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Pulmonary fibrosis (PF) in children is a rare complication of specific forms of childhood interstitial lung diseases (chILD) with extremely limited scientific evidence to guide optimal management. Whilst there continues to be significant progress in PF management for adult populations, paediatric guidelines have stagnated. New anti-fibrotic medications (nintedanib and pirfenidone) are finding regular use amongst adult PF patients but remain largely unstudied and untested in children. Although there are major differences between the two age-group populations, it is useful to learn from the evolution of adult PF management, especially in the absence of dedicated paediatric studies. Whilst there have been recent trials aimed at assessing the safety and efficacy of drugs such as nintedanib and hydroxychloroquine, there is still a dire need for more research aimed at further assessing current treatment practices and evaluating the safety and efficacy of new emerging treatments in the paediatric population., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2023. Published by Elsevier Ltd. All rights reserved.)

Objectives : Understanding the long-term consequences of sleep-disordered breathing (SDB) in neonates is crucial. A lack of consensus on diagnostic and treatment thresholds has resulted in limited research in this area. Our study aims to describe the trajectory of SDB in a cohort of high-risk neonates and their respiratory and neurodevelopmental outcomes at 3 years of age, and explore the relationship between SDB during early infancy and neurocognitive outcomes. Methods : A retrospectively identified cohort of neonates with moderate-severe SDB were prospectively followed at 3 years of age. Data collected included last polysomnography (PSG) parameters up to the age of 3 years and sleep physician's recommendations, duration of CPAP use, compliance with treatment, timing of SDB resolution, and neurodevelopmental outcomes. Univariate and multivariate logistic regression analyses were performed to evaluate the association between important respiratory and sleep breathing parameters with the developmental outcomes. Results : Eighty neonates were included. Respiratory and developmental outcomes were available for 58 (72.5%) and 56 (70%) patients, respectively. In most patients (47/58, 81%), SDB had resolved by 3 years of age. Survival without major developmental delay was seen in 32/56 (57%), but a significant proportion (21/56, 37.5%) demonstrated global developmental delay. Following univariate analysis, primary diagnosis, apnoea-hypopnoea index (AHI) at the time of last PSG and SDB outcome was significantly associated with developmental delay. However, these associations were not seen in multivariate analysis. Conclusions : Despite severity at baseline, SDB resolved in the majority of patients with time and treatment. Although statistically insignificant, logistic regression analysis identified some clinically important associations between neonatal SDB and neurodevelopmental outcomes.

Spirometry and peak cough flow testing (PCF) are commonly used in the respiratory assessment of children with a neuromuscular disorder (NMD). Testing uses two different machines, increases laboratory time, costs and resource utilisation. No studies have assessed the correlation between peak expiratory flow (PEF) obtained from spirometry and PCF in children with NMD using one device. An audit of children with a NMD managed at the Children's Hospital at Westmead in 2022-2024 aged < 20 years who performed spirometry and PCF testing on the same device (Vyaire Body Box TM , Ultrasonic flow meter-based, or Vyaire Pneumotachograph TM , Pneumotach flow meter-based; Germany) was conducted to assess the correlation between PCF and PEF. Fifty-one sets of testing were identified, and 40 subjects (9F) had reproducible testing and were included. Median (range) age was 14.95 (7.20-19.00) years. Median PEF (L/min) was 4.05 (1.22-10.26) and median PCF (L/min) was 4.29 (1.69-10.82). PEF and PCF had a strong Pearson's correlation coefficient, (R = 0.97, p = 0.03). The coefficient of determination was 0.93. If laboratory resources permit, spirometry should be the test of choice for children with NMD. On average, spirometry required multiple practices to achieve reproducibility to meet ATS/ERS standards. PCF testing can be utilised for children where performing technically acceptable spirometry is not possible., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2024. Published by Elsevier Ltd. All rights reserved.)

Competing Interests: Declaration of competing interest The author declares that he has no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant condition characterised by small telangiectasias and larger multisystem arteriovenous malformations (AVMs). Common sites of AVMs include in the nose, lungs, brain and liver. These lesions are prone to rupture, leading to complications including recurrent epistaxis and significant haemorrhage. Pulmonary hypertension (PH) can also occur. This review presents an update on the genetics, clinical manifestations, management options, and screening recommendations for children with HHT., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024. Published by Elsevier Ltd.)

Obstructive sleep apnea (OSA) due to a hypertrophy of the adenoids and/or the tonsils in otherwise healthy children is associated with neurocognitive dysfunction and behavioural disorders with various degrees of hyperactivity, aggressiveness, sometimes evolving to a label of attention-deficit hyperactivity disorder. Children with anatomical and/or functional abnormalities of the upper airways represent a very specific population which is at high risk of OSA (also called complex OSA or OSA type III). Surprisingly, the neurocognitive consequences of OSA have been poorly studied in these children, despite the fact that OSA is more common and more severe than in their healthy counterparts. This may be explained by that fact that screening for OSA and sleep-disordered breathing is not systematically performed, the performance of sleep studies and neurocognitive tests may be challenging, and the respective role of the underlining disease, OSA, but also poor sleep quality, is complex. However, the few studies that have been performed in these children, and mainly children with Down syndrome, tend to show that OSA, but even more disruption of sleep architecture and poor sleep quality, aggravate the neurocognitive impairment and abnormal behaviour in these patients, underlining the need for a systematic and early in life assessment of sleep and neurocognitive function and behaviour in children with OSA type III., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024 Elsevier Ltd. All rights reserved.)

Poor adherence is an important factor in unstable disease control and treatment failure. There are multiple ways to monitor a patient's adherence, each with their own advantages and disadvantages. The reasons for poor adherence are multi-factorial, inter-related and often difficult to target for improvement. Although practitioners can implement different methods of adherence, the ultimate aim is to improve health outcomes for the individual and the health care system. Asthma is a common airway disease, particularly diagnosed in children, often treated with inhaled corticosteroids and long-acting bronchodilators. Due to the disease's tendency for exacerbations and consequently, when severe will require unscheduled health care utilisation including hospital admissions, considerable research has been done into the effects of medication adherence on asthma control. This review discusses the difficulties in defining adherence, the reasons for and consequences of poor adherence, and the methods of recording and improving adherence in asthma patients, including an in-depth analysis of the uses of smart inhalers., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved.)

Melioidosis is a tropical infectious disease caused by the saprophytic gram-negative bacterium Burkholderia pseudomallei. Despite the infection being endemic in southeast Asia and northern Australia, the broad clinical presentations and diagnostic difficulties limit its early detection, particularly in children. Melioidosis more commonly affects the immunocompromised and adults. Melioidosis is increasingly being diagnosed around the world and whole-genome sequencing indicates that these cases are not linked with travel to endemic areas. Research has concentrated on the adult population with limited experience reported in the care of this uncommon, but potentially fatal condition in children presenting with bacteraemia and pneumonia., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023 Elsevier Ltd. All rights reserved.)

Objective: To investigate the neurodevelopmental outcomes for preterm infants born < 29 weeks gestation with/without bronchopulmonary dysplasia (BPD)., Study Design: Preterm infants < 29 weeks' gestation born 2007-2018 in New South Wales and the Australian Capital Territory, Australia, were included. Infants who died < 36 weeks' postmenstrual age and those with major congenital anomalies were excluded. Subjects were assessed at 18-42 months corrected age using the Bayley Scales of Infant Development, 3rd edition., Results: 1436 infants without BPD (non-BPD) and 1189 infants with BPD were followed. The BPD group, 69 % infants were discharged without respiratory support (BPD1), 29 % on oxygen (BPD2) and 2 % on pressure support/tracheostomy (BPD3). Moderate neurodevelopmental impairment (NDI) was evident in 5.7 % of non-BPD infants, 11 % BPD1, 15 % BPD2, 15 % BPD3 infants. Severe NDI was seen in 1.7 % non-BPD infants, 3.4 % BPD1, 7.3 % BPD2, 35 % BPD3 infants. After adjusting for confounders, infants with BPD2 (OR 2.24, 99.9 % CI 1.25 to 5.77) or BPD3 (OR 5.99, 99.9 % CI 1.27 to 46.77) were more likely to have moderate-severe NDI compared to non-BPD infants., Conclusion: The majority of infants with BPD were discharged home without respiratory support and had better neurocognitive outcomes in early childhood compared to those that required home-based oxygen or respiratory support., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2024. Published by Elsevier Ltd. All rights reserved.)

Competing Interests: Declaration of competing interest The author declares that he has no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

The impact of evolving treatment regimens, airway clearance strategies, and antibiotic combinations on the incidence and prevalence of respiratory infection in cystic fibrosis (CF) in children and adolescents remains unclear. The incidence, prevalence, and prescription trends from 2002 to 2019 with 18,339 airway samples were analysed. Staphylococcus aureus [- 3.86% (95% CI - 5.28-2.43)] showed the largest annual decline in incidence, followed by Haemophilus influenzae [- 3.46% (95% CI - 4.95-1.96)] and Pseudomonas aeruginosa [- 2.80%95% CI (- 4.26-1.34)]. Non-tuberculous mycobacteria and Burkholderia cepacia showed a non-significant increase in incidence. A similar pattern of change in prevalence was observed. No change in trend was observed in infants < 2 years of age. The mean age of the first isolation of S. aureus (p < 0.001), P. aeruginosa (p < 0.001), H. influenza (p < 0.001), Serratia marcescens (p = 0.006) and Aspergillus fumigatus (p = 0.02) have increased. Nebulised amikacin (+ 3.09 ± 2.24 prescription/year, p = 0.003) and colistin (+ 1.95 ± 0.3 prescriptions/year, p = 0.032) were increasingly prescribed, while tobramycin (- 8.46 ± 4.7 prescriptions/year, p < 0.001) showed a decrease in prescription. Dornase alfa and hypertonic saline nebulisation prescription increased by 16.74 ± 4.1 prescriptions/year and 24 ± 4.6 prescriptions/year (p < 0.001). There is a shift in CF among respiratory pathogens and prescriptions which reflects the evolution of cystic fibrosis treatment strategies over time., (© 2024. Crown.)

There is an increasing demand for the assessment of sleep-disordered breathing in children of all ages to prevent the deleterious neurocognitive and behaviour consequences of the under-diagnosis and under-treatment of obstructive sleep apnoea [OSA]. OSA can be considered in three broad categories based on predominating contributory features: OSA type 1 [enlarged tonsils and adenoids], type II [Obesity] and type III [craniofacial abnormalities, syndromal, storage diseases and neuromuscular conditions]. The reality is that sleep questionnaires or calculations of body mass index in isolation are poorly predictive of OSA in individuals. Globally, the access to testing in tertiary referral centres is comprehensively overwhelmed by the demand and financial cost. This has prompted the need for better awareness and focussed history taking, matched with simpler tools with acceptable accuracy used in the setting of likely OSA. Consequently, we present key indications for polysomnography and present scalable, existing alternatives for assessment of OSA in the hospital or home setting, using polygraphy, oximetry or contactless sleep monitoring., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024. Published by Elsevier Ltd.)

Aim: To explore the relationship between postural changes in lung function and polysomnography (PSG) in children with Duchenne muscular dystrophy (DMD)., Methods: In this prospective cross-sectional study, children with DMD performed spirometry in sitting and supine positions. A control group of age and gender matched healthy children also underwent postural lung function testing. PSG was performed within six months of spirometry., Results: Seventeen children with DMD, aged 12.3 ± 3 years performed sitting spirometry. 14 (84%) performed acceptable spirometry in the supine position. Mean FEV 1 sit and FVC sit were 77% (SD ± 22) and 74% (SD ± 20.4) respectively, with mean% ΔFVC ( sit-sup ) 9% (SD ± 11) (range 2% to 20%), and was significantly greater than healthy controls 4% (n = 30, SD ± 3, P < 0.001). PSG data on the 14 DMD children with acceptable supine spirometry showed total AHI 6.9 ± 5.9/hour (0.3 to 29), obstructive AHI 5.2 ± 4.0/hour (0.2 to 10), and REM AHI 14.1 ± -5.3/hour (0.1 to 34.7). ΔFVC(sit-sup) had poor correlation with hypoventilation on polysomnography., Conclusion: Children with DMD and mild restrictive lung disease showed greater postural changes in spirometry than healthy controls but lower supine spirometry was not predictive of sleep hypoventilation., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023. Published by Elsevier Ltd.)

With increasing competitiveness across the sporting landscape, there is a need for more research into monitoring and managing the young athlete, as the needs of a young athlete are vastly different to those of an older athlete who is already established in their respective sport. As the age of sports specialisation seems to decrease, exercise testing in the younger cohort of athletes is crucial for safety and long-term success. This article provides a comprehensive summary of available testing and monitoring methods that can be used to assist young athletes as they mature and attempt to excel in their chosen sport., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2023. Published by Elsevier Ltd. All rights reserved.)

Background: Respiratory infections caused by Staphylococcus aureus and Pseudomonas aeruginosa are a major concern for cystic fibrosis (CF) patients due to increasing antibiotic resistance. Bacteriophages, which are viruses that selectively target and kill bacteria, are being studied as an alternative treatment for these infections. This systematic review evaluates the safety and effectiveness of bacteriophages for the treatment of CF-related infections caused by S. aureus and/or P. aeruginosa. We conducted a search for original, published articles in the English language up to March 2023. Studies that administered bacteriophages via intravenous, nebulised, inhaled, or intranasal routes were included, with no comparators required. In vitro and in vivo studies were eligible for inclusion, and only animal in vivo studies that utilised a CF transmembrane conductance regulator (CFTR) animal model were included. Bacteriophage treatment resulted in a decrease in bacterial load in both humans and animals infected with P. aeruginosa. Complete eradication of P. aeruginosa was only observed in one human subject. Additionally, there was a reduction in biofilm, improvement in resistance profile, and reduced pulmonary exacerbations in individual case reports. Evidence suggests that bacteriophage therapy may be a promising treatment option for CF-related infections caused by P. aeruginosa and S. aureus. However, larger and more robust trials are needed to establish its safety and efficacy and create necessary evidence for global legislative frameworks., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023 Elsevier Ltd. All rights reserved.)

Highly effective modulator therapies (HEMTs) have revolutionised the management approach of most patients living with cystic fibrosis (CF) who have access to these therapies. Clinical trials have reported significant improvements across multiorgan systems, with patients surviving longer. However, there are accumulating case reports and observational data describing various adverse events following initiation of HEMTs including drug-to-drug interactions, drug induced liver injury, Stevens-Johnson syndrome, and neurocognitive symptoms including psychosis and depression, which have required discontinuation of therapy. Current clinical trials are assessing efficacy in younger patients with CF, yet long-term studies are also required to better understand the safety profile in the real-world setting across all ages and the impact of HEMT dose alteration or discontinuation., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2023. Published by Elsevier Ltd. All rights reserved.)

Cough medicines have been in use for over a century to treat the common and troublesome, but often helpful, symptoms of cough in children. They contain various combinations of "anti-tussive" drugs including opioids, antihistamines, herbal preparations, mucolytics, decongestants and expectorants. Whilst theoretically attractive for symptom relief when children are suffering, as time has passed these popular over the counter medicines have been shown to lack efficacy, delay more serious underlying diagnoses, and can cause complications and sometimes death. This has resulted in clinician concerns, a citizen petition to the American Food and Drug Association in 2007, some self-regulation from manufacturers and escalating restrictions on their use from regulatory agencies across the world over the last twenty years. This article will review the protective role of cough, juxtapose the conflicting treatment goals of suppressing a dry cough and promoting expectoration for a wet cough, consider the evidence basis for prescribing cough medicines in comparison to other more specific treatments such as for asthma [beta agonists] or infection [antibiotics], regulatory interventions, and conclude with the view that over counter cough medicines should not be used in children, especially young children., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023 Elsevier Ltd. All rights reserved.)

Bronchopulmonary dysplasia [BPD] is the most common complication of extremely preterm delivery and its optimal management remains challenging because of a lack of evidence to guide management. There has been improvement in the management of evolving BPD in the neonatal intensive care unit (NICU). The threshold for provision of home oxygen therapy, often occurring because of a preference for earlier discharge from the NICU, creates tensions for clincians and families. Once discharged in supplemental oxygen, the approaches for the weaning of this therapy vary considerably across the world. Regardless of guidelines and multidisciplinary team support, up to a third of families of an infant with BPD elect to withdraw home oxygen therapy independently of medical advice. There is a pressing need to derive evidence to better inform practice, generate international consensus and undertake large, appropriately funded, longitudinal studies of BPD with clinically meaningful outcomes (respiratory, cardiovascular and neurodevelopmental) from infancy to adulthood., Competing Interests: Declaration of Competing Interest The author declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2023. Published by Elsevier Ltd. All rights reserved.)

Mycobacterium abscessus is a nontuberculous mycobacterium (NTM) of particular concern in individuals with obstructive lung diseases such as cystic fibrosis (CF). Treatment requires multiple drugs and is characterised by high rates of relapse; thus, new strategies to limit infection are urgently required. This study sought to determine how Bacille Calmette-Guérin (BCG) vaccination may impact NTM infection, using a murine model of Mycobacterium abscessus infection and observational data from a non-BCG vaccinated CF cohort in Sydney, Australia and a BCG-vaccinated CF cohort in Cape Town, South Africa. In mice, BCG vaccination induced multifunctional antigen-specific CD4 + T cells circulating in the blood and was protective against dissemination of bacteria to the spleen. Prior infection with M. abscessus afforded the highest level of protection against M. abscessus challenge in the lung, and immunity was characterised by a greater frequency of pulmonary cytokine-secreting CD4 + T cells compared to BCG vaccination. In the clinical CF cohorts, the overall rates of NTM sampling during a three-year period were equivalent; however, rates of NTM colonisation were significantly lower in the BCG-vaccinated (Cape Town) cohort, which was most apparent for M. abscessus . This study provides evidence that routine BCG vaccination may reduce M. abscessus colonisation in individuals with CF, which correlates with the ability of BCG to induce multifunctional CD4 + T cells recognising M. abscessus in a murine model. Further research is needed to determine the optimal strategies for limiting NTM infections in individuals with CF.

Haemoptysis occurs in up to 25 % of young people with Cystic fibrosis (CF) [1]. We undertook a literature review and described the management approach to haemoptysis in CF between 2010 and 2020 at an Australian tertiary paediatric centre, The Children's Hospital Westmead, Sydney, New South Wales, using a retrospective review of the medical records which identified 67 episodes. Sixty episodes met inclusion criteria, including 31 patients. Using the US CF Foundation guidelines, episodes were classified as scant (53.3 %), moderate (38.3 %) or massive (8.3 %). Fifty-two percent of patients were female, mean age at presentation was 15.4 years (SD+/- 2.4) and 58 % were homozygous for the Fdel508 genotype. Twelve episodes (9 patients) required bronchial artery embolization (BAE). BAE was used in all cases of massive haemoptysis 5/5 (100 %), 6/23 (22 %) episodes of moderate and 1/32 (3 %) episode of scant haemoptysis as an elective procedure for recurrent haemoptysis. Our literature review and institutional experience highlights the need for up-to-date management guidelines in the management of haemoptysis in Cystic Fibrosis. Based on our experience, we provide a proposed algorithm to help guide the management of haemoptysis in CF., Competing Interests: Conflict of Interest The authors have no conflict of interest to disclose., (Copyright © 2022. Published by Elsevier Ltd.)

It is unclear how improvements in peripheral motor function in children with spinal muscular atrophy (SMA), treated with nusinersen, translate into clinically significant respiratory/sleep outcomes. A retrospective chart review of SMA children at the Sydney Children's Hospital Network was undertaken looking at 2 years before and after receiving their first dose of nusinersen. Polysomnography (PSG), spirometry and clinical data were collected and analysed using paired and unpaired t-tests for PSG parameters and generalised estimating equations for longitudinal lung function data. Forty-eight children (10 Type 1, 23 Type 2, 15 Type 3) at mean age 6.98 yrs (SD 5.25) for nusinersen initiation were included. There was a statistically significant improvement in oxygen nadir during sleep in individuals post nusinersen (mean of 87.9% to 92.3% (95%CI 1.24 - 7.63, p = 0.01)). Based on clinical and PSG findings, 6/21 patients (5 Type 2, 1 Type 3) ceased nocturnal NIV post nusinersen. Non-significant improvements were demonstrated in mean slope for FVC% predicted, FVC Z-score and mean FVC% predicted. Within 2 years of commencing nusinersen, stabilisation of respiratory outcomes occurred. Whilst some of the SMA type 2/3 cohort ceased NIV, there were no statistically significant improvements lung function and most PSG parameters., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023. Published by Elsevier B.V.)

This review summarises the experiences of young children and their families living with CF during the first five years of life following NBS diagnosis, as well as the options of psychosocial support available to them. We present strategies embedded within routine CF care that focus on prevention, screening, and intervention for psychosocial health and wellbeing that constitute essential components of multidisciplinary care in infancy and early childhood., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023. Published by Elsevier Ltd.)

Introduction: Cystic fibrosis (CF) is a multisystem condition that is complicated by recurrent pulmonary infections requiring aggressive antibiotic treatment. This predisposes the patient to complications such as sensorineural hearing loss, renal impairment, hypersensitivity and the development of antibiotic resistance. Pseudomonas aeruginosa is one of the more common organisms which cause recurrent infections and result in greater morbidity and mortality in people living with CF. Bacteriophages have been identified as a potential alternative or adjunct to antibiotics. We hypothesise that bacteriophage therapy is a safe and well-tolerated treatment in children with CF infected with P. aeruginosa infection in their airways., Methods: This single-arm, open-labelled, non-randomised trial will run for a maximum period of 36 months with up to 10 participants. Adolescents (≥12 years and <18 years of age) who continue to shed P.aeruginosa (within 3 months of enrolment) despite undergoing eradication therapy previously, will be considered for this trial. Non-genetically modified bacteriophages that have demonstrated obligate lytic activity against each of the study participants' P. aeruginosa strains will be selected and prepared according to a combination of established protocols (isolation, purification, sterility testing and packaging) to achieve close to good manufacturing practice recommendations. The selected bacteriophage will be administered endo-bronchially first under direct vision, followed by two times a day nebulisation for 7 days in addition to standard CF treatment (intravenous antibiotics, physiotherapy to be completed as inpatient for 10-14 days). Safety and tolerability will be defined as the absence of (1) fever above 38.5°C occurring within 1 hour of the administration of the nebulised bacteriophage, (2) a 10% decline in spirometry (forced expiratory volume in 1 s %) measured preadministration and postadministration of the first dose of nebulised bacteriophage. Clinical reviews including repeat sputum cultures and spirometry will be performed at 3, 6, 9 and 12 months following bacteriophage treatment., Ethics and Dissemination: Our clinical trial is conducted in accordance with (1) good clinical practice, (2) Australian legislation, (3) National Health and Medical Research Council guidelines for the ethical conduct of research., Trial Registration Number: Australia and New Zealand Clinical Trial Registry (ACTRN12622000767707)., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Pneumonia is the number one cause of disease and deaths in children under five years old, outside the neonatal period, with the greatest number of cases reported from resource-limited settings. The etiology is variable, with not much information on the local etiology drug resistance profile in many countries. Recent studies suggest an increasing contribution from respiratory viruses, also in children with severe pneumonia, with an increased relative contribution in settings that have good vaccine coverage against common bacterial pathogens. Respiratory virus circulation was greatly reduced during highly restrictive measures to contain the spread of COVID-19 but rebounded once COVID-19 restrictions were relaxed. We conducted a comprehensive literature review of the disease burden, pathogens, case management and current available prevention of community acquired childhood pneumonia, with a focus on rational antibiotic use, since the treatment of respiratory infections is the leading cause of antibiotic use in children. Consistent application of revised World Health Organisation (WHO) guidance that children presenting with coryzal symptoms or wheeze can be managed without antibiotics in the absence of fever, will help to reduce unnecessary antibiotic use, as will increased availability and use of bedside inflammatory marker tests, such as C-reactive protein (CRP) in children with respiratory symptoms and fever., 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., (© 2023 Nguyen, Robinson, Fitzgerald and Marais.)

Background: Children with cystic fibrosis (CF) pulmonary exacerbations receive IV tobramycin therapy, with dosing guided by either log-linear regression (LLR) or Bayesian forecasting (BF)., Objectives: To compare clinical and performance outcomes for LLR and BF., Patients and Methods: A quasi-experimental intervention study was conducted at a tertiary children's hospital. Electronic medical records were extracted (from January 2015 to September 2021) to establish a database consisting of pre-intervention (LLR) and post-intervention (BF) patient admissions and relevant outcomes. All consecutive patients treated with IV tobramycin for CF pulmonary exacerbations guided by either LLR or BF were eligible., Results: A total of 376 hospital admissions (LLR = 248, BF = 128) for CF pulmonary exacerbations were included. Patient demographics were similar between cohorts. There were no significant differences found in overall hospital length of stay, rates of re-admission within 1 month of discharge or change in forced expiratory volume in the first second (Δ FEV1) at the end of tobramycin treatment. Patients treated with LLR on average had twice the number of therapeutic drug monitoring (TDM) blood samples collected during a single hospital admission. The timeframe for blood sampling was more flexible with BF, with TDM samples collected up to 16 h post-tobramycin dose compared with 10 h for LLR. The tobramycin AUC0-24 target of ≥100 mg/L·h was more frequently attained using BF (72%; 92/128) compared with LLR (50%; 124/248) (P < 0.001). Incidence of acute kidney injury was rare in both groups., Conclusions: LLR and BF result in comparable clinical outcomes. However, BF can significantly reduce the number of blood collections required during each admission, improve dosing accuracy, and provide more reliable target concentration attainment in CF children., (© The Author(s) 2022. Published by Oxford University Press on behalf of British Society for Antimicrobial Chemotherapy. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

This review addresses regional oxygenation and perfusion changes for preterm infants and changes with body position, with or without head rotation. Future directions for improving neurodevelopmental and clinical outcomes are suggested. The MEDLINE, Embase and Scopus databases were searched up to July 2021. Fifteen out of 470 studies met the inclusion criteria. All were prospective, observational studies with a moderate risk of bias. Significant variation was found for the baseline characteristics of the cohort, postnatal ages, and respiratory support status at the time of monitoring. When placed in a non-supine position, preterm infants showed a transient reduction in cardiac output and stroke volume without changes to heart rate or blood pressure. No studies reported on long-term neurodevelopmental outcomes. Overall, side lying or prone position does not appear to adversely affect regional, and specifically cerebral, oxygenation or cerebral perfusion. The effect of head rotation on regional oxygenation and perfusion remains unclear., (Copyright © 2021 Elsevier Ltd. All rights reserved.)

Cheyne-Stokes respiration (CSA-CSR) is a form of central sleep apnea characterized by alternating periods of hyperventilation and central apneas or hypopneas. CSA-CSR develops following a cardiac insult resulting in a compensatory increase in sympathetic activity, which in susceptible patients causes hyperventilation and destabilizes respiratory control. The physiological changes that occur in CSA-CSR include hyperventilation, a reduced blood gas buffering capacity, and circulatory delay. In adults, 25% to 50% of patients with heart failure are reported to have CSA-CSR. The development of CSA-CSR in this group of patients is considered a poor prognostic sign. The prevalence, progression, and treatment outcomes of CSA-CSR in children remain unclear with only 11 children being described in the literature. The lack of data is possibly not due to the paucity of children with severe heart failure and CSA-CSR but because they may be under-recognized, compounded by the absence of routine polysomnographic assessment of children with moderate to severe heart failure. Building on much broader experience in the diagnosis and management of CSA-CSR in adult sleep medicine and our limited experience in a pediatric quaternary center, this paper will discuss the prevalence of CSA-CSR, its' treatment options, outcomes in children, and the potential future direction for research in this understudied area of pediatric sleep medicine., Competing Interests: Conflict of interest Nil for any authors., (Crown Copyright © 2022. Published by Elsevier Ltd. All rights reserved.)

Background: Parapneumonic effusions and empyema are the most frequent complication of pediatric pneumonia. Interventions include chest drain and fibrinolytics (CDF) or thoracoscopic surgery. CDF is considered less invasive, and more cost-effective though with higher rates of reintervention. We hypothesized that sonographic pleural fluid characteristics could identify cases at increased risk of reintervention following primary CDF., Methods: A retrospective cohort of complicated pneumonia managed with primary CDF (2011-2018). Cases were reviewed using ultrasound criteria to describe pleural fluid. We analyzed the correlation between ultrasound findings and reintervention., Results: We report 129 cases with a median age of 3.8 years and 44% female. A repeat intervention occurred for 24/129 (19%) cases. The interobserver reliability was moderate for the number of septations (κ 0.72, 95% CI [confidence interval]: 0.62-0.81), weak for the size of the largest locule (κ 0.55, 95% CI: 0.44-0.67), and minimal for the level of echogenicity (κ 0.24, 95% CI: 0.11-0.37), pleural thickening (κ 0.29, 95% CI: 0.17-0.42), maximum effusion depth (κ 0.37, 95% CI: 0.22-0.51), and radiologist's risk for reintervention (κ 0.34, 95% CI: 0.18-0.5). A repeat intervention was not associated with any objective sonographic variable., Conclusion: We report no association between ultrasound characteristics and repeat intervention for complicated pneumonia following primary CDF treatment. There was minimal interobserver agreement in reporting ultrasound characteristics despite more objective criteria. Clinicians rely on ultrasound findings to support decisions around intervention in pediatric empyema. This study does not support relying on ultrasound to estimate the likelihood of reintervention., (© 2022 Wiley Periodicals LLC.)

Background: Community-acquired pneumonia (CAP) is a leading cause of childhood hospitalisation. Limited data exist on factors predicting severe disease with no paediatric-specific predictive tools., Methods: Retrospective cohort (2011-2016) of hospitalised CAP cases. We analysed clinical variables collected at hospital presentation against outcomes. Stratified outcomes were mild (hospitalised), moderate (invasive drainage procedure, intensive care) or severe (mechanical ventilation, vasopressors, death)., Results: We report 3330 CAP cases, median age 2.0 years (IQR 1-5 years), with 2950 (88.5%) mild, 305 (9.2%) moderate and 75 (2.3%) severe outcomes. Moderate-severe outcomes were associated with hypoxia (SaO 2 <90%; OR 6.6, 95% CI 5.1 to 8.5), increased work of breathing (severe vs normal OR 5.8, 95% CI 4.2 to 8.0), comorbidities (4+ comorbidities vs nil; OR 8.8, 95% CI 5.5 to 14) and being indigenous (OR 4.7, 95% CI 2.6 to 8.4). Febrile children were less likely than afebrile children to have moderate-severe outcomes (OR 0.57 95% CI 0.44 to 0.74). The full model receiver operating characteristic (ROC) area under the curve (AUC) was 0.78. Sensitivity analyses showed similar results with clinical or radiological CAP definitions. We derived a clinical tool to stratify low, intermediate or high likelihood of severe disease (AUC 0.72). High scores (≥5) had nearly eight times higher odds of moderate-severe disease than those with a low (≤1) score (OR 7.7 95% CI 5.6 to 10.5)., Conclusions: A clinical risk prediction tool is needed for child CAP. We have identified risk factors and derived a simple clinical tool using clinical variables at hospital presentation to determine a child's risk of invasive or intensive care treatment with an ROC AUC comparable with adult pneumonia tools., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2022. No commercial re-use. See rights and permissions. Published by BMJ.)

The loss of an apparently healthy infant is confronting for any family, puzzling for a clinician and challenging for the pathologist charged with the task of demonstrating a cause for death. The term "cot death" evolved to "sudden infant death syndrome" [SIDS] and now "sudden unexpected death in infancy [SUDI]" as the epidemiology and pathology of infant death changed. Community interventions were successful in changing sleep practices for young babies. The current research focus is on understanding genetic predispositions to unexpected death in early childhood. Whilst much has been achieved in reducing the infant mortality rate from SUDI by between 50%, and 80% in some countries, over the last 30 years, there remain challenges for improving rates of accurate diagnosis and reaching out to more vulnerable families with clearly modifiable risk factors for SUDI. These challenges directly involve the clinician through taking a systematic and detailed history and better standardised death scene evaluations with specifically accredited assessors. Better knowledge regarding circumstances of SUDI cases will help Coroners and researchers provide answers for grieving families now, and in the future contribute to further reductions in the rate of SUDI in communities across the world., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Crown Copyright © 2021. Published by Elsevier Ltd. All rights reserved.)

In developed countries, it is projected that there will be a 70% increase in the number of adults living with Cystic Fibrosis (CF) between 2010 and 2025. This shift in demographics highlights the importance of high-quality transition programmes with developmentally appropriate integrated health care services as the individual moves through adolescence to adulthood. Adolescents living with CF face additional and unique challenges that may have long-term impacts on their health, quality of life and life-expectancy. CF specific issues around socially challenging symptoms, body image, reproductive health and treatment burden differentiate people with CF from their peers and require clinicians to identify and address these issues during the transition process. This review provides an overview of the health, developmental and psychosocial challenges faced by individuals with CF, their guardians and health care teams considering the fundamental components and tools that are required to build a transition programme that can be tailored to suit individual CF clinics., (Crown Copyright © 2020. Published by Elsevier Ltd. All rights reserved.)

Paediatric spontaneous pneumothorax (PSP) management continues to lack paediatric-specific guideline recommendations. There have been increasing reports of paediatric retrospective case studies supplemented by important well designed RCT (predominantly) adult studies. Taken together, these suggest that conservative management may have an increasing role to play in the management of PSP and that aspiration may have limited utility as a first line intervention. Our local experience, as part of a multicentre retrospective analysis and subsequent audit of management since, corroborates recent published data: it highlights an increasing trend towards conservative management in spontaneous pneumothorax with similar rates of recurrence, compared to intervention, and low use of aspiration with similarly low success rates. We have therefore updated our local practice guidelines and share these with readers. Specifically, we have removed aspiration in the management of primary spontaneous pneumothorax and reserved intervention for children who are clinically unstable or show evidence of increasing air leak irrespective of pneumothorax size. Whilst the success of this change in clinical practice will need to be reviewed in the next 5-10 years, the overall low incidence of the condition, demands a multicentre, and probably multinational, collaborative approach to allow the best chance of obtaining definitive evidence to guide clinical paediatric management., (Copyright © 2021. Published by Elsevier Ltd.)

The role of the expert witness is clear in the eyes of the court but that understanding is less consistent for the medical professionals who may be called on in this capacity. The fundamental key is the clinician's purpose in using their experience, knowledge and training as they provide the court with an unbiased assessment of the clinical issues that have arisen in the case. Using the Australian context, this article provides guidance for clinicians who are asked to provide the court with their expertise through written and spoken representations, drawing upon experience and the use of the "Expert Witness Code of Conduct"., (Copyright © 2021. Published by Elsevier Ltd.)

Physical activity is a natural part of a healthy life-style, which should be nurtured from early childhood. Regular physical activity mitigates against the global problems of overweight and obesity, hypertension, anxiety and depression. It lowers the morbidity and mortality from cardiovascular disease and provides hope for sustainable economics to support an ageing population into their retirement. This is preventative health economics that can be achieved with integrated support from families, communities, health-care professionals and governments at all levels. At present, children lack the support of those responsible for them at a societal level to adequately protect them from the physical and emotional consequences of reduced physical activity., (© 2022 Paediatrics and Child Health Division (The Royal Australasian College of Physicians).)

Face-to-face education as the traditional basis for medical education was disrupted by the COVID-19 pandemic as learners and educators were moved online with little time for preparation. Fortunately, as online learning has grown, together with medical education shifting to problem-based and team-centered learning over the last three decades, existing resources have been adapted and improved upon to meet the challenges. Effective blended learning has resulted in innovative synchronous and asynchronous learning platforms. Clearly, to do this well requires time, effort, and adjustment from clinicians, educators, and learners, but it should result in an engaging change in teaching practice. Its success will rely on an evaluation of learning outcomes, educator and learner satisfaction, and long-term retention of knowledge. It will be important to maintain ongoing assessment of all aspects of the medical education process, including how to best teach and assess theory, physiology, pathology, history-taking, physical examination, and clinical management.Conclusion: The COVID-19 pandemic triggered emergency transitional processes for teaching and assessment in medical education which built upon existing innovations in teaching medicine with the use of technology. These strategies will continue to evolve so as to provide the basis for an enduring hybrid teaching model involving blended and e-learning in medical education.. What is Known: • Most pediatricians provide clinical teaching to medical students and residents, but few have had formal training in online educational approaches and techniques. • Being able to adapt to new and innovative integrated teaching methods is of key importance when becoming a competent teacher. What is New: • This review presents an up-to-date summary of best practice in blended and e-learning and how it may be optimally delivered. • Knowledge of the principles of e-learning, and how people learn more generally, helps pediatricians shape their clinical teaching and facilitates better interaction with medical students and residents., (© 2021. Crown.)

Teenagers of today sleep less than previous generations. Technology is largely to blame for keeping people perpetually connected in the digital world which is in turn driven by changing social demands for immediacy as a form of intimacy. The consequences for teenagers are later bed times, reduced total sleep time and a degree of sleep catch up on weekends. This is termed chronic sleep deprivation or "social jetlag". The consequences of chronic sleep deprivation are underappreciated in the medical setting. They include altered mood, more somatic and psychological symptomatology, greater anxiety, more school absenteeism, reduced educational results and compromised vocational aspirations. Engagement with reluctant teenagers and their parents may be challenging and at times frustrating for all concerned. Much of the art of improving outcomes involves developing a rapport with the teenager, assisting them to gain insight into the problems associated with chronic sleep deficiency and fostering commitment from all family members to implement unpopular boundaries on the use of technology., (Crown Copyright © 2021. Published by Elsevier Ltd. All rights reserved.)

Aim: To describe the respiratory and nutritional supportive care and hospitalisations required in the real-world scenario in children with SMA type 1 treated with nusinersen., Methods: Single-centre observational cohort study of children with SMA1 commencing nusinersen from November 2016 to September 2018. Motor, respiratory and nutritional clinical characteristics and management are described from initiation of nusinersen for a minimum of two years., Results: Nine children (5 females, 4 males), median age 10.7 months (range 2.7-181.2) commenced treatment with nusinersen and outcomes were assessed over a total of 270.5 patient months and 209 hospital admissions. Supportive care in newly-diagnosed patients (n = 7) included gastrostomy insertion (n = 4) and commencement of noninvasive ventilation (n = 4) at an average of 8.3 and 4.5 months after diagnosis, respectively. The annualised hospitalisation rate was 9.3/patient/year, average length of stay (LOS) of 3.3 days (SD = 5.6). Children with two SMN2 copies required more gastrostomies (p < 0.05) and had more frequent admissions (p < 0.05). Number of total admissions halved from the first to the second year of treatment in all patients (p < 0.005)., Interpretation: Children with treated SMA1 experienced considerable respiratory and bulbar comorbidities, necessitating substantial respiratory and nutritional supportive care. Proactive respiratory and nutritional surveillance and management is essential in SMA1 patients treated with nusinersen., (Copyright © 2020 Elsevier Ltd. All rights reserved.)

World Health Organisation definitions of pneumonia severity are routinely used in research. In high income health care settings with high rates of pneumococcal vaccination and low rates of mortality, malnutrition and HIV infection, these definitions are less applicable. National guidelines from leading thoracic and infectious disease societies describe 'severe pneumonia' according to criteria derived from expert consensus rather than a robust evidence base. Contemporary cohort studies have used clinical outcomes such as intensive care therapy or invasive procedures for complicated pneumonia, to define severe disease. Describing severe pneumonia in such clinically relevant terms facilitates the identification of risk factors associated with worsened disease and the subsequently increased morbidity, and need for tertiary level care. The early recognition of children at higher risk of severe pneumonia informs site of care decisions, antibiotic treatment decisions as well as guiding appropriate investigations. Younger age, malnutrition, comorbidities, tachypnoea, and hypoxia have been identified as important associations with 'severe pneumonia' by WHO definition. Most studies have been performed in low-middle income countries and whilst they provide some insight into those at risk of mortality or treatment failure, their generalisability to the high-income setting is limited. There is a need to determine more precise definitions and criteria for severe disease in well-resourced settings and to validate factors associated with intensive care admission or invasive procedures to enhance the early recognition of those at risk., (Copyright © 2020 Elsevier Ltd. All rights reserved.)

Pneumonia is the leading cause of paediatric hospitalisation in Vietnam, placing a huge burden on the health care system. Pneumonia is also the main reason for antibiotic use in children. Unfortunately many hospital admissions for child pneumonia in Vietnam are unnecessary and inappropriate use of antibiotics is common, as in the rest of Asia, with little awareness of its adverse effects. We explored the value of an alternative approach that, instead of focusing on the identification of children with severe bacterial pneumonia, focuses on the identification of children with 'unlikely bacterial pneumonia' to improve patient care and rational antibiotic use. Implementing improved models of care require pragmatic management algorithms that are well validated, but it is ultimately dependent on financial structures, management support and evidence-based training of healthcare providers at all relevant levels. Apart from better case management, sustained reductions in the pneumonia disease burden also require increased emphasis on primary prevention., (Copyright © 2020 Elsevier Ltd. All rights reserved.)