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

Lemoine, C.; Bhardwaj, T.; Bass, L. M.; Superina, R. A.

J Pediatr Surg. 2016 Dec 6; 52(2):268-272


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+/-6mumol/L, P=0.03) and in the single PFIC-3 patient (821 to 11.2mumol/L), but not significantly in PFIC-2 patients (193+/-99 to 141+/-118mumol/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.

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