Search

Background: Bronchiolitis caused by respiratory syncytial virus (RSV) is an important cause of morbidity in ex-premature infants. In a randomised placebo controlled trial monoclonal antibody prophylaxis showed a 55% reduction in relative risk of hospital admission for these high risk infants, against a background incidence of 10.6 admissions per 100 high risk infants.Aims: To follow a cohort of high risk infants in order to assess hospitalisation rate from RSV and the potential impact of prophylaxis for these patients in a UK local health authority.Methods: A cohort of high risk infants from a local health authority were followed over the 1998/99 and 1999/2000 RSV seasons. The high risk population was defined as infants who, at the beginning of the seasons studied, were: (1) under 6 months old and born prior to 36 weeks gestation with no domiciliary oxygen requirement; or (2) under 24 months of age and discharged home in supplemental oxygen. All admissions with bronchiolitis during the season were identified.Results: A total of 370 high risk infants were identified for the 1998/99 season and 286 for the following year. Over the two years there were 68 admissions. Significantly more admissions occurred from group 2 infants. RSV was identified in 27 cases (four admissions per hundred high risk infants). Prophylaxis may have saved up to pound 195,134 in hospital costs over the two years, but would have cost pound 1.1 million in drug acquisition costs.Conclusions: Careful consideration of risk factors is needed when selecting infants for RSV prophylaxis. [ABSTRACT FROM AUTHOR]

Stratified medicine in paediatrics is increasingly becoming a reality, as our understanding of disease pathogenesis improves and novel treatment targets emerge. We have already seen some success in paediatrics in targeted therapies such as cystic fibrosis for specific cystic fibrosis transmembrane conductance regulator variants. With the increased speed and decreased cost of processing and analysing data from rare disease registries, we are increasingly able to use a systems biology approach (including '-omics') to screen across populations for molecules and genes of interest. Improving our understanding of the molecular mechanisms underlying disease, and how to classify patients according to these will lead the way for targeted therapies for individual patients. This review article will summarise how 'big data' and the 'omics' are being used and developed, and taking examples from paediatric renal medicine and rheumatology, demonstrate progress being made towards stratified medicine for children., Competing Interests: Competing interests: MAS has external consultancy roles with UCB, Retrophin and Pfizer. AVR has received speaker fees/consultancy for AbbVie, Eli Lilly, UCB and SOBI., (© Author(s) (or their employer(s)) 2019. No commercial re-use. See rights and permissions. Published by BMJ.)

This review provides paediatricians with an update on the new structure of the National Institute for Health Research's (NIHR) Clinical Research Network (CRN): Children and its role within the wider NIHR infrastructure. The network supports delivery of high-quality research within the NHS in England and supports researchers, through provision of staff and resources, with feasibility, site set-up, patient recruitment and study management. Since 2013, over 80% of commercial contract studies running within the UK sat within the UKCRN Portfolio. Of the diverse, increasing portfolio of studies supported by the network, many studies are interventional, with 33% being randomised controlled studies. Recruitment to studies supported by the network through the Children's Portfolio has consistently improved. Over 200 000 participants have been recruited to the Children's Portfolio studies to date, and there are currently approximately 500 studies open to recruitment. The CRN: Children has successfully involved patients and the public in all aspects of study design and delivery, including through the work of Generation R. Challenges remain in conducting paediatric research and the network is committed to supporting Children's research and further building on its achievements to date. Education and engagement of paediatricians within the network and research is important to further improving quality and delivery of paediatric research., Competing Interests: Competing interests: None declared., (Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.)

Systemic lupus erythematosus (SLE) is the archetypal systemic autoimmune disease, characterised by inflammation causing a wide spectrum of major clinical manifestations that may affect any organ. Childhood-onset SLE (cSLE) is more severe with greater damage and drug burden than adult-onset SLE. Understanding the pathogenesis of cSLE is a key step in directing medical management. The dysregulated immune system, that in health is usually vital in protecting the body from infection, contributes significantly to the disease process. Improved knowledge of disease mechanism will help to identify potential targets for novel agents and the identification of new biomarkers of disease activity. This review will present current knowledge of the innate and adaptive immune responses in cSLE and the optimal patient management that aims to control the disease. Innate immune dysregulation includes the overexpression of interferon-α, dendritic cell activation, neutrophil extracellular traps and phagocyte abnormalities. The classical adaptive immune system is over activated in lupus with excessive autoantibody production due to abnormalities in B and T cell regulation. Novel biologic medications are being developed to specifically target these areas with the ultimate aim of improving the long-term outlook and quality of life for children living with Lupus., (Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.)