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Journal of Cystic Fibrosis ; 20:S51, 2021.
Article in English | EMBASE | ID: covidwho-1368824


Background: Real-world, post-approval studies contribute significantly to the evidence surrounding the impact of new treatments, including CFTR modulators, but can be complex undertakings. Elexacaftor-tezacaftor-ivacaftor (ETI) was approved by EMA sooner than expected in August 2020 during a global pandemic. Method: RECOVER, a multi-centre, post-approval study examining the impact of ETI, and conducted in 8 clinical sites in Ireland and the UK over 2 years, examines important outcomes in children and adults prescribed ETI. The study will be conducted in 2 phases in line with ETI approval: 12+ and 6–11. In addition to routine data collected as part of normal care, key RECOVER endpoints include lung clearance index (LCI), spirometry-controlled CT, treatment adherence, GI symptoms, inflammation, liver disease markers, nasal inflammation and nitric oxide metabolism. Results: To date, 96 participants (56% female) out of a target of 137 in people with CFaged 12 and above, have been recruited (predominantly 12–18yrs to date). Recruitment and sample collection has been impacted by the effect of COVID-19 on CF care and CF centre attendance. Key challenges have included: Sputum collection (risks of induction and non-sputum producing participants) and coordination of study activities with limited clinic attendance. Despite this, key baseline data, prior to commencing treatment, has been successfully collected on the majority of participants to date. For subjects recruited to date, 56% have F508del/F508del and 44% F508del/minimum function mutations, mean age is 16.1 years, mean FEV1 83.6% (23–111%), mean LCI 12.2 (6.9–24.3). Recruitment and data collection is ongoing. Conclusion: Despite the impact of accelerated approval and COVID-19, we have been able to proceed with study initiation, recruitment and sample collection. Data from RECOVER and other international post-approval studies is likely to add significantly to our understanding of the impact of ETI on people with CF.

Journal of Cystic Fibrosis ; 20:S103, 2021.
Article in English | EMBASE | ID: covidwho-1361567


Objectives: Integrating mental health screening and treatment into cystic fibrosis (CF) centres has led to numerous successes: increased awareness, reduced stigma and greater access to mental health care (Quittner et al., 2020). Given that anxiety and depression screening are now part of routine care, the major aim was to identify the most important, future research priorities in mental health. Method: A 22-item online survey was sent out July 2020 to people with CF (pwCF), caregivers, and US providers in the CF community, who were given 3 weeks to respond. Questions focused on: ranking topics by importance and impact, identifying gaps in our understanding of mental health issues, and priorities and needs during COVID-19. Results: Response time was 7–11 minutes, with 693 community respondents (half pwCF, majority ages 26–45 years) and 352 CF clinicians (mainly social workers, center directors). Substantial agreement was found between community respondents and providers on the top 5 priorities:1) anxiety, 2) depression, 3) effects of psychological symptoms on physical health, 4) risk factors for anxiety/depression, and 5) effects on family functioning and parenting. Although ranked highly, differences emerged for substance misuse and disordered eating, with higher rankings endorsed by providers than community respondents. Priorities for improving outcomes were also similar: 1) greater access to mental health providers and 2) improving mental health interventions. Conclusion: A large-scale survey of CF community members and providers revealed clear consensus on the top research priorities. In addition to increased understanding of anxiety and depression (e.g., risk factors, long-term effects), new topics emerged, including substance misuse, disordered eating, and interactions between medications and mental health (e.g., modulators). Acknowledgement: Cystic Fibrosis Foundation