title: The challenges of liver transplantation in children with primary sclerosing cholangitis.
abstract: Primary sclerosing cholangitis (PSC) in children can progress to end-stage liver disease requiring liver transplantation. PSC poses many challenges beginning with evaluation and classification of ductal involvement and overlap syndromes, few options for medical management and unique risks in the post-transplant period. The construct that PSC may be an autoimmune disease is based on positive autoantibodies, association with inflammatory bowel disease, linkage to HLA type and overlap/autoimmune sclerosing cholangitis; however, PSC is not responsive to standard immunosuppression. Study of PSC and post-transplant outcomes in children may provide a unique background in which to study this challenging disease. This is particularly intriguing in the subset of patients diagnosed in the first decade of life, suggesting a strong link to predisposing genetic susceptibility and immune dysregulation. Long-term, multicenter effort is likely to be the only mechanism to study this rare disease in children and to improve outcomes in the future.
authors.affiliation: Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Pittsburgh of UPMC, Pediatrics, 4401 Penn Avenue, Pittsburgh, PA 15224, USA.
authors.forename: Veena L
authors.initials: VL
authors.lastname: Venkat
authors.forename: Sarangarajan
authors.initials: S
authors.lastname: Ranganathan
authors.forename: Rakesh
authors.initials: R
authors.lastname: Sindhi
mesh_terms: D001327:Autoimmune Diseases / Q000150:complications / Q000235:genetics / Q000276:immunology* / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D015209:Cholangitis, Sclerosing / Q000150:complications / Q000235:genetics / Q000276:immunology* / Q000601:surgery*; D058625:End Stage Liver Disease / Q000209:etiology / Q000601:surgery; D020022:Genetic Predisposition to Disease; D019693:Hepatitis, Autoimmune / Q000150:complications; D006801:Humans; D007223:Infant; D015212:Inflammatory Bowel Diseases / Q000150:complications; D016031:Liver Transplantation; D012008:Recurrence; D016896:Treatment Outcome
keywords: PSC; autoimmune liver disease; children; liver transplant; transplant outcome