WS02.04Perspectives on inhaled therapy in the post-CFTR modulator era: a survey of adults with cystic fibrosis

Abstract

Objectives

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

Methods

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

Results

Responses were received from 414 people with CF, of which 224 (54%) identified as female. Responders represented 17/26 UK adult CF centres (range 1-35 per centre). The majority were prescribed CFTRm (n=344, 83.1%) and/or inhaled therapy (n=321, 77.5%). Overall, the perceived importance of inhaled therapy was significantly lower among those on CFTRm, median of 72/100 (IQR: 48.0) compared to 88 (IQR: 45.3), p = 0.03. Among those prescribed CFTRm, 138 (40.1%) regarded inhaled therapy as less important than prior to CFTRm. This perspective was attributed to a reduction in symptoms, a lack of perceived benefit from inhaled therapy, and a desire to decrease treatment burden. Several considered inhaled therapy more appropriate for acute illness rather than routine maintenance. Conversely, 172 (50%) considered inhaled therapy equally or more important than before CFTRm. Their rationale included an awareness of cumulative lung damage, clinician recommendations, a discernible benefit from inhaled therapy, and the belief that it helps sustain the benefits of CFTRm into later life.

Conclusions

The impact of CFTRm on perspectives regarding inhaled therapy is variable amongst the awCF in the UK. Whilst population-level evidence regarding the clinical impact of changing inhaled therapy regimens for awCF 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.