Original Article|Articles in Press

Lived experiences of people with cystic fibrosis that were not eligible for elexacaftor-tezacaftor-ivacaftor (ETI): A qualitative study

Published:December 20, 2022DOI:


      • Being deemed not eligible for ETI shapes PwCF experiences and future expectations.
      • Disappointment was reported by all participants and appears to play a pivotal role in their experience.
      • Participants disclose mixed emotions: concerns for their health and happiness to see other people thrive.
      • Psychosocial support for PwCF not eligible for ETI should be a central aspect of care.



      Elexacaftor-tezacaftor-ivacaftor (ETI) represents a significant step forward in cystic fibrosis (CF) care and could change the course of CF lung disease and quality of life for many people with CF (PwCF). However, several PwCF cannot benefit from these modulators because their rare mutations are not eligible for treatment. This study aimed to investigate the lived experiences of PwCF who are not eligible for ETI.


      Data were collected through semi-structured interviews with 13 individuals with CF that were not eligible for ETI. Thematic analysis was used to identify the key themes of their experiences.


      Two main themes and six subthemes were identified. The first main theme (being deemed ineligible for ETI) had four subthemes (disappointment, information, happiness, and concerns). The second main theme (coping with a life without ETI) had two subthemes (lack of hope and continued hope).


      PwCF who are not eligible for ETI experience intense disappointment and conflicting emotions that can influence their decision-making linked to diminishing/renewal hope. Integrated care, including mental health monitoring programs, should be provided to these patients to aid them in overcoming their disappointment and to improve their coping.


      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'


      Subscribe to Journal of Cystic Fibrosis
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect


        • Kelly J.
        Environmental scan of cystic fibrosis research worldwide.
        J Cyst Fibros. 2017; 16: 367-370
        • Farrell P.
        The prevalence of cystic fibrosis in the European Union.
        J Cyst Fibros. 2008; 7: 450-453
        • Ratjen F
        • Bell S
        • Rowe S
        • Goss C
        • Quittner A
        • Bush A.
        Cystic fibrosis.
        Nat Rev Dis Primers. 2015; 1
        • Sawicki G
        • Goss C.
        Tackling the increasing complexity of CF care.
        Pediatr Pulmonol. 2015; 50: S74-S79
        • Bierlaagh M
        • Muilwijk D
        • Beekman J
        • van der Ent C.
        A new era for people with cystic fibrosis.
        Eur J Pediatr. 2021; 180: 2731-2739
        • Lopes-Pacheco M
        • Pedemonte N
        • Veit G.
        Discovery of CFTR modulators for the treatment of cystic fibrosis.
        Expert Opin Drug Discovery. 2021; : 1-17
        • Migliorisi G
        • Collura M
        • Ficili F
        • Pensabene T
        • Bongiorno D
        • Collura A
        • et al.
        Elexacaftor-tezacaftor-ivacaftor as a final frontier in the treatment of cystic fibrosis: definition of the clinical and microbiological implications in a case-control study.
        Pharmaceuticals. 2022; 15: 606
        • Wang Y
        • Ma B
        • Li W
        • Li P.
        Efficacy and safety of triple combination cystic fibrosis transmembrane conductance regulator modulators in patients with cystic fibrosis: a meta-analysis of randomized controlled trials.
        Front Pharmacol. 2022; 13
        • Campagna G
        • Amato A
        • Majo F
        • Ferrari G
        • Quattrucci S
        • Padoan R
        • Floridia G
        • Salvatore D
        • Carnovale V
        • Puppo Fornaro G
        • Taruscio D
        • Salvatore M
        Gruppo di lavoro RIFC. Registro italiano Fibrosi Cistica (RIFC). Rapporto 2017-2018 [Italian Cystic Fibrosis Registry (ICFR). Report 2017-2018]. 45. Epidemiol Prev, 2021: 1-37 (May-JunSuppl 1)
        • Page A
        • Goldenberg A
        • Matthews A.
        Lived experiences of individuals with cystic fibrosis on CFTR-modulators.
        BMC Pulmonary Med. 2022; 22
        • Aspinall S
        • Mackintosh K
        • Hill D
        • Cope B
        • McNarry M.
        Evaluating the effect of kaftrio on perspectives of health and wellbeing in individuals with cystic fibrosis.
        Int J Environ Res Public Health. 2022; 19: 6114
        • Keyte R
        • Kauser S
        • Mantzios M
        • Egan H.
        The psychological implications and health risks of cystic fibrosis pre- and post- CFTR modulator therapy.
        Chronic Illness. 2022; 174239532210990
        • Baxter Magolda M.
        Evolution of a constructivist conceptualization of epistemological reflection.
        Educ Psychologist. 2004; 39: 31-42
      1. Weenink E, Bridgman T. Taking subjectivity and reflexivity seriously: Implications of social constructionism for researching volunteer motivation. 2020.

        • O'Brien B
        • Harris I
        • Beckman T
        • Reed D
        • Cook D
        Standards for reporting qualitative research.
        Acad Med. 2014; 89: 1245-1251
        • Braun V
        • Clarke V.
        Using thematic analysis in psychology.
        Qual Res Psychol. 2006; 3: 77-101
        • Berger R.
        Now I see it, now I don't: researcher's position and reflexivity in qualitative research.
        Qual Res. 2013; 15: 219-234
        • Zeelenberg M
        • van Dijk W
        • Manstead A
        • vanr de Pligt J.
        On bad decisions and disconfirmed expectancies: the psychology of regret and disappointment.
        Cognit Emotion. 2000; 14: 521-541
        • Tzieropoulos H
        • de Peralta RG
        • Bossaerts P
        • Gonzalez Andino SL.
        The impact of disappointment in decision making: inter-individual differences and electrical neuroimaging.
        Front Hum Neurosci. 2011; 4 (Published 2011 Jan 6): 235
        • Chauveau T
        • Nalpas N
        A theory of disappointment.
        ESC-Toulouse Working Papers, 2009
        • Kramer-Golinkoff E
        • Camacho A
        • Kramer L
        • Taylor-Cousar JL.
        A survey: understanding the health and perspectives of people with CF not benefiting from CFTR modulators.
        Pediatr Pulmonol. 2022; 57: 1253-1261