A retrospective observational study of the effects of Kaftrio® on the current cohort of patients at Nottingham University Hospitals NUH trust

ABSTRACT

Background: Due to disease progression, people with cystic fibrosis (PwCF) were expected to lose 1–2% of the FEV1% predicted per year along with decreased exercise tolerance, malabsorption and weight loss. Research into the development of CFTR modulators has dominated PwCF research, and in 2020, NHSE agreed to a deal which made approximately 80% of PwCF eligible for Triple Modulator Therapy (Kaftrio®). The combination of the elexacaftor/tezacaftor/ivacaftor has shown improvements in the structure and function of the NaCl channels. PwCF reported improvements in energy, strength, breathing, sputum production, diabetes control, BMI and lung function (Edgeworth 2017).Wewere therefore interested to observe the effects of Kaftrio® on our patient group at Nottingham University Hospitals Trust (NUH) Objectives: To observe the physiological effects of Kaftrio® in regard to weight, FEV1, grip strength and 6MWT in adult PwCF at NUH Methods: A retrospective observation of 99 eligible patients who attended the NUH (Sept 20-March 21) was undertaken on initiation of on-license Kaftrio®. Patients were seen on initiation and after 3 months. Data was collected as part of their routine MDTAnnual Assessment process (Weight, FEV1, grip strength and 6MWT). Results: Of the 99 patients (aged 18–50) we observed, weight +4.14% (n = 55), FEV1 +22.5% (n = 42), grip strength − 0.48% (n = 41) and 6MWT +1.14% (n = 36). Limitations: Patient dissent to assessments, interrater reliability, variability in spirometry device, COVID-19 pandemic restrictions and side effects resulting in termination of Kaftrio®. Conclusions: Overall improvement occurred in all areas except for grip strength. It is encouraging to see a general improvement within our cohort of patients which reflects changes aligned with international research. However, the reduction in chest symptom burden does pose the question of the nature of thephysiotherapy involvement in cystic fibrosis for thefuture.