Rose Natalie R, Bailey Julianna, Anderson Justin D, Chalamalla Ashritha R, Ryan Kevin J, Acosta Edward P, Guimbellot Jennifer S
Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Department of Pediatrics, Division of Pulmonary and Sleep Medicine, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Pharmacotherapy. 2024 Dec;44(12):920-926. doi: 10.1002/phar.4630. Epub 2024 Dec 23.
Nutritional support for people with cystic fibrosis (PwCF) after the implementation of novel drug therapies is shifting from managing malnutrition through a high-fat, high-calorie diet to managing emerging incidences of obesity in this population. Additionally, dietary recommendations prescribed with elexacaftor/tezacaftor/ivacaftor (ETI) recommend taking this drug with a fat-containing meal, which is variably interpreted by patients. This pilot and feasibility study was conducted to assess dietary fat intake and body composition on ETI plasma concentrations.
Ten participants were enrolled in a 1:1 crossover design by dietary recommendations. To mimic recommendations made during routine clinical care, participants were instructed to consume either a general healthful diet (no more than 30% calories from fat) or a high-fat diet (>40% calories from fat) for a week before crossing over to the alternative diet.
This pilot study was acceptable to and feasible for study participants. Most participants increased fat intake calories when following a high-fat diet. Body composition measurements showed a trending correlation between lean mass and fat-free mass with ETI plasma concentrations. ETI compounds were quantified in plasma at 0 h (prior to the ETI morning dose) and 6 h after ingestion, and consuming a high-fat diet did not significantly impact ETI concentrations.
Consuming a higher-fat diet did not significantly impact ETI plasma concentrations, and all participants were in range for clinical effectiveness of ETI regardless of fat intake. This work provides vital pilot data to design larger studies to clarify dietary composition for optimal ETI exposure for PwCF on this therapy.
新型药物疗法实施后,囊性纤维化患者(PwCF)的营养支持正从通过高脂、高热量饮食管理营养不良转向管理该人群中日益增多的肥胖发生率。此外,依列卡福/替扎卡福/艾伐卡福(ETI)的饮食建议推荐与含脂餐一起服用此药,患者对此的理解各不相同。本试点及可行性研究旨在评估饮食脂肪摄入量和身体成分对ETI血浆浓度的影响。
按照饮食建议,10名参与者采用1:1交叉设计入组。为模拟常规临床护理中的建议,参与者在交叉至另一种饮食前,被指示食用普通健康饮食(脂肪提供的热量不超过30%)或高脂饮食(脂肪提供的热量>40%)一周。
该试点研究对研究参与者来说是可接受且可行的。大多数参与者在遵循高脂饮食时增加了脂肪摄入热量。身体成分测量显示,瘦体重和去脂体重与ETI血浆浓度之间存在趋势性相关性。在0小时(ETI晨服剂量前)和摄入后6小时对血浆中的ETI化合物进行定量,食用高脂饮食对ETI浓度没有显著影响。
食用高脂饮食对ETI血浆浓度没有显著影响,无论脂肪摄入量如何,所有参与者的ETI临床疗效均在有效范围内。这项工作提供了重要的试点数据,可用于设计更大规模的研究,以明确饮食组成,使接受该疗法的PwCF获得最佳的ETI暴露。