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 language | English (US) |
|---|---|
| Pages (from-to) | 268-272 |
| Number of pages | 5 |
| Journal | Journal of Pediatric Surgery |
| Volume | 52 |
| Issue number | 2 |
| DOIs | |
| State | Published - Feb 1 2017 |
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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
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Outcomes following partial external biliary diversion in patients with progressive familial intrahepatic cholestasis. / Lemoine, Caroline; Bhardwaj, Tanya; Bass, Lee M; Superina, Riccardo A.
In: Journal of Pediatric Surgery, Vol. 52, No. 2, 01.02.2017, p. 268-272.Research output: Contribution to journal › Article
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
UR - http://www.scopus.com/inward/record.url?scp=85007424280&partnerID=8YFLogxK
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U2 - 10.1016/j.jpedsurg.2016.11.021
DO - 10.1016/j.jpedsurg.2016.11.021
M3 - Article
VL - 52
SP - 268
EP - 272
JO - Journal of Pediatric Surgery
JF - Journal of Pediatric Surgery
SN - 0022-3468
IS - 2
ER -