Outcomes following partial external biliary diversion in patients with progressive familial intrahepatic cholestasis

Caroline Lemoine, Tanya Bhardwaj, Lee M Bass, Riccardo A Superina*

*Corresponding author for this work

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Background/purpose 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 ± 6 μmol/L, P = 0.03) and in the single PFIC-3 patient (821 to 11.2 μmol/L), but not significantly in PFIC-2 patients (193 ± 99 to 141 ± 118 μmol/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). Conclusion 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. Level of evidence Level IV.

Original languageEnglish (US)
Pages (from-to)268-272
Number of pages5
JournalJournal of Pediatric Surgery
Volume52
Issue number2
DOIs
StatePublished - Feb 1 2017

Fingerprint

Bile Acids and Salts
Transplantation
Transplants
Survival
Progressive familial intrahepatic 1 Cholestasis
Liver
Liver Diseases
Serum

Keywords

  • Bile acids
  • Liver transplantation
  • Partial external biliary drainage
  • Progressive familial intrahepatic cholestasis (PFIC) C

ASJC Scopus subject areas

  • Surgery
  • Pediatrics, Perinatology, and Child Health

Cite this

@article{316c63f1f0f74a648044173ef5ca498b,
title = "Outcomes following partial external biliary diversion in patients with progressive familial intrahepatic cholestasis",
abstract = "Background/purpose 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 ± 6 μmol/L, P = 0.03) and in the single PFIC-3 patient (821 to 11.2 μmol/L), but not significantly in PFIC-2 patients (193 ± 99 to 141 ± 118 μmol/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). Conclusion 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. Level of evidence Level IV.",
keywords = "Bile acids, Liver transplantation, Partial external biliary drainage, Progressive familial intrahepatic cholestasis (PFIC) C",
author = "Caroline Lemoine and Tanya Bhardwaj and Bass, {Lee M} and Superina, {Riccardo A}",
year = "2017",
month = "2",
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pages = "268--272",
journal = "Journal of Pediatric Surgery",
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TY - JOUR

T1 - Outcomes following partial external biliary diversion in patients with progressive familial intrahepatic cholestasis

AU - Lemoine, Caroline

AU - Bhardwaj, Tanya

AU - Bass, Lee M

AU - Superina, Riccardo A

PY - 2017/2/1

Y1 - 2017/2/1

N2 - Background/purpose 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 ± 6 μmol/L, P = 0.03) and in the single PFIC-3 patient (821 to 11.2 μmol/L), but not significantly in PFIC-2 patients (193 ± 99 to 141 ± 118 μmol/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). Conclusion 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. Level of evidence Level IV.

AB - Background/purpose 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 ± 6 μmol/L, P = 0.03) and in the single PFIC-3 patient (821 to 11.2 μmol/L), but not significantly in PFIC-2 patients (193 ± 99 to 141 ± 118 μmol/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). Conclusion 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. Level of evidence Level IV.

KW - Bile acids

KW - Liver transplantation

KW - Partial external biliary drainage

KW - Progressive familial intrahepatic cholestasis (PFIC) C

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DO - 10.1016/j.jpedsurg.2016.11.021

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JF - Journal of Pediatric Surgery

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