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Article

CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population

1
Department of Pulmonology, Allergology and Pulmonary Oncology, Poznan University of Medical Sciences, 84 Szamarzewskiego St, 60-569 Poznań, Poland
2
Chair and Department of Laboratory Diagnostics, Poznan University of Medical Sciences, 84 Szamarzewskiego St, 60-569 Poznań, Poland
*
Author to whom correspondence should be addressed.
J. Clin. Med. 2024, 13(15), 4491; https://doi.org/10.3390/jcm13154491
Submission received: 19 June 2024 / Revised: 10 July 2024 / Accepted: 26 July 2024 / Published: 31 July 2024
(This article belongs to the Special Issue Cystic Fibrosis: Clinical Manifestations and Treatment)

Abstract

Background/Objectives: Cystic fibrosis is a genetically determined disease that significantly influences and shortens life. Treatment with CFTR modulators (CFTR-T) is a new hope for patients. It can change the predictive values of a poor prognosis (e.g., exacerbation rate and FEV1 value). The aim of the study was to analyse exacerbation incidence and spirometry data before and after one year (+/− 2 weeks) of CFTR-T in 85 CF patients at the CF Centre in Poznań. To our knowledge, this is the first analysis of CFTR-T efficiency in the Central–Eastern Europe population. Methods: We retrospectively analysed the spirometry and exacerbation data of 85 CF adult patients (both men and women), who in the middle of 2022 began treatment with CFTR modulators. Results: The one-year ratio of hospitalisation caused by severe exacerbations lowered from 1.25 to 0.21 per patient per year. We also saw a 66% decline in ambulatory exacerbations. The median FEV1% increased by 9.60% in absolute values and by 460 mL. Even in the group with very severe obstruction (FEV1 < 35%), there was an increase in median FEV1% of 5.9 in absolute values. We also proved the increase in FVC% (median 17.10% in absolute value and 600 mL) in the study group. Conclusions: After one year of treatment, an impressive improvement was observed in two important predictive values of poor prognosis: exacerbation rate and FEV1 values. Further observation is needed to determine how long the improvement will be present and its influence on quality of life and life expectancy.
Keywords: cystic fibrosis; CFTR modulators; spirometry; exacerbations cystic fibrosis; CFTR modulators; spirometry; exacerbations

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MDPI and ACS Style

Winiarska, H.M.; Springer, D.; Wojtaś, F.; Wysocka, E.; Cofta, S. CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population. J. Clin. Med. 2024, 13, 4491. https://doi.org/10.3390/jcm13154491

AMA Style

Winiarska HM, Springer D, Wojtaś F, Wysocka E, Cofta S. CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population. Journal of Clinical Medicine. 2024; 13(15):4491. https://doi.org/10.3390/jcm13154491

Chicago/Turabian Style

Winiarska, Hanna M., Daria Springer, Filip Wojtaś, Ewa Wysocka, and Szczepan Cofta. 2024. "CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population" Journal of Clinical Medicine 13, no. 15: 4491. https://doi.org/10.3390/jcm13154491

APA Style

Winiarska, H. M., Springer, D., Wojtaś, F., Wysocka, E., & Cofta, S. (2024). CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population. Journal of Clinical Medicine, 13(15), 4491. https://doi.org/10.3390/jcm13154491

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