Progression characteristics of Biliary Atresia during the Waiting Period for Liver Transplantation: A Single-Center Observational Study

Chenyu Yang^1,2Jiqiang Hu^1,2Bingqian Tan^1,2Qiang Xiong^1,2Ruijue Wang^1,2Jianyang Hu^1,2Ying Le^1,2Xiaoke Dai^1,2*Mingman Zhang^1,2*

• ¹Children‘s Hospital of Chongqing Medical University, Chongqing, China
• ²Chongqing Medical University, Chongqing, China

Background Biliary atresia (BA) is a progressive, fibro-obliterative cholangiopathy of infancy that inevitably leads to cirrhosis and liver failure without intervention. While the rapid progression of BA is widely recognized, the precise dynamics of its natural history remain poorly quantified due to the confounding effects of surgical intervention. Aims This study aimed to characterize the natural progression of BA cirrhosis by analyzing the trajectory of clinical, laboratory, and histological changes during the unique window between diagnostic confirmation by intraoperative cholangiography and subsequent liver transplantation in children who did not undergo Kasai portoenterostomy. Methods In this single-center, observational study, we analyzed 153 children with cholangiography-confirmed BA who underwent living donor LT without primary Kasai portoenterostomy. The pathological progression of cholestatic cirrhosis and laboratory parameters such as hematological examination and liver function in children with hepatic cholestatic cirrhosis at the two time points were compared to find factors that can predict the progression of BA cirrhosis. Results The cohort comprised 153 children with cholangiography-confirmed BA who underwent liver transplantation. The mean age at diagnosing BA was 78.64 ± 31.43 days. The mean interval from exploration to liver transplantation (disease progression time) was 83.36 ± 28.37 days. Within this short period, 77.12% (118/153) of children progressed to end-stage (Stage IV) cirrhosis. Longitudinal analysis revealed a significant increase in white blood cells (WBC, 3.09±5.31×10⁹/L), neutrophil percentage (NE%, 7.42±13.77%), total bilirubin (TBIL, 121.9 ± 99.51 μmol/L), and serum ammonia (SA, 6.97±25.59 μmol/L), alongside a significant decrease in hemoglobin (HB; -4.7±15.83 g/L), platelets (PLT; -148.50±154.40× 10⁹/L), and albumin (ALB; -4.60±7.28 g/L) (all p < 0.05). A predictive model incorporating initial fibrosis stage, waiting time, and changes in NE% and SA demonstrated good accuracy (75.96% in training set, 72.00% in validation set) for forecasting cirrhosis progression. Conclusion This study provides unprecedented quantification of the rapid natural progression of BA cirrhosis, revealing a critical 3 month window of clinical deterioration. The dynamic changes in routinely available laboratory markers, particularly neutrophil percentage and ammonia, offer clinically accessible indicators for risk stratification and monitoring, forming a practical "dashboard" for managing BA patients awaiting transplantation in resource-varied settings.