Adult Liver Disease Prognostic Modelling for Long-term Outcomes in Biliary Atresia An Observational Cohort Study
Adult Liver Disease Prognostic Modelling for Long-term Outcomes in Biliary Atresia An Observational Cohort Study
复制标题
DOI：
10.1097/mpg.0000000000003116
复制
发表时间：
2021-07-01
影响因子：
2.9
通讯作者：
Samyn, Marianne
中科院分区：
文献类型：
--
作者：
Jain, Vandana;Burford, Charlotte;Samyn, Marianne
目的：评估预后评分系统对胆道闭锁（BA）存活的原生肝脏青少年的效用，以预测随后对肝移植（LT）的需求。方法：对1980-1996年间行Kasai门肠造口术（KP）并存活16年的397例BA患者进行单中心回顾性分析。16年（时间点16年）的实验室和临床变量用于计算(i) LT分配评分；终末期肝病模型[MELD/MELD-钠（Na）]和英国终末期肝病模型（UKELD）；（ii）梅奥原发性硬化性胆管炎风险评分（MayoPSC）和（iii）改良的儿科终末期肝病（PELD）评分。在最近的随访中，比较了16岁后需要肝移植的患者（16岁至16岁）和那些依靠天然肝脏存活的患者的评分。对12年（时间点12年）可用数据的患者进行额外的亚组分析。结果：16岁时MELD（受试者工作特征下面积[AUROC] 0.847）和UKELD （AUROC: 0.815）预测16岁时需要LT。没有证据表明MELD- na优于MELD。MELD >8.5和UKELD >47预测LT > 16年，敏感性分别为84%和79%，特异性分别为73%和73%。PELD的性能与MELD相似，但优于UKELD。MayoPSC显示预测>16年的准确性（AUROC为0.859），>评分为0.87，预测>16年的敏感性为85%，特异性为82%。在12年的时间点，MELD和MayoPSC预测了16年的LT。MELD、PELD和MayoPSC在12 - 16岁之间的变化与16岁时的LT相关。结论：成人LT分配评分可能有助于监测青少年BA的进展，但相关危险因素的遗漏限制了其在本队列中列出的效用。BA特异性预后评分将改善青少年BA的管理。
Objectives: To assess the utility of prognostic scoring systems for adolescents with biliary atresia (BA) surviving with native liver, for predicting the subsequent requirement for liver transplantation (LT). Methods: Single-centre retrospective analysis of 397 BA patients who received Kasai Portoenterostomy (KP) 1980-1996 and survived with the native liver at 16 years. Laboratory and clinical variables at 16 years (timepoint 16 years) were used to calculate (i) LT allocation scores; Model for End-Stage Liver Disease [MELD/MELD-sodium (Na)], and UK End-Stage Liver Disease (UKELD); (ii) Mayo Primary Sclerosing Cholangitis risk score (MayoPSC) and (iii) a modified Paediatric End-Stage Liver Disease (PELD) score. Scores were compared between patients requiring LT after 16 years of age (LT > 16 years), and those who survived with native liver, at the latest follow-up. Additional subgroup analysis for patients with data available at 12 years (timepoint 12 years). Results: MELD (area under the receiver operating characteristic [AUROC] 0.847) and UKELD (AUROC: 0.815) at 16 years of age predict the need for LT > 16 years. No advantage for MELD-Na over MELD was demonstrated. MELD >8.5 and UKELD >47 predicted LT > 16 years with 84% and 79% sensitivity and 73% and 73% specificity. PELD had a similar performance to MELD, but superiority to UKELD. MayoPSC revealed predictive accuracy for LT >16 years (AUROC 0.859), with a score of >0.87 predicting LT > 16 years with 85% sensitivity and 82% specificity. At timepoint 12 years, MELD and MayoPSC predicted LT >16 years. Change in MELD, PELD and MayoPSC between 12 and 16 years of age, was associated with LT >16 years. Conclusions: Adult LT allocation scores may help monitor progress in adolescent BA, but the omission of relevant risk factors limits their utility for listing in this cohort. A BA-specific prognostic score would improve the management of adolescent BA.