Perspectives on inhaled medicines in the CFTR modulator era: A national survey of adults with cystic fibrosis.

INTRODUCTION

The transformative effects of CFTR modulators (CFTRm) have led people with cystic fibrosis (PwCF) and clinicians to re-evaluate the role of inhaled medicines. Our objective was to understand the perspectives of pwCF.

METHODS

A survey was shared via two CF-specific UK email lists and social media. Participants rated the importance of inhaled medicines from 0 (least) to 100 (most) and noted any relative changes since commencing CFTRm. Free-text responses were categorised into insight-generating themes.

RESULTS

Responses were received from 414 pwCF, of whom 208 (50%) were female, representing 17 of the 26 UK adult CF centres. Most were prescribed CFTRm (n = 344, 83%) and/or inhaled medicines (n = 321, 78%). Perceived importance of inhaled medicines was significantly lower among those prescribed CFTRm (median 72/100, IQR 49-94) compared with those not prescribed CFTRm (88/100, 52-100, p = 0.03). Among those prescribed CFTRm, 138 (40%) considered inhaled medicines less important than before treatment, citing reduced symptoms, lack of perceived benefit, and a desire to decrease treatment burden. Conversely, 174 (51%) regarded inhaled medicines as equally or more important, referencing cumulative lung damage, clinician advice, perceived benefit, and sustaining long-term health.

CONCLUSION

The impact of CFTRm on the perceived importance of inhaled medicines is variable amongst pwCF. Whilst population-level evidence regarding the clinical impact of reducing inhaled medicines regimens for PwCF taking CFTRm is awaited, clinicians should be aware of the variation in patient perspective during the process of shared decision-making and individualisation of care.