Objective: Children <5 years are at higher risk of hospitalisation for severe influenza, yet uptake of the free, recommended vaccine in Australia remains low at 25.7%. Improving uptake requires tracking parental barriers over time to inform tailored approaches. The National Vaccination Insights project measured barriers in 2025 and compared with 2024 data. Methods: A national online survey of parents of children <5 years (n=2,012) was conducted in September-October 2025. Parents reported their child’s 2025 influenza vaccination status and agreement with 15 vaccination barriers. Primary analysis assessed barrier prevalence and prevalence differences between parents who did and did not vaccinate their child in 2025. Results: Compared to 2024, more parents reported almost all acceptance barriers. In 2025, not choosing to prioritise their child's influenza vaccination was the barrier with the largest difference between vaccinating and non-vaccinating parents (PD 47.9 pp, 95% CI 43.9, 51.9). Conclusions: These findings indicate growing motivational challenges to influenza vaccine uptake among parents of young children. Implications for Public Health: Strategies should strengthen parents’ understanding of influenza severity and confidence in vaccine effectiveness, while improving access. Tracking barriers over time will enable monitoring negative global factors, which can influence parental decision-making.

Background and objective: Vietnam had increasing rates of zero-dose children and sub-optimal COVID-19 vaccine booster coverage in 2022. With the Vietnamese Ministry of Health, we co-designed, implemented and evaluated a vaccine education and communication programme for health workers and community leaders to improve trust, knowledge, communication skills and intention to vaccinate for routine childhood and COVID-19 vaccines. Method: The Train-the-Trainer Vaccine Champions program was piloted in 2022 and deployed in 2023 in three low vaccine coverage provinces. Master trainers trained provincial trainers (healthcare workers and communication experts), who trained community leaders and other health workers as champions to deliver community vaccine education sessions. Using mixed methods and the RE-AIM framework, we evaluated reach, effectiveness, adoption, implementation and maintenance. Primary effectiveness outcomes included trust in vaccines and the healthcare system, vaccine knowledge, communication self-efficacy, satisfaction and community respondents’ intention to vaccinate. Secondary outcomes included changes in provincial-level coverage pre- and 6-months post-intervention. Results: The programme trained 56 provincial trainers (47% female), 286 vaccine champions (46% female) and reached 4027 community attendees. Communication self-efficacy and trust in vaccines and the health system increased significantly among provincial trainers and vaccine champions post-training, but change in knowledge was minimal. Participants reported high satisfaction with sessions and materials, and 86% of community respondents reported increased intention to vaccinate. Champions continue to run education sessions and promote other health programmes with the Ministry of Health. Conclusions: In Vietnam, a multi-level Vaccine Champions program that trained healthcare workers and community leaders to promote routine childhood and COVID-19 vaccination increased participant trust in vaccines and the health system, self-efficacy and community intention to vaccinate. The programme has potential to reduce healthcare workforce burden and has ongoing government support. Hybrid implementation-effectiveness trials are needed to determine impact on vaccine coverage and cost-effectiveness, alongside implementation outcomes, to guide scalability.

BackgroundVaccine misinformation can undermine vaccine confidence, institutional trust, and uptake. Arabic speakers in Australia are disproportionately susceptible to vaccine misinformation and have experienced adverse health outcomes. This randomized controlled trial tested the effectiveness of Cranky Uncle – Vaccine (Arabic), a gamified psychological inoculation intervention, in improving vaccine attitudes, misinformation discernment, and institutional trust among Arabic-speaking Australians.MethodsParticipants (N = 198) were randomized 1:1 to either the intervention (game) or control (educational climate change video). Outcomes were assessed immediately and 3 weeks post-activity using a combination of validated and custom instruments: (1) the Vaccine Trust Indicator (VTI); (2) a misinformation discernment scale developed by the research team, and (3) items from the Institutional Trust subscale. Analyses were conducted using multiple imputation and adjusted linear regression models. The trial protocol was registered in the Australian and New Zealand Clinical Trials Registry (registration number: ACTRN12624000934549p).ResultsNo statistically significant differences were found between the intervention and control groups at either timepoint across primary or secondary outcomes, and no subgroup effects were observed by age or education. Post-hoc item analyses found that items assessing trust in scientists and trust in pharmaceutical companies demonstrated the greatest difference between groups at the immediate post-activity timepoint.DiscussionThis trial is the first RCT of the Cranky Uncle – Vaccine game. Findings highlight methodological and measurement challenges in misinformation research. Developing and validating standardized measures that are appropriately sensitive to detect meaningful changes should be an urgent priority for the field. Future evaluation of the Cranky Uncle intervention for different populations could consider testing delivery in structured educational group settings.

Background/objectives: Influenza vaccines are recommended and free in Australia for children aged <5 years, but uptake remains low at 25.8% compared to the targets of 40% and 50%. National data on barriers hindering paediatric influenza vaccination can inform strategies to improve uptake. The aim of this study was to measure barriers to influenza vaccination in Australian children aged <5 years. Methods: A national, cross-sectional survey of parents of children aged <5 years was conducted in March/April 2024. Parents were recruited using an online panel and asked about their intention to get an influenza vaccine for their youngest child in the upcoming influenza season. An adapted version of the validated Vaccine Barriers Assessment Tool measured 14 influenza vaccination barriers. Analysis assessed the prevalence of barriers and differences between parents intending to and those unsure or not intending to vaccinate by calculating the prevalence difference and 95% confidence interval. Results: A total of 2000 parents were recruited nationally. The most common barrier was parents feeling distressed when thinking about vaccinating their child against influenza (66.1% of intending parents, 65.6% of unsure/not intending parents). The barrier with the largest difference between intending and not intending/unsure parents was not prioritising their child’s influenza vaccination (47.2% vs. 6.1%, PD = 41.1 ppts, 95% CI: 35.9%, 46.3%). Other barriers with large differences were parents not feeling guilty if their unvaccinated child got influenza (41.5% vs. 7.5%, PD = 34.0 ppts, 95% CI: 28.8%, 39.1%) and parents not believing that influenza vaccines are effective (31.3% vs. 3.0%, PD = 28.2 ppts, 95% CI: 23.6%, 32.9%). Conclusions: Parents should be encouraged and supported to prioritise influenza vaccination alongside routine childhood vaccines in campaigns that emphasise disease risk and the importance, safety and effectiveness of influenza vaccination, and by optimising access to influenza vaccination. We recommend conducting similar surveys regularly to monitor trends in parental barriers to childhood influenza vaccination.

Background:There is limited evidence describing the changing natural history of DMD in Australia. Methods:This retrospective cohort study collated information on clinical management and disease milestones from medical records of males with DMD attending a paediatric hospital between 1973 and 2019 and linked this to information from two adult tertiary hospitals. Data were stratified by decade of birth and Kaplan Meier analyses were conducted to describe median time to key disease milestones. Findings:The cohort included 356 individuals with DMD with year of birth ranging from 1958 to 2014 and median (interquartile range, IQR) follow up time from diagnosis of 10.5 (4.1, 15.7) years. Use of corticosteroids, angiotensin-converting enzyme inhibitors (ACE-I), echocardiography and respiratory support increased over time. Mean age of diagnosis decreased from 6.4 years in those born before 1970 to 3.4 years in those born 2010-2019. Median (IQR) survival increased over time from 18.2 (15.2, 20.4) years in those born before 1970 to 24.0 (20.3, 27.5) years in those born between 1990 and 1999. Increased life expectancy was observed in individuals using corticosteroids, ACE-I and respiratory support. Interpretation:Survival in individuals with DMD has increased over the last five decades, likely due to changes in clinical management. Given the increased population surviving to adulthood, there is a need to enhance clinical services and surveillance to support neuromuscular disease in Australia, especially in transitional care and adult populations. Funding:Independent Research Grant, Pfizer Australia.

INTRODUCTION:Like many countries globally, childhood immunisation coverage is below government targets in the Philippines and has worsened during the COVID-19 pandemic, leading to outbreaks of vaccine-preventable diseases. This study aimed to identify the social and behavioural drivers of routine childhood immunisation and preferred caregiver information sources to improve uptake in the Philippines. METHODS:A quantitative, cross-sectional study, as part of a larger mixed-methods study, was undertaken in three regions in the Philippines with low immunisation coverage in 2023. Data on Behavioural and Social Drivers (BeSD) of immunisation, preferred information sources and vaccination status of children under 5 years were collected, analysed and reported according to the WHO BeSD framework. RESULTS:The study included 653 caregivers and 950 children, with 65% of children fully immunised, 31.8% partially immunised and 2.5% being zero-dose or having received no vaccines. Key reasons for complete vaccination included the protection vaccines offer, healthcare worker recommendations and vaccines being free of charge. For partial vaccination, top reasons were intercurrent illness, inaccessible vaccination sites and parental belief that the child was too young to receive vaccines. The most common reason for non-vaccination was also intercurrent illness. The predictors of complete vaccination were older age of caregivers (46-59 years) (prevalence ratio (PR)=1.29; 95% CI 1.12 to 1.48) and low concerns about vaccine safety (PR=1.13; 95% CI 1.03 to 1.25). Other Christian denominations, compared with Catholics, were less likely to have fully immunised children (PR 0.82; 95% CI 0.69 to 0.97). Preferred vaccine information sources included the Department of Health, healthcare workers and mass media. CONCLUSIONS:We found that only 65% of children were fully immunised in 2023, with both access and acceptance factors impacting uptake of routine childhood vaccines. Improving service accessibility and education of healthcare workers on vaccine safety and effectiveness and more targeted messaging from health authorities and mass media about the benefits of vaccination could increase uptake in the Philippines, with learnings from this study applicable globally.

BACKGROUND:Cystic fibrosis (CF) lung disease begins early in life and progresses throughout childhood into adolescence. Children completing the Australasian CF Bronchoalveolar Lavage (ACFBAL) trial were followed longitudinally (CF-FAB study) to determine progression of CF lung disease during adolescence using visual and automatic methods and to correlate CT-derived metrics with spirometry outcomes. METHODS:CTs from start and end visits of CF-FAB (mean 24 [SD 12] months apart) were analysed using visual PRAGMA-CF scoring and automatic bronchus-artery (BA) analysis. PRAGMA-CF assessed %Disease by summing %Bronchiectasis, %Mucus plugging, % Airway wall thickening on inspiratory scans and %Trapped air on expiratory scans. The BA-analysis segments the bronchial tree, identifies segmental bronchi (G0) and distal generations (G1, G2, G3…), measures diameters of bronchial outer wall (Bout), inner wall (Bin), wall thickness (Bwt), and artery (A), and computes BA-ratios (Bout/A, Bin/A, Bwt/A, Bwa/Boa[=bronchial wall area/bronchial outer area]) to evaluate bronchial dilatation and wall thickening. RESULTS:120 children (median age 13 years, IQR 11.4-14) contributed 115 start and 105 end scans. Eleven children were treated with CFTR modulators prior to the start of the study and four received the treatment during the study. Progression was found in PRAGMA-CF %Bronchiectasis (p=0.02) and %Mucus plugging (p=0.02), and Bout/A (p=0.01), Bwt/A (p<0.001), and Bwa/Boa (p<0.001). Spirometry outcomes showed no significant decline. BA-metrics correlated more strongly with spirometry outcomes than PRAGMA-CF scores. CONCLUSION:Heterogeneous progression of structural lung disease in children with CF during adolescence was detected using visual PRAGMA-CF scores and automatic BA-analysis. Spirometry outcomes showed no significant decline.

OBJECTIVES:Data on barriers to childhood vaccine uptake are needed to understand and address declining coverage. This study aimed to measure access and acceptance barriers to routine childhood vaccination faced by parents in Australia. STUDY DESIGN:National cross-sectional online survey. METHODS:We recruited Australian parents/carers of children aged <5 years using an online panel from March-April 2024. We measured 15 access and acceptance barriers to routine childhood vaccine uptake using the validated Vaccine Barriers Assessment Tool. Parents reported their child's vaccination status (up-to-date, partially vaccinated, or unvaccinated) and demographics. We calculated prevalence of vaccination barriers and associations between barriers and parent location, financial stress, number of children, and child vaccination status. Data were weighted using the 2021 estimated resident parent population. RESULTS:Of 2000 parents surveyed, 94.0 % reported up-to-date child vaccination, 4.5 % partial, and 1.5 % unvaccinated. In all three vaccination status groups the most common barrier was feeling distressed when thinking about vaccination (60.2 % in total). Compared to parents of up-to-date children, the partially vaccinated group reported more access barriers like difficulty getting an appointment (24.8 % vs 8.5 %, PD 16.3 %, 95 % CI: 6.3-26.3) and affordability (20.5 % vs 10.4 %, PD 10.0 %, 95 % CI: 0.7-19.3). Acceptance barriers like not believing vaccines are safe were more associated with non-vaccination. CONCLUSIONS:Along with strategies to improve vaccine acceptance, interventions addressing access issues like reducing appointment costs should be prioritised for partially vaccinated children. Annual assessment of social and behavioural barriers amenable to intervention will enable comparison over time to inform policy and practice.

BACKGROUND:Long-term effects of early, recurrent human exposure to general anaesthesia remain unknown. The Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial provided an opportunity to examine this issue in children randomly assigned in infancy to either repeated bronchoalveolar-lavage (BAL)-directed therapy with general anaesthesia or standard care with no planned lavages up to 5 years of age when all children received BAL-directed therapy under general anaesthesia. METHODS:This multicentre, randomised, open-label phase 4 trial (CF-GAIN) used the original ACFBAL trial randomisation at 3·6 months (SD 1·6) to BAL-directed therapy or standard-care groups to assess the impact of general anaesthesia exposures over early childhood. Children who completed the ACFBAL trial, with a mean age of 5·1 (SD 0·18) years, received standardised neurobehavioural and health-related-quality-of-life assessment and brain MRI scans between Oct 8, 2013, and June 30, 2017, at a mean age of 12·8 (SD 1·7) years at three hospitals in Australia and one hospital in New Zealand. The primary outcome was a composite score of performance on a standardised, computer-based assessment of child attention, processing speed, and response inhibition skills (Conners Continuous Performance test, second edition). Secondary outcomes included intellectual function, other neurobehavioural measures, and brain imaging as an exploratory outcome. The trial was registered with the Australian New Zealand Clinical Trials Registry (ACTRN 12613000057785) and is completed. FINDINGS:At 2 years, the BAL-directed therapy group (n=52) and standard-care group (n=45) had a median of 2·0 (IQR 1·0-3·0) and 0·0 (0·0-0·0) exposures, respectively. At completion of the ACFBAL trial, the BAL-directed therapy group had a median of 6·0 (4·0-9·5) exposures and the standard-care group 2·0 (1·0-4·0) exposures. At CF-GAIN completion, the BAL-directed therapy group had a median of 10·0 (IQR 6·5-14·5) exposures and the standard-care group 4·0 (3·0-7·0) exposures. Cumulative general anaesthesia exposure time was not prospectively collected but, for those with complete cumulative exposure time data to the end of the ACFBAL trial, the median cumulative exposure time for the BAL-directed therapy group (n=29) was 180 (IQR 140-285) min and for the standard-care group (n=32) was 48 (30-122) min. The mean Conners Continuous Performance test, second edition composite score was 51 (SD 8·1) in BAL-directed therapy group and 53 (8·8) in the standard-care group; difference -1·7 (95% CI -5·2 to 1·7; p=0·32) with similar performance on other neurobehavioural measures, including measures of executive function, intellectual quotient scores, and brain imaging. INTERPRETATION:Our findings suggest that repeated general anaesthesia exposure in young children with cystic fibrosis is not related to functional impairment in attention, intellectual quotient, executive function, or brain structure compared with a group with fewer and shorter cumulative anaesthesia durations. FUNDING:National Health and Medical Research Council Australia, Queensland Government Health Service and Clinical Innovation Fellowship, and the Children's Hospital Foundation Queensland.

Background: Home spirometry holds promise as a primary endpoint for clinical trials.Qualitative needs assessments describing the practices and perspectives of people with cystic fibrosis (PwCF), caregivers of PwCF, and research coordinators (RCs) regarding home spirometry can inform strategies for incorporating home spirometry into clinical trials.Methods: We conducted a series of focus groups that engaged PwCF, caregivers, or RCs (separately), led by an experienced facilitator and conducted via videoconference.PwCF aged 14 and older and caregivers with experience performing home spirometry were recruited through the Cystic Fibrosis Foundation (CFF) Community Voice.RCs with experience coaching home spirometry were recruited through the CFF Therapeutics Development Network from sites participating in the PROMISE study.Participants provided informed consent and completed an online survey before the focus group to describe their demographic characteristics and home spirometry devices.Focus groups elicited current experiences and barriers to and facilitators of home spirometry across six target areas, followed by discussion and prioritization.Target areas for PwCF and caregivers included research incentives, burden of procedures, reminders, remote coaching, training, and spirometry results.Target areas for RCs included participant and RC training, remote coaching, monitoring progress, participant engagement, and institution-specific issues.Qualitative analyses followed the deductive approach of template analysis [1].Common themes identified in each session were reviewed in all PwCF or RC sessions to identify areas of consensus, which were used to formulate recommendations for future clinical trials.Results: From September to November 2021, 27 PwCF and six caregivers stratified according to age and role (teens, adults aged 18-39, adults aged ≥40, caregivers) participated in seven sessions, and 24 RCs participated in five sessions.Groups identified barriers to and facilitators of use of home spirometry.Although most PwCF and caregivers found home spirometry convenient, many experienced technical barriers, reported a learning curve to home measurement, and expressed uncertainty about the quality and reliability of measurements.Major barriers that RCs identified involved tailoring participant training to individual needs, scheduling remote coaching, and performing effective coaching remotely.Participants offered age-specific recommendations in key domains: training materials and procedures (for PwCF and RCs), remote coaching, monitoring progress, maintaining engagement, and other areas, including differences in the conduct and interpretation of research versus clinical and home versus office spirometry.Conclusions: Recommendations from this qualitative needs assessment of PwCF, caregivers, and RCs regarding home spirometry in the research setting have been incorporated into the design of OUTREACH, a CFFfunded, multicenter, prospective study of the accuracy, variability, feasibility, and acceptability of home spirometry as a clinical trial endpoint.Our results can also help inform the design of future remote clinical trials.Acknowledgements: This work was supported by

Background: There is no data exclusively on the relationship between health-related quality-of-life (HRQOL) and lung disease severity in early school-aged children with cystic fibrosis (CF). Using data from the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) we assessed the relationships between HRQOL, lung function and structure. Methods: 125 children aged 6.5-10 years enrolled in the AREST CF program were included from CF clinics at Royal Children's Hospital (RCH), Melbourne (n = 66) and Perth Children's Hospital (PCH), Perth (n = 59), Australia. Demographics, HRQOL measured by Cystic Fibrosis Questionnaire-Revised (CFQ-R), spirometry, multiple-breath washout (MBW) and chest CT were collected across two years. Correlation between CFQ-R scores and lung structure/function parameters and agreement between parent-proxy and child-reported HRQOL were evaluated. Results: No correlation was observed between most CFQ-R domain scores and FEV1 z-scores, excepting weak-positive correlation with parent CFQ-R Physical (rho = 0.21, CI 0.02-0.37), and Weight (rho = 0.21, CI 0.03-0.38) domain and child Body domain (rho = 0.26, CI 0.00-0.48). No correlation between most CFQ-R domain scores and LCI values was noted excepting weak-negative correlation with parent Respiratory (rho = -0.23, CI (-)0.41-(-)0.05), Emotional (rho = -0.24, CI (-)0.43-(-)0.04), and Physical (-0.21, CI (-)0.39-(-)0.02) domains. Furthermore, structural lung disease on CT data demonstrated little to no association with CFQ-R parent and child domain scores. Additionally, no agreement between child self-report and parent-proxy CFQ-R scores was observed across the majority of domains and visits. Conclusion: HRQOL correlated poorly with lung function and structure in early school-aged children with CF, hence clinical trials should consider these outcomes independently when determining study end-points. Crown Copyright (c) 2021 Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society. All rights reserved.

Objectives: To describe characteristics and outcomes of children requiring intensive care therapy (ICT) within 12 hours following a medical emergency team (MET) event. Design: Retrospective cohort study. Setting: Quaternary paediatric hospital. Patients: Children experiencing a MET event. Measurements and main results: Between July 2017 and March 2019, 890 MET events occurred in 566 patients over 631 admissions. Admission to intensive care followed 183/890 (21 %) MET events. 76/183 (42%) patients required ICT, defined as positive pressure ventilation or vasoactive support in intensive care, within 12 hours. Older children had a lower risk of requiring ICT than infants aged < 1 year (age 1-5 years [risk difference, -6.4%; 95% CI, -11% to -1.6%; P = 0.01] v age > 5 years [risk difference, -8.0%; 95% CI, -12% to -3.8%; P < 0.001]), while experiencing a critical event increased this risk (risk difference, 16%; 95% CI, 3.3-29%; P = 0.01). The duration of respiratory support and intensive care length of stay was approximately double in patients requiring ICT (ratio of geometric means, 2.0 [95% CI, 1.4-3.0] v 2.1 [95% CI, 1.5-2.8]; P < 0.001) and the intensive care mortality increased (risk difference, 9.6%; 95% CI, 2.4-17%; P = 0.01). Heart rate, oxygen saturation and respiratory rate were the most commonly measured vital signs in the 6 hours before the MET event. Conclusions: Approximately one-fifth of MET events resulted in intensive care admission and nearly half of these required ICT within 12 hours. This group had greater duration of respiratory support, intensive care and hospital length of stay, and higher mortality. Age < 1 year and a critical event increased the risk of ICT.

Background For Australians living with cystic fibrosis (CF), increased longevity means greater consideration needs to be given to long-term endocrine sequelae such as CF-related bone disease. Deficits in bone mass accrual are most likely to occur during childhood and adolescence. Current guidelines in Australia suggest repeat dual-energy X-ray absorptiometry (DXA) scans every 2 years. This study aims to stratify clinical factors that determine future bone health in the Australian CF population and use this to guide a more streamlined approach to bone health screening. Methods This study was a retrospective audit of all patients diagnosed with CF who were treated at the Royal Children's Hospital Melbourne, Australia from 2000 to 2016 (n = 453). Two hundred and two patients had a DXA scan in the study period (191 with height-adjusted data) and 111 patients had more than one scan (108 with height-adjusted data). An investigation into the associations between bone mineral density (BMD) Z score and potential risk factors was conducted using DXA and historical data. Results The main predictor of future BMD was the previous BMD Z score (p < .001). Other factors found to be determinants of BMD included nutritional status, lung function (FEV1), age, history of previous fracture, oral corticosteroid use, and the number of hospital admissions. However, after adjusting for previous BMD, evidence of an association remained only with nutritional status, FEV1, and number of hospital admissions. Conclusion Second yearly scans may be unnecessary in children with an adequate DXA score on the initial scan who remain clinically stable. However, clinical deterioration in those whose BMD was previously normal, may require closer monitoring of bone health. We propose a guideline for the frequency of DXA monitoring in relation to clinical risk factors.

BACKGROUND:The impact of early cystic fibrosis (CF) on health-related quality-of-life (HRQOL) in preschool children is poorly characterised, and data on relationships between HRQOL and health outcomes in young children with CF are limited. We aimed to characterise and compare parent-proxy and child-reported HRQOL and evaluate relationships with clinical outcomes at age 5-years.METHODS:Subjects were participating in the multi-centre Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial investigating BAL-directed versus standard CF therapy. Children aged 5-years and their parents rated HRQOL using the Pediatric Quality of Life Inventory (PedsQL™) and Cystic Fibrosis Questionnaire-Revised (CFQ-R) questionnaires.RESULTS:PedsQL and CFQ-R questionnaires were completed by 141 primary caregivers and 135 and 130 children, respectively. There were no differences in HRQOL between children randomised to BAL-directed versus standard CF therapy. Children with CF rated worse HRQOL than healthy children and there was poor parent-child concordance across HRQOL domains. Nutritional status, CF-CT scan score, forced expiratory volume in 1-second (FEV1), and pulmonary exacerbations correlated with HRQOL at age 5-years. FEV1 z-scores positively correlated with parent-proxy HRQOL in CFQ-R Respiratory (p = 0.018), Physical (<0.001), Emotional (p = 0.007) subscales and PedsQL Total-score (p = 0.021), Physical (p = 0.019) domains. Pulmonary exacerbation rates were inversely associated with parent-proxy CFQ-R Respiratory (p = 0.004), Physical (p = 0.022), PedsQL Total (p = 0.009) and Physical (p = 0.009) scores.CONCLUSION:Parent-reported HRQOL is a meaningful clinical endpoint to evaluate interventions in young children. Parent and child HRQOL reports provide different, complementary information. A preschool version of the CFQ-R is needed to assess relationships between HRQOL and clinical outcomes in young children.

Little is known about early predictors of later cystic fibrosis (CF) structural lung disease. This study examined early predictors of progressive structural lung abnormalities in children who completed the Australasian CF Bronchoalveolar Lavage (ACFBAL) clinical trial at age 5-years and participated in an observational follow-up study (CF-FAB).Eight Australian and New Zealand CF centres participated in CF-FAB and provided follow-up chest computed-tomography (CT) scans for children who had completed the ACFBAL study with baseline scans at age 5-years. CT scans were annotated using PRAGMA-CF scoring. Ordinal regression analysis and linear regression were used to investigate associations between PRAGMA-CF (Perth-Rotterdam Annotated Grid Morphometric Analysis for CF) outcomes at follow-up and variables measured during the ACFBAL study.99 out of 157 ACFBAL children (mean±sd age 13±1.5 years) participated in the CF-FAB study. The probability of bronchiectasis at follow-up increased with airway disease severity on the baseline CT scan. In multiple regression (retaining factors at p<0.05) the extent of bronchiectasis at follow-up was associated with baseline atelectasis (OR 7.2, 95% CI 2.4-22; p≤ 0.001), bronchoalveolar lavage (BAL) log2 interleukin (IL)-8 (OR 1.2, 95% CI 1.05-1.5; p=0.010) and body mass index z-score (OR 0.49, 95% CI 0.24-1.00; p=0.05) at age 5 years. Percentage trapped air at follow-up was associated with BAL log2 IL-8 (coefficient 1.3, 95% CI 0.57-2.1; p<0.001) at age 5 years.The extent of airway disease, atelectasis, airway inflammation and poor nutritional status in early childhood are risk factors for progressive structural lung disease in adolescence.

OBJECTIVES:To determine the association between residence and climate with risk of Pseudomonas aeruginosa (Pa) and other respiratory outcomes.METHODS:We performed regular bronchoalveolar lavage and upper airway cultures in young children with CF to identify Pa infection. Children were classified for residence as regional or metropolitan. Bronchiectasis was detected on periodic chest computed tomography scans. Multilocus sequence typing determined Pa genotype. Lung function was assessed using Multiple Breath Washout.RESULTS:Of infants diagnosed with CF between 2006 and 2017, 129 were included in the study. Seven patients moved between metropolitan and regional Victoria and were excluded from analysis. Of the remaining 122 subjects, seventy-four (61%) children resided in metropolitan areas and over half (54%) were male. There were 83 Pa episodes in the 122 children who lived consistently in a geographical location. The incidence rate was 0.15 episodes per person-years. We found weak evidence of a 15% increase in the rate of Pa episodes with increasing average annual maximum temperature (95%CI (0.98, 1.36); p = .086), while the rate of Pa acquision decreased with average annual 3 pm humidity (IRR = 0.96; 95%CI(0.92, 1.0008); p = .054). The rate of Pa episodes was 2.1 times higher in regional participants (95%CI (1.4, 3.1); p = .001) and risk of second episode was more than five times greater (HR 5.7; 95%CI 1.9, 17); p = .002). No difference between regions in lung clearance index and presence of bronchiectasis was detected.CONCLUSION:Regional residence is associated with risk of acquiring recurrent infection with Pseudomonas aeruginosa in young children with CF.

Background: Both infection and inflammation are critical to the progression of cystic fibrosis (CF) lung disease. Potential anatomical differences in lower airway infection, inflammation and bronchiectasis in young children with CF raise questions regarding the pathogenesis of early structural lung disease. Methods: A longitudinal multi-centre birth cohort study of infants newly diagnosed with CF was conducted. Paired bronchoalveolar lavage (BAL) samples were obtained from the right middle lobe (RML) and lingula bronchi. Chest computed tomography (CT) was performed biennially and analysed using the modified CF-CT scoring system. Results: One hundred and twenty-four children (0.11 - 7.0 years) contributed 527 BAL samples and underwent 388 CT chest scans. Pro-inflammatory microbes were detected in 279 BAL samples (53%), either in both lingula and RML samples (69%), in the lingula alone (24%), or in the RML alone in only 7% of samples. Overall, the prevalence of structural lung disease was greater in the setting of pro-inflammatory microbes. Although infection was less commonly isolated in the right lung, bronchiectasis was more commonly detected in the right lung compared with the left. No anatomical differences in the presence of air trapping were detected. Conclusion: Overall, the detection of pro-inflammatory microbes in the lower airways was associated with increased risk of both air trapping and bronchiectasis. However, the apparent discordance between commonest sites of isolation of pro-inflammatory microbes and the anatomical site of early bronchiectasis warrants further exploration. (C) 2019 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.