Clinical characteristics of neonatal cholestasis in a tertiary hospital and the development of a novel prediction model for mortality

Background Few studies have described the aetiologies of neonatal cholestasis, and the overall neonatal cholestasis-related mortality (NCM) rate is unclear. We investigated the aetiology and outcome of neonatal cholestasis in a ter-tiary hospital and developed an NCM prediction model for these patients.& nbsp;Methods Patients aged < 100 days with serum direct bilirubin (DB) levels of > 1.0 mg/dL were retrospectively screened. Diagnostic and laboratory data during the 8-week follow-up period after enrolment between 2005 and 2020 were extracted digitally, and medical charts were reviewed manually by clinicians. Logistic regression was used to derive a prediction model for the 1-year mortality outcome of neonatal cholestasis, and performance evaluation and external validation were conducted for the NCM prediction model.& nbsp;Findings We enrolled 4028 neonates with DB of > 1.0 mg/dL at least once. Prematurity and birth injury (35.4%), complex heart anomalies (18.6%), liver diseases (11.4%), and gastrointestinal anomalies (9.2%) were the most com-mon aetiologies; 398 (9.9%) patients died before one year of age. The peak value of DB was positively correlated to the 1-year mortality rate. In the multivariate analysis, simple laboratory indices, including platelet, prothrombin time, aspartate aminotransferase, albumin, direct bilirubin, creatinine, and C-reactive protein, were independent predictors of 1-year mortality outcome of complete-case subjects. Using these laboratory indices, a logistic regression-based NCM prediction model was constructed. It showed acceptable performances on discrimination (area under the curve, 0.916), calibration (slope, 1.04) and Brier scoring (0.072). The external validation of the sample (n = 920) from two other centres also revealed similar performance profiles of the NCM model.& nbsp;Interpretation Various aetiologies of neonatal cholestasis were identified in a tertiary hospital, resulting in unfavourable outcomes of a large proportion. The NCM prediction model may have the potential to help clinicians to be aware of high-risk neonatal cholestasis.& nbsp;Copyright (C) 2022 The Author(s). Published by Elsevier B.V.

Automated summary

This study investigated the etiology and outcome of neonatal cholestasis in a tertiary hospital and developed a prediction model for neonatal cholestasis-related mortality. The study found that prematurity, birth injury, complex heart anomalies, liver diseases, and gastrointestinal anomalies were the most common causes of neonatal cholestasis. A logistic regression-based prediction model using simple laboratory indices showed acceptable performance in predicting one-year mortality outcome. The model was externally validated in other centers and exhibited similar performance profiles.