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Background: To limit the progression of disease, people with cystic fibrosis (pwCF) perform daily respiratory physiotherapy, which is perceived as the most burdensome routine in managing their condition. The elexacaftor–tezacaftor–ivacaftor (ETI) combination has changed respiratory management. Objective: To investigate how the perceived treatment burden changed in 1 year of treatment with ETI. Design: Prospective observational study. Methods: Ad hoc questionnaires for the pwCF and for the caregivers of pwCF < 18 years were administered before the initiation of ETI therapy and then at 6–12 months. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) and the Sinonasal Outcome Test (SNOT-22) were administered to explore disease-related symptoms and social limitations. The International Physical Activity Questionnaire was used to determine levels of physical activity. Mixed-effect models were fitted to explore whether the time engaged in respiratory physiotherapy changed during 1 year. Results: The study included 47/184 pwCF aged 21.4 (5.7) years, who completed 1 year of ETI therapy. At 6 months, time on aerosol therapy was decreased by 2.5 (95% CI −32.9 to 27.8) min/day, time on airway clearance therapies (ACTs) was decreased by 8.8 (95% CI −25.9 to 8.3) min/day, and time for cleaning and disinfecting respiratory equipment was decreased by 10.6 (95% CI −26.5 to 5.3) min/day. At 1 year, gains in time saved were nearly 15 min/day on average. At 1 year, 5/47 (10.6%) pwCF reported that they had discontinued positive expiratory pressure mask. Conclusion: PwCF on ETI may note less time engaged in their daily respiratory physiotherapy routine. Nonetheless, aerosol therapy, ACTs and maintaining respiratory equipment were still perceived as time-consuming daily activities. Plain language summary: Understanding the challenges of respiratory physiotherapy in individuals with cystic fibrosis using triple therapy: a one-year study. In order to slow down the progression of their disease, people with cystic fibrosis typically do daily respiratory physiotherapy, which they find to be the most challenging part of managing their condition. The elexacaftor-tezacaftor-ivacaftor combination has changed how they manage their respiratory health. We wanted to see how the perceived difficulty of the treatment changed over one year of using elexacaftor-tezacaftor-ivacaftor. We gave questionnaires to people with cystic fibrosis and to their caregivers before they started the triple therapy and again at 6-12 months. We also used two international questionnaires to learn about symptoms and social limitations related to the disease. The International Physical Activity Questionnaire helped us understand their physical activity levels. We used statistical models to see if the time spent on respiratory physiotherapy changed over the year. Our study involved 47 individuals with cystic fibrosis, with an average age of 21 years, who completed one year of elexacaftor-tezacaftor-ivacaftor therapy. After 6 months, time spent on aerosol therapy decreased by 2.5 minutes per day, time on airway clearance therapies decreased by 8.8 minutes per day, and time for cleaning respiratory equipment decreased by 10.6 minutes per day. By the end of the year, they were saving almost 15 minutes per day on average. At one year, 5 out of 47 said they had stopped using the positive expiratory pressure mask. People with cystic fibrosis using elexacaftor-tezacaftor-ivacaftor may find that they spend less time on their daily respiratory physiotherapy routine. However, activities like aerosol therapy, airway clearance therapies, and maintaining respiratory equipment were still seen as time-consuming. [ABSTRACT FROM AUTHOR]

Background: Airway clearance techniques (ACTs) are integral to cystic fibrosis (CF) management. However, there is no consensus as to which outcome measures (OMs) are best for assessing ACT efficacy. Objectives: To summarise OMs that have been assessed for their clinimetric properties (including validity, feasibility, reliability, and reproducibility) within the context of ACT research in CF. Design and Methods: A systematic review was conducted according to Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (PRISMA) standards. Any parallel or cross-over randomised controlled trial (RCT) investigating outcome measures for ACT in the CF population were eligible for inclusion. The search was performed in five medical databases, clinicaltrials.gov, and abstracts from international CF conferences. The authors planned to independently assess study quality and risk of bias using the CO nsensus-based S tandards for the selection of health status M easurement I nstrume N ts (COSMIN) risk of bias checklist with external validity assessment based upon study details (participants and study intervention). Two review authors (GS and MJ) independently screened search results against inclusion criteria, and further data extraction were planned but not required. Results: No completed RCTs from the 187 studies identified met inclusion criteria for the primary or post hoc secondary objective. Two ongoing trials were identified. Discussion and conclusion: This empty systematic review highlights that high-quality RCTs are urgently needed to investigate and validate the clinimetric properties of OMs used to assess ACT efficacy. With the changing demographics of CF combined with the introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, an accurate assessment of the current benefit of ACT or the effect of ACT withdrawal is a high priority for clinical practice and future research; OMs which have been validated for this purpose are essential. Registration: This systematic review was registered on the PROSPERO database (CRD42020206033). [ABSTRACT FROM AUTHOR]

Background: High frequency chest wall oscillation (HFCWO) has long been used for airway clearance for patients with cystic fibrosis. Only limited research has evaluated this therapy in adult patients with non-cystic fibrosis bronchiectasis (NCFB). Methods: Data from 2596 patients from a registry of adult bronchiectasis patients using HFCWO therapy was used to evaluate hospitalization patterns before and after initiation of HFCWO therapy, as well as antibiotic use and self-reported metrics of quality of life. Self-reported outcomes were also reviewed by cross-checking with sampled patient charts and found to be consistent. Results: The number of patients who had at least one respiratory-related hospitalization decreased from 49.1% (192/391) in the year before to 24.0% (94/391) in the year after starting HFCWO therapy (p -value < 0.001). At the same time, the number of patients who required three or more hospitalizations dropped from 14.3% (56/391) to 5.6% (22/391). Patients currently taking oral antibiotics for respiratory conditions decreased from 57.7% upon initiation of therapy to 29.9% within 1 year (p < 0.001). Patients who subjectively rated their "overall respiratory health" as good to excellent increased from 13.6% upon initiation of therapy to 60.5% in 1 year (p < 0.001) and those who rated their "ability to clear your lungs" as good to excellent increased from 13.9% to 76.6% (p < 0.001). Conclusion: NCFB patients showed improved self-reported outcomes associated with the initiation of HFCWO therapy as measured by number of hospitalizations, antibiotic use, and the subjective experience of airway clearance. The improvement was observed early on after initiation of therapy and sustained for at least 1 year. The reviews of this paper are available via the supplemental material section. [ABSTRACT FROM AUTHOR]