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基于肝胆超声特征的胆道闭锁筛查列线图的开发与评估

Development and Assessment of Screening Nomogram for Biliary Atresia Based on Hepatobiliary Ultrasonographic Features.

作者信息

Dai Shu Yang, Sun Yu Qi, Wu Ying, Chen Gong, Sun Song, Dong Rui, Zheng Shan

机构信息

Shanghai Key Laboratory of Birth Defect, Department of Pediatric Surgery, Children's Hospital of Fudan University, Shanghai, China.

Key Laboratory on Public Health, Safety of the Ministry of Education, Department of Biostatistics, School of Public Health, Fudan University, Shanghai, China.

出版信息

Front Pediatr. 2021 May 17;9:625451. doi: 10.3389/fped.2021.625451. eCollection 2021.

Abstract

Biliary atresia (BA) is a rare neonatal liver disease of which the early diagnosis remains a challenge for clinicians. Our center has established a nomogram diagnostic model based on clinical characteristics and liver function characteristics. We aim to develop and validate a nomogram that includes additional ultrasound and finds hepatobiliary abnormality with better BA early screening performance. In this single-center, retrospective cohort analysis, 1,001 neonatal obstructive jaundice (NOJ) patients between 2012 and 2015 were enrolled. Multivariable analysis was used to identify clinical characteristics, laboratory liver function characteristics, and ultrasonic features that may early screen BA. A nomogram was developed to predict the probability of BA using multiple logistic regression analysis. This nomogram was subsequently validated using another cohort of 501 NOJ patients between 2015 and 2017. Calibration curve analysis and decision curve analyses were performed to evaluate and interpret the nomogram's clinical benefits. Gender, direct bilirubin (DB), alkaline phosphatase (ALP), gamma-glutamyl transpeptidase (GGT), fasting gallbladder visibility, fasting gallbladder filling, and common bile duct visibility were found to have profound statistical significance between the BA and non-BA groups ( < 0.05). The significant features were used to build the nomogram. The area under the receiver operating characteristic (ROC) curve (AUC) value of the novel nomogram (0.87) was superior to those of the former nomogram (0.83) and GGT alone (0.81) in the prediction of BA. The calibration curve revealed a close resemblance between the predicted and actual BA probabilities. Also, the net benefit from the decision curve analysis (DCA) of the nomogram (0.54) was superior to those of the former nomogram (0.49) and GGT alone (0.45) at 80% of threshold possibility. The nomogram has demonstrated better performance for BA screening by including additional information of the US finding, holding a promising future as a non-invasive method for BA patients.

摘要

胆道闭锁(BA)是一种罕见的新生儿肝脏疾病,早期诊断对临床医生来说仍是一项挑战。我们中心基于临床特征和肝功能特征建立了一种列线图诊断模型。我们旨在开发并验证一种包含额外超声检查结果且能发现肝胆异常的列线图,以具备更好的BA早期筛查性能。在这项单中心回顾性队列分析中,纳入了2012年至2015年间的1001例新生儿梗阻性黄疸(NOJ)患者。采用多变量分析来确定可能用于早期筛查BA的临床特征、实验室肝功能特征及超声特征。利用多元逻辑回归分析开发了一种列线图以预测BA的概率。随后,使用2015年至2017年间的另一组501例NOJ患者对该列线图进行验证。进行校准曲线分析和决策曲线分析以评估和解释列线图的临床益处。结果发现,BA组与非BA组在性别、直接胆红素(DB)、碱性磷酸酶(ALP)、γ-谷氨酰转肽酶(GGT)、空腹胆囊可见性、空腹胆囊充盈情况及胆总管可见性方面具有显著统计学意义(<0.05)。利用这些显著特征构建了列线图。在预测BA方面,新型列线图的受试者操作特征(ROC)曲线下面积(AUC)值(0.87)优于先前的列线图(0.83)和单独的GGT(0.81)。校准曲线显示预测的BA概率与实际概率非常接近。此外,在阈值可能性为80%时,列线图决策曲线分析(DCA)的净效益(0.54)优于先前的列线图(0.49)和单独的GGT(0.45)。该列线图通过纳入超声检查结果的额外信息,在BA筛查方面表现出更好的性能,作为一种针对BA患者的非侵入性方法具有广阔前景。

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