Gastroenterology Research

Researchers at UPMC Children’s Hospital of Pittsburgh have taken on multiple challenges in gastroenterology disorders. Pancreatitis, liver disease, biliary atresia, and inflammatory bowel disease are just a few of the many areas of focus.

With funding from the National Institutes of Health, Children’s gastroenterology laboratories provide leadership in: understanding the physiology of lipases – enzymes that digest fats – and, more specifically, the roles these proteins play in digestion and disease; characterizing the function and regulation of intestinal transporters for water and bile acids; and the pathophysiology and treatment of alpha-1-antitrypsin deficiency, the most common genetic disease of the liver in childhood.

Children’s gastroenterologists also head multiple clinical studies. Liver disease is under assault from Children’s pediatric gastroenterologists working to advance potential therapies that range from increasing our understanding of biliary atresia and acute liver failure, liver diseases often treated with liver transplantation, to transplanting hepatocytes as a possible cure for many childhood liver diseases. Clinical studies in pediatric inflammatory bowel disease are also a focus of Children’s gastroenterology researchers. These studies include participation in local and national registries to gather data about outcome and treatment of children with inflammatory bowel disease and an innovative and novel study to define the role of behavioral therapy in treating children with inflammatory bowel disease.

Children’s gastroenterology research is both local and international in scope, as the hospital has taken a leadership role in establishment of multicenter studies allowing collaboration and information sharing among experts throughout North America and beyond.

Researchers

Clinical Studies

Biliary Atresia Long-Term Observation: The BASIC Study

Biliary Atresia Study in Infants, Children and Adults

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Biliary Atresia Treatment with Maralixibat after Kasai Procedure: The EMBARK Study – Phase II

Study to Evaluate Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy

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Cholestasis Long-term Observation: The LOGIC Study

Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)

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Cholestasis Natural History Database: The PROBE Study

A Prospective Database of Infants with Cholestasis

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Cholestatic Liver Disease Treatment with Maralixibat: The RISE Study – Phase II

Study to Evaluate Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

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Crohn’s Disease Maintenance Therapy with Vedolizumab – Phase III

Study to Evaluate Vedolizumab Intravenous as Maintenance Therapy in Pediatric Subjects with Moderately to Severely Active Crohn’s Disease Who Achieved Clinical Response Following Open-Label Vedolizumab Intravenous Therapy

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Erosive Esophagitis Healing Maintenance Therapy with Protonix – Phase II

Study of Oral Pantoprazole in Pediatric Participants who Require Maintenance Therapy for Healed Erosive Esophagitis

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IBD ImproveCareNow Registry

Using Patient Data to Transform Care and Improve Outcomes for Children, Adolescents and Young Adults with Inflammatory Bowel Disease

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Liver Disease Scarring: The FORCE Study

FibroScan® in Pediatric Cholestatic Liver Disease Study

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Mitochondrial Hepatopathies Long-term Observation: The MITOHEP Study

Longitudinal Study of Mitochondrial Hepatopathies

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Nonalcoholic Fatty Liver/Steatohepatitis (NAFL/NASH) Observational Study

A 5-year Longitudinal Observational Study of Patients with Nonalcoholic Fatty Liver or Nonalcoholic Steatohepatitis

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Pancreatitis INSPPIRE 2 Study

Pediatric Longitudinal Cohort Study of Chronic Pancreatitis (INSPPIRE 2)

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Primary Sclerosing Cholangitis Observational Study

Prospective Observational Study of Primary Sclerosing Cholangitis in Children, a Protocol of the Childhood Liver Disease Research Network

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Ulcerative Colitis Maintenance Therapy with Vedolizumab – Phase III

Study to Evaluate Vedolizumab Intravenous as Maintenance Therapy in Pediatric Subjects with Moderately to Severely Active Ulcerative Colitis Who Achieved Clinical Response Following Open–Label Vedolizumab Intravenous Therapy

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Additional Researchers

Arjumand Ghazi, PhD