The cost of proband and trio exome and genome analysis in rare disease: A micro-costing study.

PURPOSE: Rare disease genomic testing is a complex process involving various resources. Accurate resource estimation is required for informed prioritization and reimbursement decisions. This study aims to analyze the costs and cost drivers of clinical genomic testing. METHODS: Based on genomic sequencing workflows we microcosted limited virtual panel analysis on exome sequencing backbone, proband and trio exome, and genome testing for proband and trio analysis in 2023 Australian Dollars ($). Deterministic and probabilistic sensitivity analyses were undertaken. RESULTS: Panel testing costs AUD $2373 ($733-$6166), and exome sequencing costs $2823 ($802-$7206) and $5670 ($2006-$11,539) for proband and trio analysis, respectively. Genome sequencing costs $4840 ($2153-$9890) and $11,589 ($5842-$16,562) for proband and trio analysis. The most expensive cost component of genomic testing was sequencing (36.9%-69.4% of total cost), with labor accounting for 27.1%-63.2% of total cost. CONCLUSION: We provide a comprehensive analysis of rare disease genomic testing costs, for a range of clinical testing types and contexts. This information will accurately inform economic evaluations of rare disease genomic testing and decision making on policy settings that assist with implementation, such as genomic testing reimbursement.

Selecting PedsQL items to derive the PedsUtil health state classification system to measure health utilities in children.

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.

Deriving the PedsUtil health state classification system to measure health utilities for pediatric populations based on the PedsQL: a confirmatory factor analysis.

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.

Microcosting diagnostic genomic sequencing: A systematic review.

PURPOSE: Microcosting can provide valuable economic evidence to inform the translation of genomic sequencing to clinical practice. A systematic literature review was conducted to identify studies employing microcosting methods to estimate the cost of genomic sequencing to diagnose cancer and rare diseases. METHODS: Four electronic databases, Medline, Embase, EconLit, and Cumulated Index to Nursing and Allied Health Literature were searched. Reference lists of identified studies were also searched. Studies were included if they had estimated the cost of genome sequencing or exome sequencing for cancer or rare disease diagnosis using microcosting methods. RESULTS: Seven studies met the inclusion criteria. Cost estimates for genome sequencing and exome sequencing ranged between US$2094 and $9706 and US$716 and $4817 per patient, respectively. All studies disaggregated resource use and cost inputs into labor, equipment, and consumables, with consumables being the main cost component. Considerable differences in the level of detail used to report the steps and resources used in each of the sequencing steps limited study comparisons. CONCLUSION: Defining a standard microcosting methodology is challenging because of the heterogeneous nature of genomic sequencing. Reporting of detailed and complete sequencing procedures, inclusion of sensitivity analyses and clear justifications of resource use, and measurement of unit costs can improve comparability, transferability, and generalizability of study findings.

TreEAT trial: Protocol for a randomized controlled trial investigating the efficacy and safety of early introduction of tree nuts for the prevention of tree nut allergy in infants with peanut allergy.

INTRODUCTION: Children with peanut allergy are at increased risk of developing tree nut allergies, which can be severe and for most lifelong. Introduction of peanut in the first year of life can reduce the risk of peanut allergy; however, prevention strategies for tree nut allergies have not been established. We aimed to test the efficacy and safety of a novel strategy, a supervised multi-nut oral food challenge (OFC) compared with standard care for tree nut allergy prevention in infants at high risk of developing tree nut allergy, TreEAT. METHODS AND ANALYSIS: TreEAT is a 2-armed, open-label, randomized, controlled trial (RCT). Infants (n = 212) aged 4-11 months with peanut allergy will be randomized 1:1 at peanut allergy diagnosis to either a hospital-based multi-tree nut (almond, cashew, hazelnut, and walnut) OFC using multi-nut butter or standard care (home introduction of individual tree nuts). All infants will be assessed at age 18 months, with questionnaires and SPT to peanut and tree nuts. Peanut and tree nut OFCs will be performed as required to determine the allergy status for each nut. The primary outcome is tree nut allergy at age 18 months. Secondary outcomes include peanut allergy resolution, proportion, and severity of adverse events related to tree nut ingestion, number and frequency of tree nuts ingested, quality of life and parental anxiety, and allergy-related healthcare visits from randomization to 18 months of age. Analyses will be performed on an intention-to-treat basis. ETHICS AND DISSEMINATION: TreEAT was approved by the Royal Children's Hospital Human Research Ethics Committee (#70489). Outcomes will be presented at scientific conferences and disseminated through publication. TRIAL REGISTRATION NUMBER: ClinicalTrials.gov ID: NCT04801823.

Test-Retest Reliability of EQ-5D-Y-3L Best-Worst Scaling Choices of Adolescents and Adults.

BACKGROUND: There is an increasing interest to obtain adolescents' own health state valuation preferences and to understand how these differ from adult preferences for the same health state. An important question in health state valuation is whether adolescents can report preferences reliably, yet research remains limited. OBJECTIVE: This study aims to investigate the test-retest reliability of best-worst scaling (BWS) to elicit adolescent preferences compared with adults. METHODS: Identical BWS tasks designed to value 3-level version of EQ-5D-Y health states were administered online in samples of 1000 adolescents (aged 11-17 years) and 1006 adults in Spain. The valuation survey was repeated approximately 3 days later. We calculated (1) simple percentage agreement and (2) kappa statistic as measures of test-retest reliability. We also compared BWS marginal frequencies and relative attribute importance between baseline and follow-up to explore similarities in the obtained preferences. RESULTS: We found that both adolescents and adults were able to report their preferences with moderate reliability (kappa: 0.46 for adolescents, 0.46 for adults) for best choices and fair to moderate reliability (kappa: 0.39 for adolescents, 0.41 for adults) for worst choices. No notable difference was observed across years of child age. Higher consistency was observed for best choices than worst in some dimensions for both populations. No significant differences were found in the relative attribute importance between baseline and follow-up in both populations. CONCLUSION: Our results suggest that BWS is a reliable elicitation technique to value 3-level version of EQ-5D-Y health states in both adolescents and adults.

Use of Utility and Disability Weights in Economic Evaluation of Pediatric Vaccines.

OBJECTIVE: To describe how utility weights and disability weights have been used in the context of quality-adjusted life-years (QALYs) and disability-adjusted life-years (DALYs)-based cost-effectiveness analysis (CEA) of pediatric vaccines for infectious diseases and assess the comparability between weights. METHODS: A systematic review was conducted of CEAs of pediatric vaccines for 16 infectious diseases, published between January 2013 and December 2020 and using QALYs or DALYs as outcome measure. Data on values and sources of weights for the estimation of QALYs and DALYs were extracted from studies and compared across similar health states. Reporting was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. RESULTS: Out of 2154 articles identified, 216 CEAs met our inclusion criteria. Of the included studies, 157 used utility weights and 59 used disability weights in their valuation of health states. In QALY studies, the source, background, who's preferences (adults'/children's) were applied and adjustments made to utility weights were poorly reported. In DALY studies, the Global Burden of Disease study was most often referenced. Valuation weights for similar health states varied within QALY studies and between DALY and QALY studies, but no systematic differences were identified. CONCLUSIONS: This review identified considerable gaps in the way valuation weights are used and reported on in CEA. The nonstandardized use of weights may lead to different conclusions about cost-effectiveness of vaccines and policy decisions.

A Qualitative Investigation to Develop an Adapted Version of the EQ-5D-Y-3L for Use in Children Aged 2-4 Years.

OBJECTIVES: Few preference-weighted health-related quality-of-life measures exist for children under 5 years of age. Young children are substantial consumers of healthcare services. This project aims to assess EQ-5D-Y-3L's appropriateness in children aged 2 to 4 years and to coproduce with parents a suitable adaptation. METHODS: Purposive sampling at the Murdoch Children's Research Institute and Royal Children's Hospital was used to recruit parents or carers of children aged 2 to 4 years in Australia. Online focus groups were conducted consisting of 13 parents of healthy children, and 6 parents of children with moderate to severe health conditions. Parents provided feedback on each dimension of the proxy EQ-5D-Y-3L. Recordings were transcribed and thematic analysis was conducted. Qualitative findings guided the design of adaptations to the instrument. The adaptations were piloted to obtain feedback and refined to improve language translatability and comparability with other EuroQol instruments. RESULTS: The adapted EQ-5D-Y-3L was considered generally acceptable by the parents. Parents provided a wide range of examples of how each domain related to their children, with varied examples provided across ages 2 to 4 years and health status. Additional or alternative wording was suggested by parents to improve the applicability of the instrument to this age group. One example of this was the change of the domain wording "walking about" to "movement"-ID5:"In this age group, movement is more important than walking." CONCLUSIONS: The adapted EQ-5D-Y-3L has improved relevance for 2-4-year olds and appears easy to complete. Further testing of the adapted instrument is required to evaluate acceptability, reliability, and validity.

Distinct developmental trajectories of health-related quality of life for boys and girls throughout childhood and adolescence; a national level longitudinal study.

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.

How do common conditions impact health-related quality of life for children? Providing guidance for validating pediatric preference-based measures.

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.

Targeted screening for congenital cytomegalovirus: A micro-costing analysis.

AIM: We aimed to determine the cost and potential cost-savings of delivering a targeted congenital cytomegalovirus (cCMV) screening programme through a universal newborn hearing screening (UNHS) programme to detect cCMV-related hearing loss in infants from Victoria, Australia. METHODS: We completed a micro-costing analysis from a health-care perspective using data from a targeted cCMV screening programme piloted between June 2019 and March 2020. The programme involved collection of saliva samples to test for cCMV in infants who: received a 'refer' result on their second newborn hearing screen; were aged 21 days or less; and born at one of four maternity hospitals in Victoria, Australia. All costs to complete targeted cCMV screening were recorded in Australian 2020 dollars. Potential costs and benefits of adding targeted cCMV screening to the pre-existing UNHS programme were compared to when no screening was available up to 18 years to determine the likely cost or cost savings. RESULTS: The cost of adding targeted cCMV screening to Victoria's UNHS is $202 per infant screened. The total cost per positive case identified is $21 456. The overall cost of adding targeted salivary cCMV screening at the point of a second 'refer' result on the UNHS programme in Victoria's four largest hospitals is estimated to be $28 966 for the first year. CONCLUSION: Targeted screening for cCMV provides families the opportunity to detect and, if appropriate, treat cCMV in the first month of life in line with current recommendations. It falls within the range between cost neutral and cost saving.

Cost-effectiveness of professional-mode flash glucose monitoring in general practice among adults with type 2 diabetes: Evidence from the GP-OSMOTIC trial.

AIM: To assess the cost-effectiveness of professional-mode flash glucose monitoring in adults with type 2 diabetes in general practice compared with usual clinical care. METHODS: An economic evaluation was conducted as a component of the GP-OSMOTIC trial, a pragmatic multicentre 12-month randomised controlled trial enrolling 299 adults with type 2 diabetes in Victoria, Australia. The economic evaluation was conducted from an Australian healthcare sector perspective with a lifetime horizon. Health-related quality of life (EQ-5D) and total healthcare costs were compared between the intervention and the usual care group within the trial period. The 'UKPDS Outcomes Model 2' was used to simulate post-trial lifetime costs, life expectancy and quality-adjusted life years (QALYs). RESULTS: No significant difference in health-related quality of life and costs was found between the two groups within the trial period. Professional-mode flash glucose monitoring yielded greater QALYs (0.03 [95% CI: 0.02, 0.04]) and a higher cost (A$3807 [95% CI: 3604, 4007]) compared with usual clinical care using a lifetime horizon under the trial-based monitoring frequency, considered not cost-effective (incremental cost-effectiveness ratio = A$120,228). The intervention becomes cost-effective if sensor price is reduced to lower than 50%, or monitoring frequency is decreased to once per year while maintaining the same treatment effect on HbA1c . CONCLUSIONS: Including professional-mode flash glucose monitoring every 3 months as part of a management plan for people with type 2 diabetes in general practice is not cost-effective, but could be if the sensor price or monitoring frequency can be reduced.

Ana o 3 sIgE and diagnostic algorithms reduce cost of cashew allergy diagnosis in children compared with skin prick test: A cost comparison analysis.

BACKGROUND: In the absence of a clear clinical history of reaction, diagnosis of cashew allergy using skin prick tests (SPT) or cashew-specific IgE requires a high number of oral food challenges (OFC). By using Ana o 3 sIgE alone, or a two-step diagnostic algorithm using cashew sIgE followed by Ana o 3 sIgE, there is a reduced need for OFC. We aimed to perform a cost comparison for both of these approaches compared with cashew SPT alone. METHODS: Pooled individual-level data from 6 studies were used to determine diagnostic accuracy and OFC rate. Two studies used cashew SPT (n = 567, 198 allergic), with 95% positive and negative predictive values of ≥12 mm and <3 mm. Four studies were included in the pathways for Ana o 3 sIgE alone or a 2-step algorithm incorporating cashew and Ana o 3 sIgE (n = 271, 156 allergic). Cut-offs used were ≥8.5kUA/L and ≤0.1kUA/L for cashew sIgE and ≥0.35kUA/L and ≤0.1kUA/L for Ana o 3 sIgE. Costs were constructed based on unit prices from hospital inpatient admissions, expenses incurred by families, individual patient data on allergic reaction types and rates, and adrenaline autoinjector carriage, applying a health system perspective. RESULTS: Modeled data through the Ana o 3 pathway resulted in a 46.43% cost reduction (€307,406/1000 patients) compared with using cashew SPT alone (€573,854/1000 patients). The 2-step algorithm resulted in a 44.94% cost reduction compared with SPT alone (€315,952.82/1000 patients). Both the Ana o 3 pathway and 2-step algorithm resulted in a 79%-80% reduction in OFCs compared with SPT. CONCLUSIONS: Using Ana o 3 as a standalone test for cashew allergy diagnosis or a 2-step algorithm incorporating cashew sIgE and Ana o 3 sIgE is accurate and results in a large reduction in both OFCs and health system costs compared with cashew SPT alone.

Out-of-hospital health care costs of childhood food allergy in Australia: A population-based longitudinal study.

BACKGROUND: Australia has one of the highest prevalence of childhood food allergy in the world, but there are no data on its economic burden in Australia. METHODS: We used data from the HealthNuts study, a population-based longitudinal study undertaken in Melbourne, Australia. Infants were recruited at age 12 months between Sept 2007 and Aug 2011 with food allergy diagnosed using oral food challenges. Health care costs of out-of-hospital services were collected through data linkage to Australia's universal health insurance scheme Medicare. Two-part model was used to compare costs after controlling for potential confounders. RESULTS: 2919 children were included, and 390 (13.4%) had challenge-confirmed food allergy at age 1 year. Compared with children without food allergy, children with food allergy had significantly higher costs for GP visits, specialist visits, tests, and prescriptions in the first four years of life. The total Medicare cost associated with food allergy from age 1 to 4 years was estimated to be AUD$889.7 (95% CI $566.1-$1188.3) or €411.0 (95% CI €261.5-€549.0) per child. This was projected into an annual Medicare cost of AUD$26.1 million (95% CI $20.1-$32.3 million) or €12.1 (95% CI €9.3-€14.9 million) based on population size in 2020. CONCLUSIONS: Childhood food allergy causes considerable Medicare costs for out-of-hospital services in the first four years after birth in Australia. These findings can help anticipate the financial impact on the health care system associated with childhood food allergy, act as a useful costing resource for future evaluations, and inform management of childhood food allergy internationally.

A Systematic Review of International Guidance for Self-Report and Proxy Completion of Child-Specific Utility Instruments.

OBJECTIVES: This study aimed to identify and summarize published guidance and recommendations for child self- and proxy assessment of existing child-specific instruments of health-related quality of life (HRQoL) that are accompanied by utilities. METHODS: A total of 9 databases plus websites of (1) health technology assessment and health economics outcomes research organizations and (2) instrument developers were systematically searched. Studies were included if they reported guidance for child self- and proxy assessment for child populations (0-18 years old). Three reviewers independently screened titles, abstracts, and full-text reviews against the inclusion criteria. Key features of the guidance identified were summarized. RESULTS: A total of 19 studies met the inclusion criteria. In general, journal articles provided little guidance on child self- and proxy assessment, with the majority focused on instrument development and psychometric performance more broadly. Instrument developers' websites provided more guidance for child self- and proxy reports with specific guidance found for the EQ-5D-Y and the Pediatric Quality of Life Inventory. This guidance included the minimum age for self-report and mode of administration; recommended proxy types, age range of child for whom proxy report can be completed, and target population; and recall period. Websites of leading organizations provided general guidance on HRQoL evaluation in children but lacked specific guidance about self- and proxy completion. CONCLUSIONS: EQ-5D-Y and Pediatric Quality of Life Inventory developers' websites provided the most comprehensive guidance for self-report and proxy report of their respective instruments. More evidence is required for developing best practice guidance on why, when, and how to use self- and proxy reports in assessing HRQoL in child populations.

Cost-effectiveness of home-based care of febrile neutropenia in children with cancer.

INTRODUCTION: Home-based treatment of febrile neutropenia (FN) in children with cancer with oral or intravenous antibiotics is safe and effective. There are limited data on the economic impact of this model of care. We evaluated the cost-effectiveness of implementing an FN programme, incorporating home-based intravenous antibiotics for carefully selected patients, in a tertiary paediatric hospital. METHODS: A decision analytic model was constructed to compare costs and outcomes of the home-based FN programme, with usual in-hospital treatment with intravenous antibiotics. The programme included a clinical decision rule to stratify patients by risk for severe infection and home-based eligibility criteria using disease, chemotherapy and patient-level factors. Health outcomes (quality of life) and probabilities of FN risk classification and home-based eligibility were based on prospectively collected data between 2017 and 2019. Patient-level costs were extracted from hospital administrative records. Cost-effectiveness was expressed as the incremental cost per quality-adjusted life year (QALY). FINDINGS: The mean health care cost of home-based FN treatment in low-risk patients was Australian dollars (A$) 7765 per patient compared to A$20,396 for in-hospital treatment (mean difference A$12,632 [95% CI: 12,496-12,767]). Overall, the home-based FN programme was the dominant strategy, being more effective (0.0011 QALY [95% CI: 0.0011-0.0012]) and less costly. Results of the model were most sensitive to proportion of children eligible for home-based care programme. CONCLUSION: Compared to in-hospital FN care, the home-based FN programme is cost-effective, with savings arising from cheaper cost of caring for children at home. These savings could increase as more patients eligible for home-based care are included in the programme.

The relationship between physical and mental health multimorbidity and children's health-related quality of life.

PURPOSE: To examine the relationships between physical health problems, and borderline or clinical levels of mental health symptoms and children's health-related quality of life (HRQoL). METHODS: Data were from the Longitudinal Study of Australian Children (2004-2018). Parents reported on their child's HRQoL (PedsQL), physical health problems and mental health symptoms (Strengths and Difficulties Questionnaire, SDQ). A pooled cross-sectional analysis using linear regressions examined the relationships between physical health and clinical/borderline mental health symptoms, individually and when multi-morbid, and children's HRQoL, and whether these relationships vary by a range of child, family and social factors. RESULTS: The sample comprised 47,567 observations of children aged 4-17 years. Borderline and clinical levels of mental health symptoms were associated with significantly lower HRQoL, equal to more than two-times (10.5 points) and more than three-times (16.8 points) the clinically meaningful difference, respectively. This was a larger difference than that associated with physical health problems (4.4 points). We found a significant interaction effect between physical health problems and clinical mental health symptoms which was associated with even poorer HRQoL after accounting for the individual relationships of both problems. Mental health problems were associated with poorer HRQoL for older versus younger children; and the interaction effect was significant for boys but not girls. CONCLUSION: Findings highlight the importance of identifying and addressing mental health symptoms in children of all ages, even if these problems do not meet formal clinical criteria. Particular attention should be paid to the mental health and HRQoL of children with physical-mental multimorbidity, who are at risk of disproportionately poorer HRQoL.

Association between 24-hour movement behaviors and health-related quality of life in children.

PURPOSE: To assess the associations between adherence to 24-hour movement behaviors guidelines and child general health and functional status measured by health-related quality of life. METHODS: The Longitudinal Study of Australian Children (2004-2016) a nationally representative sample with data available for children aged 2-15 years was used. Physical activity time, recreational screen time, and sleep time were calculated from time use diaries and classified as 'meeting guidelines' or 'not' based on the age-specific 24-h movement guidelines. Child general health and functional status were measured using the multidimensional Pediatric Quality of Life Inventory (PedsQL). Associations between meeting guidelines and PedsQL were assessed using linear mixed effects models. RESULTS: 8919 children were included. Each additional guideline met was associated with a 0.52 (95% confidence interval [CI] 0.39-0.65) increase in PedsQL total score. Compared with meeting no guidelines, the effect of meeting physical activity guidelines alone (ß = 0.93, 95% CI 0.42-1.44) was larger compared to meeting screen (ß = 0.66, 95% CI 0.06-1.27) or sleep time (ß = 0.47, 95% CI 0.04-0.89) guidelines alone. The highest increment was observed in meeting both screen time and physical activity guidelines (ß = 1.89, 95% CI 1.36-2.43). Associations were stronger in children from lower-income families (ß for meeting all versus none = 2.88, 95% CI 1.77-3.99) and children aged 14-15 years (ß = 4.44, 95% CI 2.49-6.40). CONCLUSIONS: The integration of screen time and physical activity guidelines is associated with the highest PedsQL improvement. The association between guidelines adherence and PedsQL appears stronger for adolescents and those from low-income families.

Patient preferences using telehealth during the COVID-19 pandemic in four Victorian tertiary hospital services.

BACKGROUND: The Coronavirus disease 2019 (COVID-19) pandemic has had a major impact on healthcare services with many changes to telehealth care delivery. More information is needed about the patient perspective of telehealth in hospital services and the potential costs and benefits for patients. AIM: To measure patients' evaluation of telehealth, preferences for telehealth versus in-person appointments, and potential cost savings by patient characteristics. METHODS: A cross-sectional online survey (including patient and appointment characteristics, telehealth evaluation, preferences for care and costs) of adult patients using video telehealth in four metropolitan tertiary hospital services in Melbourne, Victoria. RESULTS: A total of 1045 patients (median age 44 years; interquartile range 29-59) participated with an overall response rate of 9.2%. For 98.7% patients, telehealth was convenient, 96.4% stated that it saved time, 95.9% found telehealth acceptable to receive care and 97.0% found that telehealth improved their access to care. Most (62.6%) preferred in-person consultations, although 86.9% agreed that telehealth was equivalent to an in-person consultation. Those in regional and rural areas were less likely to prefer in-person consultations. Patients attending for medical reasons were less likely to prefer in-person consultation compared with patients with surgical reasons. Patient preference to telehealth was independent of level of education, appointment type, self-rated health status and socio economic status. Patients saved an average of A$120.9 (standard deviation A$93.0) per appointment, with greater cost savings for patients from low and middle socio economic areas and regional or rural areas. CONCLUSION: Telehealth video consultations were largely evaluated positively with most patients considering the service to be as good as in-person. Understanding patient preference is critical to consider when implementing telehealth as mainstream across hospital health services.

Trends of paediatric head injury and acute care costs in Australia.

AIM: Paediatric head injuries (PHI) are the most common cause of trauma-related emergency department (ED) presentations. This study sought to report the incidence of PHI in Australia, examine the temporal trends from 2014 to 2018 and estimate the patient and population-level acute care costs. METHODS: Taking a public-sector health-care perspective, we applied direct and indirect hospital costs for PHI-related ED visits and acute admissions. All costs were inflated to 2018 Australian dollars ($). The patient-level analysis was performed with data from 17 841 children <18 years old enrolled in the prospective Australasian Paediatric Head Injury Study. Mechanisms of injury were characterised by the total and average acute care costs. The population-level data of PHI-related ED presentations were obtained from the Independent Hospital Pricing Authority. Age-standardised incidence rates (IR) and incidence rate ratios (IRR) were calculated, and negative binomial regression examined the temporal trend. RESULTS: The age-standardised IR for PHI was 2734 per 100 000 population in 2018, with a significant increase over 5 years (IRR 1.13, 95% confidence interval (CI) 1.12-1.14; P < 0.001) and acute care costs of $154 million. Falls occurred in 70% of the study cohort, with average costs per episode of $666 (95% CI: $627-$706), accounting for 47% of acute care costs. Transportation-related injuries occurred in 4.1% of the study cohort, with average costs per episode of $8555 (95% CI: $6193-$10 917), accounting for 35% of acute care costs. CONCLUSION: PHI have increased significantly in Australia and are associated with substantial acute care costs. Population-based efforts are required for road safety and injury prevention.