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Brick T.,Great Ormond Street Hospital NHS Foundation Trust | Peters M.J.,Great Ormond Street Hospital NHS Foundation Trust | Peters M.J.,Guilford College
BMC Medicine | Year: 2014

Severe anemia contributes significantly to child mortality in sub-Saharan Africa. Blood transfusion is used in emergencies but carries risks. In BMC Medicine, Olupot-Olupot and colleagues report the findings of a phase II trial in children with severe anemia in Eastern Uganda. They provide important early safety and efficacy data supporting large volume whole blood transfusion (30 ml/kg) compared with the World Health Organization recommendation of 20 ml/kg. Large volume transfusions result in more rapid and frequent correction of severe anemia; they can be expected to reduce the risk of transfusions, and help manage the scarce resource of donor blood. However, severe anemia arises from varying combinations of acute, sub-acute and chronic etiologies.The Fluid Expansion As Supportive Therapy study reminds us that the risks and benefits of even simple interventions are complex, and that rapid normalization of physiology may not always be the best strategy. There is no substitute for high quality evidence and to this end we strongly support Olupot-Oluput and colleagues' call for a definitive trial of large volume transfusions in severe anemia.Please see related research article http://www.biomedcentral.com/1741-7015/12/67/abstract. © 2014 Brick and Peters; licensee BioMed Central Ltd.


Low D.,University of Washington | Walker I.,Great Ormond Street Hospital NHS Foundation Trust | Heitmiller E.S.,Johns Hopkins University
Paediatric Anaesthesia | Year: 2012

Summary Checklists have established themselves as a key safety process in the operating room environment. This paper describes the background and context of how checklists have evolved in medicine. It also highlights ongoing challenges with particular attention to the importance of nontechnical skills or human factors training with relation to checklist design, testing and implementation and ongoing coaching. © 2012 Blackwell Publishing Ltd.


Worth A.J.J.,Great Ormond Street Hospital NHS Foundation Trust | Booth C.,University College London | Veys P.,Great Ormond Street Hospital NHS Foundation Trust
Current Opinion in Hematology | Year: 2013

PURPOSE OF REVIEW: In this article, we summarize the recent advances in treating primary immune deficiency (PID) disorders by stem cell transplantation (SCT); we have focused on articles published in the past 2 years since the last major review of SCT for PID. RECENT FINDINGS: Analyses of the outcomes of SCT for PID by specific molecular defect have clarified which conditions are receptive to unconditioned transplants and which require more myeloablative conditioning. Improved outcomes for 'difficult' conditions [adenosine deaminase-severe combined immunodeficiency (ADA-SCID), major histocompatibility complex class II deficiency] and potential advantages of using cord blood as a stem cell source have also been described. Newborn screening for SCID identifies well babies with SCID: the optimal SCT protocol for such young infants remains to be determined. Reduced toxicity conditioning has been successfully used to treat conditions such as Wiskott-Aldrich syndrome and chronic granulomatous disease, offering curative engraftment with reduced transplant-related mortality. Similarly, treating children with familial hemophagocytic lymphohistiocytosis using reduced intensity conditioning SCT results in much improved outcomes. Advances in next generation sequencing have identified new diseases amenable to SCT, such as DOCK8 deficiency, resulting in improved quality of life and protection from malignancy. SUMMARY: Recent studies suggest that further improvements in treating PID with SCT are possible with a greater understanding of the genetics and immunobiology of these diseases, facilitating the matching of donor type and conditioning regimens, or indeed alternative therapies (such as gene therapy) to specific PID disorders.© 2013 Wolters Kluwer Health Lippincott Williams & Wilkins.


Solebo A.L.,University College London | Rahi J.,University College London | Rahi J.,Great Ormond Street Hospital NHS Foundation Trust | Rahi J.,Moorfields Eye Hospital NHS Foundation Trust
Archives of Disease in Childhood | Year: 2014

An estimated 19 million of the world's children are visually impaired, while 1.4 million are blind. Using the UK as a model for high income countries, from a population-based incidence study, the annual cumulative incidence of severe visual impairment/blindness (SVL/BL) is estimated to be 6/10 000 by age 15 years, with the incidence being highest in the first year of life. The population of visually impaired children within high, middle and lower income countries differ considerably between and within countries. The numerous and mainly uncommon disorders which can cause impaired vision result in heterogeneous population which includes a substantial proportion (for SVI/BL, the majority) of children with additional systemic disorders or impairments whose needs differ substantially from those with isolated vision impairment. Paediatricians and other paediatric professionals have a key role in early detection and multidisciplinary management to minimise the impact of visual impairment (VI) in childhood.


Harris K.A.,Great Ormond Street Hospital NHS Foundation Trust | Kenna D.T.D.,Public Health England
Journal of Medical Microbiology | Year: 2014

Mycobacterium abscessusis a significant pathogen in the cystic fibrosis patient population. PCR amplification and sequencing can provide accurate subspecies identification, and can predict macrolide susceptibility, which is becoming increasingly important for patient management. Molecular techniques for further typing of isolates provide tools for the ongoing investigations into the clinical impact of particular M. abscessusstrains. Whole-genome sequencing is likely to be the only technique that provides sufficient resolution for investigating transmission events between patients. © 2014 The Authors.


Smith P.,Great Ormond Street Hospital NHS Foundation Trust | Bailey C.R.,Guys And St Thomas Nhs Foundation Trust
Anaesthesia | Year: 2015

We performed a review of published literature comparing the i-gel™ with other supraglottic airway devices in children. Sixty-two articles were identified following a literature search; we included data from 14 randomised controlled trials and eight observational studies that compared i-gel sizes 1-2.5 with other commonly used, equivalently-sized, devices. The primary outcome in most studies was oropharyngeal leak pressure. In the 14 randomised trials the i-gel performed the same as the comparator device in five trials, significantly better in eight studies (p < 0.05) and significantly worse in one (p < 0.01). Seven studies assessed fibreoptic views of the larynx through the device; two found significantly better views through the i-gel. Three studies reported a shorter insertion time for the i-gel, whereas two reported a longer time. Insertion success rate, gastric tube placement and complications were similar for all the devices. Seven of the eight observational studies measured average oropharyngeal leak pressures of 20-27 cmH2O and all had first-time insertion success rates exceeding 90%. We conclude that the i-gel is at least equivalent to other supraglottic airway devices currently available for use in children, and may enable a higher oropharyngeal leak pressure and an improved fibreoptic view of the glottis. © 2014 The Association of Anaesthetists of Great Britain and Ireland.


Walker I.A.,Great Ormond Street Hospital NHS Foundation Trust | Reshamwalla S.,Lifebox Foundation | Wilson I.H.,Royal Devon and Exeter NHS Foundation Trust
British Journal of Anaesthesia | Year: 2012

Summary The concept of using a checklist in surgical and anaesthetic practice was energized by publication of the WHO Surgical Safety Checklist in 2008. It was believed that by routinely checking common safety issues, and by better team communication and dynamics, perioperative morbidity and mortality could be improved. The magnitude of improvement demonstrated by the WHO pilot studies was surprising. These initial results have been confirmed by further detailed work demonstrating that surgical checklists, when properly implemented, can make a substantial difference to patient safety. However, introducing surgical checklists is not as straightforward as it seems, and requires leadership, flexibility, and teamwork in a different way to that which is currently practiced. Future work should be aimed at ensuring effective implementation of the WHO Surgical Safety Checklist, which will benefit our patients on a global scale. © The Author [2012].


Buckland K.F.,University College London | Buckland K.F.,Great Ormond Street Hospital NHS Foundation Trust | Bobby Gaspar H.,University College London | Bobby Gaspar H.,Great Ormond Street Hospital NHS Foundation Trust
Advanced Drug Delivery Reviews | Year: 2014

The range of possible gene and cell therapy applications is expanding at an extremely rapid rate and advanced therapy medicinal products (ATMPs) are currently the hottest topic in novel medicines, particularly for inherited diseases. Paediatric patients stand to gain enormously from these novel therapies as it now seems plausible to develop a gene or cell therapy for a vast number of inherited diseases.There are a wide variety of potential gene and cell therapies in various stages of development. Patients who received first gene therapy treatments for primary immune deficiencies (PIDs) are reaching 10 and 15. years post-treatment, with robust and sustained immune recovery. Cell therapy clinical trials are underway for a variety of tissues including corneal, retinal and muscle repair and islet cell transplantation. Various cell therapy approaches are also being trialled to enhance the safety of bone marrow transplants, which should improve survival rates in childhood cancers and PIDs. Progress in genetic engineering of lymphocyte populations to target and kill cancerous cells is also described. If successful these ATMPs may enhance or replace the existing chemo-ablative therapy for several paediatric cancers. Emerging applications of gene therapy now include skin and neurological disorders such as epidermolysis bullosa, epilepsy and leukodystrophy. Gene therapy trials for haemophilia, muscular dystrophy and a range of metabolic disorders are underway. There is a vast array of potential advanced therapy medicinal products (ATMPs), and these are likely to be more cost effective than existing medicines. However, the first clinical trials have not been without setbacks and some of the key adverse events are discussed. Furthermore, the arrival of this novel class of therapies brings many new challenges for the healthcare industry. We present a summary of the key non-clinical factors required for successful delivery of these potential treatments. Technological advances are needed in vector design, raw material manufacture, cell culture and transduction methodology, and particularly in making all these technologies readily scalable. © 2014.


Van't Hoff W.,Great Ormond Street Hospital NHS Foundation Trust | Offringa M.,University of Toronto
Archives of Disease in Childhood | Year: 2015

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. © 2015, BMJ Publishing Group. All rights reserved.


Ivani G.,Regina Margherita Childrens Hospital | Walker I.,Great Ormond Street Hospital NHS Foundation Trust | Enright A.,Royal Jubilee Hospital
Paediatric Anaesthesia | Year: 2012

Summary Pediatric anesthesia is no longer a small subspecialty, but an important sector where developments in the new century have brought effective and safe management to children in the perioperative period. Unfortunately, what is common daily practice in the high-income countries with all the guidelines, checklists, instruments, and dedicated pediatric anesthesiologists is often only a dream in the low- and middle-income countries where the basic anesthesia services for improving the high rate of morbidity and mortality still are lacking. Anesthesia given by nonphysicians, with no monitoring, lack of elementary supplies, poor control of infections and hemorrhage, and no water or electricity are very often the 'usual' conditions. The World Health Organization is working hard to offer teams, basic equipments, and teaching and what is needed to offer children of these countries the same opportunities given in the industrialized countries. Other projects such as the Lifebox Project have a similar aim. This paper outlines some of what organizations are doing around the world, with different strategies all having the same target: safe pediatric anesthesia. © 2012 Blackwell Publishing Ltd.

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