Abstract
BACKGROUND: 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. METHODS: 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. RESULTS: 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. conclusions: nod developed clinically evident portal hypertension faster than nl without worse growth or lung disease.>
