Nutritional Status and Circulating Levels of Fat-Soluble Vitamins in Cystic Fibrosis Patients: A Cohort Study and Evaluation of the Effect of CFTR Modulators

被引：20

作者：

Francalanci, Michela ^{[1
]}

Terlizzi, Vito ^{[1
]}

Fevola, Cristina ^{[1
]}

Di Rosa, Giulia ^{[1
]}

Pierattini, Valentina ^{[2
]}

Roselli, Elena ^{[2
]}

Bonomi, Paolo ^{[3
]}

Cavicchi, Maria Chiara ^{[1
]}

Galici, Valeria ^{[1
]}

Neri, Anna Silvia ^{[1
]}

Bianchimani, Chiara ^{[1
]}

Campana, Silvia ^{[1
]}

Dolce, Daniela ^{[1
]}

Ravenni, Novella ^{[1
]}

Camera, Erica ^{[1
]}

Orioli, Tommaso ^{[1
]}

Taccetti, Giovanni ^{[1
]}

机构：

[1] Meyer Childrens Hosp IRCCS, Cyst Fibrosis Reg Reference Ctr, Dept Paediat Med, I-50139 Florence, Italy

[2] Meyer Childrens Hosp IRCCS, Profess Dietet, I-50139 Florence, Italy

[3] Freelance Statistician, I-20146 Milan, Italy

来源：

CHILDREN-BASEL | 2023年 / 10卷 / 02期

关键词：

cystic fibrosis; malnutrition; CFTR modulators; TEZACAFTOR-IVACAFTOR; EFFICACY; MUTATION; SAFETY; LUMACAFTOR; PHASE-3; F508DEL-CFTR; GUIDELINES; DIAGNOSIS;

D O I：

10.3390/children10020252

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Background: Improved therapy in CF has led to an overall improvement in nutritional status. The objectives of our study are: to cross-sectionally assess nutritional status and serum levels of fat-soluble vitamins; to retrospectively evaluate the efficacy of modulators on nutritional status and fat-soluble vitamin levels. Methods: In patients younger than 2 years of age, we evaluated growth, in patients aged 2-18 years, we assessed BMI z-scores, and in adults, we assessed absolute BMI values. Levels of 25(OH)D, vitamins A, and E were measured. Results: A cross-sectional analysis was conducted on 318 patients, 109 (34.3%) with pancreatic sufficiency. Only three patients were under 2 years old. In 135 patients aged 2-18 years, the median BMI z-score was 0.11, and 5 (3.7%) patients had malnutrition (z-score <= 2SD). In 180 adults, the median BMI was 21.8 kg/m(2). Overall, 15 (13.7%) males (M) and 18 (25.3%) females (F) were underweight (18 < BMI > 20); 3 (2.7%) M and 5 (7.0%) F had a BMI < 18. Suboptimal 25(OH)D levels were found in patients with pancreatic insufficiency. The prevalence of deficiency of vitamins A and E is low. After one year of treatment with modulators, the increase in BMI was more consistent (M: 1.58 +/- 1.25 kg/m(2) F: 1.77 +/- 1.21 kg/m(2)) in elexacaftor/tezacaftor/ivacaftor (ETI)-treated patients compared with other modulators, with a significant increase in levels of all fat-soluble vitamins. Conclusions: Malnutrition is present in a limited number of subjects. The prevalence of subjects with suboptimal 25(OH)D levels is high. ETI showed a beneficial effect on nutritional status and circulating levels of fat-soluble vitamins.

引用

页数：11

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