Standards of care for <i>CFTR</i> variant-specific therapy (including modulators) for people with cystic fibrosi

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Southern, Kevin W ORCID: 0000-0001-6516-9083, Castellani, Carlo, Lammertyn, Elise, Smyth, Alan, VanDevanter, Donald, van Koningsbruggen-Rietschel, Silke, Barben, Juerg, Bevan, Amanda, Brokaar, Edwin, Collins, Sarah et al (show 32 more authors) , Connett, Gary J, Daniels, Thomas WV, Davies, Jane, Declercq, Dimitri, Gartner, Silvia, Gramegna, Andrea, Hamilton, Naomi, Hauser, Jenny, Kashirskaya, Nataliya, Kessler, Laurence, Lowdon, Jacqueline, Makukh, Halyna, Martin, Clemence, Morrison, Lisa, Nazareth, Dilip ORCID: 0000-0002-6114-0467, Noordhoek, Jacquelien, O'Neill, Ciaran, Owen, Elizabeth, Oxley, Helen, Raraigh, Karen S, Raynal, Caroline, Robinson, Karen, Roehmel, Jobst, Schwarz, Carsten, Sermet, Isabelle, Shteinberg, Michal, Sinha, Ian, Takawira, Constance, van Mourik, Peter, Verkleij, Marieke, Waller, Michael D and Duff, Alistair (2023) Standards of care for <i>CFTR</i> variant-specific therapy (including modulators) for people with cystic fibrosi. JOURNAL OF CYSTIC FIBROSIS, 22 (1). pp. 17-30.

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Abstract

Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR modulators, the first variant-specific therapy available, have transformed the management of CF. The latest standards of care from the European CF Society (2018) did not include guidance on variant-specific therapy, as CFTR modulators were becoming established as a novel therapy. We have produced interim standards to guide healthcare professionals in the provision of variant-specific therapy for people with CF. Here we provide evidence-based guidance covering the spectrum of care, established using evidence from systematic reviews and expert opinion. Statements were reviewed by key stakeholders using Delphi methodology, with agreement (≥80%) achieved for all statements after one round of consultation. Issues around accessibility are discussed and there is clear consensus that all eligible people with CF should have access to variant-specific therapy.

Item Type:	Article
Uncontrolled Keywords:	Cystic fibrosis, CFTR, Variant-specific therapy, CFTR modulators, Guidelines
Divisions:	Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User:	Symplectic Admin
Date Deposited:	07 Dec 2022 09:28
Last Modified:	11 Oct 2023 10:36
DOI:	10.1016/j.jcf.2022.10.002
Open Access URL:	https://doi.org/10.1016/j.jcf.2022.10.002
Related URLs:	Author Publisher
URI:	https://livrepository.liverpool.ac.uk/id/eprint/3166532