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1.  Antiepileptic drug treatment of rolandic epilepsy and Panayiotopoulos syndrome: clinical practice survey and clinical trial feasibility 
Archives of Disease in Childhood  2014;100(1):62-67.
Background
The evidence base for management of childhood epilepsy is poor, especially for the most common specific syndromes such as rolandic epilepsy (RE) and Panayiotopoulos syndrome (PS). Considerable international variation in management and controversy about non-treatment indicate the need for high quality randomised controlled trials (RCT). The aim of this study is, therefore, to describe current UK practice and explore the feasibility of different RCT designs for RE and PS.
Methods
We conducted an online survey of 590 UK paediatricians who treat epilepsy. Thirty-two questions covered annual caseload, investigation and management practice, factors influencing treatment, antiepileptic drug preferences and hypothetical trial design preferences.
Results
132 responded (22%): 81% were paediatricians and 95% at consultant seniority. We estimated, annually, 751 new RE cases and 233 PS cases. Electroencephalography (EEG) is requested at least half the time in approximately 70% of cases; MRI brain at least half the time in 40%–65% cases and neuropsychological evaluation in 7%–8%. Clinicians reported non-treatment in 40%: main reasons were low frequency of seizures and parent/child preferences. Carbamazepine is the preferred older, and levetiracetam the preferred newer, RCT arm. Approximately one-half considered active and placebo designs acceptable, choosing seizures as primary and cognitive/behavioural measures as secondary outcomes.
Conclusions
Management among respondents is broadly in line with national guidance, although with possible overuse of brain imaging and underuse of EEG and neuropsychological assessments. A large proportion of patients in the UK remains untreated, and clinicians seem amenable to a range of RCT designs, with carbamazepine and levetiracetam the preferred active drugs.
doi:10.1136/archdischild-2013-304211
PMCID: PMC4283698  PMID: 25202134
Neurology; Paediatric Practice
2.  What clinical signs best identify severe illness in young infants aged 0–59 days in developing countries? A systematic review 
Archives of disease in childhood  2011;96(11):1052-1059.
Despite recent overall improvement in the survival of under-five children worldwide, mortality among young infants remains high, and accounts for an increasing proportion of child deaths in resource-poor settings. In such settings, clinical decisions for appropriate management of severely ill infants have to be made on the basis of presenting clinical signs, and with limited or no laboratory facilities. This review summarises the evidence from observational studies of clinical signs of severe illnesses in young infants aged 0–59 days, with a particular focus on defining a minimum set of best predictors of the need for hospital-level care. Available moderate to high quality evidence suggests that, among sick infants aged 0–59 days brought to a health facility, the following clinical signs—alone or in combination—are likely to be the most valuable in identifying infants at risk of severe illness warranting hospital-level care: history of feeding difficulty, history of convulsions, temperature (axillary) ≥37.5°C or <35.5°C, change in level of activity, fast breathing/respiratory rate ≥60 breaths per minute, severe chest indrawing, grunting and cyanosis.
doi:10.1136/adc.2010.186049
PMCID: PMC3081806  PMID: 21220263
3.  Marketing breast milk substitutes: problems and perils throughout the world 
Archives of Disease in Childhood  2012;97(6):529-532.
On 21 May 1981 the WHO International Code of Marketing Breast Milk Substitutes (hereafter referred to as the Code) was passed by 118 votes to 1, the US casting the sole negative vote. The Code arose out of concern that the dramatic increase in mortality, malnutrition and diarrhoea in very young infants in the developing world was associated with aggressive marketing of formula. The Code prohibited any advertising of baby formula, bottles or teats and gifts to mothers or ‘bribery’ of health workers. Despite successes, it has been weakened over the years by the seemingly inexhaustible resources of the global pharmaceutical industry. This article reviews the long and tortuous history of the Code through the Convention on the Rights of the Child, the HIV pandemic and the rare instances when substitute feeding is clearly essential. Currently, suboptimal breastfeeding is associated with over a million deaths each year and 10% of the global disease burden in children. All health workers need to recognise inappropriate advertising of formula, to report violations of the Code and to support efforts to promote breastfeeding: the most effective way of preventing child mortality throughout the world.
doi:10.1136/archdischild-2011-301299
PMCID: PMC3371222  PMID: 22419779
4.  Prospects for eradication of meningococcal disease 
Archives of Disease in Childhood  2012;97(11):993-998.
Meningococcal meningitis and septicaemia remain a serious global health threat. This review focuses on the epidemiology of meningococcal disease following the recent implementation of effective vaccines and the potential utility of a vaccine against serogroup B meningococcus.
doi:10.1136/archdischild-2012-302036
PMCID: PMC3512348  PMID: 22984187
Epidemiology; Intensive Care; Mortality; Infectious Diseases
5.  Drug treatment of inborn errors of metabolism: a systematic review 
Archives of Disease in Childhood  2013;98(6):454-461.
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.
doi:10.1136/archdischild-2012-303131
PMCID: PMC3693126  PMID: 23532493
Inborn errors of metabolism; IEM; evidence based medicine; treatment; dosage
6.  Evidence review of hydroxyurea for the prevention of sickle cell complications in low-income countries 
Archives of Disease in Childhood  2013;98(11):908-914.
Hydroxyurea is widely used in high-income countries for the management of sickle cell disease (SCD) in children. In Kenyan clinical guidelines, hydroxyurea is only recommended for adults with SCD. Yet many deaths from SCD occur in early childhood, deaths that might be prevented by an effective, disease modifying intervention. The aim of this review was to summarise the available evidence on the efficacy, effectiveness and safety of hydroxyurea in the management of SCD in children below 5 years of age to support guideline development in Kenya. We undertook a systematic review and used the Grading of Recommendations Assessment, Development and Evaluation system to appraise the quality of identified evidence. Overall, available evidence from 1 systematic review (n=26 studies), 2 randomised controlled trials (n=354 children), 14 observational studies and 2 National Institute of Health reports suggest that hydroxyurea may be associated with improved fetal haemoglobin levels, reduced rates of hospitalisation, reduced episodes of acute chest syndrome and decreased frequency of pain events in children with SCD. However, it is associated with adverse events (eg, neutropenia) when high to maximum tolerated doses are used. Evidence is lacking on whether hydroxyurea improves survival if given to young children. Majority of the included studies were of low quality and mainly from high-income countries. Overall, available limited evidence suggests that hydroxyurea may improve morbidity and haematological outcomes in SCD in children aged below 5 years and appears safe in settings able to provide consistent haematological monitoring.
doi:10.1136/archdischild-2012-302387
PMCID: PMC3812872  PMID: 23995076
Genetics; Haematology
7.  Management of Kawasaki disease 
Kawasaki disease (KD) is an acute self-limiting inflammatory disorder, associated with vasculitis, affecting predominantly medium-sized arteries, particularly the coronary arteries. In developed countries KD is the commonest cause of acquired heart disease in childhood. The aetiology of KD remains unknown, and it is currently believed that one or more as yet unidentified infectious agents induce an intense inflammatory host response in genetically susceptible individuals. Genetic studies have identified several susceptibility genes for KD and its sequelae in different ethnic populations, including FCGR2A, CD40, ITPKC, FAM167A-BLK and CASP3, as well as genes influencing response to intravenous immunoglobulin (IVIG) and aneurysm formation such as FCGR3B, and transforming growth factor (TGF) β pathway genes. IVIG and aspirin are effective therapeutically, but recent clinical trials and meta-analyses have demonstrated that the addition of corticosteroids to IVIG is beneficial for the prevention of coronary artery aneurysms (CAA) in severe cases with highest risk of IVIG resistance. Outside of Japan, however, clinical scores to predict IVIG resistance perform suboptimally. Furthermore, the evidence base does not provide clear guidance on which corticosteroid regimen is most effective. Other therapies, including anti-TNFα, could also have a role for IVIG-resistant KD. Irrespective of these caveats, it is clear that therapy that reduces inflammation in acute KD, improves outcome. This paper summarises recent advances in the understanding of KD pathogenesis and therapeutics, and provides an approach for managing KD patients in the UK in the light of these advances.
doi:10.1136/archdischild-2012-302841
PMCID: PMC3888612  PMID: 24162006
Infectious Diseases; Rheumatology
8.  What causes attention deficit hyperactivity disorder? 
Archives of Disease in Childhood  2011;97(3):260-265.
Attention deficit hyperactivity disorder (ADHD) affects around 1–3% of children. There is a high level of comorbidity with developmental and learning problems as well as with a variety of psychiatric disorders. ADHD is highly heritable, although there is no single causal risk factor and non-inherited factors also contribute to its aetiology. The genetic and environmental risk factors that have been implicated appear to be associated with a range of neurodevelopmental and neuropsychiatric outcomes, not just ADHD. The evidence to date suggests that both rare and multiple common genetic variants likely contribute to ADHD and modify its phenotype. ADHD or a similar phenotype also appears to be more common in extreme low birth weight and premature children and those exposed to exceptional early adversity. In this review, the authors consider recent developments in the understanding of risk factors that influence ADHD.
doi:10.1136/archdischild-2011-300482
PMCID: PMC3927422  PMID: 21903599
9.  Assessment and management of anxiety disorders in children and adolescents 
Archives of Disease in Childhood  2014;99(7):674-678.
Anxiety disorders in childhood and adolescence are extremely common and are often associated with lifelong psychiatric disturbance. Consistent with DSM-5 and the extant literature, this review concerns the assessment and treatment of specific phobias, separation anxiety disorder, generalised anxiety disorder, social anxiety disorder, panic disorder and agoraphobia. Evidence-based psychological treatments (cognitive behaviour therapy; CBT) for these disorders have been developed and investigated, and in recent years promising low-intensity versions of CBT interventions have been proposed that offer a means to increase access to evidence-based treatments. There is some evidence of effectiveness of pharmacological treatments for anxiety disorders in children and young people, however, routine prescription is not recommended due to concerns about potential harm.
doi:10.1136/archdischild-2013-303768
PMCID: PMC4078705  PMID: 24636957
Anxiety; Assessment; Treatment; Children; Adolescents
10.  Diagnosis and management of primary ciliary dyskinesia 
Archives of Disease in Childhood  2014;99(9):850-856.
Primary ciliary dyskinesia (PCD) is an inherited autosomal-recessive disorder of motile cilia characterised by chronic lung disease, rhinosinusitis, hearing impairment and subfertility. Nasal symptoms and respiratory distress usually start soon after birth, and by adulthood bronchiectasis is invariable. Organ laterality defects, usually situs inversus, occur in ∼50% of cases. The estimated prevalence of PCD is up to ∼1 per 10 000 births, but it is more common in populations where consanguinity is common. This review examines who to refer for diagnostic testing. It describes the limitations surrounding diagnosis using currently available techniques and considers whether recent advances to genotype patients with PCD will lead to genetic testing and screening to aid diagnosis in the near future. It discusses the challenges of monitoring and treating respiratory and ENT disease in children with PCD.
doi:10.1136/archdischild-2013-304831
PMCID: PMC4145427  PMID: 24771309
primary ciliary dyskinesia; Kartagener syndrome; treatment; diagnosis; ciliary motility disorders
11.  Common visual problems in children with disability 
Archives of Disease in Childhood  2014;99(12):1163-1168.
Children with disability are at a substantially higher risk of visual impairment (VI) (10.5% compared with 0.16%) but also of ocular disorders of all types, including refractive errors and strabismus. The aetiology of VI in children with disability reflects that of the general population and includes cerebral VI, optic atrophy, as well as primary visual disorders such as retinal dystrophies and structural eye anomalies. VI and other potentially correctable ocular disorders may not be recognised without careful assessment and are frequently unidentified in children with complex needs. Although assessment may be more challenging than in other children, identifying these potential additional barriers to learning and development may be critical. There is a need to develop clearer guidelines, referral pathways and closer working between all professionals involved in the care of children with disability and visual disorders to improve our focus on the assessment of vision and outcomes for children with disability.
doi:10.1136/archdischild-2013-305267
PMCID: PMC4251159  PMID: 25165073
Neurodisability; Ophthalmology; Paediatric Practice
12.  Paediatric clinical pharmacology in the UK 
Archives of Disease in Childhood  2014;99(12):1143-1146.
Paediatric clinical pharmacology is the scientific study of medicines in children and is a relatively new subspecialty in paediatrics in the UK. Training encompasses both the study of the effectiveness of drugs in children (clinical trials) and aspects of drug toxicity (pharmacovigilance). Ethical issues in relation to clinical trials and also studies of the pharmacokinetics and drug metabolism in children are crucial. Paediatric patients require formulations that young children in particular are able to take. The scientific evidence generated from clinical trials, pharmacokinetic studies and studies of drug toxicity all need to be applied in order to ensure that medicines are used rationally in children.
doi:10.1136/archdischild-2014-306853
PMCID: PMC4251165  PMID: 25202131
Pharmacology; Toxicology; Evidence Based Medicine
13.  Hurricanes and child health: lessons from Cuba 
Archives of Disease in Childhood  2010;96(4):328-329.
doi:10.1136/adc.2009.178145
PMCID: PMC3056292  PMID: 20861403
14.  What clinical signs best identify severe illness in young infants aged 0–59 days in developing countries? A systematic review 
Archives of Disease in Childhood  2011;96(11):1052-1059.
Despite recent overall improvement in the survival of under-five children worldwide, mortality among young infants remains high, and accounts for an increasing proportion of child deaths in resource-poor settings. In such settings, clinical decisions for appropriate management of severely ill infants have to be made on the basis of presenting clinical signs, and with limited or no laboratory facilities. This review summarises the evidence from observational studies of clinical signs of severe illnesses in young infants aged 0–59 days, with a particular focus on defining a minimum set of best predictors of the need for hospital-level care. Available moderate to high quality evidence suggests that, among sick infants aged 0–59 days brought to a health facility, the following clinical signs—alone or in combination—are likely to be the most valuable in identifying infants at risk of severe illness warranting hospital-level care: history of feeding difficulty, history of convulsions, temperature (axillary) ≥37.5°C or <35.5°C, change in level of activity, fast breathing/respiratory rate ≥60 breaths per minute, severe chest indrawing, grunting and cyanosis.
doi:10.1136/adc.2010.186049
PMCID: PMC3081806  PMID: 21220263
15.  Armed conflict and child health 
Summary
Armed conflict has a major impact on child health throughout the world. One in six children worldwide lives in an area of armed conflict and civilians are more likely to die than soldiers as a result of the conflict. In stark contrast to the effect on children, the international arms trade results in huge profits for the large corporations involved in producing arms, weapons and munitions. Armed conflict is not inevitable but is an important health issue that should be prevented.
doi:10.1136/adc.2009.178186
PMCID: PMC3237260  PMID: 21393303

Results 1-15 (15)