CF Organization of Care in the Era of Highly Effective Modulator.

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Other: Intervention mapping

• Registration Number: NCT06599892
• Lead Sponsor: Hospices Civils de Lyon

Brief Summary

Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation.

The hypothesis is that the arrival of CFTRmHEs has considerably altered the way cystic fibrosis is managed, requiring a rethink of how the CRCMs are organised, and a redefinition of the practices and missions of professionals, as well as the patient-professional relationship.

Detailed Description

The organisation of cystic fibrosis care in France, within the Muco-CFTR rare disease network, is based on 47 cystic fibrosis resource and competence centres (CRCMs), which bring together multidisciplinary resources (doctors, physiotherapists, nurses, psychologists, dieticians, social workers) to ensure the continuity and coordination of patients' care pathways, taking into account the physical, psychological, educational/professional and social consequences of the disease. The CRCMs' missions are based on the National Diagnosis and Care Protocol (PNDS 2017). The recent arrival of highly effective CFTR modulators (CFTRmHE) in the arsenal of treatments is a therapeutic revolution for 83% of patients in the French Cystic Fibrosis Registry who are eligible for this treatment. Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation. This treatment improves lung function and body mass index, and reduces pulmonary exacerbations, with good tolerability. These results have led to its approval in 2019 in the United States and 2020 in Europe. Real-life results from 245 patients confirmed a spectacular improvement in respiratory function and nutritional status, to the extent that the indication for lung transplantation was suspended in 47 of the 53 patients concerned. The majority of eligible patients started treatment in 2021. The clinical benefits have prompted some patients to abandon standard treatments (respiratory physiotherapy, bronchial fluidisers) and to reconsider the way they are managed at the CRCM (spacing of visits, teleconsultation). However, in the absence of hindsight, it is not possible at present to make recommendations on reducing the burden of care.

The hypothesis is that the arrival of CFTRmHEs has considerably altered the way cystic fibrosis is managed, requiring a rethink of how the CRCMs are organised, and a redefinition of the practices and missions of professionals, as well as the patient-professional relationship.

Study Timeline

• Study Start: 2023-09-01
• Study First Submitted: 2024-08-12
• Status Verified: 2024-09
• Study First Submitted QC: 2024-09-12
• Last Update Submitted: 2024-09-12
• Study First Posted: 2024-09-19
• Last Update Posted: 2024-09-19
• Primary Completion: 2026-12-31
• Study Completion: 2027-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5000

Inclusion Criteria

• Patients aged 6 and over with cystic fibrosis treated with CFTRmHE for at least 12 months
• Patients aged over 6 years with cystic fibrosis not eligible for CFTRmHE treatment
• Families of patients under 18 years of age
• Professionals working in a CRCM for more than 12 months, physiotherapists and community nurses involved in the management of cystic fibrosis.

Exclusion Criteria * :

• Refusal to participate
• Persons deprived of their liberty by judicial or administrative decision
• Persons under psychiatric care
• Adults under legal protection (guardianship, curatorship)
• Persons not affiliated to a social security scheme or beneficiaries of a similar scheme

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients from the 47 CF French center	Intervention mapping	National registry indicator study = 7513 patients in total, including 5000 eligible for highly effective CFTRm, for the study of pathways at national level, for which clinical and healthcare consumption data are available in the national registry linked to the SNDS
Healthcare professionels	Intervention mapping	Case study: 8 to 10 CRCMs, 7 to 8 professionals and 7 to 8 patients per CRCM, i.e. 56 to 80 professionals, 200 to 400 patients DELPHI: panel of 80 professionals and patients/parents

Primary Outcome Measures

Name	Time	Method
Obtaining a national consensus on a proposal for a new organisation of the cystic fibrosis care pathway, including the CRCMs and the liberal network	The outcome measure will be measured at the twelfth month of the survey.	It will be measured by a mixed approach based on the intervention mapping model combining patient and professional questionnaires and a qualitative survey using individual interviews with patients and focus groups with professionals to identify the obstacles and levers to change at the level of patients, professionals and organisations.; The main outcome measure is to establish a new organization of care for pwCF and HCPs in the era of HEMT by the develop an evaluation plan using a community-based participatory approach to promote external validity.

Trial Locations

Locations (1)

Groupement Hospitalier Est Pediatric CFcenter; 🇫🇷 Lyon, France