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CFTR Modulators: Does One Dose Fit All?

Department of Pulmonology, Haga Teaching Hospital, Els Borst-Eilersplein 275, 2545 AA The Hague, The Netherlands
Central Hospital Pharmacy, Charlotte Jacobslaan 70, 2545 AB The Hague, The Netherlands
Department of Pulmonology, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, The Netherlands
Author to whom correspondence should be addressed.
Academic Editor: Youssef Daali
J. Pers. Med. 2021, 11(6), 458;
Received: 17 April 2021 / Revised: 10 May 2021 / Accepted: 12 May 2021 / Published: 24 May 2021
(This article belongs to the Special Issue Cystic Fibrosis and Personalized Medicine)
For many people with cystic fibrosis (pwCF), CFTR modulators will be the cornerstone of their treatment. These modulators show robust treatment effects at group level in pwCF with specific mutations. The individual effect however, is variable. In this review we will explain reasons for reconsideration of dosing regimens of CFTR modulating therapy in order to improve treatment response and prevent side effects. Since the effect of a drug depends on pharmacodynamics and pharmacokinetics, pharmacodynamics and pharmacokinetic properties of CFTR modulators will be discussed. Pharmacokinetic-pharmacodynamic relationships will be used to gain insight in dosage response and exposure response relationships. To understand the cause of variation in drug exposure, pharmacokinetic properties that may change due to CF disease will be explained. We show that with current insight, there are conceivable situations that give reason for reconsideration of dosing regimens, however many questions need to be unravelled. View Full-Text
Keywords: cystic fibrosis; CFTR modulators; pharmacodynamics; pharmacokinetics; exposure response relationship cystic fibrosis; CFTR modulators; pharmacodynamics; pharmacokinetics; exposure response relationship
MDPI and ACS Style

van der Meer, R.; Wilms, E.B.; Heijerman, H.G.M. CFTR Modulators: Does One Dose Fit All? J. Pers. Med. 2021, 11, 458.

AMA Style

van der Meer R, Wilms EB, Heijerman HGM. CFTR Modulators: Does One Dose Fit All? Journal of Personalized Medicine. 2021; 11(6):458.

Chicago/Turabian Style

van der Meer, Renske, Erik B. Wilms, and Harry G.M. Heijerman. 2021. "CFTR Modulators: Does One Dose Fit All?" Journal of Personalized Medicine 11, no. 6: 458.

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