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Outcomes of pediatric liver transplantation for progressive familial intrahepatic cholestasis
Anu K Vasudevan1, Naresh Shanmugam1, Ashwin Rammohan1
1The Institute of Liver Disease and Transplantation, Dr.Rela Institute & Medical Centre, Bharath Institute of Higher Education & Research, Chennai, India.
Insights
Liver transplantation (LT) outcomes for Progressive Familial Intrahepatic Cholestasis (PFIC) types 1-4 were analyzed. While overall outcomes are excellent, PFIC-1 patients show poorer growth and higher complication rates, potentially improved with Total Internal Biliary Diversion (TIBD).
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Transplant Surgery
Background:
- Progressive Familial Intrahepatic Cholestasis (PFIC) comprises inherited liver disorders with unclear clinical trajectories and transplant outcomes.
- Liver transplantation (LT) is a critical intervention for end-stage liver disease in PFIC.
- This study investigates the outcomes of LT across different PFIC subtypes.
Purpose of the Study:
- To compare early and long-term outcomes of LT in children with PFIC types 1-4.
- To identify differences in outcomes and management strategies among PFIC subtypes.
- To evaluate the impact of LT on growth and complications in PFIC patients.
Main Methods:
- A decade of prospectively collected data (2011-2022) from children with PFIC undergoing LT was analyzed.
- Patients were categorized into PFIC 1, 2, 3, and 4 groups.
- Outcomes including growth, survival, morbidity, and specific complications were compared.
Main Results:
- Sixty children underwent LT for PFIC (13 PFIC-1, 11 PFIC-2, 31 PFIC-3, 5 PFIC-4).
- PFIC-1 patients experienced significantly poorer catch-up growth (44.4%) and higher rates of post-LT diarrhea (46.1%) and graft steatohepatitis (23%).
- Overall 1- and 5-year patient survival rates were high across groups (PFIC-1: 69.2%, PFIC-2: 81.8%, PFIC-3: 96.8%, PFIC-4: 100%).
Conclusions:
- LT offers excellent overall outcomes for PFIC, but PFIC-1 presents unique challenges.
- Poorer growth and complications in PFIC-1 may be mitigated by incorporating Total Internal Biliary Diversion (TIBD) during LT.
- This study provides valuable insights into managing PFIC subtypes post-liver transplantation.
Background:
Progressive familial intrahepatic cholestasis (PFIC) is a heterogenous group of inherited hepatocellular disorders and the clinical aspects, role of liver transplantation (LT), and its outcomes remain largely unelucidated. We present our data of LT for each type of PFIC and compare their early, and long-term outcomes, highlighting their individual differences and management strategies.
Methods:
Prospectively collected data over a decade (2011-2022) of children with PFIC who underwent LT was analyzed. The groups (PFIC 1-4) were compared with regard to early and long-term outcomes including attainment of catch-up growth.
Results:
Of 60 children with PFIC who underwent LT, 13, 11, 31 & 5 were of PFIC 1, 2, 3 & 4, respectively. There were no significant differences in gender, PELD scores, BMI, type of grafts, cold and warm ischemia times, intraoperative blood loss, and morbidity among the groups. Post-LT chronic diarrhea was observed in 6 (46.1%) children with PFIC-I, and of them, 3 (23%) developed graft steatohepatitis. Three of these children underwent total internal biliary diversion (TIBD) and on 1-year follow-up, their graft steatosis resolved and they attained catch-up growth. Catch-up growth was significantly poorer in the PFIC1 group (44.4% vs. 88%, 90%, 100% p < .001). Overall 1- and 5-year patient survival of the four PFIC groups (1-4) were 69.2%, 81.8%, 96.8%, 100% & 69.2%, 81.8%, 96.8%, 100%, respectively.
Conclusion:
Ours is the largest to-date series of LT for PFIC illustrating their short- and long-term outcomes. While the results for the whole cohort were excellent, those after LT for PFIC1 was relatively poorer as reflected by catch-up growth, graft steatosis, and post-LT diarrhea, which can be optimized by the addition of TIBD during LT.
