From the Pediatric Gastroenterology, Hepatology and Nutrition, University of Minnesota Masonic Children's Hospital, Minneapolis, MN.
the Division of Pediatric Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, and Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.
J Pediatr Gastroenterol Nutr. 2022 Nov 1;75(5):635-642. doi: 10.1097/MPG.0000000000003605. Epub 2022 Sep 6.
Cystic fibrosis liver disease (CFLD) begins early in life. Symptoms may be vague, mild, or nonexistent. Progressive liver injury may be associated with decrements in patient health before liver disease is clinically apparent. We examined Health-Related Quality of Life (HRQOL) in children enrolled in a multi-center study of CFLD to determine the impact of early CFLD on general and disease-specific QOL.
Ultrasound (US) patterns of normal (NL), heterogeneous (HTG), homogeneous (HMG), or nodular (NOD) were assigned in a prospective manner to predict those at risk for advanced CFLD. Parents were informed of results. We assessed parent/child-reported (age ≥5 years) HRQOL by PedsQL 4.0 Generic Core and CF Questionnaire-revised (CFQ-R) prior to US and annually. HRQOL scores were compared by US pattern at baseline (prior to US), between baseline and 1 year and at 5 years. Multivariate analysis of variance (MANOVA) with Hotelling-Lawley trace tested for differences among US groups.
Prior to US, among 515 participants and their parents there was no evidence that HTG or NOD US was associated with reduced PedsQL/CFQ-R at baseline. Parents of NOD reported no change in PedsQL/CFQ-R over the next year. Child-report PedsQL/CFQ-R (95 NL, 20 NOD) showed improvement between baseline and year 5 for many scales, including Physical Function. Parents of HMG children reported improved CFQ-R scores related to weight.
Early undiagnosed or pre-symptomatic liver disease had no impact on generic or disease-specific HRQoL, and HRQoL was remarkably stable in children with CF regardless of liver involvement.
囊性纤维化肝病（CFLD）在生命早期就开始了。症状可能是模糊的、轻微的或不存在的。进行性肝损伤可能与临床明显的肝病之前患者健康状况的下降有关。我们检查了参加多中心 CFLD 研究的儿童的健康相关生活质量（HRQOL），以确定早期 CFLD 对一般和疾病特异性 QOL 的影响。
前瞻性地将超声（US）模式分为正常（NL）、异质性（HTG）、均匀性（HMG）或结节性（NOD），以预测那些有进展性 CFLD 风险的患者。父母被告知了结果。我们在 US 之前和每年评估父母/孩子报告（年龄≥5 岁）的 HRQOL 通过 PedsQL 4.0 通用核心和 CF 问卷修订版（CFQ-R）。在基线（US 之前）、基线与 1 年之间以及 5 年时，通过多元方差分析（MANOVA）与 Hotelling-Lawley 迹线测试比较 US 模式之间的 HRQOL 评分差异。
在 515 名参与者及其父母中，在进行 US 之前，没有证据表明 HTG 或 NOD US 与基线时 PedsQL/CFQ-R 降低有关。NOD 患儿的父母在接下来的一年中报告 PedsQL/CFQ-R 没有变化。儿童报告的 PedsQL/CFQ-R（95NL，20NOD）在许多量表上都有所改善，包括身体功能。HMG 儿童的父母报告 CFQ-R 评分与体重相关的改善。
早期未诊断或无症状前肝病对一般或疾病特异性 HRQoL 没有影响，并且无论肝脏受累情况如何，CF 儿童的 HRQoL 都非常稳定。
