Systematic Review and Meta-Analysis: Partial External Biliary Diversion in Progressive Familial Intrahepatic Cholestasis

Verkade, H. J., Thompson, R. J., Arnell, H., Fischler, B., Gillberg, P-G., Mattsson, J. P., Torfgård, K. & Lindström, E., 2020, In : Journal of Pediatric Gastroenterology and Nutrition.

Research output: Contribution to journalArticleAcademicpeer-review

  • Henkjan J Verkade
  • Richard J Thompson
  • Henrik Arnell
  • Björn Fischler
  • Per-Göran Gillberg
  • Jan P Mattsson
  • Kristina Torfgård
  • Erik Lindström

OBJECTIVES: We assessed available data on impact of partial external biliary diversion (PEBD) surgery on clinical outcomes in patients with progressive familial intrahepatic cholestasis (PFIC).

METHODS: We performed a systematic literature review (PubMed) and meta-analysis to evaluate relationships between liver biochemistry parameters (serum bile acids, bilirubin, and alanine aminotransferase [ALT]) and early response (pruritus improvement) or long-term outcomes (need for liver transplant) in patients with PFIC who underwent PEBD.

RESULTS: Searches identified 175 publications before September 2018; 16 met inclusion criteria. Receiver operating characteristic (ROC) analysis examined ability of liver biochemistry parameters to discriminate patients who demonstrated early and long-term response to PEBD from those who did not. Regarding pruritus improvement in 155 included patients in aggregate, 104 (67%) were responders, 14 (9%) had partial response, and 37 (24%) were non-responders. In ROC analyses of individual patient data, post-PEBD serum concentration of bile acids, in particular, could discriminate responders from non-responders for pruritus improvement (area under the curve, 0.99; p < 0.0001; n = 42); to a lesser extent, this was also true for bilirubin (0.87; p = 0.003; n = 31), whereas ALT could not discriminate responders from non-responders for pruritus improvement (0.74; p = 0.06; n = 28). Reductions from pre-PEBD values in serum bile acid concentration (0.89; p = 0.0003; n = 32) and bilirubin (0.98; p = 0.002; n = 18) but not ALT (0.62; p = 0.46; n = 18) significantly discriminated decreased aggregate need for liver transplant.

CONCLUSIONS: Changes in bile acids seem particularly useful in discriminating early and long-term post-PEBD outcomes and may be potential biomarkers of response to interruption of enterohepatic circulation in patients with PFIC.

Original languageEnglish
JournalJournal of Pediatric Gastroenterology and Nutrition
Publication statusE-pub ahead of print - 2020

ID: 125791310