Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00209805%3A_____%2F24%3A00079826" target="_blank" >RIV/00209805:_____/24:00079826 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216224:14110/24:00135924 RIV/00216208:11130/24:10481140 RIV/61989592:15110/24:73625807 RIV/65269705:_____/24:00079880 a 2 dalších

Výsledek na webu

<a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11250406/" target="_blank" >https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11250406/</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1159/000538606" target="_blank" >10.1159/000538606</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor

Popis výsledku v původním jazyce

Introduction: Cystic fibrosis transmembrane conductance regulator modulator therapy improves nutritional status and quality of life. Clinical trials have shown pancreatic insufficiency conversion, mostly in pediatric patients treated with ivacaftor. Studies with elexacaftor/tezacaftor/ivacaftor (ETI) in older patients have not suggested restoration of exocrine pancreas function, but quality data in adults are lacking. Our aim was to show the effect of ETI in adults with cystic fibrosis (CF) on nutritional status and digestive function. We hypothesized improvement of nutritional parameters and gastrointestinal symptoms, reduction of pancreatic enzyme replacement therapy, but uncertain improvement in exocrine pancreatic function. Methods: We prospectively enrolled adults with CF treated with ETI from August 2021 to June 2022. We measured anthropometric parameters, laboratory nutritional markers, change of fecal elastase, pancreatic enzymes replacement therapy needs, and gastrointestinal symptoms. Results: In the cohort of 29 patients (mean age 29.1 years), 82.8% suffered exocrine pancreatic insufficiency. After ETI, mean BMI increased by 1.20 kg/m(2) (p &lt; 0.001), mean body weight by 3.51 kg (p &lt; 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p &lt; 0.001). Only 1 patient, initially pancreatic insufficient (4.5%, p &lt; 0.001), developed pancreatic sufficiency, indicated by increased fecal elastase from 45 mu g/g to 442.1 mu g/g. Mean change in lipase substitution decreased by 1,969 units/kg/day (p &lt; 0.001) and stools frequency by 1.18 per day (p &lt; 0.001). Conclusion: Our data suggest increased nutritional parameters, lower pancreatic substitution requirements, and improved defecation in adult CF patients on ETI. Improvement in exocrine pancreatic function might be mutation-specific and needs further study.

Název v anglickém jazyce

Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor

Popis výsledku anglicky

Introduction: Cystic fibrosis transmembrane conductance regulator modulator therapy improves nutritional status and quality of life. Clinical trials have shown pancreatic insufficiency conversion, mostly in pediatric patients treated with ivacaftor. Studies with elexacaftor/tezacaftor/ivacaftor (ETI) in older patients have not suggested restoration of exocrine pancreas function, but quality data in adults are lacking. Our aim was to show the effect of ETI in adults with cystic fibrosis (CF) on nutritional status and digestive function. We hypothesized improvement of nutritional parameters and gastrointestinal symptoms, reduction of pancreatic enzyme replacement therapy, but uncertain improvement in exocrine pancreatic function. Methods: We prospectively enrolled adults with CF treated with ETI from August 2021 to June 2022. We measured anthropometric parameters, laboratory nutritional markers, change of fecal elastase, pancreatic enzymes replacement therapy needs, and gastrointestinal symptoms. Results: In the cohort of 29 patients (mean age 29.1 years), 82.8% suffered exocrine pancreatic insufficiency. After ETI, mean BMI increased by 1.20 kg/m(2) (p &lt; 0.001), mean body weight by 3.51 kg (p &lt; 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p &lt; 0.001). Only 1 patient, initially pancreatic insufficient (4.5%, p &lt; 0.001), developed pancreatic sufficiency, indicated by increased fecal elastase from 45 mu g/g to 442.1 mu g/g. Mean change in lipase substitution decreased by 1,969 units/kg/day (p &lt; 0.001) and stools frequency by 1.18 per day (p &lt; 0.001). Conclusion: Our data suggest increased nutritional parameters, lower pancreatic substitution requirements, and improved defecation in adult CF patients on ETI. Improvement in exocrine pancreatic function might be mutation-specific and needs further study.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30219 - Gastroenterology and hepatology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2024

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Digestive diseases

ISSN

0257-2753

e-ISSN

1421-9875

Svazek periodika

42

Číslo periodika v rámci svazku

4

Stát vydavatele periodika

DE - Spolková republika Německo

Počet stran výsledku

8

Strana od-do

361-368

Kód UT WoS článku

001243080000001

EID výsledku v databázi Scopus

2-s2.0-85195096508