García-Pérez-de-Sevilla Guillermo, Blanco Velasco Ángela, Yvert Thomas, Sanz-Santiago Verónica, Tirado Ana Morales, López Neyra Alejandro, de Manuel Cristina, Ruiz Valbuena Marta, Pérez-Ruiz Margarita
Department of Physiotherapy, Faculty of Medicine, Health and Sports, European University of Madrid, 28670 Madrid, Spain.
ImFINE Research Group, Department of Health and Human Performance, Facultad de Ciencias de la Actividad Física y del Deporte, INEF Universidad Politécnica de Madrid, 28040 Madrid, Spain.
Children (Basel). 2025 Jul 3;12(7):878. doi: 10.3390/children12070878.
: The objective of this study was to analyze respiratory muscle function in children and adolescents with cystic fibrosis (CF) treated with Elexacaftor/Tezacaftor/Ivacaftor (ETI) compared to healthy individuals, based on the hypothesis that CFTR modulators may improve respiratory muscle strength. : A descriptive, observational, cross-sectional study was conducted with patients with CF treated with ETI aged 6-18 years. Lung function, maximal expiratory and inspiratory pressures (MIP and MEP), diet quality (KIDMED), and physical activity levels (PAQ) were assessed. The student's -test or the Mann-Whitney U-test was used to compare differences between groups. The effect size was calculated with Cohen's d. Significance level was set as a -value < 0.05. : A total of 48 children and adolescents (60.4% male) were analyzed in this study (24 healthy and 24 with CF). The participants with CF had mild pulmonary involvement. No significant differences were found in respiratory muscle strength between groups (MEP = 0.440, MIP = 0.206). Patients with CF showed lower KIDMED ( = 0.022) and PAQ ( = 0.010) scores. However, the MIP and MEP values observed in CF participants were higher than those reported in previous studies conducted before the introduction of ETI modulators. : Children and adolescents with CF treated with ETI showed respiratory muscle strength comparable to that of healthy controls. Despite differences in lifestyle factors, these findings may reflect a positive impact of CFTR modulators on respiratory muscle function, although further longitudinal and controlled studies are needed.
本研究的目的是基于囊性纤维化跨膜传导调节因子（CFTR）调节剂可能改善呼吸肌力量这一假设，分析接受依列卡福/替扎卡福/依伐卡福（ETI）治疗的儿童和青少年囊性纤维化（CF）患者与健康个体相比的呼吸肌功能。
对6至18岁接受ETI治疗的CF患者进行了一项描述性、观察性横断面研究。评估了肺功能、最大呼气和吸气压力（MIP和MEP）、饮食质量（KIDMED）和身体活动水平（PAQ）。使用学生t检验或曼-惠特尼U检验比较组间差异。效应大小用科恩d值计算。显著性水平设定为p值<0.05。
本研究共分析了48名儿童和青少年（60.4%为男性）（24名健康者和24名CF患者）。CF患者有轻度肺部受累。组间呼吸肌力量未发现显著差异（MEP = 0.440，MIP = 0.206）。CF患者的KIDMED（p = 0.022）和PAQ（p = 0.010）得分较低。然而，CF参与者观察到的MIP和MEP值高于ETI调节剂引入之前进行的先前研究报告的值。
接受ETI治疗的CF儿童和青少年显示出与健康对照组相当的呼吸肌力量。尽管生活方式因素存在差异，但这些发现可能反映了CFTR调节剂对呼吸肌功能的积极影响，尽管还需要进一步的纵向和对照研究。
