Enaud R, Languepin J, Lagarrigue M, Arrouy A, Macey J, Bui S, Dupuis M, Roditis L, Flumian C, Mas E, Mittaine M
University of Bordeaux, INSERM U1045, Centre de Recherche Cardio-thoracique de Bordeaux, U1045, Bordeaux, France; Bordeaux University Hospital, CRCM pédiatrique, CIC 1401, Bordeaux, France.
Limoges University Hospital, Paediatric Cystic Fibrosis Reference Center (CRCM), Limoges, France.
Clin Nutr. 2025 Jul;50:76-82. doi: 10.1016/j.clnu.2025.04.027. Epub 2025 Apr 30.
Cystic fibrosis (CF) is a genetic disorder involving multi-organ dysfunction, with nutritional status playing a crucial role in disease progression. Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulator therapy, particularly the combination Elexacaftor/Tezacaftor/Ivacaftor (ETI), has shown numerous clinical benefits, including significant improvements in nutritional status. However, the factors driving this nutritional improvement, especially the potential role of increased dietary intake, remain underexplored. This study aimed to assess changes in nutritional status and dietary intake in patients with CF (pwCF) treated with ETI.
62 pwCF (36 children, 26 adults) were analyzed in a prospective, realworld, multicenter study (https://clinicaltrials.gov/study/NCT06072365). Dietary intakes were assessed via 3-day food diaries collected at baseline (M0) and one year after ETI treatment initiation (M12).
Over the first year of ETI treatment, Body Mass Index (BMI) significantly increased with a median BMI Z-score gain of 0.2 (IQR: 0.7) for children and median BMI gain of 1.0 kg/m (IQR: 1.8) for adults. Notably, these gains occurred without a significant increase in median daily caloric intake (2216 kcal (IQR: 750) at M0 vs. 2266 (IQR: 733) kcal at M12). Pancreatic enzyme requirements and calprotectin decreased significantly with ETI (p < 0.001 and p < 0.01, respectively), indicating improved pancreatic function and intestinal inflammation in some patients. Seven patients became overweight after one year of ETI.
ETI therapy enhances nutritional status in pwCF, independently of increased caloric intake. Further research is essential to refine dietary recommendations under ETI treatment, aiming to prevent overweight and obesity while optimizing health outcomes.
囊性纤维化（CF）是一种涉及多器官功能障碍的遗传性疾病，营养状况在疾病进展中起着至关重要的作用。囊性纤维化跨膜传导调节因子（CFTR）调节剂疗法，特别是三联疗法艾列卡福/替扎卡福/依伐卡福（ETI），已显示出诸多临床益处，包括营养状况的显著改善。然而，推动这种营养改善的因素，尤其是饮食摄入量增加的潜在作用，仍未得到充分探索。本研究旨在评估接受ETI治疗的CF患者（pwCF）的营养状况和饮食摄入量的变化。
在一项前瞻性、真实世界、多中心研究（https://clinicaltrials.gov/study/NCT06072365）中分析了62例pwCF患者（36名儿童，26名成人）。通过在基线（M0）和ETI治疗开始一年后（M12）收集的3天食物日记评估饮食摄入量。
在ETI治疗的第一年，体重指数（BMI）显著增加，儿童的BMI Z评分中位数增加0.2（四分位间距：0.7），成人体重指数中位数增加1.0 kg/m²（四分位间距：1.8）。值得注意的是，这些增加并未伴随着每日热量摄入中位数的显著增加（M0时为2216千卡（四分位间距：750），M12时为2266（四分位间距：733）千卡）。ETI治疗后，胰酶需求量和钙卫蛋白显著降低（分别为p < 0.001和p < 0.01），表明部分患者的胰腺功能和肠道炎症得到改善。7例患者在接受ETI治疗一年后超重。
ETI疗法可改善pwCF患者的营养状况，与热量摄入增加无关。进一步的研究对于完善ETI治疗下的饮食建议至关重要，旨在预防超重和肥胖，同时优化健康结局。
