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Background Allergic bronchopulmonary aspergillosis (ABPA) is an allergic reaction to colonisation of the lungs with the fungus Aspergillus fumigatus and affects around 10% of people with cystic fibrosis. ABPA is associated with an accelerated decline in lung function. High doses of corticosteroids are the main treatment for ABPA; although the long-term benefits are not clear, their many side effects are well-documented. A group of compounds, the azoles, have activity against Aspergillus fumigatus and have been proposed as an alternative treatment for ABPA. Of this group, itraconazole is the most active. A separate antifungal compound, amphotericin B, has been employed in aerosolised form to treat invasive infection with Aspergillus fumigatus, and may have potential for the treatment of ABPA. Antifungal therapy for ABPA in cystic fibrosis needs to be evaluated. This is an update of a previously published review. Objectives The review aimed to test the hypotheses that antifungal interventions for the treatment of ABPA in cystic fibrosis: 1. improve clinical status compared to placebo or standard therapy (no placebo); 2. do not have unacceptable adverse effects. If benefit was demonstrated, we aimed to assess the optimal type, duration and dose of antifungal therapy. Search methods We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register which comprises references identified from comprehensive electronic database searches, handsearches of relevant journals and abstract books of conference proceedings. In addition, pharmaceutical companies were approached. Date of the most recent search of the Group's Trials Register: 29 September 2016. Selection criteria Published or unpublished randomised controlled trials, where antifungal treatments have been compared to either placebo or no treatment, or where different doses of the same treatment have been used in the treatment of ABPA in people with cystic fibrosis. Data collection and analysis Four trials were identified by the searches; none of which was judged eligible for inclusion in the review. Main results No completed randomised controlled trials were included. Authors' conclusions At present, there are no randomised controlled trials to evaluate the use of antifungal therapies for the treatment of ABPA in people with cystic fibrosis, although trials in people who do not have cystic fibrosis have shown clinical and serological evidence of improvement and a reduction in the use of corticosteroids with no increase in adverse effects. Trials with clear outcome measures are needed to properly evaluate this potentially useful treatment for cystic fibrosis.

Narcolepsy is a chronic disabling neurological sleep disorder that requires lifelong treatment. We have outlined the clinical features of narcolepsy, the assessment and diagnosis process and have summarised the existing treatment options for children and adolescents with narcolepsy. In the future, the approach to management of paediatric narcolepsy should ideally be in a multidisciplinary setting, involving specialists in sleep medicine, sleep physiology, neurologists and psychologists/psychiatrists. A multidisciplinary approach will help to manage the potential impact of narcolepsy on children and adolescents who are in a stage of their life that is critical to their physical, emotional and social development and their academic attainment.

The objective was to investigate the prevalence of Pseudomonas aeruginosa (PA) in patients with complex neurodisability and current treatment practice in our centre in order to inform future guidelines. A retrospective case note review was undertaken at a tertiary children’s hospital. One hundred sixty-two patients (mean age 11.7 years) with a primary diagnosis of neuromuscular disease (NMD) or severe cerebral palsy (CP) and a respiratory sample sent for analysis during the study period were studied. Associations between PA in respiratory samples and diagnosis, long-term ventilation, presence of a gastrostomy or a tracheostomy, antibiotic choice, clinical deterioration and adverse events were analysed. Twenty-five (15%) had one or more PA isolate in respiratory samples. There was a significant association between PA in respiratory samples and tracheostomy (pConclusions: A larger prospective study may establish clearer criteria for guideline development. Techniques such as point-of-care testing to identify virulent strains of PA may improve patient outcomes and prevent the development of antibiotic resistance in the future. What is Known:•Children with complex neurodisability are at increased risk of respiratory morbidity and of infection with gram-negative organisms such as Pseudomonas aeruginosa.•There are currently no guidelines to inform treatment choices in this group of vulnerable children. What is New:•15% children in this study population had Pseudomonas aeruginosa in respiratory samples during a 12-month period, the majority of whom did not require critical care treatment. Thirteen of these children had a tracheostomy in situ and 12 did not. •In those that deteriorated clinically or developed antibiotic resistant organisms, earlier detection and targeted treatment of Pseudomonas aeruginosa may have prevented deterioration.

Non-invasive ventilation (NIV) is assisted mechanical ventilation delivered via a facemask for people with chronic conditions that affect breathing. NIV is most commonly delivered via an interface (mask) covering the nose (nasal mask) or the nose and mouth (oronasal mask). The number of children in the UK requiring NIV is currently estimated to be around 5000. Mass-produced masks are available for both the adult and paediatric markets but masks that fit well are difficult to find for children who are small or have asymmetrical facial features. A good conforming fit between the mask and the patient's face to minimise unintentional air leakage is essential to deliver the treatment effectively; most ventilators will trigger an alarm requiring action if such leakage is detected. We present an innovative use of 3D scanning and manufacturing technologies to deliver novel mask-face interfaces to optimise mask fit to the needs of individual patients. Ahead of planned user trials with paediatric patients, the project team trialled the feasibility of the process of creating and printing bespoke masks from 3D scan data and carried out testing of the masks in adult volunteers to select the strongest design concept for the paediatric trial. The evaluation of the process of designing a bespoke mask from scan data, arranging for its manufacture and carrying out user testing has been invaluable in gaining knowledge and discovering the pitfalls and timing bottlenecks in the processes. This allowed the team to iteratively refine the techniques and methods involved, informing user trials later on in the project. It has also provided indicative cost estimates for 3D printed mask prototype components which are useful in project decision making and trial planning. The value of the process extends to considerations for future implementation of the process within a clinical pathway.

Objective To assess the value of respiratory rate (RR) as a predictor of clinical deterioration in children, compared with other vital sign measurements. Design A retrospective case-control study, comparing children who deteriorated, requiring admission to critical care with children who did not deteriorate. Methods RR, heart rate (HR), and blood pressure (BP) measurements were collected from each patient for a 48-hour duration. The 95th centile was identified for each and 5% to 30% thresholds above the 95th centile were calculated. For each threshold the sensitivity, specificity, odds ratio, positive, and negative predictive value for deterioration was calculated. Results Forty cases (age range 7 weeks-15 years) and 40 control patients matched for age, gender, and hospital location were recruited. In 30/40 patients who deteriorated at least one RR ≥ 30% above the 95th centile for their age was recorded in the 48 hours before deterioration, compared with 10/40 controls, regardless of clinical diagnosis. Only 3/40 children that deteriorated had a HR > 30% greater than the 95th centile, compared with 2/40 controls. An elevated RR was the only vital sign whose odds ratios were significant at each threshold level above the 95th centile. Maximum RR occurred 16.8 hours before deterioration. Conclusion RR is a more accurate predictor of clinical deterioration in children than other vital signs. Greater weighting and importance should be placed on RR, which is often omitted in children due to difficulties with its measurement.

Non-invasive ventilation (NIV) is assisted respiratory support delivered via facemask for people with chronic respiratory failure. Commercial NIV masks are available but masks that fit well are difficult to find for children who have small or asymmetrical facial features. Compromised ventilation can have significant health and quality of life impacts for patients and their families. The overarching aim of the project was to improve comfort, fit and performance of NIV masks for children using 3D technology. The needs of patients, parents/carers and healthcare professionals from 6 UK centres was ensured through events advising on mask design and research procedures. 3D scanning technologies were compared in vitro using workflows and deviation analysis and with 3 healthy and 19 patient volunteers. Early design concepts were tested and the final prototype selected using a test mannequin and adult volunteers. Prototype masks were manufactured using biocompatible materials to IOS 13485 quality standards and evaluated with 19 patients. Feedback on fit, comfort and facial marking and oxicapnography data to demonstrate ventilator effectiveness were collected. The potential cost-effectiveness of the customised masks was assessed using an economic model. A handheld structured light 3D scanner was selected due to superior resolution, ease of use, availability and cost. The final design was a semi-bespoke hybrid approach. 11/15 (73%) evaluation participants reported comfort and fit that was as good or better than their usual mask. 5/11 (45%) were unable to wear the mask due to poorly fitting headgear despite a well-fitting mask. All physiological ventilation parameters were improved (table 1). Preliminary health economics analysis demonstrated that to achieve a 5% improvement in fit and ventilation, a custom-made mask would be cost effective at a price below £500. A series of in vitro, user-perspective, healthy volunteer and patient evaluations informed the development of custom-made masks for children using NIV.

Background Long term ventilation (LTV) refers to mechanical support for breathing either at home or in hospital, for all or part of the day, for at least 3 months. Two national surveys carried out in 1998 and 2008, highlighted the increasing numbers of ventilator dependent children throughout the UK . Our objective was to collect current information about children receiving LTV in the UK, 10 years after the last national survey. Methods All LTV centres in the UK completed a single time point census survey on 30th September 2019 using an electronic questionnaire. Data included the child’s location, underlying diagnosis, interface and type of respiratory support, and whether disease-modifying drugs affected the decision to initiate LTV in specific groups within this target population. Results Data was collected from 25 LTV centres. The total study population was 2383 children and young people. The median age of the overall sample was x (range x-x). 40.3% female; 57.2% of those recorded were male. Diagnoses were 417 (17.5%) central nervous system, 692 (29%) musculoskeletal and 1274 (53.5%) a respiratory, of which 31.8% of the total had upper airway obstruction. Notable changes since 1998 were the decline in the use of 24-hour ventilation, negative pressure ventilation and tracheostomy as an interface, and the increase in the proportion of patients treated at home. 115 children had received a disease-modifying drug. The use of Ataluren and Myozme did not influence the decision to treat with LTV, but in 35% of the children treated with Nusinersin the clinician stated that the use of this drug had or may have influenced their decision to initiate LTV. Conclusion The number of children being treated with LTV has increased by 250% in the last 10 years with notable changes in practice over the last 20 years.

Introduction Poor sleep is associated with many adverse outcomes in childhood. The most common sleep difficulty experienced by children is behavioural insomnia which responds to behavioural therapy. The Coronavirus disease 2019 (COVID-19) global pandemic in 2020 has had a profound effect on children’s sleep patterns. This project aimed to evaluate a one-to-one sleep service delivered via on-line clinics by community sleep practitioners in the UK. Methods The study was an observational pre- and post- evaluation in a 12-month period. The intervention is derived from the behavioural aspects of Cognitive Behavioural Therapy for insomnia. The evaluation was questionnaire-based and assessed sleep parameters and wellbeing. Results 104 parents returned completed questionnaires. Average sleep onset was 1 hour, 39 minutes pre-intervention and 20 minutes post-intervention. Average nights that the child woke per week was 3.9 pre-intervention and 0.9 post-intervention; night awakenings fell from 1.9 to 0.5 and time awake after sleep onset fell from 66. 8 minutes to 5.8 minutes. The average time slept asleep per night was 8.0 hours pre-intervention and 10.2 hours post-intervention. Improvement in all sleep measures was statistically significant (p Discussion We believe that this is the first report of a video-based clinic developed for children’s behavioural insomnia during the COVID-19 pandemic in the UK. The COVID-19 pandemic has accelerated the evolution of telehealth and, in the case of children’s sleep clinics, has provided a mode of intervention delivery that is as effective as face-to-face delivery, acceptable and accessible.

Background Children and young people (CYP) with special educational needs (SEN) are more likely to experience disturbed sleep and poorer mental wellbeing. This study explored the differential impact of the COVID-19 pandemic on the sleep and mental wellbeing of CYP with and without SEN. Methods NIHR Children and Young People MedTech Co-operative, Sheffield Children’s NHS Foundation Trust, and The Sleep Charity carried out an online survey between 23 June 2020 and 17 August 2020. The 77-item survey was shared via social media platforms. Results 559 participants were included in the analyses, 15.74% of whom reported having a CYP with SEN. While sleep changes due to the pandemic were largely similar for both groups, CYP with SEN (40.91%) were more likely to get or wake up during the night than CYP without SEN (40.91% vs 27.18%). CYP with SEN were significantly more likely than children without SEN to be demotivated (61.44% vs 31.57%), sad and tearful (36.15% vs 19.35%) or anxious and stressed (43.48% vs 14.82%) during the pandemic, as well as to report that increased anxiety was more likely to contribute to poorer sleep (43.48% vs 14.82%). Conclusion While the majority of CYP in both groups reported sleep changes due to the pandemic, CYP with SEN experienced more sleep disturbance. The findings provide initial evidence to suggest that the pandemic may have had a greater impact on the sleep and mental wellbeing of CYP with SEN compared to CYP without SEN.

Achondroplasia, the most common skeletal dysplasia, is characterized by a variety of medical, functional and psychosocial challenges across the lifespan. The condition is caused by a common, recurring, gain-of-function mutation in FGFR3, the gene that encodes fibroblast growth factor receptor 3. This mutation leads to impaired endochondral ossification of the human skeleton. The clinical and radiographic hallmarks of achondroplasia make accurate diagnosis possible in most patients. However, marked variability exists in the clinical care pathways and protocols practised by clinicians who manage children and adults with this condition. A group of 55 international experts from 16 countries and 5 continents have developed consensus statements and recommendations that aim to capture the key challenges and optimal management of achondroplasia across each major life stage and sub-specialty area, using a modified Delphi process. The primary purpose of this first International Consensus Statement is to facilitate the improvement and standardization of care for children and adults with achondroplasia worldwide in order to optimize their clinical outcomes and quality of life. Achondroplasia is the most common skeletal dysplasia and is characterized by various lifelong clinical, functional and psychosocial challenges for affected individuals. This first International Consensus Statement on the care of children and adults with achondroplasia aims to facilitate the global standardization and improvement of achondroplasia clinical care.

Sleep-related Rhythmic Movement Disorder (RMD) affects around 1% of UK pre-school children. Little is known about RMD in Down syndrome (DS). We aimed to determine: (a) the prevalence of RMD in children with DS aged 1.5–8 years, (b) phenotypic and sleep quality differences between children with DS and RMD and sex- and age-matched DS controls, and (c) night-to-night variability in rhythmic movements (RMs). Parents who previously reported RMs from a DS research registry of 202 children were contacted. If clinical history suggested RMD, home videosomnography (3 nights) was used to confirm RMs and actigraphy (5 nights) was used to assess sleep quality. Phenotype was explored by demographic, strengths and difficulties, Q-CHAT-10/social communication and life events questionnaires. Eight children had confirmed RMD. Minimal and estimated maximal prevalence were 4.10% and 15.38%, respectively. Sleep efficiency was significantly lower in RMD-cases (69.1%) versus controls (85.2%), but there were no other phenotypic differences. There was considerable intra-individual night-to-night variability in RMs. In conclusion, RMD has a high prevalence in children with DS, varies from night to night and is associated with poor sleep quality but, in this small sample, no daytime phenotypic differences were found compared to controls. Children with DS should be screened for RMD, which is amenable to treatment.

BACKGROUND: The choice of antibiotic, and the use of single or combined therapy are controversial areas in the treatment of respiratory infection due to Pseudomonas aeruginosa in cystic fibrosis (CF). Advantages of combination therapy include wider range of modes of action, possible synergy and reduction of resistant organisms; advantages of monotherapy include lower cost, ease of administration and reduction of drug‐related toxicity. Current evidence does not provide a clear answer and the use of intravenous antibiotic therapy in CF requires further evaluation. This is an update of a previously published review. OBJECTIVES: To assess the effectiveness of single compared to combination intravenous anti‐pseudomonal antibiotic therapy for treating people with CF. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. Most recent search of the Group's Trials Register: 07 October 2020. We also searched online trials registries on 16 November 2020. SELECTION CRITERIA: Randomised controlled trials (RCTs) comparing a single intravenous anti‐pseudomonal antibiotic with a combination of that antibiotic plus a second anti‐pseudomonal antibiotic in people with CF. DATA COLLECTION AND ANALYSIS: Two authors independently assessed trial quality and extracted data. We assessed the certainty of the data using GRADE. MAIN RESULTS: We identified 59 trials, of which we included eight trials (356 participants) comparing a single anti‐pseudomonal agent to a combination of the same antibiotic and one other. There was a wide variation in the individual antibiotics used in each trial. In total, the trials included seven comparisons of a beta‐lactam antibiotic (penicillin‐related or third generation cephalosporin) with a beta‐lactam‐aminoglycoside combination and three comparisons of an aminoglycoside with a beta‐lactam‐aminoglycoside combination. There was considerable heterogeneity amongst these trials, leading to difficulties in performing the review and interpreting the results. These results should be interpreted cautiously. Six of the included trials were published between 1977 and 1988; these were single‐centre trials with flaws in the randomisation process and small sample size. Overall, the methodological quality was poor and the certainty of the evidence ranged from low to moderate. The review did not find any differences between monotherapy and combination therapy in either the short term or in the long term for the outcomes of different lung function measures, bacteriological outcome measures, need for additional treatment, adverse effects, quality of life or symptom scores. AUTHORS' CONCLUSIONS: The results of this review are inconclusive. The review raises important methodological issues. There is a need for an RCT which needs to be well‐designed in terms of adequate randomisation allocation, blinding, power and long‐term follow‐up. Results need to be standardised to a consistent method of reporting, in order to validate the pooling of results from multiple trials.

Children with complex neurodisability have increased risk of respiratory tract infections. Pseudomonas aeruginosa (PA) is an organism that commonly infects and colonises the airway. Treatment choices are complicated by adverse effects and development of antibiotic resistant organisms. There are no established guidelines and the treatment approach of clinicians varies. A retrospective case notes review was undertaken to look at the prevalence of PA in respiratory samples of children with neurodisability, treatment practices and outcomes. Of 226 children with a neuromuscular condition or cerebral palsy (GMFCS score 4-5), 25 had at least one PA positive sample during a 12-month period. Those using a tracheostomy were twice as likely to isolate PA; 21 (80%) had recurrent isolates, suggesting colonisation of the airway. 62 individual clinical episodes were identified. Patients most likely to deteriorate clinically were those treated with broad-spectrum antibiotics (41%) compared with PA-specific (27%) or no antibiotics (11%). All 10 patients in whom antibiotics were changed as directed by microbial results, improved clinically thereafter. No adverse effects were documented as a direct result of antibiotics. Antibiotic resistance was documented in 8/21 colonised patients with these more likely to require escalation to intensive care treatment. A guideline for management and more sophisticated methods to direct treatment choices are needed. Techniques such as the ability to identify virulent strains of PA using point-of-care testing may improve patient outcomes and prevent the development of antibiotic resistance in the future.

In order for inhaled corticosteroids to be delivered adequately to the airways they require patients to take them regularly using an effective technique. Patients often have a poor inhaler technique, and this has been shown to result in sub-optimal asthma control. It is important for all clinicians prescribing inhaled medication to be experienced in the correct technique, and take time to train children so that they have mastered corrected inhaler technique. Using Teach to Goal or teach back methodology is a simple and effective way to provide this in the clinic setting. More than one training session is typically needed before children can master correct inhaler technique. Adherence to inhaled therapy has been shown to be sub-optimal in pediatric populations, with studies showing an average rate of around 50%. Subjective methods of measuring adherence have been shown to be inaccurate and overestimate rates. The advent of new technology has allowed adherence rates to be measured electronically, and it has been shown that regular feedback of these data can be effective at improving asthma control. New mobile apps and smart technology aim to engage patients and families with their asthma care. Effective use of these apps in collaboration with health care professionals has a vast potential to improve adherence rates and inhaler technique, resulting in improved asthma control.

Study objectives Obstructive sleep apnea (OSA) is common in children with Down syndrome (DS) and is associated with adverse health and cognitive outcomes. Daytime clinical assessment is poorly predictive of OSA, so regular screening with sleep studies is recommended. However, sleep studies are costly and not available to all children worldwide. We aimed to evaluate the psychometric properties and predictive value of a newly developed screening questionnaire for OSA in this population. Methods 202 children aged 6 months to 6th birthday with DS were recruited, of whom 188 completed cardio-respiratory sleep studies to generate an obstructive apnea hypopnea index (OAHI). Parents completed the 14-item Down syndrome OSA screening questionnaire. Responses were screened, a factor analysis undertaken, internal consistency calculated and receiver operator characteristic (ROC) curves drawn to generate an area under the curve (AUC) to assess criterion related validity. Results Of 188 children who completed cardiorespiratory sleep studies; parents completed the screening questionnaire for 186. Of this study population 15.4% had moderate to severe OSA defined by an OAHI of ≥5/h. Sixty-three (33.9%) participants were excluded due to "unsure" responses or where questions were not answered. Using the remaining 123 questionnaires a four-factor solution was found, with the 1st factor representing breathing related symptoms, explaining a high proportion of the variance. Internal consistency was acceptable with a Cronbach alpha of 0.87. ROC curves for the total score generated an AUC statistic of 0.497 and for the breathing subscale an AUC of 0.603 for moderate to severe OSA. Conclusion A well designed questionnaire with good psychometric properties had limited predictive value to screen for moderate to severe OSA in young children with DS. The use of a screening questionnaire is not recommended. Screening for OSA in this population requires objective sleep study measures.

ObjectiveTo evaluate the success rates of home cardiorespiratory polygraphy in children under investigation for sleep-disordered breathing and parent perspectives on equipment use at home.DesignProspective observational study.SettingSheffield, Evelina London and Southampton Children’s Hospitals.PatientsData are reported for 194 research participants with Down syndrome, aged 0.5–5.9 years across the three centres and 61 clinical patients aged 0.4–19.5 years from one centre, all of whom had home cardiorespiratory polygraphy including respiratory movements, nasal pressure flow, pulse oximetry, body position and motion.Main outcome measuresPercentage of home cardiorespiratory studies successfully acquiring ≥4 hours of artefact-free data at the first attempt. Parental report of ease of use of equipment and preparedness to repeat home diagnostics in the future.Results143/194 (74%; 95% CI 67% to 79%) of research participants and 50/61 (82%; 95% CI 71% to 90%) of clinical patients had successful home cardiorespiratory polygraphy at the first attempt. Some children required multiple attempts to achieve a successful study. Overall, this equated to 1.3 studies per research participant and 1.2 studies per clinical child. The median artefact-free sleep time for successful research studies was 515 min (range 261–673) and for clinical studies 442 min (range 291–583). 84% of research and 87% of clinical parents expressed willingness to repeat home cardiorespiratory polygraphy in the future. 67% of research parents found the equipment ‘easy or okay’ to use, while 64% of clinical parents reported it as ‘easy’ or ‘very easy’.ConclusionsHome cardiorespiratory polygraphy offers an acceptable approach to the assessment of sleep-disordered breathing in children.

BackgroundThere is uncertainty about the most appropriate ways to manage non-respiratory sleep disturbances in children with neurodisabilities (NDs).ObjectiveTo assess the clinical effectiveness and safety of NHS-relevant pharmacological and non-pharmacological interventions to manage sleep disturbance in children and young people with NDs, who have non-respiratory sleep disturbance.Data sourcesSixteen databases, including The Cochrane Central Register of Controlled Trials, EMBASE and MEDLINE, were searched up to February 2017, and grey literature searches and hand-searches were conducted.Review methodsFor pharmacological interventions, only randomised controlled trials (RCTs) were included. For non-pharmacological interventions, RCTs, non-randomised controlled studies and before-and-after studies were included. Data were extracted and quality assessed by two researchers. Meta-analysis and narrative synthesis were undertaken. Data on parents’ and children’s experiences of receiving a sleep disturbance intervention were collated into themes and reported narratively.ResultsThirty-nine studies were included. Sample sizes ranged from 5 to 244 participants. Thirteen RCTs evaluated oral melatonin. Twenty-six studies (12 RCTs and 14 before-and-after studies) evaluated non-pharmacological interventions, including comprehensive parent-directed tailored (n = 9) and non-tailored (n = 8) interventions, non-comprehensive parent-directed interventions (n = 2) and other non-pharmacological interventions (n = 7). All but one study were reported as having a high or unclear risk of bias, and studies were generally poorly reported. There was a statistically significant increase in diary-reported total sleep time (TST), which was the most commonly reported outcome for melatonin compared with placebo [pooled mean difference 29.6 minutes, 95% confidence interval (CI) 6.9 to 52.4 minutes;p = 0.01]; however, statistical heterogeneity was extremely high (97%). For the single melatonin study that was rated as having a low risk of bias, the mean increase in TST was 13.2 minutes and the lower CI included the possibility of reduced sleep time (95% CI –13.3 to 39.7 minutes). There was mixed evidence about the clinical effectiveness of the non-pharmacological interventions. Sixteen studies included interventions that investigated the feasibility, acceptability and/or parent or clinician views of sleep disturbance interventions. The majority of these studies reported the ‘family experience’ of non-pharmacological interventions.LimitationsPlanned subgroup analysis was possible in only a small number of melatonin trials.ConclusionsThere is some evidence of benefit for melatonin compared with placebo, but the degree of benefit is uncertain. There are various types of non-pharmacological interventions for managing sleep disturbance; however, clinical and methodological heterogeneity, few RCTs, a lack of standardised outcome measures and risk of bias means that it is not possible to draw conclusions with regard to their effectiveness. Future work should include the development of a core outcome, further evaluation of the clinical effectiveness and cost-effectiveness of pharmacological and non-pharmacological interventions and research exploring the prevention of, and methods for identifying, sleep disturbance. Research mapping current practices and exploring families’ understanding of sleep disturbance and their experiences of obtaining help may facilitate service provision development.Study registrationThis study is registered as PROSPERO CRD42016034067.FundingThe National Institute for Health Research Health Technology Assessment programme.

Narcolepsy is a relatively common neurological condition affecting the regulation of normal sleep/wake cycles leading to excessive daytime sleepiness (EDS). It is almost certainly under-recognised as it has a prevalence of 20–50 per 100,000 population and most cases have an onset in adolescence. Cataplexy (attacks of muscle weakness often precipitated by strong emotions) is a hallmark of this condition and represents the intrusion of REM sleep into wakefulness. Narcolepsy is caused by destruction of hypocretin producing cells due to an autoimmune process often by an infective trigger. Hypocretin is found in the hypothalamus and plays a role in stabilisation of the transition between wake and sleep states. In establishing a diagnosis a comprehensive history to exclude other causes of EDS, including poor sleep habits, is essential. Primary sleep related conditions such as sleep apnoea should be excluded. Investigations for confirmation of the diagnosis include Actigraphy, Polysomnography (PSG), Multiple Sleep Latency Testing (MSLT) and CSF analysis. The symptoms of this debilitating condition can have a huge impact on a child's life and are often vastly underestimated. The impact of EDS on cognitive function is an important factor in difficulties at school, mood, quality of life and future career opportunities. Advances in understanding the pathophysiology have led to trials of novel treatment approaches. The aim of this article is to briefly summarise the recent advances in understanding and give an overview of this important condition for those who are involved in the care of a child with this disease.

Introduction Paper sleep diaries and actigraphy are useful tools in Sleep Medicine to evaluate sleep wake patterns in the home environment over the course of 7–14 days. In our service, the demand for actigraphy far outweighs the available resources. As such the team are investigating using an electronic sleep diary, integrating with actigraphy and simplifying the whole process. The first stage of this process was to ask the stakeholders their opinions of sleep diaries and actigraphy. Methods Stakeholders were asked about sleep diaries and actigraphy considering all aspects of the process from when to request a referral and organising collection, completing a diary and wearing actigraphy, through to analysing and interpreting results. Questions asked were appropriate to the different stakeholder groups involved. Families and local hospital staff were interviewed and British Paediatric Sleep Society (BPSA) members and families undergoing actigraphy were sent appropriate brief questionnaires relevant to their part of the pathway [Service evaluation SE1347]. Results 47 stakeholders provided feedback. Interviews were conducted with 14 staff, 9 parents and 3 young people. Additional questionnaires were completed by 8 families undergoing actigraphy and 13 BPSA responders. A summary of the most common responses is shown in table 1. Most institutions were issuing sleep diaries and actigraphy to specific patient groups due to limited resources. Discussion This stakeholder engagement provides useful information to improve the use of sleep diaries and actigraphy within our paediatric clinical setting. If the process can be simplified then more sleep diary and actigraphy usage can occur at the triage stage of the sleep referral process and prevent unnecessary sleep studies, benefitting both families and limited resources. Feedback received confirms the current solution for paediatric sleep diaries and actigraphy is not ideal and there is a need to identify new approaches to increase usability, acceptability and stakeholder’s satisfaction.

Introduction Many children with sleep difficulties are prescribed melatonin. However, melatonin is costly and evidence for its efficacy is limited. The aim of this study was to evaluate a nurse-led sleep clinic to look at outcomes in terms of melatonin prescribing. Methods All new referrals attending a nurse-led sleep clinic were examined from June 2016 to March 2017. Patients/parents attending the clinic were given an individualised sleep programme to implement with their child at home, with ongoing follow-up support. Families received an average of 3 face-to-face clinic visits and 3 follow-up telephone calls from first visit to discharge. Retrospective data was gathered from clinic records [Clinical Audit approval number CA1309]. Results 69 patients aged 1–17 years (31 males) were analysed. The primary complaint was ‘problem with sleep initiation and maintenance’ (81%), ‘problem with sleep initiation’ (13%) and ‘problem with sleep maintenance’ (6%). 84% of patients had medical co-morbidities; 54% of which were neuro-disability, the commonest being ASD and/or ADHD. 40/69 patients were successfully discharged from the clinic during the evaluation period. 65% were discharged without melatonin (23% with neurodisability; 42% without neurodisability), of which 35% were weaned off melatonin and 30% avoided melatonin prescriptions. 12% felt that the sleep intervention had been successful but still required melatonin, 15% were referred to medical/psychology clinics, and 8% were non-compliant with the behavioural sleep programme and were discharged still taking melatonin. Discussion In this cohort of paediatric insomnia patients, a brief but intensive behavioural programme with support from a specialist sleep nurse was effective in resolving sleep difficulties and reducing melatonin usage. We recommend that standardised sleep support from trained practitioners should be available prior to prescribing melatonin for children with sleep difficulties. This approach is not only beneficial in effectively resolving sleep problems but is cost-effective when compared with melatonin prescribing.

Introduction Narcolepsy occurs due to an inability to regulate the sleep-wake cycle, causing disturbed night-time sleep and excessive daytime sleepiness. Children and young people with narcolepsy have increased psychiatric illness, compared to healthy controls. A recent study of 31 paediatric narcolepsy patients found 43% had psychiatric comorbidity, compared with 10% in the general population.1 The aim of this service evaluation was to determine whether Sheffield Children’s Hospital narcolepsy patients have increased psychological morbidity in order to inform service development. Methods 43 patients, aged 4–16 years, and their parents were given Revised Children’s Anxiety and Depression Scale (RCADS) questionnaires in clinic [Service Evaulation SE1473]. Software produced by the developer was used to analyse the results, using data from a cohort of children and parents separated by gender and American school grade. Scores are divided into: Separation Anxiety, General Anxiety, Panic, Social Phobia, Obsessive Compulsive Disorder and Depression. Results 34 patients and parents completed their questionnaires. A positive score was defined by a T score>65, indicating clinically significant anxiety or depression. 16 patients scored positively based on their questionnaires and 25 scored positively based on their parent’s questionnaires, giving psychological morbidity rates of 47% and 74% respectively. The category scored positively in most frequently was depression: 15 patients and 23 parents’ questionnaires scored positively here. Scoring for anxiety was lower: 13 in the parent and 2 in the patient questionnaires. Discussion This service evaluation showed raised levels of anxiety and depression in Sheffield Children’s Hospital narcolepsy children and young people, compared with national averages. Regular psychological assessment and early intervention for patients would be appropriate, given the results shown here and these results will be put forward as part of a future business case. Reference Szakacs, et al. Psychiatric comorbidity and cognitive profile in children with narcolepsy with or without association to the H1N1 influenza vaccination. Sleep 2015.

Introduction The current gold standard sensors to measure airflow in apnoea and hypopnoea detection are the oronasal thermal airflow sensor and the nasal pressure transducer, respectively.1 Due to the contact nature of these sensors in the nasal region, they are poorly tolerated by children. In a recent audit, we found that 50% of children refused the nasal pressure sensor due to its invasiveness, and of those that accepted it, 64% removed the prongs over the course of the night. The aim of the current study was to evaluate a non-contact method to monitor respiration by developing infrared thermal imaging, whereby temperature fluctuations associated with respiration are measured and correlated with airflow. Methods 11 healthy adult volunteers participated following University Ethics. Respiratory signals were recorded over four simulated apnoea scenarios resembling normal respiration; central; obstructive and hypopnoeic pauses. Simulated apnoeas were measured via nasal pressure cannula and respiratory inductance bands [SOMNOtouchTM RESP; SOMNOmedics, Germany] versus thermal imaging techniques. Results 70% of the apnoea episodes correlated with airflow sensor readings (Example trace in figure 1). In 16% of recordings the subject’s head position did not allow correct identification of the region of interest (i.e. the nostrils). For the remaining 14% of cases there was partial agreement between the thermal measurements and nasal pressure readings. Discussion These results indicate thermal imaging may be valuable as a detection tool for sleep apnoea, particularly in the case of paediatric patients whose tolerance for contact nasal sensors is poor. Reference Berry RB, Albertario CL, Harding SM, et al. for the American Academy of Sleep Medicine. The AASM Manual for the Scoring of Sleep and Associated Events: Rules, Terminology and Technical Specifications. Version 2.5. Darien, IL: American Academy of Sleep Medicine, 2018.

Introduction Narcolepsy is a chronic neurological condition in which the brain’s ability to regulate the sleep-wake cycle is disturbed and can result in reduced quality of life (QOL). A positive association between exercise and QOL has been found in children. The aim of this study was to assess the feasibility of implementation of an exercise intervention with a mobile app to monitor outcomes in children with narcolepsy. Methods This feasibility study received service evaluation (SE1308), and University of Sheffield ethical approval (project number: 022995). A patient and parent engagement exercise to inform study design was carried out during the ‘Sheffield Children’s Hospital Annual Narcolepsy Family Day’. An exercise intervention was designed based upon NHS recommendations and tailored to the child’s activity levels.1 Weekly goals were set and progress was reviewed weekly, using an app specifically developed by Aparito Ltd (Wrexham, UK). QOL was monitored using the Pediatric Quality of Life Inventory (PedsQL). Patients were asked for feedback at the end of the intervention, to assess its feasibility and acceptability. Results 117 children and parents took part in the engagement exercise. 12 patients took part in the feasibility study (mean age 12.9 years). The focus group highlighted the importance of motivation, timing and tailoring exercise interventions to the individual. Children said exercise should be fun to help motivation. Average physical activity increased from 229 minutes to 254 minutes. Of those who had successfully increased their exercise, baseline average PedsQL score increased from 70.6 to 77.6 (figure 1). Feedback on the app and exercise intervention were both positive. Conclusion This feasibility study has shown that a targeted exercise intervention can improve QOL in children with narcolepsy. The Aparito app, it is being developed further for children with narcolepsy. Reference Health Survey for England 2015: Physical Activity for Children

Objective/Background: children with Down syndrome (DS) commonly experience difficulties with executive function (EF). They are also vulnerable to obstructive sleep apnoea (OSA). OSA is associated with EF deficits in typically developing children. A recent study reported an association between OSA and cognitive deficits in 38 school-aged children with DS. We experimentally investigated EF behaviours in young children with DS, and their association with OSA. Participants and Methods: children with DS were recruited to take part in a larger study of OSA (N=202). Parents of 80 children (50 male) aged 36 to 71 months (M = 56.90, SD = 10.19 months) completed the Behavior Rating Inventory of Executive Function - Preschool Version (BRIEF-P). Of these 80 children, 69 were also successfully studied overnight with domiciliary cardiorespiratory polygraphy to diagnose OSA. Results: obstructive apnoea/hypopnoea index was in the normal range (0-1.49/h) for 28 children but indicated OSA (≥1.5/h) in 41 children. Consistent with previous research, we found a large effect for children experiencing particular weaknesses in working memory, planning and organising, whilst emotional control was a relative strength. OSA was associated with poorer working memory (β=.23, R2=.05, p=.025), emotional control (β=.20, R2=.04, p=.047) and shifting (β=.24, R2=.06, p=.023). Conclusions: findings suggest that known EF difficulties in DS are already evident at this young age. Children with DS already have limited cognitive reserve and can ill afford additional EF deficit associated with OSA. OSA is amendable to treatment and should be actively treated in these children to promote optimal cognitive development.

ObjectivesTo assess whether a core outcome set is required for studies evaluating the effectiveness of interventions for non-respiratory sleep disturbances in children with neurodisabilities.DesignSurvey of outcome measures used in primary studies identified by a systematic review.Data sourcesASSIA, CENTRAL, Cochrane Database of Systematic Reviews, Conference Proceedings Citation Index, CINAHL, DARE, Embase, HMIC, MEDLINE, MEDLINE In-Process, PsycINFO, Science Citation Index, Social Care Online, Social Policy & Practice, ClinicalTrials.gov, WHO International Clinical Trials Registry Platform and the UK Clinical Trials Gateway were searched up to February 2017.Eligibility criteriaStudies evaluating pharmacological or non-pharmacological interventions for children (≤18 years old) with a neurodisability and experiencing non-respiratory sleep disturbance.Data extraction and synthesisOutcome measures were listed from each study and categorised into domains.ResultsThirty-nine studies assessed five core outcome areas: child sleep, other child outcomes, parent outcomes, adverse events and process measures. There were 54 different measures of child sleep across five domains: global measures; sleep initiation; maintenance; scheduling; and other outcomes. Fifteen non-pharmacological (58%) and four pharmacological studies (31%) reported child outcomes other than sleep using 29 different measures.One pharmacological and 14 non-pharmacological (54%) studies reported parent outcomes (17 different measures). Eleven melatonin studies (85%) recorded adverse events, with variation in how data were collected and reported. One non-pharmacological study reported an explicit method of collecting on adverse events. Several process measures were reported, related to adherence, feasibility of delivery, acceptability and experiences of receiving the intervention.ConclusionsThere is a lack of consistency between studies in the outcome measures used to assess the effectiveness of interventions for non-respiratory sleep disturbances in children with neurodisabilities. A minimum core outcome set, with international consensus, should be developed in consultation with parents, children and young people, and those involved in supporting families.PROSPERO registration numberCRD42016034067

Aim Many children with sleep difficulties are prescribed melatonin, a hormone playing a key role in the timing of sleep-wake cycle, however the drug is costly and evidence for its efficacy is limited. The aim of this study was to evaluate a nurse-led sleep clinic, in which a behavioural sleep programme is delivered, to look at outcomes in terms of melatonin prescribing. Methods All new referrals attending a nurse-led Sleep Clinic were examined over a 9 month period from June 2016 to March 2017. Patients attending the clinic were given an individualised sleep programme for the parent to implement with their child at home, with ongoing support from the sleep nurse. Families received an average of 3 face to face clinic visits and 3 follow up telephone calls from first visit to discharge. Retrospective data was gathered from clinic records. Melatonin prescribing data was analysed. Results 69 patients aged 1–17 years (45% male; 55% female) were analysed. The primary complaint was ‘problem with sleep initiation and maintenance’ in 81%, ‘problem with sleep initiation’ in 13% and ‘problem with sleep maintenance’ in 6%. Of the 69 patients, 84% had medical co-morbidities. 54% of which were neuro-disability, the commonest being Autistic Spectrum Disorder and/or Attention Deficit Hyperactivity Disorder. 40/69 patients were successfully discharged from the clinic during the study period. 65% were discharged without melatonin (23% with neurodisability; 42% without neurodisability), of which 35% were weaned off melatonin and 30% avoided melatonin prescriptions. 12% felt that the sleep intervention had been successful but still required melatonin, 15% were referred to medical/psychology clinics, and 8% were non-compliant with the behavioural sleep programme and were discharged still taking melatonin. Conclusion In this cohort of patients with sleep initiation and maintenance problems despite taking melatonin, a brief but intensive behavioural programme with support from a sleep nurse was effective in resolving the sleep problems. We recommend that standardised sleep support from trained practitioners should be available prior to prescribing melatonin for children suffering sleep difficulties. This approach is not only beneficial in effectively resolving sleep problems but is cost-effective when compared with melatonin prescribing costs.

Aim A partnership involving a Children’s Trust, the City Council and a Sleep Charity evaluated a behavioural intervention to provide support to parent/carers and young people to improve sleep patterns. Methods The intervention entailed basic education about sleep, a one-to-one session with a sleep practitioner to create an individual sleep programme and telephone support to empower the parent to carry out the sleep programme at home. Results 39 children completed the intervention and evaluation, median age 8.56 years (range 1.82–15.75 years). 79.5% were male. 75% had a diagnosis of ADHD or were awaiting assessment, the remaining 25% were Looked After or Adopted Children (of whom 10% also had ADHD). Parents’ ratings of their child’s ability to self-settle to sleep improved from 1.13/10 to 6.73/10 after the intervention (MD 5.62, 95% confidence intervals 4.56–6.69, p The primary word used to describe the mood of the child on wakening before the intervention was ‘grumpy’ and after the intervention was ‘happy’. The impact of sleep deprivation on the parents’ wellbeing improved for all measures. The overall WEMWBS score improved significantly following the intervention (MD 8.84, 95% CI 5.32–12.36, p Conclusion The evaluation gave us confidence in the delivery model. ‘Regular telephone calls and support’ and ‘Learning about sleep’ were the main positive factors. Our partnership working brought together the individual strengths, drive and passion that were critical for delivery, planning, and influencing better provision for families. We have established a strategic group to support local implementation and produced a draft delivery model which we believe is replicable for other areas.

Aims To compare sleep in infants and toddlers with Down syndrome (DS) to typically developing controls, including differences in snoring and sleep ecology (sleep setting and parent behaviors).MethodsParents of 104 children with DS and 489 controls aged 6-36 months completed the Brief Infant Sleep Questionnaire. We explored group differences, controlling for demographic variables.Results Parents of children with DS reported more sleep problems (45% v 19%), snoring (19% vs 2%), room-sharing (37% vs 17%), and less night-time sleep (55 mins) and total sleep over 24 hours (38 mins). They were more likely to be present when their child fell asleep (OR 4.40). Snoring increased night waking but did not limit night-time/24-hour sleep. However, parental presence was associated with 55 minutes less night-time and 64 minutes less 24-hour sleep. After controlling for snoring and parental presence, children with DS slept less at night (38 mins) but more in the day (21 mins) with no significant difference in 24-hour sleep. ConclusionsOverall, significant differences in sleep patterns, problems, and ecology were found between children with DS and controls. Parental presence at settling, not snoring, explained most differences, including over an hour’s less 24-hour sleep. Early intervention programmes that promote self-soothing skills could prevent the burden of sleep loss in young children with DS.

AimsWe describe how patient and public involvement (PPI) was integrated into the design of an intervention for a randomised controlled trial (RCT) based within primary care. The RCT, known as the PLEASANT trial, aimed to reduce unscheduled medical contacts in children with asthma associated with start of the new school year in September with a simple postal intervention, highlighting the importance of maintaining asthma medication for helping to prevent increased asthma exacerbations.BackgroundPPI is a key feature of UK health research policy, and is often a requirement of funding from the National Institute for Health Research. There are few detailed accounts of PPI in the design and conduct of clinical trials in the PPI literature for researchers to learn from.MethodsWe held PPI consultation events to determine whether the proposed intervention for the trial was acceptable to children with asthma and their parents, and to ascertain whether enhancements should be made. Two PPI consultation events were held with children with asthma and their parents, prior to the research commencing. Detailed field notes were taken by the research team at each consultation event.FindingsAt the first consultation event, parents and children endorsed the trial’s rationale, made suggestions to the wording of the trial intervention letter, and made recommendations about to whom the letter should be sent out. At the second consultation event, parents discussed the timing of the intervention, commented on the lay summary of the Research Ethics Application, and were invited to join the trial’s steering committee, while the children selected a logo for the study. PPI has resulted in enhancements to the PLEASANT study’s intervention. A further PPI consultation event is scheduled for the end of the trial, in order for children with asthma and their parents to contribute to the trial’s dissemination strategy.

Background: Respiratory rate is a vital physiological measurement used in the immediate assessment of unwell children and adults. Convenient electronic devices exist for the measurement of pulse, blood pressure, oxygen saturation, and temperature. Although devices which measure respiratory rate exist, none have entered everyday clinical practice for acute assessment of children and adults. An accurate and practical device which has no physical contact with the patient is important to ensure readings are not affected by distress caused by the assessment method. Objective: The aim of this study was to evaluate the use of a thermal imaging method to monitor the respiratory rate in children and adults. Methods: Facial thermal images of adult volunteers and children undergoing elective polysomnography were included. Respiration was recorded for at least 2 min with the camera positioned 1 m from the subject’s face. Values obtained using the thermal imaging camera were compared with those obtained from contact methods such as the nasal thermistor, respiratory inductance plethysmography, nasal airflow, and end tidal CO2. Results: A total of 61 subjects, including 41 adults (age range 27–46 years) and 20 children (age range 0.5–18 years) were enrolled. The correlation between the respiratory rate measured using thermal imaging and the contact method was r = 0.94. Sequential refinements to the thermal imaging algorithms resulted in the ability to perform real-time measurements and an improvement of the correlation to r = 0.995. Conclusion: This exploratory study shows that thermal imaging-derived respiratory rates in children and adults correlate closely with the best performing standard method. With further refinements, this method could be implemented in both acute and chronic care in children and adults.

We read with interest the article by Leong et al on the use of polysomnography (PSG) in children,1 covering indications for PSG, along with limitations of oximetry, and clearly outlining how to undertake and interpret PSG in paediatric patients. It briefly discusses limited channel recordings (respiratory polygraphy, RP) and concludes that this ‘is not standard practice’. In many paediatric centres RP is standard practice, and routinely used for assessment of sleep-disordered breathing (SDB) in children, with the most common diagnosis being obstructive sleep apnoea (OSA). In a recent survey …

Aim To evaluate the effectiveness of pharmacological interventions for managing non‐respiratory sleep disturbances in children with neurodisabilities. Method We performed a systematic review and meta‐analyses of randomized controlled trials (RCTs). We searched 16 databases, grey literature, and reference lists of included papers up to February 2017. Data were extracted and assessed for quality by two researchers (B.B., C.M., G.S., A.S., A.P.). Results Thirteen trials were included, all evaluating oral melatonin. All except one were at high or unclear risk of bias. There was a statistically significant increase in diary‐reported total sleep time for melatonin compared with placebo (pooled mean difference 29.6min, 95% confidence interval [CI] 6.9–52.4, p=0.01). Statistical heterogeneity was high (97%). For the single RCT with low risk of bias, the unadjusted mean difference in total sleep time was 13.2 minutes (95% CI −13.3 to 39.7) favouring melatonin, while the mean difference adjusted for baseline total sleep time was statistically significant (22.4min, 95% CI 0.5–44.3, p=0.04). Adverse event profile suggested that melatonin was well‐tolerated. Interpretation There is a paucity of evidence on managing sleep disturbances in children with neurodisabilities, and it is mostly of limited scope and poor quality. There is evidence of the benefit and safety of melatonin compared with placebo, although the extent of this benefit is unclear. What this paper adds Melatonin for the management of non‐respiratory sleep disturbances in children with neurodisabilities was well tolerated with minimal adverse effects.The extent of benefit and which children might benefit most from melatonin use is uncertain.Benefit may be greatest in those with autism spectrum disorder; however, this finding should be interpreted with caution., What this paper adds Melatonin for the management of non‐respiratory sleep disturbances in children with neurodisabilities was well tolerated with minimal adverse effects.The extent of benefit and which children might benefit most from melatonin use is uncertain.Benefit may be greatest in those with autism spectrum disorder; however, this finding should be interpreted with caution. This article's abstract has been translated into Spanish and Portuguese. Follow the links from the abstract to view the translations.

Aim To describe existing evidence on non-pharmacological interventions to manage sleep disturbance in children with neurodisabilities. Method We systematically reviewed non-pharmacological interventions aimed at improving non-respiratory sleep disturbance in children with neurodisability. Sixteen databases, grey literature, and reference lists of included papers were searched up to February 2017. Two researchers (B.B., C.M., G.S., A.S., A.P.) undertook screening, data extraction, and quality appraisal. Results Twenty-five studies were included: 11 randomized controlled trials and 14 before-and-after studies. All studies were at high or unclear risk of bias. Parent-directed interventions were categorized as comprehensive tailored interventions (n=9), comprehensive non-tailored interventions (n=8), and non-comprehensive interventions (n=2). Six 'other' non-pharmacological interventions were included. Seventy-one child and parent sleep-related outcomes were measured across the included studies. We report the two most commonly measured outcomes: the Child Sleep Habits Questionnaire and sleep onset latency. Five studies reported significant improvements on at least one of these outcomes. Interpretation Various types of non-pharmacological intervention for managing sleep disturbance have been evaluated. Clinical heterogeneity and poor study quality meant we could not draw definitive conclusions on the effectiveness of these interventions. Current clinical guidance recommends parent-directed interventions as the first approach to managing sleep disturbance; prioritizing research in this area is recommended. What this paper adds Existing evidence on non-pharmacological interventions to manage sleep disturbance in children with neurodisabilities is predominately of poor quality. Most included studies evaluated parent-directed interventions of varying content and intensity. There was very little consistency between studies in the outcome measures used. There is some evidence that parent-directed interventions may improve child outcomes.

Objective To determine the inter-observer agreement of a respiratory rate (RR) count on a child when assessed by three independent observers. Design The RR of 169 children (age range: 3 days to 15 years) was measured by three independent observers over a 3-month period. The first RR was taken by different healthcare professionals (HCPs) from within the hospital using their own preferred method of measurement. A further count of RR was then taken by two observers from the research team simultaneously within 30 min of the first measurement, using the WHO-recommended method of measurement. Results 507 RR measurements were taken on 169 children. Median RR showed a 4 beats per minute (bpm) difference between the HCP (median RR 32 bpm) and the researchers (median RR 28 bpm). The 95% limits of agreement between the first measurement and second and third measurements were −10.2 to 17.7 bpm and −11.4 to 18.7 bpm, respectively. For simultaneous measurements, the 95% limits of agreement were −7.1 to 7.0 bpm. 81 children had a RR > 95th centile for their age and an even poorer level of agreement was seen in these children than in those whose RR was within normal range. In only 27 of these 81 children (33%) did all three observers agree on the presence of a raised RR. Conclusions Inter-observer agreement for the measurement of RR in children is poor. The effect that this variation has on the clinical assessment and subsequent management of a child may be significant. These findings highlight the need for a robust review of our current measurement methods and interpretation of an important vital sign.

Objective Children with Down syndrome are at high risk of obstructive sleep apnoea (OSA) and screening is recommended. Diagnosis of OSA should be confirmed with multichannel sleep studies. We aimed to determine whether home pulse oximetry (HPO) discriminates children at high risk of OSA, who need further diagnostic multichannel sleep studies.Design Cross-sectional prospective study in a training sample recruited through three UK centres. Validation sample used single-centre retrospective analysis of clinical data.Patients Children with Down syndrome aged 0.5–6 years.Intervention Diagnostic multichannel sleep study and HPO.Main outcome measures Sensitivity and specificity of HPO to predict moderate-to-severe OSA.Results 161/202 children with Down syndrome met quality criteria for inclusion and 25 had OSA. In this training sample, the best HPO parameter predictors of OSA were the delta 12 s index >0.555 (sensitivity 92%, specificity 65%) and 3% oxyhaemoglobin (SpO2) desaturation index (3% ODI)>6.15 dips/hour (sensitivity 92%, specificity 63%). Combining variables (delta 12 s index, 3% ODI, mean and minimum SpO2) achieved sensitivity of 96% but reduced specificity to 52%. All predictors retained or improved sensitivity in a clinical validation sample of 50 children with variable loss of specificity, best overall was the delta 12 s index, a measure of baseline SpO2 variability (sensitivity 92%; specificity 63%). Conclusions HPO screening could halve the number of children with Down syndrome needing multichannel sleep studies and reduce the burden on children, families and health services alike. This approach offers a practical universal screening approach for OSA in Down syndrome that is accessible to the non-specialist paediatrician.

Robert W Morton,1,2 Heather E Elphick,2 Elaine Edwards,1 William J Daw,1,2 Noreen S West1 1Cystic Fibrosis Unit, Sheffield Children’s Hospital, 2Academic Unit of Child Health, University of Sheffield, Sheffield, UK Background: Children with cystic fibrosis (CF) often have suboptimal adherence rates to nebulized medication. Adherence barriers cited include forgetting to take the nebulizers, due to busy home and social lives. Text message reminders have been shown to be effective at improving adherence rates in other chronic diseases such as asthma and diabetes. Objective: The objective of this study was to assess the feasibility and efficacy of sending text reminders for a prolonged period of time to children with CF. Materials and methods: Children with CF aged 5–16 years taking at least one medication via a nebulizer with an electronic adherence monitor were consented for the study. Text message reminders were sent to participants and/or parents via the hospital’s automated text service, up to twice a day, for 6months. The adherence rates for the 6-month text period were compared to the previous 6months before the study. Rates were calculated for weekdays, weekends, and school holidays. Results: Seventeen participants were recruited to the study, with a mean age of 12 years and a mean forced expiratory volume in 1 second (FEV1) of 81% predicted. Fifteen children ­completed the 6-month text period, and I-neb data were accurately analyzed for 13 participants. The mean adherence rate in the 6 months receiving texts was 80%, compared to 81% in the prior 6 months. Overall adherence rates on weekdays, weekends, and school holidays were equivalent during the 2 time periods. A subgroup of patients with moderate baseline adherence showed increased adherence during the text period, particularly at weekends. Conclusion: It is feasible to send text message reminders to children with CF, and they are amenable to this approach. Although text reminders do not increase rates in patients with existing optimal adherence, they may be of value in patients with more moderate baseline rates. Keywords: cystic fibrosis, adherence, telehealth

Background Respiratory rate (RR) is an important vital sign used in the initial and ongoing assessment of unwell children. Measuring respiratory rate can be difficult and time consuming, especially in an uncooperative child. There are concerns that there may be high levels of inconsistencies in measurements of respiratory rate. This may impact greatly on the child, their clinical assessment and subsequent management, and the accurate identification of possible deterioration. Aim To determine the reliability of a respiratory rate count on a child when assessed by three independent observers. Method From August 2016 to October 2016 the respiratory rate of 169 children was measured by three independent observers in one tertiary children’s hospital. The first respiratory rate was taken by different healthcare professionals from within the hospital using their own method of measurement. A further count of respiratory rate was then taken by two different observers simultaneously within 30 min of the first measurement. They measured the respiratory rate using the WHO recommended method of measurement by observing chest movements over 60 s. All observers were blinded to each of the others’ measurements. Results A total of 507 respiratory rate measurements were taken on 169 children aged between 3 days and 15 years. The 95% limits of agreement between the first RR measurement and second and third measurements was 10.15 to 17.68 and 11.36 to 18.73 respectively. For simultaneous measurements the 95% limits of agreement were 7.11 to 6.95, meaning that that the difference in measurements could have been anything from 7 breaths less to 6 breaths more. Significantly, in 25% of the children the difference in the measured RR was such that the child would not have been assessed as being tachypnoeic by one or more of the three observers. Conclusion There exists a large variation in respiratory rate measurements taken in children. The agreement between measurements and the identification of tachypnoea was poor. The effect that this variation may have on the clinical assessment of a child is significant. These findings highlight the need for a robust review of our current reliance and interpretation of such an important vital sign.

Cystic fibrosis (CF) is a life-limiting, multisystem disease characterized by thick viscous secretions leading to recurrent lung infections, bronchiectasis, and progressive deterioration in lung function. CF is caused by loss or dysfunction of the CF transmembrane conductance regulator (CFTR) protein which is responsible for transepithelial chloride and water transport. Improved understanding of CFTR protein dysfunction has allowed the development of mutation-specific small-molecule compounds which directly target the underlying CFTR defect. Ivacaftor is the first licensed small-molecule compound for CF patients which targets the CFTR gating mutation Gly551Asp (previously termed G551D) and has the potential to be truly disease-modifying. Ivacaftor is an oral medication given twice daily and has shown benefit in terms of an increase in lung function, decreased sweat chloride, weight gain, improvement in patient-reported quality of life, and reduction in number of respiratory exacerbations in clinical trials. Although ivacaftor is currently only licensed for use in approximately 5% of the CF population (those who have at least one Gly551Asp mutation), the developmental pathway established by ivacaftor paves the way for other CFTR modulators that may benefit many more patients. In particular, a CFTR modulator for those with the Phe508del deletion (previously ∆F508) would allow 90% of the CF population to benefit from disease-modifying treatment.

BackgroundAsthma episodes and deaths are known to be seasonal. A number of reports have shown peaks in asthma episodes in school-aged children associated with the return to school following the summer vacation. A fall in prescription collection in the month of August has been observed, and was associated with an increase in the number of unscheduled contacts after the return to school in September.ObjectiveThe primary objective of the study was to assess whether or not a NHS-delivered public health intervention reduces the September peak in unscheduled medical contacts.DesignCluster randomised trial, with the unit of randomisation being 142 NHS general practices, and trial-based economic evaluation.SettingPrimary care.InterventionA letter sent (n = 70 practices) in July from their general practitioner (GP) to parents/carers of school-aged children with asthma to remind them of the importance of taking their medication, and to ensure that they have sufficient medication prior to the start of the new school year in September. The control group received usual care.Main outcome measuresThe primary outcome measure was the proportion of children aged 5–16 years who had an unscheduled medical contact in September 2013. Supporting end points included the proportion of children who collected prescriptions in August 2013 and unscheduled contacts through the following 12 months. Economic end points were quality-adjusted life-years (QALYs) gained and costs from an NHS and Personal Social Services perspective.ResultsThere is no evidence of effect in terms of unscheduled contacts in September. Among children aged 5–16 years, the odds ratio (OR) was 1.09 [95% confidence interval (CI) 0.96 to 1.25] against the intervention. The intervention did increase the proportion of children collecting a prescription in August (OR 1.43, 95% CI 1.24 to 1.64) as well as scheduled contacts in the same month (OR 1.13, 95% CI 0.84 to 1.52). For the wider time intervals (September–December 2013 and September–August 2014), there is weak evidence of the intervention reducing unscheduled contacts. The intervention did not reduce unscheduled care in September, although it succeeded in increasing the proportion of children collecting prescriptions in August as well as having scheduled contacts in the same month. These unscheduled contacts in September could be a result of the intervention, as GPs may have wanted to see patients before issuing a prescription. The economic analysis estimated a high probability that the intervention was cost-saving, for baseline-adjusted costs, across both base-case and sensitivity analyses. There was no increase in QALYs.LimitationThe use of routine data led to uncertainty in the coding of medical contacts. The uncertainty was mitigated by advice from a GP adjudication panel.ConclusionsThe intervention did not reduce unscheduled care in September, although it succeeded in increasing the proportion of children both collecting prescriptions and having scheduled contacts in August. After September there is weak evidence in favour of the intervention. The intervention had a favourable impact on costs but did not demonstrate any impact on QALYs. The results of the trial indicate that further work is required on assessing and understanding adherence, both in terms of using routine data to make quantitative assessments, and through additional qualitative interviews with key stakeholders such as practice nurses, GPs and a wider group of children with asthma.Trial registrationCurrent Controlled Trials ISRCTN03000938.Funding detailsThis project was funded by the NIHR Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 93. See the HTA programme website for further project information.

BackgroundChildren with Down syndrome (DS) are vulnerable to obstructive sleep apnoea (OSA) because of their unique craniofacial anatomy and hypotonia. Understanding the predictors of OSA in DS may enable targeted screening.MethodsChildren with DS (n = 202) aged from six months to below six years (110 boys) were recruited from three UK children's hospitals. The clinical assessment included height, weight and tonsillar size. The parents either set up cardiorespiratory polygraphy at home or chose laboratory studies. Studies with less than four hours of interpretable data were repeated where possible. American Academy of Sleep Medicine (AASM) 2012 scoring criteria were used to derive an obstructive apnoea/hypopnoea index (OAHI). Predictors of moderate to severe OSA were examined.ResultsIn total, 188/202 (93%) participants were successfully studied. Of these, 169 studies were completed at home and 19 in a sleep laboratory. Moderate to severe OSA, defined by an OAHI of >5/h, was found in 14% and mild to moderate OSA (1/h≥OAHI ConclusionsModerate to severe OSA is common in very young children with DS. Examination of tonsillar size did not predict OSA severity. Population-based screening for OSA is recommended in these children, and domiciliary cardiorespiratory polygraphy is an acceptable screening approach. Further research is required to understand the natural history, associated morbidity, optimal screening methodology and treatment modality for OSA in these children.

Dysfunctional breathing is a significant cause of morbidity, adversely affecting an individual's quality of life. There is currently no data from paediatric centres on the impact of breathing retraining for dysfunctional breathing. Symptoms and quality of life were measured in 34 subjects referred sequentially for breathing retraining to the first dedicated paediatric dysfunctional breathing clinic in the UK. Data were obtained prior to the first intervention (time point 1), at discharge (time point 2) and by post 6 months later (time point 3). The mean (interquartile range) age of participants was 13.3 (9.1–16.3) years, with 52% female. Data were obtained at time points 2 and 3 in 23 and 13 subjects, respectively. Statistically significant improvements were observed in symptom scores, child quality of life and parental proxy quality of life between time points 1 and 2 (p, Breathing retraining improves the quality of life and symptoms of children with dysfunctional breathing http://ow.ly/qg1c302sYAF

Background: Respiratory rate (RR) is an important vital sign used in the initial and ongoing assessment of acutely ill children. It is also used as a predictor of serious deterioration in a patient9s clinical condition. Convenient electronic devices exist for measurement of pulse, blood pressure, oxygen saturation and temperature. Although devices which measure RR exist, none has entered everyday clinical practice. Aims: We have developed a contactless portable respiratory rate monitor (CPRM) and aimed to evaluate the agreement in RR measurements between existing methods and our new device. Method: RR data were collected from 30 children undergoing polysomnography sleep studies at a tertiary children9s hospital. The RR of each participant was measured simultaneously by 3 different methods. Respiratory impedance plethysmography (RIP) the established contact method and gold standard. Visual counting of chest movements, and method used most in clinical settings. The contactless portable respiratory monitor (CPRM), which is the new method. Two data sets were collected from each child. Results: Data showed substantial agreement between measurements from the CPRM and the gold standard RIP, with intra-class correlation coefficient: 0.762, mean difference -0.212 and 95% limits of agreement of -6.842 to 6.419. Conclusion: A contactless device for accurately and quickly measuring RR will be an important tool in the assessment of unwell children. More testing is needed to explore the reasons for outlying measurements and to evaluate in different clinical settings. Further development and modification of the device and software are ongoing.

Reports have shown peaks in asthma episodes in school-aged children associated with the return to school following the summer vacation (Julious SA et al. Public Health 2007;121(6):482-4). A drop in prescription collection in the month of August has been observed which was associated with an increase in the number of unscheduled contacts after the return to school in September. The aim of the study was to assess whether a primary care intervention reduced the number of unscheduled medical contacts after the school return. A letter was sent from the GP to parents/carers of children aged 5-16 years with asthma at the start of school summer holidays reminding them to take their preventer medication prior to the start of the new school year. The study was a cluster randomised trial with a control group that received usual care. The intervention did not reduce unscheduled care in September (odds-ratio 1.09; 95% CI 0.96 to 1.25) against the intervention. It did succeed in increasing the proportion of children collecting prescriptions in August (odds-ratio 1.43; 95% CI 1.24 to 1.64) as well as scheduled contacts in the same month. For the wider time intervals (Sep-Dec 2013 and Sep-Aug 2014) there was weak evidence of effect. The economic analysis estimated a high probability that the intervention was cost saving. A higher uptake of prescriptions and scheduled contacts in August may indicate that the letter triggered parents to arrange asthma reviews in children whose prescriptions had lapsed. GPs and commissioning groups may wish to implement this intervention in areas with high rates of asthma and low rates of scheduled asthma reviews.

Respiratory rate is an important vital sign used for diagnosing illnesses in children as well as prioritising\ud patient care. All children presenting acutely to hospital should have a respiratory rate measured as part of their initial and ongoing assessment. However measuring the respiratory rate remains a subjective assessment and in children can be liable to measurement error especially if the child is uncooperative. Devices to measure respiratory rate exist but many provide only an estimate of respiratory rate due to the associated methodological complexities. Some devices are used within the intensive care, post-operative or more specialised investigatory settings none however have made their way into the everyday clinical setting. A non-contact device may be better tolerated in children and not cause undue stress distorting the measurement. Further validation and adaption to the acute clinical setting is needed before such devices can supersede current methods.

Respiration rate is an important indicator of a person's health, and thus it is monitored when performing clinical evaluations. There are different approaches for respiration monitoring, but generally they can be classed as contact or noncontact. For contact methods, the sensing device (or part of the instrument containing it) is attached to the subject's body. For noncontact approaches the monitoring is performed by an instrument that does not make any contact with the subject. In this article a review of respiration monitoring approaches (both contact and noncontact) is provided. Concerns related to the patient's recording comfort, recording hygiene, and the accuracy of respiration rate monitoring have resulted in the development of a number of noncontact respiration monitoring approaches. A description of thermal imaging based and vision based noncontact respiration monitoring approaches we are currently developing is provided.