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.
Original language | English |
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Pages (from-to) | 17-30 |
Number of pages | 14 |
Journal | Journal of Cystic Fibrosis |
Volume | 22 |
Issue number | 1 |
DOIs | |
Publication status | Published - Jan 2023 |
Keywords
- CFTR
- CFTR modulators
- Cystic fibrosis
- Guidelines
- Variant-specific therapy