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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

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Kevin W. Southern

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

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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


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.


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.

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 Nov 10, 2022
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
Keywords Pulmonary and Respiratory Medicine, Pediatrics, Perinatology and Child Health
Public URL
Publisher URL


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