Abstract：
BACKGROUND: Non-invasive and sensitive clinical endpoints are needed to monitor onset and progression of early lung disease in children with cystic fibrosis (CF). We compared lung clearance index (LCI), FEV1, functional and structural lung magnetic resonance imaging (MRI) outcomes in Swiss children with CF diagnosed following newborn screening.
METHODS: Lung function (LCI, FEV1) and unsedated functional and structural lung MRI was performed in 79 clinically stable children with CF (3 - 8 years) and 75 age-matched healthy controls. Clinical information was collected throughout childhood.
RESULTS: LCI, ventilation and perfusion defects, and structural MRI scores were significantly higher in children with CF compared with controls, but FEV1 was not different between groups. Lung MRI outcomes correlated significantly with LCI (morphology score (r = 0.56, p < 0.001); ventilation defects (r = 0.43, p = 0.001); perfusion defects (r = 0.64, p < 0.001), but not with FEV1. Lung MRI outcomes were more sensitive to detect impairments in children with CF (abnormal ventilation and perfusion outcomes in 47 %, morphology score in 30 %) compared with lung function (abnormal LCI in 21 % and FEV1 in 4.8 %). Pulmonary exacerbations, respiratory hospitalizations, and increase in patient-reported cough was associated with higher LCI and higher structural and functional MRI outcomes.
CONCLUSIONS: The LCI and lung MRI outcomes non-invasively detect even mild early lung disease in young children with CF diagnosed following newborn screening. Pulmonary exacerbations and early respiratory symptoms were risk factors for structural and functional impairment in childhood.
METHODS: Lung function (LCI, FEV1) and unsedated functional and structural lung MRI was performed in 79 clinically stable children with CF (3 - 8 years) and 75 age-matched healthy controls. Clinical information was collected throughout childhood.
RESULTS: LCI, ventilation and perfusion defects, and structural MRI scores were significantly higher in children with CF compared with controls, but FEV1 was not different between groups. Lung MRI outcomes correlated significantly with LCI (morphology score (r = 0.56, p < 0.001); ventilation defects (r = 0.43, p = 0.001); perfusion defects (r = 0.64, p < 0.001), but not with FEV1. Lung MRI outcomes were more sensitive to detect impairments in children with CF (abnormal ventilation and perfusion outcomes in 47 %, morphology score in 30 %) compared with lung function (abnormal LCI in 21 % and FEV1 in 4.8 %). Pulmonary exacerbations, respiratory hospitalizations, and increase in patient-reported cough was associated with higher LCI and higher structural and functional MRI outcomes.
CONCLUSIONS: The LCI and lung MRI outcomes non-invasively detect even mild early lung disease in young children with CF diagnosed following newborn screening. Pulmonary exacerbations and early respiratory symptoms were risk factors for structural and functional impairment in childhood.
摘要：
背景：需要非侵入性且敏感的临床终点来监测囊性纤维化（CF）儿童早期肺部疾病的发作和进展。我们比较了肺清除指数（LCI），FEV1，经新生儿筛查诊断为CF的瑞士儿童的功能性和结构性肺磁共振成像（MRI）结果。
方法：对79名临床稳定的CF患儿（3-8岁）和75名年龄匹配的健康对照者进行了肺功能（LCI，FEV1）和未镇静的功能性和结构性肺MRI检查。在整个儿童时期收集临床信息。
结果：LCI，通气和灌注缺陷，与对照组相比，CF患儿的结构MRI评分明显更高，但FEV1在组间没有差异。肺部MRI结果与LCI（形态学评分（r=0.56，p<0.001）;通气缺陷（r=0.43，p=0.001）;灌注缺陷（r=0.64，p<0.001），但不是FEV1。肺部MRI结果对CF患儿的损伤检测更敏感（47％的患者为异常通气和灌注结果，形态学评分为30％）与肺功能（异常LCI为21％，FEV1为4.8％）相比。肺加重,呼吸道住院,患者报告的咳嗽增加与较高的LCI和较高的结构和功能MRI结局相关.
结论：在新生儿筛查后诊断为CF的幼儿中，LCI和肺部MRI结果无创地检测到甚至轻度的早期肺部疾病。肺加重和早期呼吸道症状是儿童期结构和功能损害的危险因素。
方法：对79名临床稳定的CF患儿（3-8岁）和75名年龄匹配的健康对照者进行了肺功能（LCI，FEV1）和未镇静的功能性和结构性肺MRI检查。在整个儿童时期收集临床信息。
结果：LCI，通气和灌注缺陷，与对照组相比，CF患儿的结构MRI评分明显更高，但FEV1在组间没有差异。肺部MRI结果与LCI（形态学评分（r=0.56，p<0.001）;通气缺陷（r=0.43，p=0.001）;灌注缺陷（r=0.64，p<0.001），但不是FEV1。肺部MRI结果对CF患儿的损伤检测更敏感（47％的患者为异常通气和灌注结果，形态学评分为30％）与肺功能（异常LCI为21％，FEV1为4.8％）相比。肺加重,呼吸道住院,患者报告的咳嗽增加与较高的LCI和较高的结构和功能MRI结局相关.
结论：在新生儿筛查后诊断为CF的幼儿中，LCI和肺部MRI结果无创地检测到甚至轻度的早期肺部疾病。肺加重和早期呼吸道症状是儿童期结构和功能损害的危险因素。
