Overview
PERCE-MUCO is a prospective, multicentre, mixed-methods observational study investigating the perceptions, expectations and treatment-change intentions of adults with cystic fibrosis who are currently treated with elexacaftor/tezacaftor/ivacaftor (ETI) or whose treatment has been medically discontinued. The study focuses on patients' stated willingness to switch from ETI to vanzacaftor/tezacaftor/deutivacaftor (VTD), and on the factors associated with this intention, including treatment satisfaction, knowledge and expectations regarding VTD, quality of life, treatment adherence, reported adverse effects, access-related concerns and desired support from an advanced practice nurse. The quantitative component uses a study-specific questionnaire and routine-care CFQ-R 14+ and Morisky-8 data. A qualitative component consists of semi-structured interviews at the Roscoff cystic fibrosis centre.
Description
Cystic fibrosis is a rare genetic disease for which CFTR modulator therapy has substantially changed treatment and patients' daily lives. Although elexacaftor/tezacaftor/ivacaftor (ETI) has improved respiratory outcomes and reduced exacerbations for many eligible patients, some patients experience intolerance or a suboptimal response. Vanzacaftor/tezacaftor/deutivacaftor (VTD) is a newer CFTR modulator combination, and patients' willingness to change from a familiar treatment to a new option cannot be assumed.
The primary objective is to assess the intention of adults with cystic fibrosis treated with ETI to change to VTD.
The primary outcome is the response to a single-choice question asking what the patient would do if their physician offered CTD: accept immediately, need time to think, or refuse.
Secondary objectives are to identify determinants of acceptance; assess the relationship between quality of life and intention to change; assess the relationship of adherence and reported tolerability with intention to change; describe the role and desired support of an advanced practice nurse during a possible transition; and explore psychological and logistical barriers through qualitative interviews.
The quantitative component is conducted across participating French cystic fibrosis centres. Participants complete the PERCE-MUCO questionnaire during a routine follow-up visit or planned hospital stay. The most recent routine-care CFQ-R 14+ and Morisky-8 questionnaires available near inclusion, together with selected clinical and treatment characteristics and adverse effects documented in the medical record, are collected in pseudonymised form.
The qualitative component is conducted at the Roscoff centre and uses individual semi-structured face-to-face interviews lasting approximately 30-45 minutes. Interviews are audio-recorded with the participant's agreement, transcribed verbatim and pseudonymised before thematic analysis.
The quantitative primary outcome is analysed descriptively; secondary analyses include descriptive statistics and comparisons using ANOVA or Kruskal-Wallis, and chi-square or Fisher's exact tests as appropriate.
Qualitative data are analysed thematically using a combination of deductive and inductive coding. The protocol estimates a quantitative sample of 343 participants and approximately 8-12 qualitative interviews, with interview recruitment continuing until data saturation.
Eligibility
Inclusion Criteria:
- Diagnosis of cystic fibrosis
- Currently treated with ETI for at least 3 months, OR previously treated with ETI but treatment discontinued for medical reasons
Exclusion Criteria:
- Patient objects to the use of their data for this project\\n•
- Currently receiving the new CFTR modulator VTD
