Kevin W. Southern
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
Authors
Carlo Castellani
Elise Lammertyn
Alan Smyth
Donald VanDevanter
Silke van Koningsbruggen-Rietschel
Jürg Barben
Amanda Bevan
Edwin Brokaar
Sarah Collins
Gary J. Connett
Thomas W.V. Daniels
Jane Davies
Dimitri Declercq
Silvia Gartner
Andrea Gramegna
Naomi Hamilton
Jenny Hauser
Nataliya Kashirskaya
Laurence Kessler
Jacqueline Lowdon
Halyna Makukh
Clémence Martin
Lisa Morrison
Dilip Nazareth
Jacquelien Noordhoek
Ciaran O'Neill
Elizabeth Owen
Helen Oxley
Karen S. Raraigh
Caroline Raynal
KAREN ROBINSON karen.robinson@nottingham.ac.uk
Professor in Gastrointestinal Infection and Immunity
Jobst Roehmel
Carsten Schwarz
Isabelle Sermet
Michal Shteinberg
Ian Sinha
Constance Takawira
Peter van Mourik
Marieke Verkleij
Michael D. Waller
Alistair Duff
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.
Citation
Southern, K. W., Castellani, C., Lammertyn, E., Smyth, A., VanDevanter, D., van Koningsbruggen-Rietschel, S., …Duff, A. (2023). Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis. Journal of Cystic Fibrosis, 22(1), 17-30. https://doi.org/10.1016/j.jcf.2022.10.002
Journal Article Type | Article |
---|---|
Acceptance Date | Oct 10, 2022 |
Online Publication Date | Oct 27, 2022 |
Publication Date | 2023-01 |
Deposit Date | Nov 4, 2022 |
Publicly Available Date | Mar 28, 2024 |
Journal | Journal of Cystic Fibrosis |
Print ISSN | 1569-1993 |
Electronic ISSN | 1873-5010 |
Publisher | Elsevier BV |
Peer Reviewed | Peer Reviewed |
Volume | 22 |
Issue | 1 |
Pages | 17-30 |
DOI | https://doi.org/10.1016/j.jcf.2022.10.002 |
Keywords | Pulmonary and Respiratory Medicine, Pediatrics, Perinatology and Child Health |
Public URL | https://nottingham-repository.worktribe.com/output/13181522 |
Publisher URL | https://www.cysticfibrosisjournal.com/article/S1569-1993(22)01385-6/fulltext |
Files
Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis
(1.2 Mb)
PDF
Publisher Licence URL
https://creativecommons.org/licenses/by-nc-nd/4.0/
You might also like
Complex roles of TGF-β signaling pathways in lung development and bronchopulmonary dysplasia
(2023)
Journal Article
Future therapies for cystic fibrosis
(2023)
Journal Article
Wheeze in the time of COVID-19: overcoming obstacles to an unusual diagnosis
(2022)
Journal Article
Downloadable Citations
About Repository@Nottingham
Administrator e-mail: digital-library-support@nottingham.ac.uk
This application uses the following open-source libraries:
SheetJS Community Edition
Apache License Version 2.0 (http://www.apache.org/licenses/)
PDF.js
Apache License Version 2.0 (http://www.apache.org/licenses/)
Font Awesome
SIL OFL 1.1 (http://scripts.sil.org/OFL)
MIT License (http://opensource.org/licenses/mit-license.html)
CC BY 3.0 ( http://creativecommons.org/licenses/by/3.0/)
Powered by Worktribe © 2024
Advanced Search