Consensus documents

2023 Standards of care for CFTR variant-specific therapy for people with CF

We are proud to announce that four of our CF editors and several more of our Group’s contributors, supported by the European Cystic Fibrosis Society, have written and published detailed standards of care for CFTR variant-specific therapy (including modulators) for people with CF. The recently published paper is freely available to access here.

In recent years, therapy tailored to specific variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene has transformed the management of CF and consequently the lives of people living with CF. Taken orally, variant-specific therapy corrects the molecular defect that causes the condition, rather than treating the clinical issues resulting from the condition; thus a relatively simple treatment can greatly ease the treatment burden for those people with CF whose genetic variants can be treated with this therapy. This is still an emerging area of research, but the CFGD Group’s up-to-date Cochrane Reviews underpin the new evidence-based pragmatic guidance for people with CF and their CF teams. The paper also highlights the importance of continued standard treatment for those whose genotypes are as yet ineligible for variant-specific therapy and of continued research to identify and develop therapy for those genotypes.