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

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) (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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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
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