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Objectives: This study aimed to (1) gauge patients understanding and expectations of real-world data and evidence (RWDE) research and (2) use this understanding and patients lived experience to co-create resources and a framework for embedding meaningful patient and public involvement and engagement (PPIE) in RWDE research within the pharmaceutical industry setting., Setting and Participants: An academic organisation, a pharmaceutical company and a PPIE panel of 12 patients or carers partnered to form the project team. The PPIE panel was purposively selected to maximise diversity., Design: Participatory and co-design methods were used to engender an understanding of the PPIE perspective on RWDE research and the PPIE role within that. Interactive workshops explored understanding and expectations of RWDE research as well as perceived barriers and facilitators of PPIE within each stage of the RWDE research cycle. Workshops were audio and video recorded, with notes captured. Summaries were analysed thematically and shared back with the PPIE panel for validation and further reflection., Results: We identified a lack of trust and understanding of real-world data, its collection and use and the need to educate the public and researchers. Four themes were identified for meaningful PPIE in RWDE research; equality, diversity and inclusion; feeling valued; ownership and understanding and evaluating impact. We co-created learning resources (video, infographic) and a novel PPIE framework, incorporating potential PPIE activities, resources and support needs for use by researchers conducting RWDE research., Conclusions: To our knowledge, this is the first project to explore the practicalities of PPIE in RWDE research from the perspective of patients and carers. Some findings confirm PPIE experience and guidance derived from other areas, with some specific insights into the pharmaceutical industry. These underpin the PPIE framework to enable robust and meaningful PPIE in RWDE research. This article includes a plain language summary in the supplement., Competing Interests: Competing interests: SAD, RB, ACB, MA and VW are employees of Pfizer and hold Pfizer stock. HM was an employee of Pfizer at the time of writing this manuscript. PW, LC and SM are employees of the University of Newcastle, which received funding from Pfizer in connection with the development of this manuscript., (© Author(s) (or their employer(s)) 2025. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ Group.)

Our international team highlights issues with efficacy reports in several studies on DMG with the new drug ONC201.

The effects of air pollution on health outcomes are well-established. However, little is known about perceptions of air pollution and how it may influence exercise behaviours. The aim of this study was to understand the perceived importance of air pollution during planned exercise, and where relevant, explore how those perceptions may differ between those living in different locations. A questionnaire was disseminated to several running and cycling clubs in the United Kingdom, covering population characteristics to determine urban, rural or coastal residents and exercisers, self-reported asthma, perceptions of air quality during active travel, planned exercise and attitudes towards learning more about the topic. Anonymised responses were gained from 381 adult participants (≥18 years and 60% female), 119 of whom answered questions related to active travel. More than half (54%) of all respondents think about the air quality they are exposed to during exercise and how it may affect their health. More urban than rural respondents (59% vs. 47% and p = 0.03) think about air quality and the impact it may have on their health when exercising. There were insufficient coastal respondents for direct comparison. Most survey respondents (57%) state that they would take the most severe course of action regarding exercise behaviour by avoiding it altogether during periods of heavy road traffic. Individuals with self-reported asthma (n = 60), irrespective of place of residence, are the most likely to be conscious of the potential effects of air pollution on their health and exercise performance compared to counterparts without self-reported asthma., (© 2024 The Author(s). European Journal of Sport Science published by Wiley‐VCH GmbH on behalf of European College of Sport Science.)

Objectives: To explore the acceptability and feasibility of detection of atrial fibrillation (AF) by emergency medical services (EMS) and identify potential barriers and facilitators to implementing a formal pathway to facilitate follow-up in primary care, which could reduce the risk of AF-related stroke., Design: Qualitative study using focus groups and one-to-one interviews guided by a semistructured topic guide., Setting: North East England., Participants: Focus groups with 18 members of the public and one-to-one online interviews with 11 healthcare and service providers (six paramedics and five experts representing cardiology, general practice (GP), public health, research, policy and commissioning)., Results: All participant groups were supportive of a role of EMS in identifying AF as part of routine assessment and formalising the response to AF detection. However, this should not create delays for EMS since rate-controlled AF is non-urgent and alternative community mechanisms exist to manage it. Public participants were concerned about communication of the AF diagnosis and whether this should be 'on scene' or in a subsequent GP appointment. Paramedics reported frequent incidental identification of AF, but it is not always clear 'on scene' that this is a new diagnosis, and there is variation in practice regarding whether (and how) this is communicated to the GP. Paramedics also focused on ensuring the safety of non-conveyed patients and a perceived need for an 'active' reporting process, so that a finding of AF was actioned. Field experts felt that a formal pathway would be useful and favoured a simple intervention without adding to time pressures unnecessarily., Conclusions: There is support for the development of a formal pathway to ensure follow-up for people with AF that is incidentally detected by EMS. This has the potential to improve anticoagulation rates and reduce the risk of stroke., Competing Interests: Competing interests: CW has previously held an unrestricted research grant from Bristol-Myers-Squibb, unrelated to this work. The other coauthors have nothing to declare., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: Asthma is one of the most frequent reasons children visit a general practitioner (GP). The diagnosis of childhood asthma is challenging, and a variety of diagnostic tests for asthma exist. GPs may refer to clinical practice guidelines when deciding which tests, if any, are appropriate, but the quality of these guidelines is unknown., Objectives: To determine (i) the methodological quality and reporting of paediatric guidelines for the diagnosis of childhood asthma in primary care, and (ii) the strength of evidence supporting diagnostic test recommendations., Design: Meta-epidemiological study of English-language guidelines from the United Kingdom and other high-income countries with comparable primary care systems including diagnostic testing recommendations for childhood asthma in primary care. The AGREE-II tool was used to assess the quality and reporting of the guidelines. The quality of the evidence was assessed using GRADE., Results: Eleven guidelines met the eligibility criteria. The methodology and reporting quality varied across the AGREE II domains (median score 4.5 out of 7, range 2-6). The quality of evidence supporting diagnostic recommendations was generally of very low quality. All guidelines recommended the use of spirometry and reversibility testing for children aged ≥5 years, however, the recommended spirometry thresholds for diagnosis differed across guidelines. There were disagreements in testing recommendations for 3 of the 7 included tests., Conclusions: The variable quality of guidelines, lack of good quality evidence, and inconsistent recommendations for diagnostic tests may contribute to poor clinician adherence to guidelines and variation in testing for diagnosing childhood asthma., (© The Author(s) 2023. Published by Oxford University Press.)

Introduction: Central venous access devices (CVADs) are commonly used for the treatment of paediatric cancer patients. Catheter locking is a routine intervention that prevents CVAD-associated adverse events, such as infection, occlusion and thrombosis. While laboratory and clinical data are promising, tetra-EDTA (T-EDTA) has yet to be rigorously evaluated or introduced in cancer care as a catheter lock., Methods and Analysis: This is a protocol for a two-arm, superiority type 1 hybrid effectiveness-implementation randomised controlled trial conducted at seven hospitals across Australia and New Zealand. Randomisation will be in a 3:2 ratio between the saline (heparinised saline and normal saline) and T-EDTA groups, with randomly varied blocks of size 10 or 20 and stratification by (1) healthcare facility; (2) CVAD type and (3) duration of dwell since insertion. Within the saline group, there will be a random allocation between normal and heparin saline. Participants can be re-recruited and randomised on insertion of a new CVAD. Primary outcome for effectiveness will be a composite of CVAD-associated bloodstream infections (CABSI), CVAD-associated thrombosis or CVAD occlusion during CVAD dwell or at removal. Secondary outcomes will include CABSI, CVAD-associated-thrombosis, CVAD failure, incidental asymptomatic CVAD-associated-thrombosis, other adverse events, health-related quality of life, healthcare costs and mortality. To achieve 90% power (alpha=0.05) for the primary outcome, data from 720 recruitments are required. A mixed-methods approach will be employed to explore implementation contexts from the perspective of clinicians and healthcare purchasers., Ethics and Dissemination: Ethics approval has been provided by Children's Health Queensland Hospital and Health Service Human Research Ethics Committee (HREC) (HREC/22/QCHQ/81744) and the University of Queensland HREC (2022/HE000196) with subsequent governance approval at all sites. Informed consent is required from the substitute decision-maker or legal guardian prior to participation. In addition, consent may also be obtained from mature minors, depending on the legislative requirements of the study site. The primary trial and substudies will be written by the investigators and published in peer-reviewed journals. The findings will also be disseminated through local health and clinical trial networks by investigators and presented at conferences., Trial Registration Number: ACTRN12622000499785., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Aim: Uptake of nasal high-flow therapy in infants with bronchiolitis has grown in the last decade with some evidence suggesting a reduction in escalation of care. The effect of the implementation of recent available evidence on clinical practice remains unclear., Methods: In a prospective observational study over 6 months in six metropolitan hospitals in Australia, we investigated the clinical practice of high-flow in infants admitted with bronchiolitis and an oxygen requirement. To assess the choice by clinicians of the initial oxygen therapy (standard oxygen or high-flow) the disease severity was measured by physiological parameters obtained prior to oxygen therapy commencement. Additional secondary outcomes were hospital length of stay and transfers to intensive care., Results: Two hundred thirty-five infants with bronchiolitis were admitted for oxygen therapy over 6 months during the winter season. Infants who received high-flow on admission to hospital displayed significantly higher respiratory rates, higher heart rates and higher early warning tool scores with more severe work of breathing than those commenced on standard oxygen therapy as a first line of oxygen therapy. A significantly longer hospital length of stay of 0.6 days occurred in infants commenced on high-flow. A significantly greater proportion on high-flow (23.3%) were admitted to intensive care compared to infants commenced on SOT (10.4%) despite the severity of disease in both groups being similar., Conclusions: Infants with bronchiolitis presenting with greater disease severity are more likely to receive high-flow therapy. Escalation of care in an intensive care unit occurred more frequently on infants on high-flow., Trial Registration: This trial is registered in the Australian New Zealand Clinical Trial Registry ACTRN12618001206213., (© 2024 The Authors. Journal of Paediatrics and Child Health published by John Wiley & Sons Australia, Ltd on behalf of Paediatrics and Child Health Division (The Royal Australasian College of Physicians).)

Objective: The objective of this systematic literature review (SLR) was to summarize the latest studies evaluating the burden of illness in endogenous Cushing's syndrome (CS), including the impact of CS on overall and domain-specific health-related quality of life (HRQoL) and the economic burden of CS to provide a holistic understanding of disease and treatment burden., Methods: An SLR was conducted in PubMed, MEDLINE and Embase using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) checklist to identify peer-reviewed manuscripts and conference abstracts published in English from 2015 to December 4, 2020., Results: Forty-five publications were eligible for inclusion; data were extracted from 37 primary studies while 8 SLRs were included for reference only. Thirty-one studies reported HRQoL using validated patient reported outcome (PRO) measures in pre- or post-surgery, radiotherapy and pharmacotherapy patients. Overall, this SLR found that patients with CS have worse outcomes relative to healthy populations across specific dimensions, such as depression, despite an improvement in HRQoL post-treatment. These findings reveal that CS symptoms are not fully resolved by the existing care paradigm. Few studies report on the economic burden of CS and currently available data indicate a high direct healthcare system cost burden., Conclusions: Patients with CS experience a significant, complex and multifactorial HRQoL burden. Symptom-specific burden studies are sparse in the literature and the understanding of long-term CS symptomatic burden and economic burden is limited. This review intends to provide an updated reference for clinicians, payers and other stakeholders on the burden of CS as reported in published literature and to encourage further research in this area., (© 2023 The Authors. Endocrinology, Diabetes & Metabolism published by John Wiley & Sons Ltd.)

Paediatric palliative care is pivotal for addressing the complex needs of children with incurable diseases and their families. While home-based care offers a familiar and supportive environment, delivering comprehensive services in this context is challenging. The existing literature on home-based palliative care lacks detailed guidance for its organization and implementation. This qualitative narrative inquiry explores the organization and provision of home-based paediatric palliative care. Data were collected from healthcare practitioners using conversations, storytelling, and reflective journaling. Schwind's Narrative Reflective Process was applied to synthesize the data, resulting in an in-depth case description. The narrative approach illuminates the complexities of home-based paediatric palliative, end-of-life, and after-death care. Key findings encompass the importance of early-care coordination, interprofessional collaboration, effective symptom management, emotional and psychosocial support, and comprehensive end-of-life planning. Through the case study of the child patient, the challenges and strategies for providing holistic, family-centred care within the home environment are described. Practical insights gained from this report can inform the development and improvement of home-based palliative care programs, benefiting researchers, practitioners, and policymakers seeking to optimize care for children and families in similar contexts.

Objective: The objective of this scoping review is to map, from wound assessment tools and other literature, the current methods used to assess wound odour in order to answer the following question: Which methods of assessment, validated or otherwise, are currently used in wound assessment tools to assess wound odour? Introduction: Wound assessment includes not only details of the condition of the wound bed but also evaluation of symptoms associated with the wound including that of odour. Odour is cited by clinicians, patients and carers as one of the most distressing wound symptoms. However, there is no consensus on a preferred method to assess odour thus negatively impacting the internal and external validity of many clinical trials and minimising the ability to perform meta-analysis. Eligibility criteria: Any wound assessment tool or framework that includes assessment of wound odour in any wound aetiology and in any care setting. Any systematic or scoping review that includes assessment of wound odour in any wound aetiology and in any care setting. No limits on date of publication or language will be applied. Methods: We will employ the Preferred Reporting Items for Systematic Review and Meta-Analyses extension for scoping reviews (PRISMA-ScR) guidelines for this scoping review and base its structure on the framework proposed by Arksey and O'Malley. Results: A narrative format will summarise extracted data and provide an overview of tools used to assess wound odour. A PRISMA diagram will outline the results of the search strategy. The identified tools will be summarised in table format and stratified according to methods used. Conclusion: The result of this scoping review will be a list of methods used to assess odour in wounds and will be used to inform a subsequent Delphi study to gain consensus on the preferred method to assess wound odour., Competing Interests: Competing interests: BMcG has received consultancy/advisory board honoraria from Novartis and UCB. No more competing interests are declared., (Copyright: © 2023 Gethin G et al.)

This report provides guidance for the implementation of online treatment monitoring (OTM) solutions in radiotherapy (RT), with a focus on modulated treatments. Support is provided covering the implementation process, from identification of an OTM solution to local implementation strategy. Guidance has been developed by a RT special interest group (RTSIG) working party (WP) on behalf of the Institute of Physics and Engineering in Medicine (IPEM). Recommendations within the report are derived from the experience of the WP members (in consultation with manufacturers, vendors and user groups), existing guidance or legislation and a UK survey conducted in 2020 (Stevens et al 2021). OTM is an inclusive term representing any system capable of providing a direct or inferred measurement of the delivered dose to a RT patient. Information on each type of OTM is provided but, commensurate with UK demand, guidance is largely influenced by in vivo dosimetry methods utilising the electronic portal imager device (EPID). Sections are included on the choice of OTM solutions, acceptance and commissioning methods with recommendations on routine quality control, analytical methods and tolerance setting, clinical introduction and staffing/resource requirements. The guidance aims to give a practical solution to sensitivity and specificity testing. Functionality is provided for the user to introduce known errors into treatment plans for local testing. Receiver operating characteristic analysis is discussed as a tool to performance assess OTM systems. OTM solutions can help verify the correct delivery of radiotherapy treatment. Furthermore, modern systems are increasingly capable of providing clinical decision-making information which can impact the course of a patient's treatment. However, technical limitations persist. It is not within the scope of this guidance to critique each available solution, but the user is encouraged to carefully consider workflow and engage with manufacturers in resolving compatibility issues., (© 2023 Institute of Physics and Engineering in Medicine.)

Aim: To determine the quality of paediatric guidelines relevant to diagnosis of three of the most common conditions in primary care: fever, gastroenteritis and constipation., Methods: We undertook a meta-epidemiological study of paediatric guidelines for fever, gastroenteritis and gastroenteritis. We systematically searched MEDLINE, Embase, Trip Database, Guidelines International Network, the National Guideline Clearinghouse and WHO from February 2011 to September 2022 for guidelines from high-income settings containing diagnostic recommendations. We assessed the quality of guideline reporting for included guidelines using the AGREE II tool., Results: We included 16 guidelines: fever (n = 7); constipation (n = 4) and gastroenteritis (n = 5). The overall quality across the three conditions was graded moderate (median AGREE II score 4.5/7, range 2.5-6.5) with constipation guidelines rated the highest (median 6/7), and fever rated the lowest (median 3.8/7). Major methodological weaknesses included consideration of guideline applicability. Half of the guidelines did not report involving parent representatives, and 56% did not adequately declare or address their competing interests., Conclusions: Substantial variations exist in the quality of paediatric guidelines related to the diagnosis of primary care presentations. Better quality guidance is needed for general practitioners to improve diagnosis for children in primary care., (© 2023 The Authors. Journal of Paediatrics and Child Health published by John Wiley & Sons Australia, Ltd on behalf of Paediatrics and Child Health Division (The Royal Australasian College of Physicians).)

Introduction: Endogenous Cushing's syndrome (CS) is a rare endocrine condition caused by chronic oversecretion of cortisol, resulting in a diverse constellation of symptoms. This study examined the ongoing burden of illness (BOI), from the first appearance of symptoms through treatment, which is currently not well evaluated., Methods: A quantitative, cross-sectional, web-enabled survey including 5 validated patient reported outcomes (PRO) measures was conducted in patients with CS who had been diagnosed ≥ 6 months prior and who had received ≥ 1 treatment for their endogenous CS at the time of the survey., Results: Fifty-five patients participated in this study; 85% were women. The mean age was 43.4 ± 12.3 years (± standard deviation, SD). On average, respondents reported a 10-year gap between the first occurrence of symptoms and diagnosis; 80% underwent surgical treatment for CS. Respondents experienced symptoms on 16 days in a typical month, and their health-related quality of life was moderately impacted based on the CushingQoL score. Weight gain, muscle fatigue, and weakness were the most common symptoms and 69% percent of patients reported moderate or severe fatigue using the Brief Fatigue Inventory. Following treatment, the occurrence of most symptoms declined over time, although anxiety and pain did not significantly decrease. Overall, 38% of participants reported an annual average of 25 missed workdays due to CS symptoms., Conclusions: These results demonstrate a BOI in CS despite ongoing treatment and illustrate the need for interventions to address persistent symptoms, particularly weight gain, pain, and anxiety., (© 2023. The Author(s).)

In newborn screening, false-negative results can be disastrous, leading to disability and death, while false-positive results contribute to parental anxiety and unnecessary follow-ups. Cutoffs are set conservatively to prevent missed cases for Pompe and MPS I, resulting in increased falsepositive results and lower positive predictive values. Harmonization has been proposed as a way to minimize false-negative and false-positive results and correct for method differences, so we harmonized enzyme activities for Pompe and MPS I across laboratories and testing methods (Tandem Mass Spectrometry (MS/MS) or Digital Microfluidics (DMF)). Participating states analyzed proofof- concept calibrators, blanks, and contrived specimens and reported enzyme activities, cutoffs, and other testing parameters to Tennessee. Regression and multiples of the median were used to harmonize the data. We observed varied cutoffs and results. Six of seven MS/MS labs reported enzyme activities for one specimen for MPS I marginally above their respective cutoffs with results classified as negative, whereas all DMF labs reported this specimen's enzyme activity below their respective cutoffs with results classified as positive. Reasonable agreement in enzyme activities and cutoffs was achieved with harmonization; however, harmonization does not change how a value would be reported as this is dependent on the placement of cutoffs.

Objective: The chronic complications of ageing with HIV are not well studied in sub-Saharan Africa (SSA) where general healthcare resources are limited. We aimed to collaborate with individuals living with HIV aged ≥ 50 years, and community elders (aged ≥ 60 years) living with non-communicable diseases in the Kilimanjaro region of Tanzania in a health research priority-setting exercise., Methods: We conducted structured workshops based on broad questions to aid discussion and group-based patient priority setting, alongside discussion of the feasibility of future community research engagement. Participant priorities were tallied and ranked to arrive at core priorities from consensus discussion., Results: Thirty older people living with HIV and 30 community elders attended separate priority setting workshops. Both groups reported motivation to participate in, conduct, and oversee future studies. In this resource-limited setting, basic needs such as healthcare access were prioritised much higher than specific HIV-complications or chronic disease. Stigma and social isolation were highly prioritised in those living with HIV., Conclusions: Community engagement and involvement in HIV and ageing research appears feasible in Tanzania. Ageing and non-communicable disease research should consider the wider context, and lack of basic needs in low-income settings. A greater impact may be achieved with community involvement., (© 2023. The Author(s).)

Importance: Nasal high-flow oxygen therapy in infants with bronchiolitis and hypoxia has been shown to reduce the requirement to escalate care. The efficacy of high-flow oxygen therapy in children aged 1 to 4 years with acute hypoxemic respiratory failure without bronchiolitis is unknown., Objective: To determine the effect of early high-flow oxygen therapy vs standard oxygen therapy in children with acute hypoxemic respiratory failure., Design, Setting, and Participants: A multicenter, randomized clinical trial was conducted at 14 metropolitan and tertiary hospitals in Australia and New Zealand, including 1567 children aged 1 to 4 years (randomized between December 18, 2017, and March 18, 2020) requiring hospital admission for acute hypoxemic respiratory failure. The last participant follow-up was completed on March 22, 2020., Interventions: Enrolled children were randomly allocated 1:1 to high-flow oxygen therapy (n = 753) or standard oxygen therapy (n = 764). The type of oxygen therapy could not be masked, but the investigators remained blinded until the outcome data were locked., Main Outcomes and Measures: The primary outcome was length of hospital stay with the hypothesis that high-flow oxygen therapy reduces length of stay. There were 9 secondary outcomes, including length of oxygen therapy and admission to the intensive care unit. Children were analyzed according to their randomization group., Results: Of the 1567 children who were randomized, 1517 (97%) were included in the primary analysis (median age, 1.9 years [IQR, 1.4-3.0 years]; 732 [46.7%] were female) and all children completed the trial. The length of hospital stay was significantly longer in the high-flow oxygen group with a median of 1.77 days (IQR, 1.03-2.80 days) vs 1.50 days (IQR, 0.85-2.44 days) in the standard oxygen group (adjusted hazard ratio, 0.83 [95% CI, 0.75-0.92]; P < .001). Of the 9 prespecified secondary outcomes, 4 showed no significant difference. The median length of oxygen therapy was 1.07 days (IQR, 0.50-2.06 days) in the high-flow oxygen group vs 0.75 days (IQR, 0.35-1.61 days) in the standard oxygen therapy group (adjusted hazard ratio, 0.78 [95% CI, 0.70-0.86]). In the high-flow oxygen group, there were 94 admissions (12.5%) to the intensive care unit compared with 53 admissions (6.9%) in the standard oxygen group (adjusted odds ratio, 1.93 [95% CI, 1.35-2.75]). There was only 1 death and it occurred in the high-flow oxygen group., Conclusions and Relevance: Nasal high-flow oxygen used as the initial primary therapy in children aged 1 to 4 years with acute hypoxemic respiratory failure did not significantly reduce the length of hospital stay compared with standard oxygen therapy., Trial Registration: anzctr.org.au Identifier: ACTRN12618000210279.

Background: Newborn screening (NBS) by means of T cell receptor excision circles (TREC) is now universal in the United States, Puerto Rico, and the Navajo Nation as a strategy to identify severe combined immunodeficiency (SCID) in newborns. Owing to the characteristics of adenosine deaminase (ADA) deficiency, a small but important number of cases can be missed by this screening., Objective: To evaluate the results of the first year statewide NBS for ADA by means of dried blood spot NBS., Methods: On October 7, 2019, the state of Michigan began screening newborn dried blood spots for ADA deficiency by means of the Neobase-2 tandem mass spectroscopy (TMS) kit. We report 1 known case of ADA deficiency in the 18 months before screening. We then reviewed the results of the first 2 years of TMS ADA screening in Michigan., Results: There was 1 patient with ADA deficiency known to our centers in the 18 months before initiation of TMS ADA screening; this patient died of complications of their disease. In the first 2 years of TMS ADA NBS, 206,321 infants were screened, and 2 patients had positive ADA screen results. Both patients had ADA deficiency confirmed through biochemical and genetic testing. One patient identified also had a positive TREC screen and was confirmed to have ADA-SCID., Conclusion: In our first 2 years, TMS NBS for ADA deficiency identified 2 patients with ADA deficiency at negligible cost, including 1 patient who would not have been identified by TREC NBS. This report provides initial evidence of the value of specific NBS for ADA deficiency., (Copyright © 2022 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.)

Objective: It is theorised that adhesive-free wound care developed specifically for patients with hidradenitis suppurativa (HS) can improve their quality of life (QoL). Our study aimed to investigate the impact of a novel wound care device on Dermatology Life Quality Index (DLQI) scores, and other factors related to experienced pain, time spent changing dressings, comfort, ease of use and body image., Method: A 21-day, single-arm, unblinded, pilot trial was conducted to assess ease of use and the impact of effective wound care on various aspects of wound management in patients with HS. Participants were provided two trial garments and trial dressings as required, to use over a 21-day period in the home setting. A seven-item questionnaire and the DLQI questionnaire was completed on days 0, 7, 14 and 21., Results: All 15 participants were female, aged >18 years old and with a diagnosis of HS. Mean DLQI score at baseline (day 0) was 19.3, which was reduced to 4.53 on day 21, a significant improvement in 100% of participants (p<0.001). High levels of dressing-related pain, assessed using an 11-point Visual Analogue Scale, reduced from 5.53 at baseline to 0.8 on day 21. Other significant improvements in terms of patient comfort, time spent on changing dressings, body confidence and the dressing's ability to retain exudate were also noted., Conclusion: The results illustrated the improvement made to study participants' day-to-day activities and QoL when effective HS-specific wound care products were provided. Wound care is an essential component in the treatment journey of patients.

Advancements in technology and communication have revolutionised the twenty-first century with the introduction of mobile phones and smartphones. These phones are known to be platforms harbouring microbes with recent research shedding light on the abundance and broad spectrum of organisms they harbour. Mobile phone use in the community and in professional sectors including health care settings is a potential source of microbial dissemination. To identify the diversity of microbial genetic signature present on mobile phones owned by hospital medical staff. Twenty-six mobile phones of health care staff were swabbed. DNA extraction for downstream next generation sequencing shotgun metagenomic microbial profiling was performed. Survey questionnaires were handed to the staff to collect information on mobile phone usage and users' behaviours. Each of the 26 mobile phones of this study was contaminated with microbes with the detection of antibiotic resistance and virulent factors. Taken together the sum of microbes and genes added together across all 26 mobile phones totalised 11,163 organisms (5714 bacteria, 675 fungi, 93 protists, 228 viruses, 4453 bacteriophages) and 2096 genes coding for antibiotic resistance and virulent factors. The survey of medical staff showed that 46% (12/26) of the participants used their mobile phones in the bathroom. Mobile phones are vectors of microbes and can contribute to microbial dissemination and nosocomial diseases worldwide. As fomites, mobile phones that are not decontaminated may pose serious risks for public health and biosecurity., (© 2022. The Author(s).)

Objective: To synthesize existing qualitative research exploring the experiences of parents caring for children with cancer during the end-of-life phase, and the factors that influence parental decision-making when choosing the location of end-of-life care and death for their child., Results: This review included 15 studies of 460 parents of 333 children and adolescents who died from progressive cancer. Where reported, the majority (58%) of children died at home or in a hospital (39%), with only a small fraction dying in a hospice. Factors impacting decision-making for the location of care included the quality of communication and the quality of care available. Themes related to choosing home for end-of-life care and death included honoring the child's wishes, the familiarity of home, and parents' desire to be their child's primary carer. Preference for the location of death in the hospital included trust in hospital staff, practical logistics, and the safety of the hospital environment., (© 2022 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)

Aim: The Streptococcus anginosus group (SAG) comprises three bacterial species colonising the mouth and gastrointestinal and genitourinary tracts and capable of serious pyogenic infections. Although well-described in adults, studies in children are limited. Here, we characterise paediatric SAG infections from a single Australian centre., Methods: Hospitalised patients aged ≤18 years with positive SAG cultures from January 2009 to December 2019 were identified from Pathology Queensland's Gold Coast Laboratory database and their medical records were reviewed., Results: Two-hundred children (62% male), median age 12 years (interquartile range 6-16), with positive SAG cultures were identified. Overall, 90% received intravenous antibiotics, 89% underwent surgical drainage, 23% were readmitted and 15% required additional surgery. The most common sites were the abdomen (39%), soft tissues (36%) and head and neck regions (21%). Since 2011, Pathology Queensland reported SAG at the species level (n = 133). Of these, S. anginosus was the most prevalent (39%), then S. constellatus (34%) and S. intermedius (27%). Compared with the other two species, S. intermedius was most commonly associated with head and neck infections (relative risk (RR) = 2.2, 95% confidence interval (CI) 1.4-3.5), while S. constellatus (RR = 1.7, 95% CI 1.2-2.4) and S. anginosus (RR = 1.5, 95% CI 1.0-2.0) were each associated with a higher risk of intra-abdominal infection than S. intermedius. Since February 2015, the number of children admitted with SAG-associated intra-abdominal infection per 1000 hospitalisations increased by 29% annually compared with an annual decline of 8% in previous years., Conclusions: SAG infections occur at various anatomical sites. Despite antibiotics and surgical management, almost one-quarter are re-hospitalised for further treatment., (© 2021 Paediatrics and Child Health Division (The Royal Australasian College of Physicians).)

Importance: There is a paucity of high-quality evidence on the effect of care coordination on health-related quality of life among children with chronic noncomplex medical conditions (non-CMCs)., Objective: To examine whether care coordination delivered by an Allied Health Liaison Officer results in improved quality-of-life (QOL) outcomes for children with chronic non-CMCs and their families., Design, Setting and Participants: This multicenter, open label, randomized clinical trial was conducted in pediatric outpatient clinics at 3 Australian hospitals with tertiary- and secondary-level pediatric care facilities. A total of 81 children with chronic non-CMCs and their families participated in the trial for a period of up to 12 months between October 2017 to October 2020. Primary care reviews were offered at 1 week, 3 months, and 6 months after diagnosis., Interventions: Eligible children were randomized 1:1 to receive care coordination or standard care. Families of children receiving care coordination were provided access to an Allied Health Liaison Officer, who was responsible for facilitation of health care access across hospital, education, primary care, and community sectors., Main Outcomes and Measures: The primary outcomes were scores on the Pediatric Quality of Life Inventory (PedsQL), version 4.0, and the PedsQL Family Impact Module, version 2.0, measured at 6 and 12 months. An intent-to-treat approach was used to analyze the data., Results: Of 81 children (mean [SD] age, 8.2 [3.5] years; 55 [67.9%] male), 42 (51.9%) were randomized to care coordination and 39 (48.1%) to standard care. Compared with standard care, care coordination resulted in greater improvements in overall PedsQL scores (difference in score changes between groups, 7.10; 95% CI, 0.44-13.76; P = .04), overall PedsQL Family Impact Module scores (difference in score changes between groups, 8.62; 95% CI, 1.07-16.16; P = .03), and family functioning QOL (difference in score changes between groups, 15.83; 95% CI, 5.05-26.62; P = .004) at 12 months after diagnosis., Conclusions and Relevance: In this randomized clinical trial, care coordination improved the quality of life of children with chronic non-CMCs and their families. Further studies should explore specific non-CMCs that may benefit most from care coordination and whether an orientation among health services to provide such a coordination model could lead to longer-term improved clinical outcomes., Trial Registration: http://anzctr.org.au Identifier: ACTRN12617001188325.

Hidradenitis suppurativa (HS) is an under-reported, poorly understood and often mismanaged inflammatory skin condition that manifests itself as painful, inflamed areas or boils. Although blind at first, it develops into exuding lesions often with tracking sinuses, leaving painfully stretched and scarred skin. While advances have been made in surgical and pharmaceutical interventions to interrupt the disease and prevent infection, effective wound management, evidence and guidelines remain limited. This article describes the experiences of an HS sufferer, in her quest for a satisfactory and acceptable wound management solution. It is hoped that the author's criteria for the ideal wound management system for HS will be the starting point for discussion and development of sound HS wound care guidelines.

Fluoroquinolones are reported to have peripheral nerve toxic effects that can be severe and permanent in adults. However, limited data exist for children. We describe a case of acute-onset peripheral neuropathy associated with ciprofloxacin in a 13-year-old boy who was characterized by severe dysesthesia and erythema of both hands and feet, but which resolved promptly after ceasing the antibiotic., Competing Interests: The authors have no funding or conflicts of interest to disclose., (Copyright © 2021 Wolters Kluwer Health, Inc. All rights reserved.)

Background: Recurring nodules, abscesses, and lesions characterise hidradenitis suppurativa (HS): a chronic, inflammatory skin disorder. Globally the prevalence of HS is estimated to be around 1% of the population. Leakage, pain, and odour from HS wounds require substantial management. Little is known of the personal burdens that routine wound management imposes on the patient., Objectives: To evaluate how routine HS wound management impacts patients in terms of the time spent changing dressings, the number of dressings required per day, pain experienced during dressing changes, and negative impact on various domains of their personal lives., Methods: An anonymous online questionnaire was posted on closed social media patient support groups between April and May 2019. Pearson χ2 test was used to evaluate if Hurley stages influenced the personal impact of wound care routines on patients. Statistical significance was determined as p value <0.05., Results: In total, 908 people from 28 countries responded. Of these, 81% (n = 734) reported that regular dressing changes negatively impacted on their quality of life. Most patients, 82% (n = 744), experience pain during dressing changes. 16% (n = 142) of patients required five or more dressings daily, and 12% (n = 108) spend over 30 min daily tending to wounds. Patients indicated high levels of dissatisfaction with currently available wound dressings., Conclusion: HS wound management imposes a substantial personal burden on patients. There is a clear unmet need for HS-specific wound dressings and wound care provisions, and a greater awareness of the condition and its impact is needed among clinicians., (© 2021 S. Karger AG, Basel.)

Objective: To evaluate population-level implementation of Confident Body, Confident Child (CBCC); an evidence-based program providing parenting strategies to promote healthy eating, physical activity and body satisfaction in children aged 2-6 years; with community child health nurses (CHNs)., Methods: This study utilised an implementation-effectiveness hybrid design, with dual focus on assessing: (a) CBCC implementation into Child Health Centres at a regional health service in Queensland, Australia (process evaluation); and (b) CBCC's effect on CHNs' knowledge and attitudes (outcomes evaluation). Process (CBCC reach, dose, fidelity) and outcome data (CHN knowledge of child body image; and attitudes towards higher body weights) were collected during implementation, and pre- and post-intervention delivery to CHNs, respectively., Results: Twenty-six CHNs (all female; mean age 52.7 ± 9.5 years) participated in the study by attending a 1-day CBCC training workshop and completing demographic and outcome surveys. Process evaluation found that CBCC was implemented as planned and reached 56% of CHNs across the health service. Outcome evaluation showed small but non-significant improvements in CHN knowledge (P = .077) and attitudes towards overweight (using Anti-Fat Attitudes scale; significant improvements on willpower sub-scale only (P < .05))., Discussion: This is the first study to evaluate population-wide CBCC implementation in a real-world health service setting with CHNs. Findings highlight the potential for using pragmatic, implementation-focused methodologies to translate preventive eating disorder programs into community child health services., (© 2021 Australian Health Promotion Association.)

Numerous commercial technologies for online treatment monitoring (OTM) in radiotherapy (RT) are currently available including electronic portal imaging device (EPID) in vivo dosimetry (IVD), transmission detectors and log files analysis. Despite this, in the UK there exists limited guidance on how to implement and commission a system for clinical use or information about the resources required to set up and maintain a service. A Radiotherapy Special Interest Group working party, established by Institute of Physics and Engineering in Medicine was formed with a view to reassess the current practice for OTM in the UK and an aim to develop consensus guidelines for the implementation of a system. A survey distributed to Heads of Medical Physics at 71 UK RT departments investigated: availability of OTM in the UK; estimates of workload; clinical implementation; methods of analysis; quality assurance; and opinions on future directions. The survey achieved a 76% response rate and demonstrated that OTM is widely supported in the UK, with 87% of respondents indicating all patients should undergo OTM. EPID IVD (EIVD) was the most popular form of OTM. An active EIVD service was reported by 37% of respondents, with 84% believing it was the optimal solution. This demonstrates a steady increase in adoption since 2012. Other forms of OTM were in use but they had only been adopted by a minority of centres. Financial barriers and the increase of staff workload continue to hinder wider implementation in other centres. Device automation and integration is a key factor for successful future adoption and requires support between treatment machine and OTM manufacturers. The survey has provided an updated analysis on the use of OTM methods across the UK. Future guidance is recommended on commissioning, adoption of local tolerances and root-cause analysis strategies to assist departments intending to implement OTM., (© 2021 Institute of Physics and Engineering in Medicine.)

Introduction: Mobile phones are used the world over, including in healthcare settings. This study aimed to investigate the viable microbial colonisation of mobile phones used by healthcare personnel., Methods: Swabs collected on the same day from 30 mobile phones belonging to healthcare workers from three separate paediatric wards of an Australian hospital were cultured on five types of agar plate, then colonies from each phone were pooled, extracted and sequenced by shotgun metagenomics. Questionnaires completed by staff whose phones were sampled assisted in the analysis and interpretation of results., Results and Discussion: All phones sampled cultured viable bacteria. Overall, 399 bacterial operational taxonomic units were identified from 30 phones, with 1432 cumulative hits. Among these were 58 recognised human pathogenic and commensal bacteria (37 Gram-negative, 21 Gram-positive). The total number of virulence factor genes detected was 347, with 1258 cumulative hits. Antibiotic resistance genes (ARGs) were detected on all sampled phones and overall, 133 ARGs were detected with 520 cumulative hits. The most important classes of ARGs detected encoded resistance to beta-lactam, aminoglycoside and macrolide antibiotics and efflux pump mediated resistance mechanisms., Conclusion: Mobile phones carry viable bacterial pathogens and may act as fomites by contaminating the hands of their users and indirectly providing a transmission pathway for hospital-acquired infections and dissemination of antibiotic resistance. Further research is needed, but meanwhile adding touching mobile phones to the five moments of hand hygiene is a simple infection control strategy worth considering in hospital and community settings. Additionally, the implementation of practical and effective guidelines to decontaminate mobile phone devices would likely be beneficial to the hospital population and community at large., (Copyright © 2021 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Background: The number of people living with dementia worldwide is increasing, particularly in low- and middle-income countries (LMICs) where little is known about existing post-diagnostic care and support. This study aimed to better understand healthcare provision for people living with dementia in Malaysia, and to identify priorities for providing timely, quality, and accessible care and support to all. Methods: This is a qualitative interview study on care providers and facilitators (health and community care professionals, paid carers, traditional medicine practitioners, faith healers, community leaders, non-governmental organisations). A topic guide, piloted in Malaysia and peer reviewed by all LMIC partners, elicited the understanding of dementia and dementia care and barriers and facilitators to care for people living with dementia and carers, and perceptions of key priorities for developing efficient, feasible, and sustainable dementia care pathways. Verbatim transcription of audio-recorded interviews was followed by iterative, thematic data analysis. Results: Twenty interviews were conducted (11 healthcare professionals, 4 traditional medicine practitioners, and 5 social support providers). The findings indicate that dementia care and support services exist in Malaysia, but that they are not fully utilised because of variations in infrastructure and facilities across the country. Despite a locally recognised pathway of care being available in an urban area, people with dementia still present to the healthcare system with advanced disease. The interviewees linked this to a public perception that symptoms of dementia, in particular, are normal sequelae of ageing. Earlier detection of dementia is commonly opportunistic when patients present to GPs, government clinic staff, and general physicians with other ailments. Dementia may only be identified by practitioners who have some specialist interest or expertise in it. Workforce factors that hindered early identification and management of dementia included lack of specialists, overburdened clinics, and limited knowledge of dementia and training in guideline use. Post-diagnostic social care was reported to be largely the domain of families, but additional community-based support was reported to be available in some areas. Raising awareness for both the public and medical professionals, prevention, and more support from the government are seen as key priorities to improve dementia management. Conclusions: This qualitative study provides novel insight into the availability, delivery, and use of post-diagnostic care and support in Malaysia from the perspective of care providers. The respondents in this study perceived that while there was a provision for dementia care in the hospital and community settings, the different care sectors are largely unaware of the services each provides. Future work should explore how care provision across different service sectors and providers can be supported to better facilitate patient access and referral between primary, secondary, and social care. The importance of supporting families to understand dementia and its progression, and strategies to help them care for relatives was emphasised. There is also a need for broad workforce training and development, at both the postgraduate and undergraduate levels, as well as improved general awareness in the community to encourage earlier help-seeking for symptoms of dementia. This will enable the use of preventive strategies and access to specialist services to optimise care and quality of life for people living with dementia in Malaysia., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2021 Goodson, McLellan, Rosli, Tan, Kamaruzzaman, Robinson and Moloney.)

Introduction: Patients presenting with undifferentiated illnesses provide valuable learning opportunities for medical students. Evidence detailing the factors that affect patient participation in undergraduate medical education is limited. This study examines how patients regard their participation in teaching consultations in primary care., Methods: We conducted a cross-sectional questionnaire survey in four GP practices. We tested the relationship between variables of interest and willingness to participate, using hierarchical logistic regression., Results: We analysed 525 questionnaires. 88% of respondents were willing to have students take part in their consultation, and 72% were willing to see a student alone before seeing the doctor. Older patients and those with less sensitive clinical problems were more likely to participate. Willingness to participate was also associated with patients' perceptions of certain costs and benefits of participation. Respondents had poor knowledge about medical education, and a sizeable minority perceived a lack of autonomy about the presence of students in their encounters. More than one-third of respondents expressed the presence of a clinician as a precondition for approval of students' performing some active roles., Discussion: The findings have identified potential interventions to enhance patient involvement including patient education, respecting patient autonomy, and ensuring appropriate student supervision.

Combining a historical institutionalism approach with institutional isomorphism and punctuated equilibrium, this article analyzes quarantine policy change across 120 years of Australian quarantine history. By anchoring its analysis within specific time periods (the years before the Spanish flu, seven decades of inaction, and multiple post-1997 pandemic updates and responses), the authors highlight when and why policies did or did not change and how the constant push-and-pull between state and Commonwealth institutional ownership altered policy possibilities. The heart of the analysis showcases how Australia's successful COVID-19 response is a unique output of prior quarantine policies, institutional evolution, and mid-pandemic alterations of key national pandemic response plans., (© 2020 by The American Society for Public Administration.)

Objective: To determine the extent to which care received by Australian children presenting with croup is in agreement with Clinical Practice Guidelines (CPGs)., Design: Retrospective population-based sample survey. Croup clinical indicators were derived from CPGs., Data Sources/study Setting: Medical records from three healthcare settings were sampled for selected visits in 2012 and 2013 in three Australian states., Data Collection: Data were collected by nine experienced paediatric nurses, trained to assess eligibility for indicator assessment and adherence to CPGs. Surveyors undertook criterion-based medical record reviews using an electronic data collection tool., Results: Documented guideline adherence was lower for general practitioners (65.9%; 95% CI: 60.8-70.6) than emergency departments (91.1%; 95% CI: 89.5-92.5) and inpatient admissions (91.3%; 95% CI: 88.1-93.9). Overall adherence was very low for a bundle of 10 indicators related to assessment (4.5%; 95% CI: 2.4-7.6) but higher for a bundle of four indicators relating to the avoidance of inappropriate therapy (83.1%; 95% CI: 59.5-96.0)., Conclusions: Most visits for croup were characterized by appropriate treatment in all healthcare settings. However, most children had limited documented clinical assessments, and some had unnecessary tests or inappropriate therapy, which has potential quality and cost implications. Universal CPG and clinical assessment tools may increase clinical consistency., (© The Author(s) 2019. Published by Oxford University Press in association with the International Society for Quality in Health Care. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Introduction: Acute hypoxaemic respiratory failure (AHRF) in children is the most frequent reason for non-elective hospital admission. During the initial phase, AHRF is a clinical syndrome defined for the purpose of this study by an oxygen requirement and caused by pneumonia, lower respiratory tract infections, asthma or bronchiolitis. Up to 20% of these children with AHRF can rapidly deteriorate requiring non-invasive or invasive ventilation. Nasal high-flow (NHF) therapy has been used by clinicians for oxygen therapy outside intensive care settings to prevent escalation of care. A recent randomised trial in infants with bronchiolitis has shown that NHF therapy reduces the need to escalate therapy. No similar data is available in the older children presenting with AHRF. In this study we aim to investigate in children aged 1 to 4 years presenting with AHRF if early NHF therapy compared with standard-oxygen therapy reduces hospital length of stay and if this is cost-effective compared with standard treatment., Methods and Analysis: The study design is an open-labelled randomised multicentre trial comparing early NHF and standard-oxygen therapy and will be stratified by sites and into obstructive and non-obstructive groups. Children aged 1 to 4 years (n=1512) presenting with AHRF to one of the participating emergency departments will be randomly allocated to NHF or standard-oxygen therapy once the eligibility criteria have been met (oxygen requirement with transcutaneous saturation <92%/90% (dependant on hospital standard threshold), diagnosis of AHRF, admission to hospital and tachypnoea ≥35 breaths/min). Children in the standard-oxygen group can receive rescue NHF therapy if escalation is required. The primary outcome is hospital length of stay. Secondary outcomes will include length of oxygen therapy, proportion of intensive care admissions, healthcare resource utilisation and associated costs. Analyses will be conducted on an intention-to-treat basis., Ethics and Dissemination: Ethics approval has been obtained in Australia (HREC/15/QRCH/159) and New Zealand (HDEC 17/NTA/135). The trial commenced recruitment in December 2017. The study findings will be submitted for publication in a peer-reviewed journal and presented at relevant conferences. Authorship of all publications will be decided by mutual consensus of the research team., Trial Registration Number: ACTRN12618000210279., Competing Interests: Competing interests: DF, SG, AS and SD received travel support from Fisher and Paykel Healthcare. All other authors have no conflicts to disclose. Fisher and Paykel have provided equipment and consumables for the study but have had no input in the study design., (© Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: Bronchiolitis is the most common reason for hospital admission in infants, with one third requiring oxygen therapy due to hypoxemia. It is unknown what proportion of hypoxemic infants with bronchiolitis can be managed with nasal high-flow in room air and their resulting outcomes. Objectives and Settings: To assess the effect of nasal high-flow in room air in a subgroup of infants with bronchiolitis allocated to high-flow therapy in a recent multicenter randomized controlled trial. Patients and Interventions: Infants allocated to the high-flow arm of the trial were initially treated with room air high-flow if saturations were ≥85%. Subsequently, if oxygen saturations did not increase to ≥92%, oxygen was added and FiO 2 was titrated to increase the oxygen saturations. In this planned sub-study, infants treated during their entire hospital stay with high-flow room air only were compared to infants receiving either standard-oxygen or high-flow with oxygen. Baseline characteristics, hospital length of stay and length of oxygen therapy were compared. Findings: In the per protocol analysis 64 (10%) of 630 infants commenced on high-flow room air remained in room air only during the entire stay in hospital. These infants on high-flow room air were on average older and presented with moderate hypoxemia at presentation to hospital. Their length of respiratory support and length of stay was also significantly shorter. No pre-enrolment factors could be identified in a multivariable analysis. Conclusions: In a small sub-group of hypoxemic infants with bronchiolitis hypoxemia can be reversed with the application of high-flow in room air only. Trial registration: ACTRN12615001305516., (Copyright © 2019 Franklin, Babl, Gibbons, Pham, Hasan, Schlapbach, Oakley, Craig, Furyk, Neutze, Moloney, Gavranich, Shirkhedkar, Kapoor, Grew, Fraser, Dalziel, Schibler and PARIS and PREDICT.)

Background: Children with chronic health conditions have better health-related outcomes when their care is managed in a personalised and coordinated way. However, increased demand on Australian ambulatory care hospital services has led to longer waitlist times to access specialists and appropriate intervention services; placing vulnerable children at increased risk of poorer short-term (e.g. social difficulties) and long-term (e.g. convictions) health and social outcomes. Traditional approaches to increasing frequency and service of delivery are expensive and can have minimal impact on caregiver burden. A community based service-integration approach, rather than self-directed care is proposed as increased service linkages are more likely to occur and improve the health outcomes of children with a chronic health condition., Methods: An open, unblinded, multi-centre randomised controlled trial in two Australian public hospitals. 112 children (0-16 years) fulfilling the inclusion criteria will be randomised to one of two clinical pathways for management of their chronic health condition: (1) integrated children's care clinic (ICCC) or (2) self-directed care pathway. All children and caregivers will be interviewed at 1 week, and 3, 6 and 12 month time intervals. Primary outcome measures include the Pediatric Quality of Life (PedQOL) questionnaire, Subjective Units of Distress Scale, Child Behaviour Checklist (CBCL) and Rotter's Locus of Control Scale. Secondary outcome measures include the total number of medical appointments, school days missed and quantity of services accessed. Our main objectives are to determine if the ICCC results in better health and economics outcomes compared to the self-directed care pathway., Discussion: The success of a health systems approach needs to be balanced against clinical, mortality and cost-effectiveness data for long-term sustainability within a publicly funded health system. A clinical pathway that is sustainable, cost-effective, provides efficient evidence-based care and improves the quality of life outcomes for children with chronic health conditions has the potential to reduce waitlist times, improve access to health services, increase consumer satisfaction; and prevent costs associated with poorly managed chronic health conditions into adulthood. This study will be the first to provide clinical and health economics data on an integrated care pathway for the management of chronic health conditions in children. On a broader scale, results from this study will help guide care coordination frameworks for children with chronic health conditions; particularly with the introduction and implementation of a National Disability Insurance Scheme (NDIS) across Australia., Trial Registration: Australia and New Zealand Clinical Trials Register (ANZCTR) ACTRN12617001188325 . Registered: 14th August, 2017.

Background: Neonatal central diabetes insipidus (NCDI) remains a therapeutic challenge, as extremely low doses of enteral desmopressin cannot be titrated with current preparations. The aim of this study was to describe the use of orally administered dilute desmopressin in NCDI., Methods: Nasal desmopressin (100 μg/mL) was diluted in 0.9% saline to 10 μg/mL. Infants were treated with 1-5 μg and doses were titrated to a twice-daily regimen. The feed volume was 150 mL/kg/day and titrated according to weight gain., Results: Five infants aged 6-105 days were included. Stabilizing treatment doses ranged from 2 to 5 μg twice daily in neonates, and 12 μg twice daily in the older infant who was diagnosed at 105 days., Conclusions: Dilution of nasal desmopressin with saline facilitates safe administration and dose titration in NCDI. We recommend considering this therapeutic approach to NCDI, particularly in small infants or where alternative treatment regimens have been unsuccessful.

Objective: More is known about the experience of occupational therapists than the experience of patients during the profession's early years. We examined soldiers' experiences of occupational therapy in American Base Hospital 9 in France during World War I through analysis of a 53-line poem by Corporal Frank Wren contained in the unpublished memoir of occupational therapy reconstruction aide Lena Hitchcock., Method: Historical documentary research methods and thematic analysis were used to analyze the poem, the memoir, and the hospital's published history., Results: The poem describes the activities engaged in during occupational therapy, equipment used, and the context of therapy. It articulates positive dimensions of the experience of engaging in activities, including emotional benefits, diversion, and orthopedic benefits., Conclusion: Previous historical research has identified core philosophical premises about the use of occupational therapy; in this article, the enactment of these principles is established through the analysis of a soldier's account of receiving occupational therapy., (Copyright © 2017 by the American Occupational Therapy Association, Inc.)

The study objective was to determine paediatricians' experience with female genital mutilation (FGM) in Australian children and adolescents. A cross-sectional, pilot-tested national survey of paediatricians practising in Australia and contributing to the Australian Paediatric Surveillance Unit was conducted. Clinicians' knowledge, attitudes and clinical experience with FGM, awareness of clinical guidelines and education/training needs were recorded. Of 1311 paediatricians surveyed, 497 (38%) responded. Fifty-seven percent were aged 50 years or more, and 51.3% were males. Over half believed that FGM was performed in children in Australia and most were aware of its complications, but few asked about or examined for FGM. Fifty (10.3%) had seen at least one case of FGM in girls aged <18 years during their clinical career, including 16 (3.3%) in the past 5 years. Most were aware that FGM is illegal in Australia (93.9%), agreed all types of FGM were harmful (97.4%) and agreed that FGM violated human rights (98.2%). Most (87.6%) perceived FGM as a traditional cultural practice, although 11.6% thought it was required by religion. The majority (81.8%) knew notification of FGM to child protection authorities was mandatory. Over half (62.0%) were aware of the WHO Statement on FGM, but only 22.0% knew the WHO classification of FGM. These novel data indicate a minority of paediatricians in Australia have clinical experience with or education about FGM. Educational programs, best-practice clinical guidelines and policies are required to address knowledge gaps and help paediatricians identify, manage and prevent FGM in children., (Copyright © 2016 Elsevier Ltd. All rights reserved.)