A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators.

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

SUBMITTER: Kramer-Golinkoff E 

PROVIDER: S-EPMC9314897 | biostudies-literature | 2022 May

REPOSITORIES: biostudies-literature