Evaluating the Effect of Kaftrio on Perspectives of Health and Wellbeing in Individuals with Cystic Fibrosis

Abstract

Background: Modulator therapy represents a significant step forward in CF care and is expected to have a significant impact on the health and mortality of many individuals with CF. Studies have predominantly explored the physiological effects of modulator therapy on clinical outcomes, with little consideration of the individual lived experience of modulator therapy among adults with Cystic Fibrosis. Methods: To explore this, semi-structured interviews were conducted with 12 individuals currently taking Kaftrio, which were subsequently thematically analysed. Results: Three overarching themes were identified: (i) positive perception of Kaftrio, (ii) negative perception of Kaftrio, and (iii) the relationships with the clinical team. The experience of modulator therapy should be recognised as being unique to the individual, with perceptions of illness, self-identity, and outcomes strongly dictating the lived experience. Conclusions: There is a consensus that, while for many, the quality of life is evidently increased through the use of Kaftrio, this is not without its own challenges. This highlights the need for both individuals with CF and their clinical teams to learn to navigate this new disease landscape.

Survey of exercise testing and training in cystic fibrosis clinics in the UK: a decade of progress

“… 34 The importance of experienced and qualified staff is recognised in recent clinical exercise testing guidelines, 11 and supported by a nationwide effort in the UK to establish clinical exercise physiologists within the healthcare workforce. 35 Considering this increased value of exercise professionals is alongside increasing recognition for the health benefits associated with exercise by society in general, perceived benefit of CFTR modulator treatments on perspectives on quality of life and health status 36 and the recognised benefit of exercise professionals by pwCF themselves; 37 CF MDTs should consider the incorporation of exercise specialists 27 or clinical exercise physiologists 35 into their teams. 38 These roles exist in countries outside of the UK, 39–41 and it is likely their prevalence will increase within the National Health Service, given a recent charter and push for standardised roles.…”

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

“…Using the SNOT questionnaire, Bode and collaborators also reported improved scores in nasal, otologic, sleep, and emotional subdomains in children and adults with CF receiving ETI therapy [ 77 ]. Aspinall and collaborators used a different approach and interviewed 12 PwCF who were receiving ETI therapy for at least six months [ 142 ]. Overall, an improvement in self-relating quality of life in daily routine was observed.…”

“…The reduction in the frequency of exacerbation and the number of hospitalizations led these PwCF to feel healthier, which resulted in less anxiety. They also reported a feeling of more independence and hope [ 142 ].…”

“…An improvement in objective sleep measures, such as oxygen saturation during sleep and apnea/hypopnea index, was observed, which may be associated with the increase in ppFEV 1 and respiratory rates [ 143 ]. It is noteworthy that by increasing sleep quality, PwCF also observed an improvement in other aspects of their lives, including quality of life [ 142 ].…”

“…On the other hand, it has been suggested that ETI may cross the blood–brain barrier due to their lipophilic affinity and might contribute to some psychiatric and sleep disorders that have been reported after the start of the therapy [ 142 , 144 , 145 , 146 ]. Zhang and collaborators investigated the potential impact of ETI therapy on depression and anxiety of 100 PwCF [ 147 ].…”