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

Abstract

Background/purposePFIC 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. MethodsAll 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. ResultsThirty-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). ConclusionPEBD 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 evidenceLevel IV.