The long-term effect of elexacaftor/tezacaftor/ivacaftor on
cardiorespiratory fitness in adolescent patients with cystic
fibrosis: a pilot observational study

J 2024

The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study

ŠŤASTNÁ, Nela, Lenka HRABOVSKÁ, Pavel HOMOLKA, Lukáš HOMOLA, Michal SVOBODA et. al.

Basic information

Original name

The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study

Authors

ŠŤASTNÁ, Nela (203 Czech Republic, belonging to the institution), Lenka HRABOVSKÁ (203 Czech Republic, belonging to the institution), Pavel HOMOLKA (203 Czech Republic, belonging to the institution), Lukáš HOMOLA (203 Czech Republic, belonging to the institution), Michal SVOBODA (203 Czech Republic, belonging to the institution), Kristián BRAT (703 Slovakia, belonging to the institution) and Libor FILA (203 Czech Republic)

Edition

BMC Pulmonary Medicine, LONDON, BMC, 2024, 1471-2466

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30203 Respiratory systems

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.100 in 2022

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1186/s12890-024-03069-8

UT WoS

001233958000001

Keywords in English

Cystic fibrosis; Cardiopulmonary exercise testing; Elexacaftor/tezacaftor/ivacaftor

Abstract

V originále

Background Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO2peak) is an appropriate tool to evaluate the physical activity in these patients. However, there are several other valuable CPET parameters describing exercise tolerance (Wpeak, VO2VT1, VO2VT2, VO2/HRpeak, etc.), and helping to better understand the effect of specific treatment (VE, VT, VD/VT etc.). Limited data showed ambiguous results of this improvement after CFTR modulator treatment. Elexacaftor/tezacaftor/ivacaftor medication improves pulmonary function and quality of life, whereas its effect on CPET has yet to be sufficiently demonstrated. Methods We performed a single group prospective observational study of 10 adolescent patients with cystic fibrosis who completed two CPET measurements between January 2019 and February 2023. During this period, elexacaftor/tezacaftor/ivacaftor treatment was initiated in all of them. The first CPET at the baseline was followed by controlled CPET at least one year after medication commencement. We focused on interpreting the data on their influence by the novel therapy. We hypothesized improvements in cardiorespiratory fitness following treatment. We applied the Wilcoxon signed-rank test. The data were adjusted for age at the time of CPET to eliminate bias of aging in adolescent patients. Results We observed significant improvement in peak workload, VO2 peak, VO2VT1, VO2VT2, VE/VCO2 slope, VE, VT, RQ, VO2/HR peak and RR peak. The mean change in VO2 peak was 5.7 mL/kg/min, or 15.9% of the reference value (SD ± 16.6; p= 0.014). VO2VT1 improved by 15% of the reference value (SD ± 0.1; p= 0.014), VO2VT2 improved by 0.5 (SD ± 0.4; p= 0.01). There were no differences in other parameters. Conclusion Exercise tolerance improved after elexacaftor/tezacaftor/ivacaftor treatment initiation. We suggest that the CFTR modulator alone is not enough for recovering physical decondition, but should be supplemented with physical activity and respiratory physiotherapy. Further studies are needed to examine the effect of CFTR modulators and physical therapy on cardiopulmonary exercise tolerance.

Citovat

ŠŤASTNÁ, Nela, Lenka HRABOVSKÁ, Pavel HOMOLKA, Lukáš HOMOLA, Michal SVOBODA, Kristián BRAT and Libor FILA. The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study. BMC Pulmonary Medicine. LONDON: BMC, 2024, vol. 24, No 1, p. 1-6. ISSN 1471-2466. Available from: https://dx.doi.org/10.1186/s12890-024-03069-8.

@article{2407839,
   author = {Šťastná, Nela and Hrabovská, Lenka and Homolka, Pavel and Homola, Lukáš and Svoboda, Michal and Brat, Kristián and Fila, Libor},
   article_location = {LONDON},
   article_number = {1},
   doi = {http://dx.doi.org/10.1186/s12890-024-03069-8},
   keywords = {Cystic fibrosis; Cardiopulmonary exercise testing; Elexacaftor/tezacaftor/ivacaftor},
   language = {eng},
   issn = {1471-2466},
   journal = {BMC Pulmonary Medicine},
   title = {The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study},
   url = {https://bmcpulmmed.biomedcentral.com/articles/10.1186/s12890-024-03069-8},
   volume = {24},
   year = {2024}
}

TY  - JOUR
ID  - 2407839
AU  - Šťastná, Nela - Hrabovská, Lenka - Homolka, Pavel - Homola, Lukáš - Svoboda, Michal - Brat, Kristián - Fila, Libor
PY  - 2024
TI  - The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study
JF  - BMC Pulmonary Medicine
VL  - 24
IS  - 1
SP  - 1-6
EP  - 1-6
PB  - BMC
SN  - 14712466
KW  - Cystic fibrosis
KW  - Cardiopulmonary exercise testing
KW  - Elexacaftor/tezacaftor/ivacaftor
UR  - https://bmcpulmmed.biomedcentral.com/articles/10.1186/s12890-024-03069-8
N2  - Background Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO2peak) is an appropriate tool to evaluate the physical activity in these patients. However, there are several other valuable CPET parameters describing exercise tolerance (Wpeak, VO2VT1, VO2VT2, VO2/HRpeak, etc.), and helping to better understand the effect of specific treatment (VE, VT, VD/VT etc.). Limited data showed ambiguous results of this improvement after CFTR modulator treatment. Elexacaftor/tezacaftor/ivacaftor medication improves pulmonary function and quality of life, whereas its effect on CPET has yet to be sufficiently demonstrated. Methods We performed a single group prospective observational study of 10 adolescent patients with cystic fibrosis who completed two CPET measurements between January 2019 and February 2023. During this period, elexacaftor/tezacaftor/ivacaftor treatment was initiated in all of them. The first CPET at the baseline was followed by controlled CPET at least one year after medication commencement. We focused on interpreting the data on their influence by the novel therapy. We hypothesized improvements in cardiorespiratory fitness following treatment. We applied the Wilcoxon signed-rank test. The data were adjusted for age at the time of CPET to eliminate bias of aging in adolescent patients. Results We observed significant improvement in peak workload, VO2 peak, VO2VT1, VO2VT2, VE/VCO2 slope, VE, VT, RQ, VO2/HR peak and RR peak. The mean change in VO2 peak was 5.7 mL/kg/min, or 15.9% of the reference value (SD ± 16.6; p= 0.014). VO2VT1 improved by 15% of the reference value (SD ± 0.1; p= 0.014), VO2VT2 improved by 0.5 (SD ± 0.4; p= 0.01). There were no differences in other parameters. Conclusion Exercise tolerance improved after elexacaftor/tezacaftor/ivacaftor treatment initiation. We suggest that the CFTR modulator alone is not enough for recovering physical decondition, but should be supplemented with physical activity and respiratory physiotherapy. Further studies are needed to examine the effect of CFTR modulators and physical therapy on cardiopulmonary exercise tolerance.
ER  -

ŠŤASTNÁ, Nela, Lenka HRABOVSKÁ, Pavel HOMOLKA, Lukáš HOMOLA, Michal SVOBODA, Kristián BRAT and Libor FILA. The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study. \textit{BMC Pulmonary Medicine}. LONDON: BMC, 2024, vol.~24, No~1, p.~1-6. ISSN~1471-2466. Available from: https://dx.doi.org/10.1186/s12890-024-03069-8.

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