Ruth García Romero , Concepción López Cárdenes , Elena Crehuá Gaudiza , Marina Álvarez Beltrán , Mercedes Murray Hurtado , Carlos Tutau Gómez , Inés Loverdos Eseverri , Encarni Torcuato Rubio , Camila García Volpe , Enrique Salcedo Lobato , María Medina Martínez , Carmen Martin Fernández , Ana Moreno Álvarez , Ana Reyes Domínguez , David González Jiménez , Grupo de trabajo de Fibrosis Quística y Páncreas de la SEGHNP
{"title":"Mejoría de la inflamación intestinal tras tratamiento con moduladores de la proteína CFTR en pacientes con fibrosis quística","authors":"Ruth García Romero , Concepción López Cárdenes , Elena Crehuá Gaudiza , Marina Álvarez Beltrán , Mercedes Murray Hurtado , Carlos Tutau Gómez , Inés Loverdos Eseverri , Encarni Torcuato Rubio , Camila García Volpe , Enrique Salcedo Lobato , María Medina Martínez , Carmen Martin Fernández , Ana Moreno Álvarez , Ana Reyes Domínguez , David González Jiménez , Grupo de trabajo de Fibrosis Quística y Páncreas de la SEGHNP","doi":"10.1016/j.anpedi.2025.503836","DOIUrl":null,"url":null,"abstract":"<div><h3>Introduction</h3><div>Treatments with CFTR protein modulators have improved respiratory and digestive health in patients with cystic fibrosis.</div></div><div><h3>Objective</h3><div>To assess changes in intestinal inflammation through the analysis of fecal calprotectin in patients with cystic fibrosis during treatment with CFTR modulators.</div></div><div><h3>Material and methods</h3><div>Prospective multicenter study of changes in fecal calprotectin in patients with cystic fibrosis treated with CFTR modulators, comparing double combinations (lumacaftor/ivacaftor or tezacaftor/ivacaftor) and triple combinations (elexacaftor/tezacaftor/ivacaftor). We collected data before treatment initiation and at 6<!--> <!-->and 12<!--> <!-->months.</div></div><div><h3>Results</h3><div>Analysis of 117 patients (69% with F508del/F508del). The median baseline fecal calprotectin level was 49<!--> <!-->μg/g (IQR: 23-108); 48.7% had median levels greater than 50<!--> <!-->μg/g and 11% levels greater than 250<!--> <!-->μg/g. Fecal calprotectin decreased in both groups, with a greater decrease in patients treated with elexacaftor/tezacaftor/ivacaftor. We found a progressive decrease in abnormal values (><!--> <!-->50<!--> <!-->μg/g) at 6<!--> <!-->months (48.7% vs 33.1%; <em>P</em> <!-->=<!--> <!-->.0067) and at 12<!--> <!-->months (54% vs 33.5%; <em>P</em> <!-->=<!--> <!-->.0218). In the elexacaftor/tezacaftor/ivacaftor group, only two patients at 6<!--> <!-->months and one patient at 12<!--> <!-->months had levels greater than 250<!--> <!-->μg/g. The estimated change at 12<!--> <!-->months in the triple therapy group compared to the other group was −133<!--> <!-->μg/g (95%<!--> <!-->CI: −<!--> <!-->254 to −<!--> <!-->13; <em>P</em> <!-->=<!--> <!-->.030); and, adjusting for sex, probiotics and <em>Pseudomonas aeruginosa</em>, −<!--> <!-->130<!--> <!-->μg/g (−259 to −<!--> <!-->1; <em>P</em> <!-->=<!--> <!-->.049).</div></div><div><h3>Conclusions</h3><div>Treatment with CFTR modulators reduces intestinal inflammation in patients with cystic fibrosis, with a greater decrease in patients treated with triple therapy.</div></div>","PeriodicalId":7783,"journal":{"name":"Anales de pediatria","volume":"102 5","pages":"Article 503836"},"PeriodicalIF":1.5000,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Anales de pediatria","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1695403325000712","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0
Abstract
Introduction
Treatments with CFTR protein modulators have improved respiratory and digestive health in patients with cystic fibrosis.
Objective
To assess changes in intestinal inflammation through the analysis of fecal calprotectin in patients with cystic fibrosis during treatment with CFTR modulators.
Material and methods
Prospective multicenter study of changes in fecal calprotectin in patients with cystic fibrosis treated with CFTR modulators, comparing double combinations (lumacaftor/ivacaftor or tezacaftor/ivacaftor) and triple combinations (elexacaftor/tezacaftor/ivacaftor). We collected data before treatment initiation and at 6 and 12 months.
Results
Analysis of 117 patients (69% with F508del/F508del). The median baseline fecal calprotectin level was 49 μg/g (IQR: 23-108); 48.7% had median levels greater than 50 μg/g and 11% levels greater than 250 μg/g. Fecal calprotectin decreased in both groups, with a greater decrease in patients treated with elexacaftor/tezacaftor/ivacaftor. We found a progressive decrease in abnormal values (> 50 μg/g) at 6 months (48.7% vs 33.1%; P = .0067) and at 12 months (54% vs 33.5%; P = .0218). In the elexacaftor/tezacaftor/ivacaftor group, only two patients at 6 months and one patient at 12 months had levels greater than 250 μg/g. The estimated change at 12 months in the triple therapy group compared to the other group was −133 μg/g (95% CI: − 254 to − 13; P = .030); and, adjusting for sex, probiotics and Pseudomonas aeruginosa, − 130 μg/g (−259 to − 1; P = .049).
Conclusions
Treatment with CFTR modulators reduces intestinal inflammation in patients with cystic fibrosis, with a greater decrease in patients treated with triple therapy.
期刊介绍:
La Asociación Española de Pediatría tiene como uno de sus objetivos principales la difusión de información científica rigurosa y actualizada sobre las distintas áreas de la pediatría. Anales de Pediatría es el Órgano de Expresión Científica de la Asociación y constituye el vehículo a través del cual se comunican los asociados. Publica trabajos originales sobre investigación clínica en pediatría procedentes de España y países latinoamericanos, así como artículos de revisión elaborados por los mejores profesionales de cada especialidad, las comunicaciones del congreso anual y los libros de actas de la Asociación, y guías de actuación elaboradas por las diferentes Sociedades/Secciones Especializadas integradas en la Asociación Española de Pediatría.