NCT06599892

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

Rethinking CF Organization of Care in the Era of Highly Effective Modulator: a Nationwide Research Program HORIZON.

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 5,000 (estimated)
• Sponsor: Hospices Civils de Lyon · Academic / Other
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
OTHER	Intervention mapping	Intervention mapping is a 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.

Timeline

• Start date: 2023-09-01
• Primary completion: 2026-12-31
• Completion: 2027-06-30
• First posted: 2024-09-19
• Last updated: 2024-09-19

Locations

1 site across 1 country: France