Exploring the impact of elexacaftor-tezacaftor-ivacaftor treatment on opinions regarding airway clearance techniques and nebulisers: TEMPO a qualitative study in children with cystic fibrosis, their families and healthcare professionals

Abstract

BackgroundCystic fibrosis (CF) is a genetic condition caused by variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that primarily impacts the lungs. Treatments historically have been symptomatic to improve airway clearance and treat infection. However, CFTR modulator drugs have recently been developed that target the underlying defect. The triple combination of elexacaftor-tezacaftor-ivacaftor (ETI) was approved in 2020 in England for over 80% of people with CF aged over 12 years and in 2022 extended to those over 6 years. ETI treatment is associated with substantial improvements in lung function. The experience of children with CF starting on ETI or their views regarding future treatments have not been well studied. This study aimed to explore the opinions of children with CF, their parents/carers and healthcare professionals (HCPs) on the impact of ETI, airway clearance techniques (ACTs) and nebulised treatments.MethodsSemistructured qualitative interviews were performed with 10 children with CF, 7 parents/carers and 10 HCPs. Audio recordings were transcribed and analysed using reflexive thematic analysis.ResultsFour main themes were identified: ‘Kaftrio changed my life’, ‘Your entire life is dictated by the CF timetable’, ‘Simplifying treatment-hopes and fears’ and ‘Kaftrio is a game-changer’ along with several subthemes and an overarching theme of ‘I still can’t get my head around how three tablets can do what Kaftrio done’.ConclusionsDespite the highly positive impact of ETI on the health of children with CF some concerns remain about the longer-term outcomes of reducing ACTs or nebulised treatments. ETI has prompted a shift in treatment for many and offers an opportunity to personalise approaches.

Perceived burden of respiratory physiotherapy in people with cystic fibrosis taking elexacaftor–tezacaftor–ivacaftor combination: a 1-year observational study

“…During the first year of ETI therapy, neither the frequency nor the modality of ACT was changed; this decision was shared by other colleagues and caregivers from the CF community, 22 who agree upon the lack of short- and long-term evidence in favour of simplifying treatment. We believe that the rationale for time-consuming and low-grade evidence-based ACT is no longer tenable.…”

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

“…Most participants indicated being free from exacerbations since the start of ETI therapy and also reported improvements in various extra-pulmonary symptoms [ 72 ]. In another qualitative study, interviews were performed to explore the impact of ETI therapy on the lives of children with CF and their attitudes toward nebulization and airway clearance techniques [ 86 ]. Overall, ETI therapy was indicated to improve the health of children with CF and reduced the daily treatment burden, despite some concerns about the benefits in the long term.…”

Chronic daily respiratory care needs in people with cystic fibrosis treated with highly effective cystic fibrosis transmembrane conductance regulator modulators

“…Now that ETI is available to children with cystic fibrosis (cwCF) as young as 2 years of age in the United States and as young as 6 years of age in Europe, eliciting the opinions of cwCF and their families about treatment simplification is important. Recognizing that ACTs and nebulized medication are the most burdensome to pwCF, investigators in the UK conducted the TEMPO study [46] to evaluate opinions about the impact of ETI, ACTs, and nebulized medications on cwCF, parents of cwCF, and healthcare professionals (HCPs). Ten cwCF, 7 parents or caregivers, and 10 HCPs completed semi-structured qualitative interviews.…”