A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators. Issue 5 (28th March 2022)

• Record Type: Journal Article
• Authors: Kramer‐Golinkoff, Emily; Camacho, Amanda; Kramer, Liza; Taylor‐Cousar, Jennifer L.
• Is Part Of: Pediatric pulmonology. Volume 57:Issue 5(2022)
• Page Start: 1253
• Page End: 1261
• Publication Date: 2022-03-28
• Subjects: 10% -- CFTR modulator -- cystic fibrosis -- nonsense mutation -- orphan disease; Pediatric respiratory diseases -- Periodicals; Pediatrics -- Periodicals; 618.922
• DOI: 10.1002/ppul.25859
• Languages: English
• ISSNs: 8755-6863

Abstract

Abstract: Background: While the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use has improved daily life and long‐term prognosis of CF for many with approved CFTR mutations, approximately 10% of people with CF (pwCF) have only symptomatic treatments available. Methods: Between June 10 and July 1, 2021, Emily's Entourage distributed a 38‐question anonymous survey targeted at pwCF not benefitting from approved modulators via social media and email to pwCF and CF advocacy groups in and outside the United States regarding health status, impact of CF, unmet needs, and clinical research interest. Results: There were 431 survey respondents representing pwCF on five continents. The majority of pwCF had moderate lung disease (50.3%). Ineligibility based on CFTR mutation (64.1%) was the most frequently reported reason pwCF were not on modulators. PwCF reported the most impacted aspects of life were mental (66.7%) and physical (40.7%) health. Financial concerns and feelings of isolation were commonly reported. Witnessing improvements for peers with access to modulators was both uplifting and disheartening. The majority of pwCF would be interested in participating in future clinical research (77.6%), although some living outside of the United States cited lack of opportunity to participate in clinical trials as a barrier. Conclusions: PwCF who are ineligible, intolerant, or lack access to modulators have a high burden of disease impacting their physical and mental health. Although most are happy for those who are benefiting from modulators, they are eager for the opportunity to experience similar improvements for themselves, and willing to participate in clinical trials of new therapies.