on children with a number of genetic disorders that affect both liver and kidney development.
  • FORWARD (FOntan Rehabilitation, Wellness, Activity and Resilience Development) – This multidisciplinary program cares for children with single ventricle circulation, many of whom have liver involvement.
  • Non-alcoholic Fatty Liver Disease Clinic
  • Viral Hepatitis Clinical Care Program
  • Liver Transplant Program

For more information about any of our liver programs, visit

Kathleen M. Loomes, MD, is co-director of the Fred and Suzanne Biesecker Pediatric Liver Center, a leader in the Liver Transplant Program and a pediatric gastroenterologist in the Division of Gastroenterology, Hepatology and Nutrition at CHOP.

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