目的评估监测elexacaftor-tezacaftor-ivacaftor(ETI)治疗对囊性纤维化(CF)患者肺通气和灌注的影响的可行性。使用相位分辨功能肺(PREFUL)MRI。材料和方法这项对多中心前瞻性研究的二次分析是在2020年8月至2021年3月之间进行的,包括12岁或以上接受PREFULMRI检查的CF患者。肺活量测定,汗液氯化物试验,ETI治疗前和治疗后8-16周的肺清除指数评估。对于PREFUL导出的通气和灌注参数提取,使用自动定量管道评估二维冠状动态梯度回波MR图像。目视评估T1和T2加权MR图像以及PREFUL灌注图的半定量Eichinger评分。Wilcoxon符号秩检验比较了ETI治疗前后的临床参数和PREFUL值。参数的相关性计算为Spearmanρ相关系数。结果23名参与者(平均年龄,18年[IQR:14-24.5年];包括13名女性)。定量参数,艾希格得分,ETI治疗后,临床参数(肺清除指数=21)显着改善。局部通气的通气缺陷百分比从18%(IQR:14%-25%)降至9%(IQR:6%-17%)(P=.003),灌注缺陷百分比从26%(IQR:18%-36%)降至19%(IQR:13%-24%)(P=.002)。正常(健康)通气和灌注匹配的区域从52%(IQR:47%-68%)增加到73%(IQR:61%-83%)。目视评估的灌注评分与PREFUL灌注(P=.11)无关,也与通气-灌注匹配值(P=.38)无关。结论该研究证明了PREFULMRI用于半自动定量评估CF患者对ETI治疗反应的灌注和通气变化的可行性。关键词:儿科,MR功能成像,肺,肺肺,比较研究,囊性纤维化,Elexacaftor-Tezacaftor-Ivacaftor治疗,傅里叶分解,PREFUL,自由呼吸质子核磁共振,肺部MRI,灌注,功能磁共振成像,CFTR,调制器治疗,Kaftrio临床试验登记号NCT04732910补充材料可用于本文。©RSNA,2024.
Purpose To assess the feasibility of monitoring the effects of elexacaftor-tezacaftor-ivacaftor (ETI) therapy on lung ventilation and perfusion in people with cystic fibrosis (CF), using phase-resolved functional lung (PREFUL) MRI. Materials and Methods This secondary analysis of a multicenter prospective
study was carried out between August 2020 and March 2021 and included participants 12 years or older with CF who underwent PREFUL MRI, spirometry, sweat chloride test, and lung clearance index assessment before and 8-16 weeks after ETI therapy. For PREFUL-derived ventilation and perfusion parameter extraction, two-dimensional coronal dynamic gradient-echo MR images were evaluated with an automated quantitative pipeline. T1- and T2-weighted MR images and PREFUL perfusion maps were visually assessed for semiquantitative Eichinger scores. Wilcoxon signed rank test compared clinical parameters and PREFUL values before and after ETI therapy. Correlation of parameters was calculated as Spearman ρ correlation coefficient. Results Twenty-three participants (median age, 18 years [IQR: 14-24.5 years]; 13 female) were included. Quantitative PREFUL parameters, Eichinger score, and clinical parameters (lung clearance index = 21) showed significant improvement after ETI therapy. Ventilation defect percentage of regional ventilation decreased from 18% (IQR: 14%-25%) to 9% (IQR: 6%-17%) (P = .003) and perfusion defect percentage from 26% (IQR: 18%-36%) to 19% (IQR: 13%-24%) (P = .002). Areas of matching normal (healthy) ventilation and perfusion increased from 52% (IQR: 47%-68%) to 73% (IQR: 61%-83%). Visually assessed perfusion scores did not correlate with PREFUL perfusion (P = .11) nor with ventilation-perfusion match values (P = .38). Conclusion The
study demonstrates the feasibility of PREFUL MRI for semiautomated quantitative assessment of perfusion and ventilation changes in response to ETI therapy in people with CF. Keywords: Pediatrics, MR-Functional Imaging, Pulmonary, Lung, Comparative Studies, Cystic Fibrosis, Elexacaftor-Tezacaftor-Ivacaftor Therapy, Fourier Decomposition, PREFUL, Free-Breathing Proton MRI, Pulmonary MRI, Perfusion, Functional MRI,
CFTR, Modulator Therapy, Kaftrio Clinical
trial registration no. NCT04732910 Supplemental material is available for this article. © RSNA, 2024.