Samal Munidasa, Faiyza Alam, Brandon Zanette, Daniel Li, Wallace Wee, Sharon Braganza, Jason Woods, Felix Ratjen, Giles Santyr
{"title":"相分辨功能肺（PREFUL）和超极化129Xe MRI纵向监测儿童囊性纤维化患者肺功能的比较，在elexaftor /tezacaftor/ivacaftor后。","authors":"Samal Munidasa, Faiyza Alam, Brandon Zanette, Daniel Li, Wallace Wee, Sharon Braganza, Jason Woods, Felix Ratjen, Giles Santyr","doi":"10.1007/s00247-025-06390-4","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Phase-resolved functional lung (PREFUL) MRI offers a more accessible alternative to hyperpolarized <sup>129</sup>Xe MRI (Xe-MRI) for monitoring treatment response in pediatric cystic fibrosis (CF), but longitudinal comparisons are limited.</p><p><strong>Objective: </strong>To assess longitudinal lung function changes following elexacaftor/tezacaftor/ivacaftor (ETI) treatment initiation in CF children using PREFUL MRI, in comparison with Xe-MRI and pulmonary function tests (PFTs).</p><p><strong>Materials and methods: </strong>PREFUL MRI, Xe-MRI, and PFTs were performed in 14 CF patients (median [IQR] age 15 [14-16.5] years old) at baseline and 1 month, 6 months, 12 months, and 24 months following initiation of ETI treatment. Ventilation and defect percentage (VDP) was derived from PREFUL MRI (regional ventilation VDP, VDP<sub>RVent</sub>; regional-flow volume loop cross-correlation VDP, VDP<sub>CC</sub>; and the combination of VDP<sub>RVent</sub> and VDP<sub>CC</sub>, VDP<sub>combined</sub>) and Xe-MRI (VDP<sub>Xe</sub>) maps. Perfusion defect percentage (QDP) was derived from normalized perfusion maps and, with VDP<sub>combined</sub>, determined the percentage of healthy ventilation-perfusion matching (VQM). Significance of 1-month treatment changes was determined using the Wilcoxon-signed rank test and was correlated between metrics using Spearman ranked correlation.</p><p><strong>Results: </strong>All PREFUL measures significantly improved (P < 0.01) 1-month post-treatment in agreement with changes in Xe-MRI VDP and PFTs (P < 0.03). The absolute change in VDP<sub>CC</sub> and VDP<sub>combined</sub> significantly correlated with VDP<sub>Xe</sub> (r ≥ 0.62, P < 0.02), unlike VDP<sub>RVent</sub> (P = 0.35). The change in QDP did not correlate with any metric (P > 0.10). PREFUL MRI and Xe-MRI measures showed minimal changes 1 to 24 months post-treatment (median changes = -2.3% to 1.4%), in agreement with PFTs.</p><p><strong>Conclusion: </strong>PREFUL MRI detects longitudinal treatment-related changes in pulmonary ventilation and perfusion in CF children post ETI.</p>","PeriodicalId":19755,"journal":{"name":"Pediatric Radiology","volume":" ","pages":""},"PeriodicalIF":2.3000,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Comparison of phase-resolved functional lung (PREFUL) and hyperpolarized <sup>129</sup>Xe MRI for longitudinal monitoring of lung function in pediatric cystic fibrosis following elexacaftor/tezacaftor/ivacaftor.\",\"authors\":\"Samal Munidasa, Faiyza Alam, Brandon Zanette, Daniel Li, Wallace Wee, Sharon Braganza, Jason Woods, Felix Ratjen, Giles Santyr\",\"doi\":\"10.1007/s00247-025-06390-4\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Phase-resolved functional lung (PREFUL) MRI offers a more accessible alternative to hyperpolarized <sup>129</sup>Xe MRI (Xe-MRI) for monitoring treatment response in pediatric cystic fibrosis (CF), but longitudinal comparisons are limited.</p><p><strong>Objective: </strong>To assess longitudinal lung function changes following elexacaftor/tezacaftor/ivacaftor (ETI) treatment initiation in CF children using PREFUL MRI, in comparison with Xe-MRI and pulmonary function tests (PFTs).</p><p><strong>Materials and methods: </strong>PREFUL MRI, Xe-MRI, and PFTs were performed in 14 CF patients (median [IQR] age 15 [14-16.5] years old) at baseline and 1 month, 6 months, 12 months, and 24 months following initiation of ETI treatment. Ventilation and defect percentage (VDP) was derived from PREFUL MRI (regional ventilation VDP, VDP<sub>RVent</sub>; regional-flow volume loop cross-correlation VDP, VDP<sub>CC</sub>; and the combination of VDP<sub>RVent</sub> and VDP<sub>CC</sub>, VDP<sub>combined</sub>) and Xe-MRI (VDP<sub>Xe</sub>) maps. Perfusion defect percentage (QDP) was derived from normalized perfusion maps and, with VDP<sub>combined</sub>, determined the percentage of healthy ventilation-perfusion matching (VQM). Significance of 1-month treatment changes was determined using the Wilcoxon-signed rank test and was correlated between metrics using Spearman ranked correlation.</p><p><strong>Results: </strong>All PREFUL measures significantly improved (P < 0.01) 1-month post-treatment in agreement with changes in Xe-MRI VDP and PFTs (P < 0.03). The absolute change in VDP<sub>CC</sub> and VDP<sub>combined</sub> significantly correlated with VDP<sub>Xe</sub> (r ≥ 0.62, P < 0.02), unlike VDP<sub>RVent</sub> (P = 0.35). The change in QDP did not correlate with any metric (P > 0.10). PREFUL MRI and Xe-MRI measures showed minimal changes 1 to 24 months post-treatment (median changes = -2.3% to 1.4%), in agreement with PFTs.</p><p><strong>Conclusion: </strong>PREFUL MRI detects longitudinal treatment-related changes in pulmonary ventilation and perfusion in CF children post ETI.</p>\",\"PeriodicalId\":19755,\"journal\":{\"name\":\"Pediatric Radiology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2025-09-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric Radiology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s00247-025-06390-4\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Radiology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s00247-025-06390-4","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PEDIATRICS","Score":null,"Total":0}
Comparison of phase-resolved functional lung (PREFUL) and hyperpolarized 129Xe MRI for longitudinal monitoring of lung function in pediatric cystic fibrosis following elexacaftor/tezacaftor/ivacaftor.
Background: Phase-resolved functional lung (PREFUL) MRI offers a more accessible alternative to hyperpolarized 129Xe MRI (Xe-MRI) for monitoring treatment response in pediatric cystic fibrosis (CF), but longitudinal comparisons are limited.
Objective: To assess longitudinal lung function changes following elexacaftor/tezacaftor/ivacaftor (ETI) treatment initiation in CF children using PREFUL MRI, in comparison with Xe-MRI and pulmonary function tests (PFTs).
Materials and methods: PREFUL MRI, Xe-MRI, and PFTs were performed in 14 CF patients (median [IQR] age 15 [14-16.5] years old) at baseline and 1 month, 6 months, 12 months, and 24 months following initiation of ETI treatment. Ventilation and defect percentage (VDP) was derived from PREFUL MRI (regional ventilation VDP, VDPRVent; regional-flow volume loop cross-correlation VDP, VDPCC; and the combination of VDPRVent and VDPCC, VDPcombined) and Xe-MRI (VDPXe) maps. Perfusion defect percentage (QDP) was derived from normalized perfusion maps and, with VDPcombined, determined the percentage of healthy ventilation-perfusion matching (VQM). Significance of 1-month treatment changes was determined using the Wilcoxon-signed rank test and was correlated between metrics using Spearman ranked correlation.
Results: All PREFUL measures significantly improved (P < 0.01) 1-month post-treatment in agreement with changes in Xe-MRI VDP and PFTs (P < 0.03). The absolute change in VDPCC and VDPcombined significantly correlated with VDPXe (r ≥ 0.62, P < 0.02), unlike VDPRVent (P = 0.35). The change in QDP did not correlate with any metric (P > 0.10). PREFUL MRI and Xe-MRI measures showed minimal changes 1 to 24 months post-treatment (median changes = -2.3% to 1.4%), in agreement with PFTs.
Conclusion: PREFUL MRI detects longitudinal treatment-related changes in pulmonary ventilation and perfusion in CF children post ETI.
期刊介绍：
Official Journal of the European Society of Pediatric Radiology, the Society for Pediatric Radiology and the Asian and Oceanic Society for Pediatric Radiology
Pediatric Radiology informs its readers of new findings and progress in all areas of pediatric imaging and in related fields. This is achieved by a blend of original papers, complemented by reviews that set out the present state of knowledge in a particular area of the specialty or summarize specific topics in which discussion has led to clear conclusions. Advances in technology, methodology, apparatus and auxiliary equipment are presented, and modifications of standard techniques are described.
Manuscripts submitted for publication must contain a statement to the effect that all human studies have been reviewed by the appropriate ethics committee and have therefore been performed in accordance with the ethical standards laid down in an appropriate version of the 1964 Declaration of Helsinki. It should also be stated clearly in the text that all persons gave their informed consent prior to their inclusion in the study. Details that might disclose the identity of the subjects under study should be omitted.
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