학술논문
A prospective study to assess the impact of a novel CFTR therapy combination on body composition in patients with cystic fibrosis with F508del mutation.
Document Type
Academic Journal
Author
Knott-Torcal C; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: cknott.hup@gmail.com.; Sebastián-Valles F; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: fer.sebas.valles@gmail.com.; Girón Moreno RM; Department of Respiratory Medicine, Adult Cystic Fibrosis Unit, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: rmgiron@gmail.com.; Martín-Adán JC; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: jmadan@salud.madrid.org.; Jiménez-Díaz J; Department of Endocrinology and Nutrition, Hospital Universitario de Torrejón, C. Mateo Inurria, 28850 Torrejón de Ardoz, Madrid, Spain. Electronic address: jessica_j30@hotmail.com.; Marazuela M; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: monica.marazuela@uam.es.; Sánchez de la Blanca N; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: nsdelablanca.96@gmail.com.; Fernández-Contreras R; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: raufer03@ucm.es.; Arranz-Martín A; Department of Endocrinology and Nutrition, Health Research Institute, Hospital Universitario de La Princesa, Universidad Autónoma de Madrid, 28006 Madrid, Spain. Electronic address: alfarranz@hotmail.com.
Source
Publisher: Elsevier Country of Publication: England NLM ID: 8309603 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1532-1983 (Electronic) Linking ISSN: 02615614 NLM ISO Abbreviation: Clin Nutr Subsets: MEDLINE
Subject
Language
English
Abstract
Background & Aim: Malnutrition is a prevalent condition in Cystic Fibrosis (CF) and can result in worsening of pulmonary function and other comorbidities. Cystic fibrosis transmembrane regulator (CFTR) modulator therapies are improving the CF-related care and outcomes. Body Mass Index (BMI) is the most commonly used parameter to assess nutritional status, albeit it is a very unspecific indicator. Hence, current guidelines recommend body composition analysis as a part of nutritional assessment. The aim of our study was to evaluate the impact of elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) treatment on body composition and respiratory function.
Methods: We recruited patients with CF from University Hospital La Princesa, with follow-up in the Adult Cystic Fibrosis Unit. All patients were eligible to initiate ELX/TEZ/IVA therapy. Body composition was assessed with a Bioelectrical Impedance Analysis (BIA) and spirometry data were obtained before and after 6 months of treatment.
Results: Our study sample was composed of 36 patients with CF. We observed a significant increase in BMI after 6 months of treatment (p < 0.001), as well as an increase in fat mass (p = 0.008) and visceral fat area (p = 0.026). The other body composition parameters did not yield significant changes. Overall, %FEV1 increased from 72.67 % (±17.39) to 84.74 % (±18.18) after 6 months of treatment. Interestingly, we found an inverse correlation between %FEV1 and fat mass (r = -0,476; p = 0,0058), %FEV1 and age (r = -0,411; p = 0,0196) and between %FEV1 and visceral fat area (r = -0,515; p = 0,0025). On the contrary, we found a direct correlation between %FEV1 and body cell mass (r = 0,367; p = 0,038).
Conclusions: Novel CFTR modulators are emerging for the treatment of CF. Specifically, triple combination with ELX/TEZ/IVA has shown to effectively improve both pulmonary and nutritional status in patients with CF with F508del mutation. Body composition should be a part of the routine assessment for patients with CF.
Competing Interests: Conflicts of interest The authors have no conflict of interest to declare.
(Copyright © 2023. Published by Elsevier Ltd.)
Methods: We recruited patients with CF from University Hospital La Princesa, with follow-up in the Adult Cystic Fibrosis Unit. All patients were eligible to initiate ELX/TEZ/IVA therapy. Body composition was assessed with a Bioelectrical Impedance Analysis (BIA) and spirometry data were obtained before and after 6 months of treatment.
Results: Our study sample was composed of 36 patients with CF. We observed a significant increase in BMI after 6 months of treatment (p < 0.001), as well as an increase in fat mass (p = 0.008) and visceral fat area (p = 0.026). The other body composition parameters did not yield significant changes. Overall, %FEV1 increased from 72.67 % (±17.39) to 84.74 % (±18.18) after 6 months of treatment. Interestingly, we found an inverse correlation between %FEV1 and fat mass (r = -0,476; p = 0,0058), %FEV1 and age (r = -0,411; p = 0,0196) and between %FEV1 and visceral fat area (r = -0,515; p = 0,0025). On the contrary, we found a direct correlation between %FEV1 and body cell mass (r = 0,367; p = 0,038).
Conclusions: Novel CFTR modulators are emerging for the treatment of CF. Specifically, triple combination with ELX/TEZ/IVA has shown to effectively improve both pulmonary and nutritional status in patients with CF with F508del mutation. Body composition should be a part of the routine assessment for patients with CF.
Competing Interests: Conflicts of interest The authors have no conflict of interest to declare.
(Copyright © 2023. Published by Elsevier Ltd.)