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. 2023 Apr 24;9(5):e15756.
doi: 10.1016/j.heliyon.2023.e15756. eCollection 2023 May.

Elexacaftor - Tezacaftor - Ivacaftor treatment improves systemic infection parameters and Pseudomonas aeruginosa colonization rate in patients with cystic fibrosis a monocentric observational study

Alexander Schnell  1 , Hannah Hober  1 , Natalie Kaiser  1 , Renate Ruppel  1 , Annika Geppert  2 , Christina Tremel  1 , Julia Sobel  1 , Erika Plattner  1 , Joachim Woelfle  1 , André Hoerning  1
Affiliations

Elexacaftor - Tezacaftor - Ivacaftor treatment improves systemic infection parameters and Pseudomonas aeruginosa colonization rate in patients with cystic fibrosis a monocentric observational study

Alexander Schnell et al. Heliyon. .

Abstract

Background & aims: The CFTR-modulating therapy Elexaftor - Tezacaftor - Ivacaftor (ETI) has been widely prescribed since its approval in 2020 in the European Union. The aim of this study was to methodically evaluate the effects of an ETI treatment on clinical, biochemical data and Pseudomonas colonization in order to demonstrate its efficacy.

Methods: This prospective monocentric study comprised 69 patients diagnosed with cystic fibrosis aged at least 12 years and treated with ETI between September 2020 and November 2021. Clinical and laboratory data of each patient and study visit were collected before and after 24 weeks of ETI treatment. Follow-up status of Pseudomonas aeruginosa (PsA) colonization was assessed after one year of therapy by regularly determined sputum or throat swab samples.

Results: Marked improvements biochemical markers of systemic inflammation as white blood cell count, levels of immunoglobulins A, G and M and albumin within 24 weeks of therapy were observed. ETI treatment proved to be effective as seen by amelioration of lung function and sweat chloride concentration. Assessment of PsA colonization status revealed a conversion from a positive to negative detection in 36% of the cases after one year of therapy.

Conclusions: ETI treatment effectively improves systemic inflammation parameters and shows promising results in PsA status conversion.

Keywords: CFTR modulator; Clinical data; Cystic fibrosis; ETI; Elexacaftor; Inflammation markers; Ivacaftor; Kaftrio; Tezacaftor.

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Conflict of interest statement

AS is stock owner of Vertex Pharmaceuticals. AH has received research grants for clinical studies, speaker’s fees, honoraria or travel expenses from Abbvie, Astellas, MSD, Novartis, Nutricia and Shire/Takeda.

Figures

Fig. 1
Fig. 1
Study flow.
Fig. 2
Fig. 2
Proportion of Pseudomonas aeruginosa (PsA) status change after one year of ETI therapy.
See this image and copyright information in PMC

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