Wellness in the modulator era: An observational study of the impact of CFTR modulator therapy on the well-being of people with cystic fibrosis.

BACKGROUND: People with cystic fibrosis (PwCF) have experienced substantial improvements in health following use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies. However, less is known about how modulator therapies impact well-being. METHODS: We used a cross-sectional observational study to identify relationships between CFTR modulator therapies, health-related quality of life (HRQoL), and well-being. Adult PwCF and caregivers of children with CF completed the Wellness in the Modulator Era (Well-ME) survey between June 22 and July 31, 2022. HRQoL was measured with PROMIS Global 10/Global 7 + 2 Parent Proxy. We used a mixed methods analysis to compare experiences and concerns of PwCF who currently (n = 665), no longer (n = 51), or never (n = 184) took modulator therapy. RESULTS: Adult PwCF taking a modulator (n = 416) reported better PROMIS global physical health than those who no longer (n = 37) or never took a modulator (n = 94) and better PROMIS global mental health than those who never took a modulator. Caregiver-reported HRQoL was similar across children with CF who currently, no longer, or never took a modulator. PwCF taking a modulator reported larger improvements in physical health, quality of life, social well-being, and treatment burden than those who no longer or never took a modulator. Nearly one-quarter (23 %) of PwCF taking modulator therapy reported worsening of mental well-being. CONCLUSIONS: This study expands our knowledge of well-being among PwCF in the CFTR modulator era as reported by patients and parents. Findings lay the groundwork for establishing future research priorities, policy efforts, and communications in areas that improve well-being for PwCF.

The Cystic Fibrosis Learning Network: A mixed methods evaluation of program goals, attributes, and impact.

Introduction: The Cystic Fibrosis (CF) Foundation sponsored the design, pilot testing, and implementation of the CF Learning Network (CFLN) to explore how the Foundation's Care Center Network (CCN) could become a learning health system. Six years after the design, the Foundation commissioned a formative mixed methods evaluation of the CFLN to assess: CFLN participants' understanding of program goals, attributes, and perceptions of current and future impact. Methods: We performed semi-structured interviews with CFLN participants to identify perceived goals, attributes, and impact of the network. Following thematic analyses, we developed and distributed a survey to CFLN members and a matched sample of CCN programs to understand whether the themes were unique to the CFLN. Results: Interviews with 24 CFLN participants were conducted. Interviewees identified the primary CFLN goal as improving outcomes for people living with CF, with secondary goals of providing training in quality improvement (QI), creating a learning community, engaging all stakeholders in improvement, and spreading best practices to the CCN. Project management, use of data, common QI methods, and the learning community were seen as critical to success. Survey responses were collected from 103 CFLN members and 25 CCN members. The data revealed that CFLN respondents were more likely than CCN respondents to connect with other CF programs, routinely use data for QI, and engage patient and family partners in QI. Conclusions: Our study suggests that the CFLN provides value beyond that achieved by the CCN. Key questions remain about whether spread of the CFLN could improve outcomes for more people living with CF.