背景:囊性纤维化(CF)中的结节性肝(NOD)提示晚期CF肝病(aCFLD);在检测到超声NOD后,对肝病(LD)的进展知之甚少。
方法:临床,实验室,和超声(US)数据来自CFLD研究参与者在筛查或随访时将NOD与正常(NL)进行比较。线性混合效应模型用于LD进展的危险因素,Kaplan-Meier估计用于事件发生时间。
结果:54例NOD患儿(22例筛查,32次随访)和112NL进行了评估。用力呼气量的基线(BL)和轨迹,强制肺活量,NOD与NL的身高/BMIz评分相似.BL时NOD中的血小板较低(250比331×103/微升;p<0.001),NL中的血小板减少8600/年比2500。平均AST与血小板比率指数(1.1vs0.4;p<0.001),纤维化-4指数(0.4vs0.2,p<0.001),BL时NOD的脾脏大小z评分(SSZ)[1.5vs0.02;p<0.001]较高;NOD中SSZ增加0.5单位/年,NL中增加0.1单位/年。通过瞬时弹性成像的中位肝硬度(LSM)在NOD中较高(8.2kPa,IQR6-11.8)与NL(5.3,4.2-7,p<0.0001)。超过6.3年的随访(1.3-10.3),6NOD有食管静脉曲张(10年累积发生率:20%;95%CI:0.0%,40.0%),2有静脉曲张出血,2例接受肝移植;无腹水或肝性脑病。无NL经历肝脏相关事件。
结论:NOD比NL更快地发展为临床上明显的门脉高压,而没有较差的生长或肺部疾病。
Nodular liver (NOD) in cystic fibrosis (CF) suggests advanced CF liver disease (aCFLD); little is known about progression of liver disease (LD) after detection of sonographic NOD.
Clinical, laboratory, and ultrasound (US) data from Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in CFLD Study participants with NOD at screening or follow-up were compared with normal (NL). Linear mixed effects models were used for risk factors for LD progression and Kaplan-Meier estimator for time-to-event.
54 children with NOD (22 screening, 32 follow-up) and 112 NL were evaluated. Baseline (BL) and trajectory of forced expiratory volume, forced vital capacity, height/BMI z-scores were similar in NOD vs NL. Platelets were lower in NOD at BL (250 vs 331×103/microL; p < 0.001) and decreased by 8600/year vs 2500 in NL. Mean AST to Platelet Ratio Index (1.1 vs 0.4; p < 0.001), Fibrosis-4 Index (0.4 vs 0.2, p < 0.001), and spleen size z-score (SSZ) [1.5 vs 0.02; p < 0.001] were higher in NOD at BL; SSZ increased by 0.5 unit/year in NOD vs 0.1 unit/year in NL. Median liver stiffness (LSM) by transient elastography was higher in NOD (8.2 kPa, IQR 6-11.8) vs NL (5.3, 4.2-7, p < 0.0001). Over 6.3 years follow-up (1.3-10.3), 6 NOD had esophageal varices (cumulative incidence in 10 years: 20%; 95% CI: 0.0%, 40.0%), 2 had variceal bleeding, and 2 underwent liver transplantation; none had ascites or hepatic encephalopathy. No NL experienced liver-related events.
NOD developed clinically evident portal hypertension faster than NL without worse growth or lung disease.