Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis
Southern, Kevin W.; Castellani, Carlo; Lammertyn, Elise; Smyth, Alan; VanDevanter, Donald; van Koningsbruggen-Rietschel, Silke; Barben, Jürg; Bevan, Amanda; Brokaar, Edwin; Collins, Sarah; Connett, Gary J.; Daniels, Thomas W.V.; Davies, Jane; Declercq, Dimitri; Gartner, Silvia; Gramegna, Andrea; Hamilton, Naomi; Hauser, Jenny; Kashirskaya, Nataliya; Kessler, Laurence; Lowdon, Jacqueline; Makukh, Halyna; Martin, Clémence; Morrison, Lisa; Nazareth, Dilip; 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.; Duff, Alistair
(2023) Journal of Cystic Fibrosis, volume 22, issue 1, pp. 17 - 30
(Article)
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
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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.
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Keywords: CFTR, CFTR modulators, Cystic fibrosis, Guidelines, Variant-specific therapy, Pediatrics, Perinatology, and Child Health, Pulmonary and Respiratory Medicine
ISSN: 1569-1993
Publisher: Elsevier
Note: Publisher Copyright: © 2022
(Peer reviewed)