Caroline Lemoine1, Tanya Bhardwaj1, Lee M Bass2, Riccardo A Superina3. 1. Division of Transplant Surgery, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA. 2. Division of Gastroenterology, Hepatology, and Nutrition, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA. 3. Division of Transplant Surgery, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA. Electronic address: RSuperina@luriechildrens.org.
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±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). 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.
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-1patients (1724±3215 to 11±6μmol/L, P=0.03) and in the single PFIC-3patient (821 to 11.2μmol/L), but not significantly in PFIC-2patients (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). CONCLUSION: PEBD is an effective procedure to reduce total BA levels and improve symptoms in PFICpatients. 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-2patients. LEVEL OF EVIDENCE: Level IV.
Authors: Irena Jankowska; Joanna Pawłowska; Marek Szymczak; Hor Ismail; Dorota Broniszczak; Joanna Cielecka-Kuszyk; Piotr Socha; Dorota Jarzębicka; Piotr Czubkowski Journal: Am J Case Rep Date: 2021-07-20
Authors: Paula M Hertel; Laura N Bull; Richard J Thompson; Nathan P Goodrich; Wen Ye; John C Magee; Robert H Squires; Lee M Bass; James E Heubi; Grace E Kim; Sarangarajan Ranganathan; Kathleen B Schwarz; Molly A Bozic; Simon P Horslen; Matthew S Clifton; Yumirle P Turmelle; Frederick J Suchy; Riccardo A Superina; Kasper S Wang; Kathleen M Loomes; Binita M Kamath; Ronald J Sokol; Benjamin L Shneider Journal: J Pediatr Gastroenterol Nutr Date: 2021-08-01 Impact factor: 3.288