Mallinckrodt Institute of Radiology, Washington University School of Medicine, St Louis, MO, USA.
Division of Gastroenterology, Hepatology and Nutrition, Texas Children's Hospital, Department of Pediatrics, Baylor College of Medicine, Houston TX, USA.
J Cyst Fibros. 2023 Jul;22(4):745-755. doi: 10.1016/j.jcf.2023.03.019. Epub 2023 Apr 7.
This study examines whether heterogeneous (HTG) pattern on liver ultrasound (US) identifies children at risk for advanced cystic fibrosis liver disease (aCFLD).
Prospective 6-year multicenter case-controlled cohort study. Children with pancreatic insufficient cystic fibrosis (CF) aged 3-12 years without known cirrhosis underwent screening US. Participants with HTG were matched (by age, Pseudomonas infection status and center) 1:2 with participants with normal (NL) US pattern. Clinical status and laboratory data were obtained annually and US bi-annually for 6 years. Primary endpoint was development of nodular (NOD) US pattern consistent with aCFLD.
722 participants underwent screening US, with 65 HTG and 592 NL. Final cohort included 55 HTG and 116 NL with ≥ 1 follow-up US. ALT, AST, GGTP, FIB-4, GPR and APRI were higher, and platelets were lower in HTG compared to NL. HTG had a 9.5-fold increased incidence (95% confidence interval [CI]:3.4, 26.7, p<0.0001, 32.7% vs 3.4%) of NOD versus NL. HTG had a sensitivity of 82% and specificity of 75% for subsequent NOD. Negative predictive value of a NL US for subsequent NOD was 96%. Multivariate logistic prediction model that included baseline US, age, and log(GPR) improved the C-index to 0.90 compared to only baseline US (C-index 0.78). Based on survival analysis, 50% of HTG develop NOD after 8 years.
Research US finding of HTG identifies children with CF with a 30-50% risk for aCFLD. A score based on US pattern, age and GPR may refine the identification of individuals at high risk for aCFLD.
Prospective Study of Ultrasound to Predict Hepatic Cirrhosis in CF: NCT 01,144,507 (observational study, no consort checklist).
本研究旨在探讨肝脏超声（US）的不均匀（HTG）模式是否能识别出患有进展性囊性纤维化肝病（aCFLD）风险的儿童。
这是一项前瞻性的 6 年多中心病例对照队列研究。年龄在 3-12 岁、胰腺功能不全的囊性纤维化（CF）患儿，无已知肝硬化，接受了筛查性 US。HTG 组的患者与正常（NL）US 模式的患者进行了年龄、铜绿假单胞菌感染状态和中心匹配（1:2）。临床状态和实验室数据每年获得一次，US 每两年获得一次，共进行 6 年。主要终点是出现符合 aCFLD 的结节性（NOD）US 模式。
722 名参与者接受了筛查性 US，其中 65 名患者为 HTG，592 名患者为 NL。最终的队列包括 55 名 HTG 和 116 名 NL，他们有≥1 次的随访 US。与 NL 相比，HTG 的 ALT、AST、GGTP、FIB-4、GPR 和 APRI 更高，血小板更低。与 NL 相比，HTG 发生 NOD 的发病率增加了 9.5 倍（95%置信区间[CI]：3.4，26.7，p<0.0001，32.7%vs3.4%）。HTG 对随后发生 NOD 的敏感度为 82%，特异度为 75%。NL US 对随后发生 NOD 的阴性预测值为 96%。纳入基线 US、年龄和 log(GPR)的多变量逻辑预测模型，与仅基于基线 US 的模型相比，C 指数提高到 0.90（C 指数 0.78）。基于生存分析，HTG 中有 50%的患者在 8 年后发生 NOD。
研究 US 发现的 HTG 可识别出 CF 患儿发生 aCFLD 的风险为 30-50%。基于 US 模式、年龄和 GPR 的评分可能会细化对高风险个体发生 aCFLD 的识别。
前瞻性研究超声预测 CF 患者的肝纤维化：NCT 01,144,507（观察性研究，无 CONSORT 清单）。
