Year: 2026
Type: article
Study design: Observational Study
Abstract: Cystic fibrosis transmembrane conductance regulator modulators (CFTRm) represent a major therapeutic advance for patients with cystic fibrosis (pwCF). Given their oral administration and high efficacy, they may be better suited than other cystic fibrosis treatments to optimize long-term adherence, which is crucial to ensuring effectiveness. To examine adherence patterns to CFTRm and their determinants, we conducted a population-based retrospective cohort study using data from the French CF Registry linked to the French National Health Data System. We included pwCF (>6 years) who initiated at least one CFTRm (ivacaftor or lumacaftor/ivacaftor) between 2012 and 2020 and were treated for at least six months. Implementation was measured using the continuous multiple-interval measure of medication availability version 7 (CMA-7), and longitudinal K-means clustering identified adherence trajectories based on monthly CMA-7 values over one-year follow-up. Multinomial logistic regression assessed associations between adherence patterns and sociodemographic and clinical characteristics. Among 1362 patients, three adherence trajectories were identified: optimal adherence (68%), gradual decline (16%), and initial drop-off followed by return to optimal adherence after 3-5 months (16%). Compared to optimal adherence, gradual decline trajectory was associated with age 15-25 years (reference: >25), while drop-off and rebound trajectory was linked to being younger than 15 and P. aeruginosa bronchial colonization. Socioeconomic disadvantage was also associated with nonoptimal adherence. This study highlights the need for age-tailored interventions to support long-term adherence, revealing a decline among adolescents and young adults, along with a specific pattern among children, which may relate to prescribing practices at treatment initiation.
Source: Respiratory Medicine
Authors: Oriane Talabart, Elia Eid, Marie Viprey, Violaine Fernandez, Manon Belhassen, Antoine Bessou, Isabelle Durieu, Quitterie Reynaud, Cécile Payet, Cinthia RAMES, Françoise Troussier, Bénédicte Richaud Thiriez, Marie-Laure Dalphin, Stéphanie Bui, Julie Macey, Muriel Laurans, Sylvie Montcouquiol, Ralph Epaud, Frédéric Huet, Manuela Scalbert, Laurent Mely, Rébecca Hamidfar, Catherine Llerena, Camille Audousset, Nathalie Wizla-Derambure, Jane Languepin, Philippe Reix, Jean-Christophe Dubus, Martine Reynaud Gaubert, Raphaël Chiron, Aurélie Tatopoulos, Anne Guillaumot, Isabelle Danner Boucher, Tiphaine Bihouee, Sylvie Leroy, Pierre-Régis Burgel, Isabelle Sermet, Véronique Houdouin, Harriet Corvol, Katia Bessaci-Kabouya, Chantal Belleguic, Éric Deneuville, Sophie Ramel, Christophe Marguet, Nathalie Allou, Caroline Perisson, Benjamin Renaud-Picard, Laurence Weiss, Dominique Grenet, Léa Roditis, Marlène Murris Espin, Julie Mankikian, Laure Cosson, Baptiste Arnouat, Asma Gabsi
Language: English
Subfield: Family Practice
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,630
OpenAlex ID: W7204667747
