Management of Individual Patient Expectations When Starting with Highly Effective CFTR Modulators

Bente L Aalbers; Inez Bronsveld; Regina W Hofland; Harry G M Heijerman

doi:10.3390/jpm11080811

Management of Individual Patient Expectations When Starting with Highly Effective CFTR Modulators

Bente L Aalbers, Inez Bronsveld, Regina W Hofland, Harry G M Heijerman

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Abstract

Highly effective CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ELE/TEZ/IVA will become available for an increasing number of people with cystic fibrosis (pwCF) in the near future. Before the start of this therapy, many questions may arise concerning the expected effects. We assembled the currently available data from the literature about ELE/TEZ/IVA that focused on commonly asked questions from patients. Overall, the literature so far presents a very hopeful prospect of effects, not only on lung function, but also on nutritional status, sinonasal symptoms and quality of life. The effects in patients with pwCF with severe lung damage are also favorable. Treatment is generally well tolerated. In some cases, patient-derived cell models can help in predicting the effects for individual patients.

Original language	English
Article number	811
Number of pages	7
Journal	Journal of Personalized Medicine
Volume	11
Issue number	8
DOIs	https://doi.org/10.3390/jpm11080811
Publication status	Published - 19 Aug 2021

Keywords

CFTR modulators
Corrector
Cystic fibrosis
Emerging therapies
Patient expectations
Po-tentiator

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10.3390/jpm11080811

jpm-11-00811-v2Final published version, 223 KB

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title = "Management of Individual Patient Expectations When Starting with Highly Effective CFTR Modulators",

abstract = "Highly effective CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ELE/TEZ/IVA will become available for an increasing number of people with cystic fibrosis (pwCF) in the near future. Before the start of this therapy, many questions may arise concerning the expected effects. We assembled the currently available data from the literature about ELE/TEZ/IVA that focused on commonly asked questions from patients. Overall, the literature so far presents a very hopeful prospect of effects, not only on lung function, but also on nutritional status, sinonasal symptoms and quality of life. The effects in patients with pwCF with severe lung damage are also favorable. Treatment is generally well tolerated. In some cases, patient-derived cell models can help in predicting the effects for individual patients.",

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AU - Hofland, Regina W

AU - Heijerman, Harry G M

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Management of Individual Patient Expectations When Starting with Highly Effective CFTR Modulators

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