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Background and Aims: During the last decades, much attention has been paid to off-label and unlicensed prescriptions in paediatrics. However, on-label prescribing can also cause health issues. In this paper, the case of first-generation H1-antihistamines is investigated, notably the range of indications for which products are licensed in different European countries and the evidence base (or lack thereof) for each indication, as well as reported adverse drug reactions.Methods: Review of the Summary of Product Characteristics of first-generation H1-antihistamines with a focus on paediatric use. This is plotted against the evidence available in the literature.Results: This investigation shows a large variability in labelled indications and licensing ages when compared in five different European countries. Moreover, most of the indications are not based on clinical trials evaluating efficacy and safety of these drugs in children.Conclusions: Many of the licensed indications of first-generation antihistamines do not appear to be evidence based. [ABSTRACT FROM AUTHOR]

One-third of the global population is aged under 20 years. For children with life-limiting conditions, palliative care services are required. However, despite 80% of global need occurring in low- and middle-income countries (LMICs), the majority of children's palliative care (CPC) is provided in high-income countries. This paper reviews the status of CPC services in LMICs--highlighting examples of best practice among service models in Malawi, Indonesia and Belarus--before reviewing the status of the extant research in this field. It concludes that while much has been achieved in palliative care for adults, less attention has been devoted to the education, clinical practice, funding and research needed to ensure children and young people receive the palliative care they need. [ABSTRACT FROM AUTHOR]

This review seeks to explore and explain what health policy research is and why it matters, through pooling the evidence and providing case examples. Alongside examining the types of research involved, and their rationale the paper identifies the challenges taking part in this sort of research may create. Finally the paper suggests how to make clinical research more accessible to policy makers. [ABSTRACT FROM AUTHOR]

Background: The treatment of inborn errors of metabolism (IEM) has seen significant advances over the last decade. Many medicines have been developed and the survival rates of some patients with IEM have improved. Dosages of drugs used for the treatment of various IEM can be obtained from a range of sources but tend to vary among these sources. Moreover, the published dosages are not usually supported by the level of existing evidence, and they are commonly based on personal experience., Methods: A literature search was conducted to identify key material published in English in relation to the dosages of medicines used for specific IEM. Textbooks, peer reviewed articles, papers and other journal items were identified. The PubMed and Embase databases were searched for material published since 1947 and 1974, respectively. The medications found and their respective dosages were graded according to their level of evidence, using the grading system of the Oxford Centre for Evidence-Based Medicine., Results: 83 medicines used in various IEM were identified. The dosages of 17 medications (21%) had grade 1 level of evidence, 61 (74%) had grade 4, two medications were in level 2 and 3 respectively, and three had grade 5., Conclusions: To the best of our knowledge, this is the first review to address this matter and the authors hope that it will serve as a quickly accessible reference for medications used in this important clinical field.

Health care professionals are increasingly required to base clinical decisions on the best available evidence. Evidence based medicine (EBM) is a systematic approach to clinical problem solving which allows the integration of the best available research evidence with clinical expertise and patient values. This paper explains the concept of EBM and introduces the five step EBM model: formulation of answerable clinical questions; searching for evidence; critical appraisal; applicability of evidence; evaluation of performance. Subsequent articles will focus on the principles and critical appraisal of randomised controlled trials, systematic reviews, and meta-analyses, and provide a practical demonstration of the five step EBM model using a real life clinical scenario.

Introduction: Global developmental delay (GDD) affects 1%-3% of the population of children under 5 years of age, making it one of the most common conditions presenting in paediatric clinics; causes are exogenous, genetic (non-metabolic) or genetic (metabolic). Recent advances in biotechnology and genetic testing mean that the investigations available to perform for children under 5 years are increasing and are more sensitive than previously. This change in availability and type of testing necessitates an update in the recommendations for investigating GDD.Methods: We conducted a review of the literature from 2006 to 2016 to identify articles with evidence relating to the investigation of developmental delay in children under the age of 5 years. We collated the evidence into first-line and second-line investigations and, where available, on their yield and cost implications.Results: We have provided up-to-date guidance for first-line and second-line investigations for children with GDD under the age of 5 years. Recent evidence demonstrates that genetic testing for all children with unexplained GDD should be first line, if an exogenous cause is not already established. Our review of the literature demonstrates that all patients, irrespective of severity of GDD, should have investigations for treatable conditions. Evidence demonstrates that the yield for treatable conditions is higher than previously thought and that investigations for these metabolic conditions should be considered as first line. Additional second-line investigations can be led by history, examination and developmental trajectories.Discussion: We may need to update present recommendations in the UK for investigation of developmental delay. This would include microarray testing as first line and a more thorough approach to investigations for metabolic disorders that can be treated. Clinical assessment remains vital for guiding investigations. [ABSTRACT FROM AUTHOR]

Cognitive-behavioural therapy (CBT) is a practical, goal-focused approach that helps children understand the relationship between their thoughts, feelings and behaviours. The aim is to identify the dysfunctional and distorted cognitions associated with their psychological problems and to create more functional and balanced cognitive patterns that create less emotional distress and more helpful behaviours. CBT has strong evidence as an effective intervention for children and adolescents with emotional problems. The benefits for children with physical health and chronic conditions appear promising, although further research is required to substantiate these gains. [ABSTRACT FROM AUTHOR]

Developmental disabilities, emotional disorders and disruptive behaviour disorders are the leading mental health-related causes of the global burden of disease in children aged below 10 years. This article aims to address the treatment gap for child mental disorders through synthesising three bodies of evidence: the global evidence base on the treatment of these priority disorders; the barriers to implementation of this knowledge; and the innovative approaches taken to address these barriers and improve access to care. Our focus is on low-resource settings, which are mostly found in low- and middle-income countries (LMIC). Despite the evidence base on the burden of child mental disorders and their long-term consequences, and the recent mental health Gap Action Programme guidelines which testify to the effectiveness of a range of pharmacological and psychosocial interventions for these disorders, the vast majority of children in LMIC do not have access to these interventions. We identify three major barriers for the implementation of efficacious treatments: the lack of evidence on delivery of the treatments, the low levels of detection of child mental disorders and the shortage of skilled child mental health professionals. The evidence based on implementation, although weak, supports the use of screening measures for detection of probable disorders, coupled with a second-stage diagnostic assessment and the use of nonspecialist workers in community and school settings for the delivery of psychosocial interventions. The most viable strategy to address the treatment gap is through the empowerment of existing human resources who are most intimately concerned with child care, including parents, through innovative technologies, such as mobile health, with the necessary skills for the detection and treatment of child mental disorders. [ABSTRACT FROM AUTHOR]

The article presents an evidence-based answer to a question on the use of bubble baths in children with urinary tract infections (UTI). There is a lack of conclusive evidence supporting the advice of general pediatricians to avoid the use of bubble bath to prevent UTI. However, the common sense approach is advised. The enjoyment of bubble baths outweighs the limited evidence of their supposed harm.

Background: Optimal fluid resuscitation in children with major burns is crucial to prevent or minimise burn shock and prevent complications of over-resuscitation.Objectives: To identify studies using endpoints to guide fluid resuscitation in children with burns, review the range of reported endpoint targets and assess whether there is evidence that targeted endpoints impact on outcome.Design: Systematic review.Methods: Medline, Embase, Cinahl and the Cochrane Central Register of Controlled Trials databases were searched with no restrictions on study design or date. Search terms combined burns, fluid resuscitation, endpoints, goal-directed therapy and related synonyms. Studies reporting primary data regarding children with burns (<16 years) and targeting fluid resuscitation endpoints were included. Data were extracted using a proforma and the results were narratively reviewed.Results: Following screening of 777 unique references, 7 studies fulfilled the inclusion criteria. Four studies were exclusively paediatric. Six studies used urine output (UO) as the primary endpoint. Of these, one set a minimum UO threshold, while the remainder targeted a range from 0.5-1.0 mL/kg/hour to 2-3 mL/kg/hour. No studies compared different UO targets. Heterogeneous study protocols and outcomes precluded comparison between the UO targets. One study targeted invasive haemodynamic variables, but this did not significantly affect patient outcome.Conclusions: Few studies have researched resuscitation endpoints for children with burns. Those that have done so have investigated heterogeneous endpoints and endpoint targets. There is a need for future randomised controlled trials to identify optimal endpoints with which to target fluid resuscitation in children with burns. [ABSTRACT FROM AUTHOR]

Objective: To compare the predictive performance of 18 paediatric early warning systems (PEWS) in predicting critical deterioration.Design: Retrospective case-controlled study. PEWS values were calculated from existing clinical data, and the area under the receiver operator characteristic curve (AUROC) compared.Setting: UK tertiary referral children's hospital.Patients: Patients without a 'do not attempt resuscitation' order admitted between 1 January 2011 and 31 December 2012. All patients on paediatric wards who suffered a critical deterioration event were designated 'cases' and matched with a control closest in age who was present on the same ward at the same time.Main Outcome Measures: Respiratory and/or cardiac arrest, unplanned transfer to paediatric intensive care and/or unexpected death.Results: 12 'scoring' and 6 'trigger' systems were suitable for comparative analysis. 297 case events in 224 patients were available for analysis. 244 control patients were identified for the 311 events. Three PEWS demonstrated better overall predictive performance with an AUROC of 0.87 or greater. Comparing each system with the highest performing PEWS with Bonferroni's correction for multiple comparisons resulted in statistically significant differences for 13 systems. Trigger systems performed worse than scoring systems, occupying the six lowest places in the AUROC rankings.Conclusions: There is considerable variation in the performance of published PEWS, and as such the choice of PEWS has the potential to be clinically important. Trigger-based systems performed poorly overall, but it remains unclear what factors determine optimum performance. More complex systems did not necessarily demonstrate improved performance. [ABSTRACT FROM AUTHOR]

Background: There is little evidence about either prevention or treatment of childhood arterial ischaemic stroke (AIS). However, drugs that regulate the immune and inflammatory response could theoretically prevent occurrence or recurrence of AIS. Additionally, as an acute treatment, they may limit the neurological damage caused by AIS. Here, we systematically review the evidence on the use of immunotherapy in childhood AIS.Design: A systematic review of publications in databases Embase and Medline from inception. All types of evidence were included from trials, cohorts, case-control and cross-sectional studies and case reports.Results: 34 reports were included: 32 observational studies and 2 trials. Immunotherapy was used in two key patient groups: arteriopathy and acute infection. The majority were cases of varicella and primary angiitis of the central nervous system. All three cohorts and 80% of the case studies were treated with steroids. Recurrence rates were low. Analytical studies weakly associated steroids with lower odds of new stroke and neurological deficits, and better cognitive outcomes in the context of Moyamoya disease and tuberculosis.Conclusions: Immunotherapies are used in children with AIS, mainly as steroids for children with arteriopathy. However, there is currently little robust evidence to either encourage or discourage this practice. There is weak evidence consistent with the hypothesis that in certain children at risk, steroids may both reduce the risk of occurrent/recurrent stroke and enhance neurological outcomes. As the potential benefit is still uncertain, this indicates that a trial of steroids in childhood AIS may be justified. [ABSTRACT FROM AUTHOR]

As many drugs in paediatrics are used off-label, prescribers face a lack of evidence-based dosing guidelines. A Dutch framework was developed to provide dosing guidelines based on best available evidence from registration data, investigator-initiated research, professional guidelines, clinical experience and consensus. This has clarified the scientific grounds of drug use for children and encouraged uniformity in prescribing habits in the Netherlands. The developed framework and the current content of the Dutch Paediatric Formulary could be used as basis for similar initiatives worldwide, preferably in a concerted effort to ultimately provide children with effective and safe drug therapy. [ABSTRACT FROM AUTHOR]

The article presents a case study of a three-year-old girl who was treated for acute lymphoblastic leukaemia (ALL). Topics discussed include the risk of transfusional iron overload (IO), the packed red blood cell (PRBC) transfusions, and the use of liver biopsy to diagnose IO. Information regarding the management and monitoring of IO is also provided.

Nephrotic syndrome is one of the most common paediatric glomerular diseases, with an incidence of around two per 100,000 children per year. Corticosteroids are the mainstay of treatment, with 85%-90% of children going into remission with an 8-week course of treatment. Unfortunately, nephrotic syndrome follows a relapsing and remitting course in the majority, with 90% relapsing at least once. About half will progress to frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS). Different initial steroid regimens have been evaluated since the first trials in Europe and America in the 1960s. Most trials have been designed to evaluate the optimal duration of the initial therapy, rather than different cumulative doses of corticosteroid, or the management of relapses. Until recently, these data suggested that an initial treatment duration of up to 6 months reduced the number of children developing a relapse, without evidence of increased steroid toxicity. Recently, three large, well-designed randomised control trials were published, which demonstrated no significant reduction in risk of relapse or of developing FRNS by extended treatment compared with 2 or 3 months. While there are few trial data to guide the treatment of individual relapses in steroid-sensitive nephrotic syndrome (SSNS), there is some evidence that a short course of corticosteroid therapy during upper respiratory tract infection may prevent relapse. In patients with FRNS or SDNS who continue to relapse despite low-dose alternate-day steroids a number of non-corticosteroid, steroid-sparing immunosuppressive agents (cyclophosphamide, ciclosporin, tacrolimus, mycophenolate mofetil, levamisole, rituximab) have been shown to reduce the risk of relapse and of FRNS. However, there are limited head-to-head data to inform which agent should be preferred. In this article, we review recent data from randomised trials to update paediatricians on the current evidence supporting interventions in SSNS. [ABSTRACT FROM AUTHOR]

The health benefits of probiotics have been the subject of extensive research. Still, some questions are being repeatedly asked: should one use or not use probiotics? If yes, how and when should probiotics be used? The purpose of this review is to summarise current evidence on specific probiotics' efficacy and safety to help healthcare professionals make evidence-based decisions on the indications for using specific probiotic strains or combinations in children. To identify relevant data, searches of MEDLINE and the Cochrane Library databases were performed in July 2015 to locate randomised controlled trials or their meta-analyses published in the last five years. The MEDLINE database also was searched for evidence-based clinical practice guidelines, developed by scientific societies. Considering that probiotics have strain-specific effects, the main focus was on data on individual probiotic strains, not on probiotics in general. [ABSTRACT FROM AUTHOR]

Context: Follow-up strategies after emergency department (ED) discharge, alias safety netting, is often based on the gut feeling of the attending physician.Objective: To systematically identify evaluated safety-netting strategies after ED discharge and to describe determinants of paediatric ED revisits.Data Sources: MEDLINE, Embase, CINAHL, Cochrane central, OvidSP, Web of Science, Google Scholar, PubMed.Study Selection: Studies of any design reporting on safety netting/follow-up after ED discharge and/or determinants of ED revisits for the total paediatric population or specifically for children with fever, dyspnoea and/or gastroenteritis. Outcomes included complicated course of disease after initial ED visit (eg, revisits, hospitalisation).Data Extraction: Two reviewers independently assessed studies for eligibility and study quality. As meta-analysis was not possible due to heterogeneity of studies, we performed a narrative synthesis of study results. A best-evidence synthesis was used to identify the level of evidence.Results: We summarised 58 studies, 36% (21/58) were assessed as having low risk of bias. Limited evidence was observed for different strategies of safety netting, with educational interventions being mostly studied. Young children, a relevant medical history, infectious/respiratory symptoms or seizures and progression/persistence of symptoms were strongly associated with ED revisits. Gender, emergency crowding, physicians' characteristics and diagnostic tests and/or therapeutic interventions at the index visit were not associated with revisits.Conclusions: Within the heterogeneous available evidence, we identified a set of strong determinants of revisits that identify high-risk groups in need for safety netting in paediatric emergency care being related to age and clinical symptoms. Gaps remain on intervention studies concerning specific application of a uniform safety-netting strategy and its included time frame. [ABSTRACT FROM AUTHOR]

Focuses on pediatricians' use of evidence-based medicine. Integration of highest quality scientific evidence with clinical expertise; Management of diseases in children.

Allergic rhinitis (AR), the most common chronic disease in childhood is often ignored, misdiagnosed and/or mistreated. Undertreated AR impairs quality of life, exacerbates asthma and is a major factor in asthma development. It can involve the nose itself, as well as the organs connected with the nose manifesting a variety of symptoms. Evidence-based guidelines for AR therapy improve disease control. Recently, paediatric AR guidelines have been published by the European Academy of Allergy and Clinical Immunology and are available online, as are a patient care pathway for children with AR and asthma from the Royal College of Paediatrics and Child Health. Management involves diagnosis, followed by avoidance of relevant allergens, with additional pharmacotherapy needed for most sufferers. This ranges, according to severity, from saline sprays, through non-sedating antihistamines, oral or topical, with minimally bioavailable intranasal corticosteroids for moderate/severe disease, possibly plus additional antihistamine or antileukotriene. The concept of rhinitis control is emerging, but there is no universally accepted definition. Where pharmacotherapy fails, allergen-specific immunotherapy, which is uniquely able to alter long-term disease outcomes, should be considered. The subcutaneous form (subcutaneous immunotherapy) in children has been underused because of concerns regarding safety and acceptability of injections. Sublingual immunotherapy is both efficacious and safe for grass pollen allergy. Further studies on other allergens in children are needed. Patient, carer and practitioner education into AR and its treatment are a vital part of management. [ABSTRACT FROM AUTHOR]

Background: Children with chronic physical illness are significantly more likely to develop common psychiatric symptoms than otherwise healthy children. These children therefore warrant effective integrated healthcare yet it is not established whether the known, effective, psychological treatments for symptoms of common childhood mental health disorders work in children with chronic physical illness. Methods: EMBASE, MEDLINE, PsycINFO and CINAHL databases were searched with predefined terms relating to evidence-based psychological interventions for psychiatric symptoms in children with chronic physical illness. We included all studies (randomised and non-randomised Design:s investigating interventions aimed primarily at treating common psychiatric symptoms in children with a chronic physical illness in the review. Two reviewers independently assessed the relevance of abstracts identified, extracted data and undertook quality analysis. Results: Ten studies (209 children, including 70 in control groups) met the criteria for inclusion in the review. All studies demonstrated some positive outcomes of cognitive behavioural therapy for the treatment of psychiatric symptoms in children with chronic physical illness. Only two randomised controlled trials, both investigating interventions for symptoms of depression, were found. Conclusions: There is preliminary evidence that cognitive behavioural therapy has positive effects in the treatment of symptoms of depression and anxiety in children with chronic physical illness. However, the current evidence base is weak and fully powered randomised controlled trials are needed to establish the efficacy of psychological treatments in this vulnerable population. [ABSTRACT FROM AUTHOR]

There has been a huge upsurge in clinical research in children in the last decade, stimulated in England by dedicated research infrastructure and support through the National Institute for Health Research. This infrastructure offering research design, expert review, trial management, research nurse, data support and dedicated facilities enables paediatricians to conduct more and better research. The challenge is how to design and conduct trials that will make a real difference to children's health. Standards for Research (StaR) in Child Health was founded in 2009 to address the paucity and shortcomings of paediatric clinical trials. This global initiative involves methodologists, clinicians, patient advocacy groups and policy makers dedicated to developing practical, evidence-based standards for enhancing the reliability and relevance of paediatric clinical research. In this overview, we highlight the contribution of StaR to this agenda, describe the international context, and suggest how StaR's future plans could be integrated with new and existing support for research. [ABSTRACT FROM AUTHOR]

Research understanding the lives of children with disabilities in low-income and middle-income countries has predominantly focused on prevalence studies with little progress on evidence-based service development. At the same time, global attention in child health has shifted from child survival strategies to those that bring child survival and development together. This review examines whether intervention research can be better aligned with current theoretical constructs of disability and international guidelines that advocate for the realisation of rights for children with disabilities and inclusive early childhood development. [ABSTRACT FROM AUTHOR]