Effects on growth, weight and body composition after CFTR modulators in children with cystic fibrosis.

IF 2.7 3区医学 Q1 PEDIATRICS Pediatric Pulmonology Pub Date : 2024-12-01 Epub Date: 2024-09-24 DOI:10.1002/ppul.27272

C M López Cárdenes, A Merino Sánchez-Cañete, S Vicente Santamaría, C Gascón Galindo, N Merino Sanz, A Tabares González, E Blitz Castro, A Morales Tirado, M Garriga García, M López Rozas, T Ramos Riesgo, M Álvarez Beltrán, J R Gutiérrez Martínez, M Suárez González, R García Romero, A De la Mano Hernández, M R Muñoz Codoceo, C Martín Fernández, C Tutau Gómez, E Torcuato Rubio, P Ortiz Pérez, I Loverdos Eseverri, C García Volpe, E Salcedo Lobato, A Martín Rivada, A M Castro Millan, R Del Brio Castillo, S Sierra San Nicolás, M Murray Hurtado, E Crehuá Gaudiza, M Medina Martínez, D González Jiménez

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引用次数: 0

Abstract

Background: Cystic Fibrosis (CF) is associated with compromised nutrition status, which is responsible for morbidity and mortality along with lung function decline. This study was designed to examine changes in anthropometric markers and body composition parameters by bioelectrical impedance analysis after CFTR modulator (CFTRm) treatment.

Methods: We compared anthropometric parameters and body composition before and after 6 and 12 months of CFTRm treatment. Results are stratified into subgroups according to the modulator used with dual therapy with lumacaftor + ivacaftor or tezacaftor + ivacaftor (LUMA/TEZ + IVA) or triple therapy with elexacaftor + tezacaftor + ivacaftor (ELE + TEZ + IVA). Body composition data are available in patients treated with ELE + TEZ + IVA.

Results: Two hundred and thirty-four children (55.1% male) were recruited. The median age was 13.6 years (inter-quartile range [IQR] 10.7-16.1). We can observe a statistically significant increase in the weight Z score and BMI Z score after CFTRm. In terms of changes in body composition, we observe a significant increase in fat mass (FM) expressed both in kilograms and as a percentage at 6 months (p < .05; Wilcoxon-test), with no such differences found at 12 months. We also observe a statistically significant increase in fat-free-mass (FFM), expressed in kilograms at 6 and 12 months (p < .05; Wilcoxon-test).

Conclusion: Weight status improved and changes in body composition occurred in children after CFTRm therapy, including an increase of fat mass. Further studies are needed to confirm these changes in body composition and their impact on disease progression.

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使用 CFTR 调节剂对囊性纤维化儿童的生长、体重和身体成分的影响。

背景：囊性纤维化（CF）与营养状况受损有关，营养状况受损会导致发病率和死亡率以及肺功能下降。本研究旨在通过生物电阻抗分析法检测CFTR调节剂（CFTRm）治疗后人体测量指标和身体成分参数的变化：我们比较了 CFTRm 治疗 6 个月和 12 个月前后的人体测量指标和身体成分。根据所使用的调节剂将结果分成不同的亚组，包括Lumacaftor + ivacaftor或tezacaftor + ivacaftor的双重疗法（LUMA/TEZ + IVA）或Elexacaftor + tezacaftor + ivacaftor的三重疗法（ELE + TEZ + IVA）。采用 ELE + TEZ + IVA 治疗的患者可获得身体成分数据：共招募了 234 名儿童（55.1% 为男性）。中位年龄为 13.6 岁（四分位数间距 [IQR] 10.7-16.1）。我们可以观察到，接受 CFTRm 治疗后，体重 Z 值和体重指数 Z 值均有统计学意义上的显著增加。在身体组成的变化方面，我们观察到脂肪量（FM）在 6 个月时以千克和百分比表示均有显著增加（P 结论：脂肪量的增加与体重的增加有关：接受 CFTRm 治疗后，儿童的体重状况有所改善，身体成分也发生了变化，其中包括脂肪量的增加。需要进一步的研究来证实这些身体成分的变化及其对疾病进展的影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pediatric Pulmonology 医学-呼吸系统

CiteScore

6.00

自引率

12.90%

发文量

468

审稿时长

3-8 weeks

期刊介绍： Pediatric Pulmonology (PPUL) is the foremost global journal studying the respiratory system in disease and in health as it develops from intrauterine life though adolescence to adulthood. Combining explicit and informative analysis of clinical as well as basic scientific research, PPUL provides a look at the many facets of respiratory system disorders in infants and children, ranging from pathological anatomy, developmental issues, and pathophysiology to infectious disease, asthma, cystic fibrosis, and airborne toxins. Focused attention is given to the reporting of diagnostic and therapeutic methods for neonates, preschool children, and adolescents, the enduring effects of childhood respiratory diseases, and newly described infectious diseases. PPUL concentrates on subject matters of crucial interest to specialists preparing for the Pediatric Subspecialty Examinations in the United States and other countries. With its attentive coverage and extensive clinical data, this journal is a principle source for pediatricians in practice and in training and a must have for all pediatric pulmonologists.