{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T22:18:29Z","timestamp":1793053109685,"version":"4.5.0"},"reference-count":23,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"4","license":[{"start":{"date-parts":[[2023,3,30]],"date-time":"2023-03-30T00:00:00Z","timestamp":1680134400000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/ http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":[],"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>\n                      Bile salt export pump (\n                      <jats:italic toggle=\"yes\">ABCB11<\/jats:italic>\n                      ) deficiency [Progressive familial intrahepatic cholestasis (PFIC2)] is the most common genetic cause of PFIC and is associated with pruritus and progressive liver disease. Surgical biliary diversion or pharmacological [ileal bile acid transporter inhibitor (IBATi)] approaches can be used to block the recirculation of bile acids to the liver. There is a paucity of detailed data on the natural history and, in particular, the longitudinal evolution of bile acid levels to predict treatment response. Cross-sectional data from large international consortia suggested a maximum cutoff value of bile acids after the intervention to predict a successful outcome.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>\n                      This retrospective, single-center, cohort study included all patients with confirmed biallelic pathogenic\n                      <jats:italic toggle=\"yes\">ABCB11<\/jats:italic>\n                      genotype PFIC2 treated at our institution with \u22652 years follow-up. The outcomes of interventions and predictors of long-term health were analyzed.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Forty-eight cases were identified with PFIC2. Eighteen received partial external biliary diversion (PEBD) surgery, and 22 patients underwent liver transplantation. Two patients developed HCC and 2 died. Improved survival with native liver was closely associated with genotype, complete normalization of serum bile acids following PEBD, and alleviation of pruritus. Persistence of mild-to-moderate elevation of bile acids or a secondary rise following normalization was associated with liver disease progression and led to transplantation, suggesting that any prolonged elevation of bile acids worsens the chance of native liver survival. Higher-grade fibrosis at the time of PEBD was not associated with reduced long-term native liver survival. Patients with PFIC2 benefit from PEBD even at a stage of advanced fibrosis.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Serum bile acid levels are an early predictor of treatment response and might serve as the gold standard in the evaluation of novel therapies including IBATi.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1097\/hc9.0000000000000092","type":"journal-article","created":{"date-parts":[[2023,3,30]],"date-time":"2023-03-30T14:00:30Z","timestamp":1680184830000},"update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":12,"title":["Native liver survival in bile salt export pump deficiency: results of a retrospective cohort study"],"prefix":"10.1097","volume":"7","author":[{"given":"Eva-Doreen","family":"Pfister","sequence":"first","affiliation":[{"name":"Division of Paediatric Gastroenterology and Hepatology, Hannover Medical School, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Veronika K","family":"Jaeger","sequence":"additional","affiliation":[{"name":"Institute of Epidemiology and Social Medicine, University of Muenster, Muenster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9","family":"Karch","sequence":"additional","affiliation":[{"name":"Institute of Epidemiology and Social Medicine, University of Muenster, Muenster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Denys","family":"Shay","sequence":"additional","affiliation":[{"name":"Institute of Epidemiology and Social Medicine, University of Muenster, Muenster, Germany"},{"name":"Department of Epidemiology, Harvard T.H. 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