Mejoría de la inflamación intestinal tras tratamiento con moduladores de la proteína CFTR en pacientes con fibrosis quística

IF 1.5 4区 医学 Q2 PEDIATRICS
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
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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.

Abstract Image

囊性纤维化患者使用CFTR蛋白调节剂治疗后的肠道炎症改善
CFTR蛋白调节剂治疗可改善囊性纤维化患者的呼吸和消化健康。目的通过分析囊性纤维化患者在CFTR调节剂治疗期间粪便钙保护蛋白的变化,评估肠道炎症的变化。材料和方法前瞻性多中心研究CFTR调节剂治疗囊性纤维化患者粪便钙保护蛋白的变化,比较双联(lumacaftor/ivacaftor或tezacaftor/ivacaftor)和三联(elexaftor /tezacaftor/ivacaftor)。我们在治疗开始前、6个月和12个月收集数据。结果117例患者(69%为F508del/F508del)。粪钙保护蛋白基线水平中位数为49 μg/g (IQR: 23-108);48.7%的中位浓度大于50 μg/g, 11%的中位浓度大于250 μg/g。两组患者的粪便钙保护蛋白水平均有所下降,其中以elexaftor /tezacaftor/ivacaftor治疗的患者下降幅度更大。我们发现异常值逐渐减少(>;50 μg/g) (48.7% vs 33.1%;P = 0.0067)和12个月时(54% vs 33.5%;p = .0218)。在elexaftor /tezacaftor/ivacaftor组中,只有2例患者在6个月时和1例患者在12个月时的水平大于250 μg/g。与其他组相比,三联治疗组在12个月时的估计变化为- 133 μg/g (95% CI: - 254至- 13;p = .030);益生菌和铜绿假单胞菌按性别调整为- 130 μg/g (- 259 ~ - 1;p = .049)。结论CFTR调节剂治疗可减轻囊性纤维化患者的肠道炎症,三联治疗效果更明显。
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来源期刊
Anales de pediatria
Anales de pediatria 医学-小儿科
CiteScore
2.10
自引率
4.80%
发文量
155
审稿时长
44 days
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.
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