对非侵入性生物标志物进行纵向评估，以诊断和预测囊性纤维化相关肝病。
A longitudinal assessment of non-invasive biomarkers to diagnose and predict cystic fibrosis-associated liver disease.
机构信息
Division of Pediatric Gastroenterology, Nutrition, and Hepatology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Division of Pediatric Gastroenterology, Nutrition, and Hepatology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN, USA.
出版信息
J Cyst Fibros. 2020 Jul;19(4):546-552. doi: 10.1016/j.jcf.2020.05.002. Epub 2020 May 30.
BACKGROUND & AIMS: A practical, inexpensive, and non-invasive biomarker of liver fibrosis is needed as a reliable screening test for cystic fibrosis-associated liver disease (CFLD). Studies have shown the utility of AST to Platelet Ratio Index (APRI), fibrosis index based on 4 factors (FIB-4), and gamma-glutamyl transferase (GGT) as good biomarkers for identifying CFLD. The goal of the study was to evaluate the effectiveness of APRI, FIB-4, AST/ALT ratio, platelet count, GGT, and GGT platelet ratio (GPR) in predicting CFLD development.
METHODS
Data was collected from CF Foundation Patient Registry for patients aged 3-21 years at Johns Hopkins from January 1, 2002 to December 31, 2014. Collected data included demographic characteristics, presence of splenomegaly, hepatomegaly, ascites, and variceal bleeding, AST, ALT, GGT, platelet count, and FEV. The sensitivity and specificity of each biomarker were analyzed and reported by the area under receiver operating characteristic (AUROC) curve.
RESULTS
By the end of the study, 144 "healthy" CF, 12 CFLD, 19 CF-associated pulmonary disease (CFPD), and 4 CFLD with CFPD cases were identified. APRI scores were higher in CFLD, 0.85 versus 0.28 in "healthy" CF and 0.23 in CFPD groups (p<0.001). GPR had the highest AUROC curve at 0.91.
CONCLUSIONS
GPR, GGT, APRI score, and platelet count were potentially useful biomarkers while FIB-4 did not predict CFLD development. Cost-effectiveness studies are needed to analyze the utility of these biomarkers in clinical practice.
背景与目的
需要一种实用、廉价、非侵入性的肝纤维化生物标志物，作为囊性纤维化相关肝病（CFLD）的可靠筛查试验。研究表明，天冬氨酸转氨酶与血小板比率指数（APRI）、基于 4 个因素的纤维化指数（FIB-4）和γ-谷氨酰转移酶（GGT）作为识别 CFLD 的良好生物标志物是有用的。本研究的目的是评估 APRI、FIB-4、AST/ALT 比值、血小板计数、GGT 和 GGT 血小板比（GPR）在预测 CFLD 发展中的有效性。
方法
从 2002 年 1 月 1 日至 2014 年 12 月 31 日，从约翰霍普金斯 CF 基金会患者登记处收集年龄在 3-21 岁的 CF 患者的数据。收集的数据包括人口统计学特征、脾肿大、肝肿大、腹水和静脉曲张出血、AST、ALT、GGT、血小板计数和 FEV。通过接收者操作特征（ROC）曲线下面积（AUROC）分析和报告每种生物标志物的敏感性和特异性。
结果
在研究结束时，确定了 144 例“健康”CF、12 例 CFLD、19 例 CF 相关肺疾病（CFPD）和 4 例 CFLD 合并 CFPD 病例。CFLD 患者的 APRI 评分较高，为 0.85，而“健康”CF 患者和 CFPD 患者的评分分别为 0.28 和 0.23（p<0.001）。GPR 的 AUROC 曲线最高，为 0.91。
结论
GPR、GGT、APRI 评分和血小板计数可能是有用的生物标志物，而 FIB-4 不能预测 CFLD 的发展。需要进行成本效益研究，以分析这些生物标志物在临床实践中的应用。
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