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

J 2024

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

ŠŤASTNÁ, Nela, Lumír KUNOVSKÝ, Michal SVOBODA, Eva POKOJOVÁ, Lukáš HOMOLA et. al.

Basic information

Original name

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

Authors

ŠŤASTNÁ, Nela (203 Czech Republic, belonging to the institution), Lumír KUNOVSKÝ (203 Czech Republic, belonging to the institution), Michal SVOBODA (203 Czech Republic, belonging to the institution), Eva POKOJOVÁ (203 Czech Republic, belonging to the institution), Lukáš HOMOLA (203 Czech Republic, belonging to the institution), Miriam MALÁ (203 Czech Republic, belonging to the institution), Žaneta GRÁCOVÁ (203 Czech Republic), Barbora JEŘÁBKOVÁ (203 Czech Republic), Jana SKŘIČKOVÁ (203 Czech Republic, belonging to the institution) and Jan TRNA (203 Czech Republic, belonging to the institution)

Edition

Digestive Diseases, BASEL, KARGER, 2024, 0257-2753

Other information

Language

English

Type of outcome

Article in a journal

Field of Study

30219 Gastroenterology and hepatology

Country of publisher

Switzerland

Confidentiality degree

is not subject to a state or trade secret

References:

URL

Impact factor

Impact factor: 2.000 in 2023

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1159/000538606

UT WoS

001243080000001

Keywords in English

Cystic fibrosis; Elexacaftor/tezacaftor/ivacaftor; Nutrition; Exocrine pancreas function; Pancreatic enzyme replacement therapy

Abstract

V originále

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/m2 (p < 0.001), mean body weight by 3.51 kg (p < 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p < 0.001). Only 1 patient, initially pancreatic insufficient (4.5%, p < 0.001), developed pancreatic sufficiency, indicated by increased fecal elastase from 45 μg/g to 442.1 μg/g. Mean change in lipase substitution decreased by 1,969 units/kg/day (p < 0.001) and stools frequency by 1.18 per day (p < 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.

Citovat

ŠŤASTNÁ, Nela, Lumír KUNOVSKÝ, Michal SVOBODA, Eva POKOJOVÁ, Lukáš HOMOLA, Miriam MALÁ, Žaneta GRÁCOVÁ, Barbora JEŘÁBKOVÁ, Jana SKŘIČKOVÁ and Jan TRNA. Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor. Digestive Diseases. BASEL: KARGER, 2024, vol. 42, No 4, p. 361-368. ISSN 0257-2753. Available from: https://dx.doi.org/10.1159/000538606.

@article{2398637,
   author = {Šťastná, Nela and Kunovský, Lumír and Svoboda, Michal and Pokojová, Eva and Homola, Lukáš and Malá, Miriam and Grácová, Žaneta and Jeřábková, Barbora and Skřičková, Jana and Trna, Jan},
   article_location = {BASEL},
   article_number = {4},
   doi = {http://dx.doi.org/10.1159/000538606},
   keywords = {Cystic fibrosis; Elexacaftor/tezacaftor/ivacaftor; Nutrition; Exocrine pancreas function; Pancreatic enzyme replacement therapy},
   language = {eng},
   issn = {0257-2753},
   journal = {Digestive Diseases},
   title = {Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor},
   url = {https://karger.com/ddi/article/doi/10.1159/000538606/906676/Improved-Nutritional-Outcomes-and-Gastrointestinal},
   volume = {42},
   year = {2024}
}

TY  - JOUR
ID  - 2398637
AU  - Šťastná, Nela - Kunovský, Lumír - Svoboda, Michal - Pokojová, Eva - Homola, Lukáš - Malá, Miriam - Grácová, Žaneta - Jeřábková, Barbora - Skřičková, Jana - Trna, Jan
PY  - 2024
TI  - Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor
JF  - Digestive Diseases
VL  - 42
IS  - 4
SP  - 361-368
EP  - 361-368
PB  - KARGER
SN  - 02572753
KW  - Cystic fibrosis
KW  - Elexacaftor/tezacaftor/ivacaftor
KW  - Nutrition
KW  - Exocrine pancreas function
KW  - Pancreatic enzyme replacement therapy
UR  - https://karger.com/ddi/article/doi/10.1159/000538606/906676/Improved-Nutritional-Outcomes-and-Gastrointestinal
N2  - 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/m2 (p < 0.001), mean body weight by 3.51 kg (p < 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p < 0.001). Only 1 patient, initially pancreatic insufficient (4.5%, p < 0.001), developed pancreatic sufficiency, indicated by increased fecal elastase from 45 μg/g to 442.1 μg/g. Mean change in lipase substitution decreased by 1,969 units/kg/day (p < 0.001) and stools frequency by 1.18 per day (p < 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.
ER  -

ŠŤASTNÁ, Nela, Lumír KUNOVSKÝ, Michal SVOBODA, Eva POKOJOVÁ, Lukáš HOMOLA, Miriam MALÁ, Žaneta GRÁCOVÁ, Barbora JEŘÁBKOVÁ, Jana SKŘIČKOVÁ and Jan TRNA. Improved Nutritional Outcomes and Gastrointestinal Symptoms in Adult Cystic Fibrosis Patients Treated with Elexacaftor/Tezacaftor/Ivacaftor. \textit{Digestive Diseases}. BASEL: KARGER, 2024, vol.~42, No~4, p.~361-368. ISSN~0257-2753. Available from: https://dx.doi.org/10.1159/000538606.

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