title: Outcomes following partial external biliary diversion in patients with progressive familial intrahepatic cholestasis.
abstract: OBJECTIVE
  PFIC is a family of bile acid (BA) transport disorders that may result in serious liver disease requiring transplantation. We reviewed our experience with PEBD as a method to improve liver function and avoid transplantation.
  
  
  METHODS
  All patients with PFIC were reviewed. Outcomes included changes in serum BA, conversion to ileal bypass (IB), and survival without transplantation. Statistics were obtained using paired t-test and Wilcoxon test.
  
  
  RESULTS
  Thirty-five patients with PFIC were identified. Data were available in 24. Twenty-four children (12 males) underwent PEBD: 10 PFIC-1, 13 PFIC-2, and one PFIC-3. BA levels decreased in PFIC-1 patients (1724±3215 to 11±6μmol/L, P=0.03) and in the single PFIC-3 patient (821 to 11.2μmol/L), but not significantly in PFIC-2 patients (193±99 to 141±118μmol/L, P=0.15). Seven patients were converted to IB. There were no significant changes in BA levels following conversion. Five-year transplant-free survival was 100% in PFIC-1 and PFIC-3, but only 38% (5/13) in PFIC-2 (P=0.004).
  
  
  CONCLUSIONS
  PEBD is an effective procedure to reduce total BA levels and improve symptoms in PFIC patients. However, it appears to be less efficacious in the PFIC-2 group. The higher BA levels could contribute to ongoing liver damage, and thus a higher transplant rate in PFIC-2 patients.
  
  
  METHODS
  Level IV.
authors.affiliation: Division of Transplant Surgery, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
authors.forename: Caroline
authors.initials: C
authors.lastname: Lemoine
authors.affiliation: Division of Transplant Surgery, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
authors.forename: Tanya
authors.initials: T
authors.lastname: Bhardwaj
authors.affiliation: Division of Gastroenterology, Hepatology, and Nutrition, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
authors.forename: Lee M
authors.initials: LM
authors.lastname: Bass
authors.affiliation: Division of Transplant Surgery, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA. Electronic address: RSuperina@luriechildrens.org.
authors.forename: Riccardo A
authors.initials: RA
authors.lastname: Superina
mesh_terms: D018435:ATP Binding Cassette Transporter, Subfamily B / Q000172:deficiency*; D000714:Anastomosis, Surgical; D001662:Biliary Tract Surgical Procedures / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D002780:Cholestasis, Intrahepatic / Q000601:surgery*; D005260:Female; D005704:Gallbladder / Q000601:surgery; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007583:Jejunum / Q000601:surgery; D008297:Male; D012189:Retrospective Studies; D054047:Surgical Stomas; D016896:Treatment Outcome
chemical_list: D018435:ATP Binding Cassette Transporter, Subfamily B
keywords: Bile acids; Liver transplantation; Partial external biliary drainage; Progressive familial intrahepatic cholestasis (PFIC) C