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Abstract Background The purpose of this study was to identify and describe common views of people with lived experience of mental health challenges – consumers and carers, families and supporters – of what they consider the most important measures to include in health economic evaluations which assess the incremental value of competing options in mental health care. Methods Participants (n = 111) were people living in the state of Victoria, Australia, who identified as consumers of mental healthcare (n = 38); carers, family members and/or supporters (n = 43); or both (n = 30). Factor analysis based on Q-Methodology was used to identify clusters of people who hold similar viewpoints. Common viewpoints were described in terms of the characteristics of the group, and a qualitative interpretation was conducted on the basis of distinguishing statements and quotes provided in participants’ own words. Results We identified four common views: (1) safety before all else, prioritizing physical, sexual and psychological safety; (2) hope and partnership in processes of care; (3) physical and emotional health and wellbeing; and (4) care access, continuity and partnership with families. Although different priorities were identified for each viewpoint, key priority areas that were common to all views were having an environment in the health service that fosters respect and dignity, and that consumers feel heard and listened to. In sub-group and qualitative analyses, differences were observed regarding the likelihood of consumers and carers holding each of the views, as well as by age group. Conclusions While some differences were noted between the views of consumers and carers and different age groups, there was also common ground regarding what outcomes are of most importance to measure. Including these measures in evaluation frameworks would provide a way of focussing mental healthcare decisions on the aspects of mental healthcare that are of most value to consumers and carers, thereby addressing an important shortcoming of current approaches to decision-making in mental healthcare.

Abstract Background An important methodological challenge in conducting pediatric economic evaluations is estimating the preference-based health-related quality of life (HRQoL) of children. Current methods are highly variable and there is no single instrument available to value HRQoL consistently across multiple pediatric age groups. The Pediatric Quality of Life Inventory (PedsQL) is a non-preference-based generic HRQoL instrument validated for children 2–18 years, but it cannot be directly used in economic evaluations. The aim of this study was to establish the core dimension structure of the PedsUtil health state classification system using confirmatory factor analysis, which is the first step of deriving a preference-based measure of HRQoL based on the PedsQL. Methods Four competing dimension structures of the PedsUtil health state classification system were developed based on published literature and expert opinion. Using data from the Longitudinal Study of Australian Children (LSAC) (n = 45,207), the 4 dimension structures were evaluated using the robust weighted least squares estimation method. The analyses were stratified by 2-year age intervals (from 2 to 17 years) to reflect the study design of the LSAC, as well as special healthcare needs status of the child. Model fit was evaluated by examining standardized factor loadings and various fit indices including the comparative fit index (CFI), Tucker-Lewis Index (TLI), and the root mean square error of approximation (RMSEA). Modification indices and residual correlations were examined to re-specify the models to improve model fit when necessary. Results The findings supported a 7-dimension structure (i.e., Physical Functioning, Pain, Fatigue, Emotional Functioning, Social Functioning, School Functioning, and School Absence) of the PedsUtil health state classification system. The 7-dimension model exhibited adequate fit across subgroups with CFI values that ranged from 0.929 to 0.954, TLI values from 0.916 to 0.946, and RMSEA values from 0.058 to 0.102. Conclusions This study established the core dimension structure of the PedsUtil health state classification system using confirmatory factor analysis. The 7-dimension structure was found to be applicable across diverse pediatric populations. Research is currently ongoing to select the most representative item within each dimension of the PedsUtil health state classification system and valuation surveys will be fielded to estimate the PedsUtil scoring system.

Abstract Background There is a lack of preference-based health-related quality of life (HRQoL) measures that consistently value health across a full range of child age groups. The PedsQL is a generic HRQoL instrument validated for children 2–18 years, but it is not preference-based. The objective of this study was to derive the PedsUtil health state classification system from the PedsQL as a basis for a preference-based HRQoL measure for children. Methods A two-step process was used to select PedsQL items to include in the health state classification system: 1) exclude poorly functioning items according to Rasch analysis in each of the previously established seven dimensions of the PedsUtil health state classification system and 2) select a single item to represent each dimension based on Rasch and psychometric analyses, as well as input from child health experts and parents. All secondary analyses were conducted using data from the Longitudinal Study of Australian Children (LSAC). Analyses were stratified by age group (i.e., 2–5 years, 6–13 years, and 14–17 years) to represent the different developmental stages of children and to reflect the study design of the LSAC. Rasch analyses were also performed on five random subsamples for each age group to enhance robustness of results. Results Twelve items were excluded from the PedsUtil health state classification system after the first step of the item selection process. An additional four items were excluded in the second step, resulting in seven items that were selected to represent the seven dimensions of the PedsUtil health state classification system: Physical Functioning (“participating in sports activity or exercise”), Pain (“having hurts or aches”), Fatigue (“low energy level”), Emotional Functioning (“worrying about what will happen to them”), Social Functioning (“other kids not wanting to be their friend”), School Functioning (“keeping up with schoolwork”), and School Absence (“missing school because of not feeling well”). Conclusions The PedsUtil health state classification system was derived from the PedsQL based on several criteria and was constructed to be applicable to children two years and older. Research is ongoing to elicit preferences for the PedsUtil health state classification system to construct the PedsUtil scoring system.

Abstract Background A recent randomised trial demonstrated [18F]fluorodeoxyglucose positron-emission tomography in combination with low-dose CT (FDG-PET/CT), compared to standard of care computed tomography (CT) imaging, positively impacted antimicrobial management and outcomes of acute leukaemia and haematopoietic stem cell transplant recipients with persistent and recurrent neutropenic fever. We conducted an economic evaluation from a healthcare perspective alongside the clinical trial. Methods Unit costs in Australian dollars were applied to all resources used (antimicrobials, diagnostic tests, ICU and hospital bed days). Effectiveness was measured as number of patients with antimicrobial rationalisation, 6-month mortality and quality-adjusted life years (QALYs) derived from patient-reported trial-based health-related quality-of-life. Generalised linear models were used to analyse costs and outcomes. Incremental cost-effectiveness ratios (ICERs) for all outcomes and net monetary benefit (NMB) for QALYs were calculated. We performed bootstrapping with 1000 replications using the recycled predictions method. Results The adjusted healthcare costs were lower for FDG-PET/CT (mean $49,563; 95%CI 36,867, 65,133) compared to CT (mean $57,574; 95% CI 44,837, 73,347). The difference in QALYs between the two groups was small (0.001; 95% CI -0.001, 0.004). When simulated 1000 times, FDG-PET/CT was the dominant strategy as it was cheaper with better outcomes than the standard CT group in 74% of simulations. The estimated NMBs at willingness-to-pay thresholds of $50,000 and $100,000 per QALY were positive, thus FDG-PET/CT remained cost-effective at these thresholds. Conclusions FDG-PET/CT is cost effective when compared to CT for investigation of persistent/recurrent neutropenic fever in high-risk patients, providing further support for incorporation of FDG-PET/CT into clinical guidelines and funding. Trial registration This trial is registered with ClinicalTrials.gov, NCT03429387.

Abstract Background To identify and describe distinct developmental trajectories of health-related quality of life (HRQoL) in a national level Australian population sample, overall and separately for boys and girls. Methods Data were from the Longitudinal Study of Australian Children (LSAC). Participants were children aged 4–5 years recruited in 2004 and followed through to age 16–17 years in 2016, and their caregivers. Group-based trajectory modelling was used to identify groups of children that follow qualitatively distinct developmental trajectories of HRQoL. Results Three distinct trajectories were identified for the total sample: (1) high-stable (52.2% of children); (2) middle-stable (38.0%); and (3) low-declining (9.8%). These trajectories differed for boys, who saw increasing HRQoL in the highest trajectory group; a middle-stable trajectory; and declining and rebounding HRQoL in the lowest trajectory group. In contrast, girls saw no increasing or rebounding trajectories; approximately half of girls had high-stable HRQoL and the remaining half had either steadily or rapidly declining HRQoL from age 4–5 to 16–17 years. Conclusions Our results highlight the importance of considering the distinct trajectories for girls and boys and not relying on population mean levels of HRQoL for decision-making. The presence of developmentally distinct trajectories of HRQoL, and differences in the trajectories faced by boys and girls, should be considered when assessing the effectiveness of treatments and interventions impacting upon HRQoL throughout childhood and adolescence. Failure to account for these pre-existing trajectories may over- or under-estimate treatment effects.

Objective To compared the cost-effectiveness of coadministration of a probiotic adjuvant with peanut oral immunotherapy (PPOIT) with placebo (no treatment) in children with peanut allergy.Design Prospectively planned cost-effectiveness analysis alongside a randomised control trial.Setting The Royal Children’s Hospital, Melbourne, Australia.Participants 56 children with peanut allergy aged 1–10 years at recruitment.Intervention A daily dose of probiotic Lactobacillus rhamnosus CGMCC 1.3724 (NCC4007) and peanut oral immunotherapy administered for 1.5 years.Main outcomes measures Costs were considered from a healthcare system perspective and included costs of treatment delivery and adverse events. Effectiveness outcomes included rate of sustained unresponsiveness (SU) and quality-adjusted life years (QALYs). The cost-effectiveness of PPOIT versus placebo was analysed using patient-level data. Time horizon was 10 years from commencement of PPOIT treatment, comprising 1.5 years of treatment (actual data), 4 years of post-treatment follow-up (actual data), and 4.5 years of extrapolation thereafter (modelling).Results Healthcare cost per patient over 10 years was higher for PPOIT compared with placebo ($A9355 vs $A1031, p

Objective To estimate traumatic brain injuries (TBIs) and acute care costs due to sports activities.Methods A planned secondary analysis of 7799 children from 5 years old to

Abstract Background Recruitment of participants is crucial to the success of randomised control trials (RCTs) but can be challenging and expensive. Current research on trial efficiency is often focused at the patient-level with an emphasis on effective recruitment strategies. Less is known about selection of study sites to optimise recruitment. We examine site-level factors that are associated with patient recruitment and cost efficiency using data from an RCT conducted across 25 general practices (GP) in Victoria, Australia. Methods Data on number of participants screened, excluded, eligible, recruited, and randomised from each study site were extracted from a clinical trial. Details regarding site characteristics, recruitment practices, and staff time commitment were collected using a three-part survey. The key outcomes assessed were recruitment efficiency (ratio of screened to randomised), average time, and cost for each participant recruited and randomised. To identify practice-level factors associated with efficient recruitment and lower cost, outcomes were dichotomised (25th percentile vs others) and each practice-level factor assessed against the outcomes to determine its association. Results Across 25 GP study sites, 1968 participants were screened of which 299 (15.2%) were recruited and randomised. The mean recruitment efficiency was 7.2, varying from 1.4 to 19.8 across sites. The strongest factor associated with efficiency was assigning clinical staff to identify potential participants (57.14% vs. 22.2%). The more efficient sites were smaller practices and were more likely to be rural locations and in areas of lower socioeconomic status. The average time used for recruitment was 3.7 h (SD2.4) per patient randomised. The mean cost per patient randomised was $277 (SD161), and this varied from $74 to $797 across sites. The sites identified with the 25% lowest recruitment cost (n = 7) were more experienced in research participation and had high levels of nurse and/or administrative support. Conclusion Despite the small sample size, this study quantified the time and cost used to recruit patients and provides helpful indications of site-level characteristics that can help improve feasibility and efficiency of conducting RCT in GP settings. Characteristics indicative of high levels of support for research and rural practices, which often tends to be overlooked, were observed to be more efficient in recruiting.

Introduction Genomic testing is a relatively new, disruptive and complex health technology with multiple clinical applications in rare diseases, cancer and infection control. Genomic testing is increasingly being implemented into clinical practice, following regulatory approval, funding and adoption in models of care, particularly in the area of rare disease diagnosis. A significant barrier to the adoption and implementation of genomic testing is funding. What remains unclear is what the cost of genomic testing is, what the underlying drivers of cost are and whether policy differences contribute to cost variance in different jurisdictions, such as the requirement to have staff with a medical license involved in testing. This costing study will be useful in future economic evaluations and health technology assessments to inform optimal levels of reimbursement and to support comprehensive and comparable assessment of healthcare resource utilisation in the delivery of genomic testing globally.Methods A framework is presented that focuses on uncovering the process of genomic testing for any given laboratory, evaluating its utilisation and unit costs, and modelling the cost drivers and overall expenses associated with delivering genomic testing. The goal is to aid in refining and implementing policies regarding both the regulation and funding of genomic testing. A process-focused (activity-based) methodology is outlined, which encompasses resources, assesses individual cost components through a combination of bottom-up and top-down microcosting techniques and allows disaggregation of resource type and process step.Ethics and dissemination The outputs of the study will be reported at relevant regional genetics and health economics conferences, as well as submitted to a peer-reviewed journal focusing on genomics. Human research ethics committee approval is not required for this microcosting study. This study does not involve research on human subjects, and all data used in the analysis are either publicly available.

BackgroundUnaddressed family adversity has potentially modifiable, negative biopsychosocial impacts across the life course. Little is known about how Australian health and social practitioners identify and respond to family adversity in community and primary health settings.ObjectiveTo describe, in two Australian community health services: (1) the number of adversities experienced by caregivers, (2) practitioner identification of caregivers experiencing adversity, (3) practitioner response to caregivers experiencing adversity, and (4) caregiver uptake of referrals.MethodsSurvey of caregivers of children aged 0–8 years attending community health services in Victoria and New South Wales (NSW). Analysis described frequencies of caregiver self-reported: (1) experiences of adversity, (2) practitioner identification of adversity, (3) practitioner response to adversity, and (4) referral uptake. Analyses were sub-grouped by three adversity domains and site.Results349 caregivers (Victoria: n = 234; NSW: n = 115) completed the survey of whom 88% reported experiencing one or more family adversities. The median number of adversities was 4 (2–6). Only 43% of participants were directly asked about or discussed an adversity with a practitioner in the previous 6 months (Victoria: 30%; NSW: 68%). Among caregivers experiencing adversity, 30% received direct support (Victoria: 23%; NSW: 43%), and 14% received a referral (Victoria: 10%; NSW: 22%) for at least one adversity. Overall, 74% of caregivers accepted referrals when extended.ConclusionThe needs of Australian families experiencing high rates of adversity are not systematically identified nor responded to in community health services. This leaves significant scope for reform and enhancement of service responses to families experiencing adversity.

Abstract Background There is increasing interest in the validation of pediatric preference-based health-related quality of life measurement instruments. It is critical that children with various degrees of health-related quality of life (HRQoL) impact are included in validation studies. To inform patient sample selection for validation studies from a pragmatic perspective, this study explored HRQoL impairments between known-groups and HRQoL changes over time across 27 common chronic child health conditions and identified conditions with the largest impact on HRQoL. Methods The health dimensions of two common preference-based HRQoL measures, the EQ-5D-Y and CHU9D, were constructed using Pediatric Quality of Life Inventory items that overlap conceptually. Data was from the Longitudinal Study of Australian Children, a nationally representative sample with over 10,000 children at baseline. Seven waves of data were included for the analysis, with child age ranging from 2 to18 years. Impacts to specific health dimensions and overall HRQoL between those having a specific condition versus not were compared using linear mixed effects models. HRQoL changes over time were obtained by calculating the HRQoL differences between two consecutive time points, grouped by “Improved” and “Worsened” health status. Comparison among various health conditions and different age groups (2–4 years, 5–12 years and 13–18 years) were made. Results Conditions with the largest statistically significant total HRQoL impairments of having a specific condition compared with not having the condition were recurrent chest pain, autism, epilepsy, anxiety/depression, irritable bowel, recurrent back pain, recurrent abdominal pain, and attention deficit hyperactivity disorder (ADHD) for the total sample (2–18 years). Conditions with largest HRQoL improvement over time were anxiety/depression, ADHD, autism, bone/joint/muscle problem, recurrent abdominal pain, recurrent pain in other part, frequent headache, diarrhea and day-wetting. The dimensions included in EQ-5D-Y and CHU9D can generally reflect HRQoL differences and changes. The HRQoL impacts to specific health dimensions differed by condition in the expected direction. The conditions with largest HRQoL impacts differed by age group. Conclusions The conditions with largest HRQoL impact were identified. This information is likely to be valuable for recruiting patient samples when validating pediatric preference-based HRQoL instruments pragmatically.

Introduction Adolescence is a period of major transition in physical, cognitive, social and emotional development, and the peak time for the onset of mental health conditions, substance use disorders and sexual and reproductive health risks. Prevention and treatment during this time can improve health and well-being now and into the future. However, despite clinical guidelines recommending annual preventive health assessments for young people, health professionals cite lack of consultation time and adequate funding as key barriers. This trial aims to determine whether a specific fee-for-service (‘rebate payment’) for a young person’s health assessment, is effective and cost-effective at increasing the detection and management of health risk behaviours and conditions among young people.Methods and analysis This cluster randomised controlled trial will be conducted in Australian general practice. 42 general practices (clusters) will be randomly allocated 1:1 to either an intervention arm where general practitioners receive a rebate payment for each annual health assessment undertaken for 14–24-year-olds during a 2 year study period, or a control arm (no rebate). The rebate amount will be based on the Medical Benefits Schedule (Australia’s list of health professional services subsidised by the Australian Government) currently available for similar age-based assessments. Our primary outcome will be the annual rate of risk behaviours and health conditions recorded in the patient electronic health record (eg, alcohol/drug use, sexual activity and mental health issues). Secondary outcomes include the annual rate of patient management activities related to health risks and conditions identified (eg, contraception prescribed, sexually transmitted infection tests ordered). A process evaluation will assess acceptability, adoption, fidelity and sustainability of the rebate; an economic evaluation will assess its cost-effectiveness. Analyses will be intention-to-treat.Ethics and dissemination Ethics approval has been obtained from University of Melbourne Human and Research Ethics Committee (2022-23435-29990-3). Findings will be published in peer-reviewed journals.Trial registration number ACTRN12622000114741

Background: Collecting data using paediatric health-related quality of life (HRQoL) instruments is complex, and there is a paucity of evidence regarding the comparative performance of paediatric HRQoL instruments. The Australian Paediatric Multi-Instrument Comparison (P-MIC) study was conducted to address this paucity of evidence. This study aims to understand the (1) feasibility of collecting data using paediatric HRQoL instruments in a research setting and (2) acceptability and feasibility for children and their caregivers to complete common paediatric HRQoL instruments using data from the Australian P-MIC study. Methods: Data were from children aged 5–18 years from the Australian P-MIC study. Demographics, cost and time for data collection, dropout rates, and inconsistent responses were used to assess Aim 1. Participant-reported difficulty and completion time were used to assess Aim 2. Subgroup analyses included child age, report type (self/proxy), sample recruitment pathway (hospital/online), and online panel sample type (general population/condition groups). Results: Overall, 5945 P-MIC participants aged 5–18 years completed an initial survey, of these, 2346 also completed the follow-up survey (39.5% response rate). Compared with online panel recruitment, hospital recruitment was more costly and time-consuming and had higher follow-up completion (33.5% versus 80.4%) (Aim 1). Data were of similar good quality (based on inconsistent responses) for both recruitment pathways (Aim 1). Participants completed each instrument in 70% reported each instrument as easy to complete (Aim 2). Conclusions: The Australian P-MIC study was able to collect good-quality data using both online panel and hospital recruitment pathways. All instruments were acceptable and feasible to children and their caregivers.

Introduction Australia’s current healthcare system for children is neither sustainable nor equitable. As children (0–4 years) comprise the largest proportion of all primary care-type emergency department presentations, general practitioners (GPs) report feeling undervalued as an integral member of a child’s care, and lacking in opportunities for support and training in paediatric conditions. This Strengthening Care for Children (SC4C) randomised trial aims to evaluate a novel, integrated GP-paediatrician model of care, that, if effective, will improve GP quality of care, reduce burden to hospital services and ensure children receive the right care, at the right time, closer to home.Methods and analysis SC4C is a stepped wedge cluster randomised controlled trial (RCT) of 22 general practice clinics in Victoria and New South Wales, Australia. General practice clinics will provide control period data before being exposed to the 12-month intervention which will be rolled out sequentially each month (one clinic per state) until all 22 clinics receive the intervention. The intervention comprises weekly GP-paediatrician co-consultation sessions; monthly case discussions; and phone and email paediatrician support, focusing on common paediatric conditions. The primary outcome of the trial is to assess the impact of the intervention as measured by the proportion of children’s (0–

Introduction Advances in the care of patients with single-ventricle congenital heart disease have led to a new generation of individuals living with a Fontan circulation. For people with Fontan physiology, physical, psychological and neurodevelopmental challenges are common. The objective of this study is to describe and develop a deeper understanding of the factors that contribute to quality of life (QOL) among children, adolescents and adults living with a Fontan circulation across Australia and New Zealand, their parents and siblings.Methods and Analysis This article presents the protocol for the Australian and New Zealand Fontan Registry (ANZFR) QOL Study, a cross-sectional, population-based study designed to examine QOL among people of all ages with a Fontan circulation, their parents and siblings. Study eligibility criteria includes (1) individuals with a Fontan circulation aged ≥6 years, at least 12 months post-Fontan procedure and enrolled in the ANZFR; (2) parents of individuals enrolled in the ANZFR; and (3) siblings aged ≥6 years of an individual enrolled in the ANZFR. A novel, online research platform is used to distribute personalised assessments tailored to participant age and developmental stage. A suite of validated psychometric self-report and parent-proxy report instruments capture potential correlates and predictors of QOL, including symptoms of psychological distress, personality attributes, coping and cognitive appraisals, family functioning, healthcare experiences and costs, access to emotional support and socioeconomic factors. Clinical characteristics are captured via self-report and parent-proxy report, as well as the ANZFR. Descriptive analyses and multilevel models will be used to examine QOL across groups and to investigate potential explanatory variables.Ethics and Dissemination Approval has been obtained from all relevant Human Research Ethics Committees (HRECs), including the Sydney Children’s Hospitals Network and the Royal Children’s Hospital Melbourne HRECs. Study findings will be published in peer-reviewed journals and presented at national and international meetings and seminars.

Introduction Integrated community healthcare Hubs may offer a ‘one stop shop’ for service users with complex health and social needs, and more efficiently use service resources. Various policy imperatives exist to implement Hub models of care, however, there is a dearth of research specifically evaluating Hubs targeted at families experiencing adversity. To contribute to building this evidence, we propose to co-design, test and evaluate integrated Hub models of care in two Australian community health services in low socioeconomic areas that serve families experiencing adversity: Wyndham Vale in Victoria and Marrickville in New South Wales.Methods and analysis This multisite convergent mixed-methods study will run over three phases to (1) develop the initial Hub programme theory through formative research; (2) test and, then, (3) refine the Hub theory using empirical data. Phase 1 involves co-design of each Hub with caregivers, community members and practitioners. Phase 2 uses caregiver and Hub practitioner surveys at baseline, and 6 and 12 months after Hub implementation, and in-depth interviews at 12 months. Two stakeholder groups will be recruited: caregivers (n=100–200 per site) and Hub practitioners (n=20–30 per site). The intervention is a co-located Hub providing health, social, legal and community services with no comparator. The primary outcomes are caregiver-reported: (i) identification of, (ii) interventions received and/or (iii) referrals received for adversity from Hub practitioners. The study also assesses child, caregiver, practitioner and system outcomes including mental health, parenting, quality of life, care experience and service linkages. Primary and secondary outcomes will be assessed by examining change in proportions/means from baseline to 6 months, from 6 to 12 months and from baseline to 12 months. Service linkages will be analysed using social network analysis. Costs of Hub implementation and a health economics analysis of unmet need will be conducted. Thematic analysis will be employed to analyse qualitative data.Ethics and dissemination Royal Children’s Hospital and Sydney Local Health District ethics committees have approved the study (HREC/62866/RCHM-2020). Participants and stakeholders will receive results through meetings, presentations and publications.Trial registration number ISRCTN55495932.

Abstract Background General Practice Optimising Structured Monitoring to Improve Clinical Outcomes in Type 2 Diabetes (GP-OSMOTIC) is a multicentre, individually randomised controlled trial aiming to compare the use of intermittent retrospective continuous glucose monitoring (r-CGM) to usual care in patients with type 2 diabetes attending general practice. The study protocol was published in the British Medical Journal Open and described the principal features of the statistical methods that will be used to analyse the trial data. This paper provides greater detail on the statistical analysis plan, including background and justification for the statistical methods chosen, in accordance with SPIRIT guidelines. Objective To describe in detail the data management process and statistical methods that will be used to analyse the trial data. Methods An overview of the trial design and primary and secondary research questions are provided. Sample size assumptions and calculations are explained, and randomisation and data management processes are described in detail. The planned statistical analyses for primary and secondary outcomes and sub-group analyses are specified along with the intended table layouts for presentation of the results. Conclusion In accordance with best practice, all analyses outlined in the document are based on the aims of the study and have been pre-specified prior to the completion of data collection and outcome analyses. Trial registration Australian New Zealand Clinical Trials Registry, ACTRN12616001372471. Registered on 3 August 2016.

Introduction While most children recover from a concussion shortly after injury, approximately 30% experience persistent postconcussive symptoms (pPCS) beyond 1-month postinjury. Existing research into the treatment of pPCS have evaluated unimodal approaches, despite evidence suggesting that pPCS likely represent an interaction across various symptom clusters. The primary aim of this study is to evaluate the effectiveness of a multimodal, symptom-tailored intervention to accelerate symptom recovery and increase the proportion of children with resolved symptoms at 3 months postconcussion.Methods and analysis In this open-label, assessor-blinded, randomised clinical trial, children with concussion aged 8–18 years will be recruited from The Royal Children’s Hospital (The RCH) emergency department, or referred by a clinician, within 17 days of initial injury. Based on parent ratings of their child’s PCS at ~10 days postinjury, symptomatic children (≥2 symptoms at least 1-point above those endorsed preinjury) will undergo a baseline assessment at 3 weeks postinjury and randomised into either Concussion Essentials (CE, n=108), a multimodal, interdisciplinary delivered, symptom-tailored treatment involving physiotherapy, psychology and education, or usual care (UC, n=108) study arms. CE participants will receive 1 hour of intervention each week, for up to 8 weeks or until pPCS resolve. A postprogramme assessment will be conducted at 3 months postinjury for all participants. Effectiveness of the CE intervention will be determined by the proportion of participants for whom pPCS have resolved at the postprogramme assessment (primary outcome) relative to the UC group. Secondary outcome analyses will examine whether children receiving CE are more likely to demonstrate resolution of pPCS, earlier return to normal activity, higher quality of life and a lower rate of utilisation of health services, compared with the UC group.Ethics and dissemination Ethics were approved by The RCH Human Research Ethics Committee (HREC: 37100). Parent, and for mature minors, participant consent, will be obtained prior to commencement of the trial. Study results will be disseminated at international conferences and international peer-reviewed journals.Trial registration number ACTRN12617000418370; pre-results.

Background Cancer patients are at significant risk of developing sepsis due to underlying malignancy and necessary treatments. Little is known about the economic burden of sepsis in this high-risk population. We estimate the short- and long-term healthcare costs of care of cancer patients with and without sepsis using individual-level linked-administrative data. Methods We conducted a population-based matched cohort study of cancer patients aged ≥18, diagnosed between 2010 and 2017. Cases were identified if diagnosed with sepsis during the study period, and were matched 1:1 by age, sex, cancer type and other variables to controls without sepsis. Mean costs (2018 Canadian dollars) for patients with and without sepsis up to 5 years were estimated adjusted using survival probabilities at partitioned intervals. We estimated excess cost associated with sepsis presented as a cost difference between the two cohorts. Haematological and solid cancers were analysed separately. Results 77,483 cancer patients with sepsis were identified and matched. 64.3% of the cohort were aged ≥65, 46.3% female and 17.8% with haematological malignancies. Among solid tumour patients, the excess cost of care among patients who developed sepsis was $29,081 (95%CI, $28,404-$29,757) in the first year, rising to $60,714 (95%CI, $59,729-$61,698) over 5 years. This was higher for haematology patients; $46,154 (95%CI, $45,505-$46,804) in year 1, increasing to $75,931 (95%CI, $74,895-$76,968). Conclusions Sepsis imposes substantial economic burden and can result in a doubling of cancer care costs, particularly during the first year of cancer diagnosis. These estimates are helpful in improving our understanding of burden of sepsis along the cancer pathway and to deploy targeted strategies to alleviate this burden.

Introduction Emerging evidence indicates that rehabilitation can improve ataxia, mobility and independence in everyday activities in individuals with hereditary cerebellar ataxia. However, with the rarity of the genetic ataxias and known recruitment challenges in rehabilitation trials, most studies have been underpowered, non-randomised or non-controlled. This study will be the first, appropriately powered randomised controlled trial to examine the efficacy of an outpatient and home-based rehabilitation programme on improving motor function for individuals with hereditary cerebellar ataxia.Methods and analysis This randomised, single-blind, parallel group trial will compare a 30-week rehabilitation programme to standard care in individuals with hereditary cerebellar ataxia. Eighty individuals with a hereditary cerebellar ataxia, aged 15 years and above, will be recruited. The rehabilitation programme will include 6 weeks of outpatient land and aquatic physiotherapy followed immediately by a 24- week home exercise programme supported with fortnightly physiotherapy sessions. Participants in the standard care group will be asked to continue their usual physical activity. The primary outcome will be the motor domain of the Functional Independence Measure. Secondary outcomes will measure the motor impairment related to ataxia, balance, quality of life and cost-effectiveness. Outcomes will be administered at baseline, 7 weeks, 18 weeks and 30 weeks by a physiotherapist blinded to group allocation. A repeated measures mixed-effects linear regression model will be used to analyse the effect of the treatment group for each of the dependent continuous variables. The primary efficacy analysis will follow the intention-to-treat principle.Ethics and dissemination The study has been approved by the Monash Health Human Research Ethics Committee (HREC/18/MonH/418) and the Human Research Ethics Committee of the Northern Territory Department of Health and Menzies School of Health Research (2019/3503). Results will be published in peer-reviewed journals, presented at national and/or international conferences and disseminated to Australian ataxia support groups.Trial registration number ACTRN12618000908235.

Abstract Background Cost-effectiveness analysis (CEA) is frequently used as an input for guiding priority setting in health. However, CEA seldom incorporates information about trade-offs between total health gains and equity impacts of interventions. This study investigates to what extent equity considerations have been taken into account in CEA in low- and middle-income countries (LMICs), using rotavirus vaccination as a case study. Methods Specific equity-related indicators for vaccination were first mapped to the Guidance on Priority Setting in Health Care (GPS-Health) checklist criteria. Economic evaluations of rotavirus vaccine in LMICs identified via a systematic review of the literature were assessed to explore the extent to which equity was considered in the research objectives and analysis, and whether it was reflected in the evaluation results. Results The mapping process resulted in 18 unique indicators. Under the ‘disease and intervention’ criteria, severity of illness was incorporated in 75% of the articles, age distribution of the disease in 70%, and presence of comorbidities in 5%. For the ‘social groups’ criteria, relative coverage reflecting wealth-based coverage inequality was taken into account in 30% of the articles, geographic location in 27%, household income level in 8%, and sex at birth in 5%. For the criteria of ‘protection against the financial and social effects of ill health’, age weighting was incorporated in 43% of the articles, societal perspective in 58%, caregiver’s loss of productivity in 45%, and financial risk protection in 5%. Overall, some articles incorporated the indicators in their model inputs (20%) while the majority (80%) presented results (costs, health outcomes, or incremental cost-effectiveness ratios) differentiated according to the indicators. Critically, less than a fifth (17%) of articles incorporating indicators did so due to an explicit study objective related to capturing equity considerations. Most indicators were increasingly incorporated over time, with a notable exception of age-weighting of DALYs. Conclusion Integrating equity criteria in CEA can help policy-makers better understand the distributional impact of health interventions. This study illustrates how equity considerations are currently being incorporated within CEA of rotavirus vaccination and highlights the components of equity that have been used in studies in LMICs. Areas for further improvement are identified.

Background: There is a lack of psychometric evidence about pediatric health-related quality of life (HRQoL) instruments. Evidence on cost effectiveness, involving the measurement of HRQoL, is used in many countries to make decisions about pharmaceuticals, technologies, and health services for children. Additionally, valid instruments are required to facilitate accurate outcome measurement and clinical decision making. A pediatric multi instrument comparison (P-MIC) study is planned to compare the psychometric performance and measurement characteristics of pediatric HRQoL instruments. Methods: The planned P-MIC study will collect data on approximately 6100 Australian children and adolescents aged 2–18 years via The Royal Children’s Hospital Melbourne and online survey panels. Participants will complete an initial survey, involving the concurrent collection of a range of pediatric HRQoL instruments, followed by a shorter survey 2–8 weeks later, involving the collection of a subset of instruments from the initial survey. Children aged ≥7 years will be asked to self-report HRQoL. Psychometric performance will be assessed at the instrument, domain, and item level. Conclusions: This paper describes the methodology of the planned P-MIC study, including benefits, limitations, and likely challenges. Evidence from this study will guide the choice of HRQoL measures used in clinical trials, economic evaluation, and other applications.

Abstract Background A considerable number of people with a mental illness are parents caring for dependent children. For those with a mental illness, parenting can provide a sense of competence, belonging, identity and hope and hence is well aligned to the concept of personal recovery. However, little research has focused on the recovery journey of those who are parents and have a mental illness. This randomised controlled trial aims to (i) evaluate the effectiveness of an intervention model of recovery for parents (Let’s Talk about Children) in three different mental health service sectors and (ii) examine the economic value of a larger roll out (longer term) of the parent recovery model. Methods A two arm parallel randomised controlled trial will be used with participants, who are being treated for their mental illness in adult mental health, non-government community mental health or family welfare services. The study will involve 192 parents, who are considered by their treating practitioner to be sufficiently well to provide informed consent and participate in an intervention (Let’s Talk about Children) or control group (treatment as usual). Participant randomisation will occur at the level of the treating practitioner and will be based on whether the randomised practitioner is trained in the intervention. Outcomes are compared at pre, post intervention and six-month follow-up. Recovery, parenting and family functioning, and quality of life questionnaires will be used to measure parent wellbeing and the economic benefits of the intervention. Discussion This is the first randomised controlled trial to investigate the efficacy of a parenting intervention on recovery outcomes and the first to provide an economic evaluation of an intervention for parents with a mental illness. An implementation model is required to embed the intervention in different sectors. Trial registration The trial was retrospectively registered: ACTRN12616000460404 on the 8/4/2016.

Objective To develop and implement a whole of hospital clinical pathway for the management of sepsis (SP) in a specialised cancer hospital and to measure the impact on patient outcomes and healthcare utilisation.Methods A multidisciplinary sepsis working party was established. Process mapping of practices for recognition and management of sepsis was undertaken across all clinical areas. A clinical pathway document that supported nurse-initiated sepsis care, prompt antibiotic and fluid resuscitation was implemented. Process and outcome measures for patients with sepsis were collected preimplementation (April–December 2012), postimplementation cohorts (April–December 2013), and from January to December 2014.Results 323 patients were evaluated (111 preimplementation, 212 postimplementation). More patients with sepsis had lactate measured (75.0% vs 17.2%) and appropriate first dose antibiotic (90.1% vs 76.1%) (all p