Arab J Gastroenterol. 2026 Jul 8:S1687-1979(26)00065-1. doi: 10.1016/j.ajg.2026.06.001. Online ahead of print.
ABSTRACT
BACKGROUND: PFIC comprises a heterogeneous group of rare genetic cholestatic liver disorders that lead to progressive liver damage, cirrhosis, and the need for liver transplantation. Understanding its clinical course and survival outcomes is essential for improving management, particularly in resource-limited settings like Iran. This study aimed to evaluate the clinical characteristics, survival outcomes, and predictors of mortality in children with PFIC.
METHODS: This cohort study was conducted as part of the Shiraz Pediatric Liver Cirrhosis Cohort Study (SPLCCS), initiated in 2018. Data on demographic characteristics, clinical features, and laboratory findings were collected. Cox regression analysis was used to identify risk factors for mortality.
RESULTS: A total of 100 children with PFIC were included, with a mortality rate of 35%. Younger age at diagnosis and higher Pediatric End-Stage Liver Disease (PELD) scores were significantly associated with increased mortality risk. Liver transplantation (LTx) was performed in 50% of cases, with post-transplant survival rates of 74%, 70%, and 66% at 12, 24, and 60 months, respectively. Elevated white blood cell (WBC) counts, aspartate aminotransferase (AST), and direct bilirubin levels were also significant predictors of mortality.
CONCLUSIONS: Early diagnosis and timely liver transplantation are critical for improving survival in children with PFIC. Higher PELD scores, WBC counts, AST, and direct bilirubin levels are strongly associated with increased mortality risk, highlighting the need for early intervention and consistent monitoring.
PMID:42420132 | DOI:10.1016/j.ajg.2026.06.001