Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis

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 KesslerJacqueline 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, Jobst Roehmel, Carsten Schwarz, Isabelle Sermet, Michal Shteinberg, Ian Sinha, Constance Takawira, Peter van Mourik, Marieke Verkleij, Michael D. Waller, Alistair Duff

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)17-30
Number of pages14
JournalJournal of Cystic Fibrosis
Issue number1
StatePublished - Jan 2023


  • CFTR
  • CFTR modulators
  • Cystic fibrosis
  • Guidelines
  • Variant-specific therapy

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Pediatrics, Perinatology, and Child Health


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