Bronchiectasis is a neglected chronic respiratory condition. In children optimal appropriate management can halt the disease process, and in some cases reverse the radiological abnormality. This requires many facets, including parental/carer bronchiectasis-specific knowledge, for which there is currently no such published data. Further, the importance of patient voices in guiding clinical research is becoming increasingly appreciated. To address these issues, we aimed to describe the voices of parents of children with bronchiectasis relating to (a) burden of illness and quality of life (QoL), (b) their major worries/concerns and (c) understanding/management of exacerbations. The parents of 152 children with bronchiectasis (median age = 5.8 years, range 3.5–8.4) recruited from the Queensland Children’s Hospital (Australia) completed questionnaires, including a parent-proxy cough-specific QoL. We found that parents of children with bronchiectasis had impaired QoL (median 4.38, range 3.13–5.63) and a high disease burden with median 7.0 (range 4.0–10.0) doctor visits in 12-months. Parental knowledge varied with only 41% understanding appropriate management of an exacerbation. The highest worry/concern expressed were long-term effects (n = 42, 29.8%) and perceived declining health (n = 36, 25.5%). Our study has highlighted the need for improved education, high parental burden and areas of concern/worry which may inform development of a bronchiectasis-specific paediatric QoL tool.

Background: The parent-proxy paediatric chronic cough quality of life questionnaire (PC-QoL) is a commonly used measure of spillover quality of life in parents of children with chronic cough. To date, spillover health utility in these parents is not routinely estimated largely due to the lack of a suitable instrument. Their perspective is not included in economic evaluations of interventions for their children. We explored developing a health state classification system based on the PC-QoL for measuring health utility spill over in this population. Methods: This study included PC-QoL 8-item responses of 653 parents participating in a prospective cohort study about paediatric chronic cough. Exploratory factor analysis (EFA) and Rasch analysis were used to examine dimensionality and select potential items and level structure. Results: EFA indicated that the PC-QoL had one underlying domain. Rasch analysis indicated threshold disordering in all items which improved when items were collapsed from seven to four levels. Two demonstrated differential item functioning (DIF) by diagnosis or ethnicity and were excluded from the final scale. This scale satisfied Rasch assumptions of local independence and unidimensionality and demonstrated acceptable fit to the Rasch model. It was presented to and modified by an expert panel and a consumer panel. The resulting classification system had six items, each with four levels. Discussion: The PC-QoL can conform to a Rasch model with minor modifications. It may be a good basis for the classification system of a child cough-specific PBM. A valuation study is required to estimate preference weights for each item and to estimate health utility in parents of children with chronic cough.

Background: Quality of life (QoL), the highest prioritised outcome by children with bronchiectasis and their parents, is a patient-reported outcome measure increasingly considered essential when evaluating health and interventions. Despite this, there are no validated instruments that specifically measure QoL related to bronchiectasis in children. We aimed to develop and validate a new bronchiectasis child-specific parent-proxy QoL instrument (BC-QoL). Methods: We developed a draft 44-item BC-QoL and subsequently conducted a prospective cohort study where 142 parents completed draft BC-QoL and six other measures: two validated cough scores, parent-proxy children's acute cough-specific QoL, depression, anxiety and stress 21-item scale, RAND-36 and paediatric QoL (PedsQL ^TM 4.0) to assess the convergent and discriminant validity of the BC-QoL. The questionnaires were completed over three weeks at different phases of their child's illness (stable state, exacerbation and/or recovery). Responses were analysed using psychometric and clinical impact techniques to reduce items and determine the instrument's reliability and validity. Minimally important difference (MID) was also calculated. Results: The final 23-item BC-QoL instrument with its three domains (emotional, physical, social well-being) demonstrated high split half reliability (0.95), Cronbach's alpha (0.97), repeatability (intraclass coefficient=0.74, 95%CI 0.62–0.82), validity and responsiveness. BC-QoL's domains significantly correlated with that of PedsQL ^TM 4.0 (Spearman correlations: emotional=0.43, social=0.41, physical=0.47). BC-QoL's MID ranged from 0.74 to 1.32. Conclusion: BC-QoL, the first bronchiectasis child-specific QoL instrument, has evidence demonstrating its validity and reliability, and can be used to evaluate the impact and effectiveness of interventions and better understand the disease burden in children with bronchiectasis.

Background: Quality of life (QoL), the highest prioritised outcome by children with bronchiectasis and their parents, is a patient-reported outcome measure increasingly considered essential when evaluating health and interventions. Despite this, there are no validated instruments that specifically measure QoL related to bronchiectasis in children. We aimed to develop and validate a new bronchiectasis child-specific parent-proxy QoL instrument (BC-QoL). Methods: We developed a draft 44-item BC-QoL and subsequently conducted a prospective cohort study where 142 parents completed draft BC-QoL and six other measures: two validated cough scores, parent-proxy children’s acute cough-specific QoL, depression, anxiety and stress 21-item scale, RAND-36 and paediatric QoL (PedsQL ^TM 4.0) to assess the convergent and discriminant validity of the BC-QoL. The questionnaires were completed over three weeks at different phases of their child’s illness (stable state, exacerbation and/or recovery). Responses were analysed using psychometric and clinical impact techniques to reduce items and determine the instrument’s reliability and validity. Minimally important difference (MID) was also calculated. Results: The final 23-item BC-QoL instrument with its three domains (emotional, physical, social well-being) demonstrated high split half reliability (0.95), Cronbach’s alpha (0.97), repeatability (intraclass coefficient=0.74, 95%CI 0.62-0.82), validity and responsiveness. BC-QoL’s domains significantly correlated with that of PedsQL ^TM 4.0 (Spearman correlations: emotional=0.43, social=0.41, physical=0.47). BC-QoL’s MID ranged from 0.74 to 1.32. Conclusion: BC-QoL, the first bronchiectasis child-specific QoL instrument, has evidence demonstrating its validity and reliability, and can be used to evaluate the impact and effectiveness of interventions and better understand the disease burden in children with bronchiectasis.

"Background: The Australasian Teletrial Model decentralises clinical trial delivery by enabling patient participation from satellite sites, supported by telehealth and central oversight. While improving access, transporting Investigational Medicinal Products (IMPs) across dispersed sites presents challenges, with no standardised technical guidance currently available. Objective: To map existing evidence on IMP transport in decentralised trials, identify operational and regulatory gaps, and inform technical guidance for Western Australia and broader national use. Study Design and Setting: A systematic search was conducted across PubMed, MEDLINE, Embase, Web of Science, CINAHL, and Scopus for literature published between January 2000 and March 2025. Grey literature, including regulatory guidelines, policy documents, and operational reports, was also reviewed. Records were included if they addressed IMP transport in decentralised or multi-site clinical trials. Results: Of 3,950 records screened, 85 documents were included (27 from academic sources and 58 from grey and other sources). Most documents originated from the United States, international bodies, and Australia. Common themes included temperature-control methods, such as cool packs and dry ice, as well as digital innovations like real-time monitoring. Only 32% addressed rural or resource-limited settings. Key gaps included inconsistent governance around labelling, repackaging, and chain-of-custody protocols. Conclusion: This scoping review provides a solid evidence base for technical guidance on IMP transport in decentralised trials. It highlights a strong regulatory focus and product integrity, while also revealing operational gaps, particularly in low-resource settings. Harmonised, scalable logistics frameworks are needed to improve trial feasibility, safety, and equity across diverse research contexts."