Abstract

Introduction: The CFTR modulators ivacaftor/tezacaftor/elexacaftor (ETI) significantly improved pulmonary function and quality of life (QoL) in eligible cystic fibrosis (CF) patients.

Aim: The aim of this research is to determine the effectiveness of ETI on the patient's subjective assessment of health.

Methods: CF patients were recruited from adult CF center in Zagreb, Croatia. They answered 22 short questions related to their current state of health and subjective perception of QoL compared to the period before taking ETI.

Results: The research was conducted on a sample of 46 adult CF patients (25 F, 21M) who were on triple therapy. 83% of patients were on ETI for more than 6 months, 4% less than 6 months and 13% were for more than a year. Mean age was 25.7 (18-38).

83% (38/46) CF patients tolerate physical activities and exercise training better than before ETI. 65% (30/46) patients describe their health as excellent, although a quarter of patients (11/46) reduced or stopped their chronic therapy without consulting a doctor.

91% (42/46) of patients bodyweight increased and 65% (30/46) report that they cough less. 85% of patients (39/46) confirmed significant improvements in breathing, 59% (27/46) physical improvements and 72% (33/46) feel like they have excess energy.

Almost the entire group, 93% (43/46) rate the QoL as significantly improved after they started taking ETI. 

Conclusion: Considering all the benefits of ETI, it is no surprise that most of patients rated all aspects of their wellbeing much better. ETI has a significant impact on the CF treatment but a big challenge still exists in patients? awareness of adherence.