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关键词： child   infectious   skin disease   sporotrichosis  

摘要： Aim Sporotrichosis is a dermatomycosis caused by the dimorphic fungus, Sporothrix schenckii, with various outbreaks across Australia attributed to mouldy hay. Our objective was to investigate the clinical presentation and management of cutaneous sporotrichosis in a paediatric population of Western Australia. Methods A retrospective case review was performed for S. schenckii infections in children below 18 years, between January 2000 and November 2017. Cases were identified from the state-wide laboratory database and additional clinical data obtained from medical records. Results Thirty-two cases of microbiologically proven S. schenckii infection were identified, mostly from rural areas (n = 20, 63%). Complete clinical data were available for 11 cases (34%). The most common risk factors were exposure to farm animals and hay, arthropod bites and outdoor activities. The median duration from symptom onset to correct diagnosis was 6 weeks (interquartile range: 4-7 weeks). Most cases were initially treated with multiple, broad-spectrum antibacterial agents (n = 7, 64%). Targeted therapy (itraconazole) was used in all cases once the diagnosis was made, with a median treatment duration of 5 months (interquartile range: 4-6 months). Morbidity included scarring (n = 4, 31%), itraconazole associated diarrhoea (n = 1, 8%) and mild hepatotoxicity (n = 1, 8%). Conclusion Summarising the clinical experience of these cases is a useful guide for clinical recognition and may serve to shorten the interval between onset and diagnosis, and avoid the need for antibacterial therapy. These data highlight the importance of recognising Sporotrichosis in children outside an outbreak setting, leading to timely diagnosis and appropriate treatment with antifungal agents.

关键词： Paediatric oncology   Immune checkpoint inhibitors   Medicinal product development  

摘要： The third multistakeholder Paediatric Strategy Forum organised by ACCELERATE and the European Medicines Agency focused on immune checkpoint inhibitors for use in combination therapy in children and adolescents. As immune checkpoint inhibitors, both as monotherapy and in combinations have shown impressive success in some adult malignancies and early phase trials in children of single agent checkpoint inhibitors have now been completed, it seemed an appropriate time to consider opportunities for paediatric studies of checkpoint inhibitors used in combination. Among paediatric patients, early clinical studies of checkpoint inhibitors used as monotherapy have demonstrated a high rate of activity, including complete responses, in Hodgkin lymphoma and hypermutant paediatric tumours. Activity has been very limited, however, in more common malignancies of childhood and adolescence. Furthermore, apart from tumour mutational burden, no other predictive biomarker for monotherapy activity in paediatric tumours has been identified. Based on these observations, there is collective agreement that there is no scientific rationale for children to be enrolled in new monotherapy trials of additional checkpoint inhibitors with the same mechanism of action of agents already studied (e.g. anti-PD1, anti-PDL1 anti-CTLA-4) unless additional scientific knowledge supporting a different approach becomes available. This shared perspective, based on scientific evidence and supported by paediatric oncology cooperative groups, should inform companies on whether a paediatric development plan is justified. This could then be proposed to regulators through the available regulatory tools. Generally, an academic-industry consensus on the scientific merits of a proposal before submission of a paediatric investigational plan would be of great benefit to determine which studies have the highest probability of generating new insights. There is already a rationale for the evaluation of combinations of chec

关键词： quality improvement   children   parents   experience measures  

摘要： Objective: Patient experience is a core component of quality and service improvement. In the case of child health, it is parents who often report on their experiences of the child's healthcare. However, these parent experiences are rarely used to drive change. Our aim was to develop and implement the systematic collection of parent experience data across a range of cardiothoracic services, using quality improvement methodology to implement change. Design: Plan-Do-Study-Act methodology was used to develop parent-reported experience measures. Setting: Specialist children's hospital. Participants: Parents of children under the care of one of a number of cardiothoracic services at the hospital. Intervention(s): Completion of questionnaires about hospital experience comprising quantitative and qualitative questions. Main Outcome Measure(s): Response rates and completion of a specific-measurable-achievable-realistic-timely (SMART) action plan by the individual clinical teams. Results: During the first cycle 17 surveys were developed for different clinical services and questionnaires sent/given to 1876 parents. The overall response rate was 30% (15-100%). Clinical teams developed SMART plans following eight (47%) surveys;three teams have reviewed and reported back on their progress against their plan and started on the second cycle of questionnaires. Challenges included lack of resources (time, finances and electronic data collection tools) and problems with actioning SMART plans. However, teams engaged positively, recognizing the importance of parent experience and actively seeking solutions to problems. Conclusions: Parent-reported experience measures are practical and acceptable quality improvement tools in healthcare but challenges need to be addressed, primarily related to resources, training and sustainability.

关键词： adults   China   clinical study   epidemiology   fungus infection   paediatrics   Sporothrix schenckii   sporotrichosis  

摘要： Sporotrichosis is endemic in Jilin Province of Northeast China. While paediatric cases make a substantial contribution to the epidemiological profile of sporotrichosis, the differences in the epidemiology and clinical manifestations of sporotrichosis between paediatric and adult patients remain unclear. We retrospectively reviewed the clinical records of 2968 cases of sporotrichosis (2113 adult patients aged >= 15 years and 855 paediatric patients aged < 15 years) over a nine-year period (01/01/2010-31/12/2018-). All the patients were diagnosed with sporotrichosis based on fungal culture of material from a skin lesion. In paediatric patients, the male:female ratio was 1.3:1, the incidence of sporotrichosis in the cold seasons was high (79.0%), most lesions occurred in the facial region (92.2%), and there was a preponderance of fixed cutaneous sporotrichosis (86.8%). In adult patients, the male:female ratio was 1:2.4, the incidence of sporotrichosis in the cold seasons was 66.0%, most lesions affected the extremities (48.6%) and the face (44.9%), and fixed cutaneous sporotrichosis was common (69.3%). The results indicate there were significant differences in the distribution of paediatric and adult sporotrichosis patients by sex, season with the highest occurrence of sporotrichosis, lesion sites and clinical types. Our results suggest that the epidemiology and clinical manifestations between paediatric and adult patients were different, and the route of infection of sporotrichosis in children may differ from that of adults in Jilin Province.

关键词： anaesthesia   outcome and process assessment   outpatient   paediatric   perioperative care   tertiary care centre  

摘要： Aim The role of pre-operative anaesthetic clinics (POAC) in adult practice is well-established and is developing in paediatric hospitals in the UK. Methods We carried out a retrospective survey of all patients assessed in our POAC by a consultant, determining the pre-operative problems and the perioperative anaesthetic interventions and outcomes. Results In 2016, 537 patients were seen by a consultant: the median age was 5.5 years (interquartile range 2.2-10.2) and median weight was 18.7 kg (interquartile range 12-28.7). 77% were ASA3 and 4% were ASA4. Seventy-five percent of patients referred for consultant assessment had a problem with at least one of the following four major body systems: cardiac (37%), respiratory (26%), airway (18%) and neurodevelopment (14%), Fifteen percent of these patients had two of these systems affected, and 3% had three or more. The rate of cancellation due to significant risk was 2.6% (n = 14): nine had significant cardiac risk and five had respiratory reasons. The rate of serious perioperative problems was 2.8% (n = 15): six were cardiac, six were respiratory, two neurological and one coagulation. Cardiac and airway problems occurred during anaesthesia, whereas problems relating to respiratory and neurological disease were post-operative. Of the 15 patients with three or more body system problems, 5 were cancelled or had a perioperative complication causing deterioration, 5 had a major complication but recovered fully and 5 proceeded to general anaesthesia (GA) without serious event. There were no deaths during GA or within 30 days. Conclusion In the POAC, consultants identified a wide range of GA-related potential problems enabling patients to be investigated, informed and prepared (or cancelled because of excess risk), and for appropriate resources to be allocated to achieve efficient and safe perioperative care.

关键词： Unintentional njuries   Management   Risk factors   Paediatric emergencies   Prevention  

摘要： Introduction: Thirty-eight million patients with injuries are treated in Emergency Departments every year, 90% of them being in the form of unintentional injuries (UIs). There are currently no global records of its management in Spain, or the risk factors that may be associated with them. The objective of this study is to describe the management of UIs in Spanish paediatric emergency departments, and to analyse factors related to the presence of serious injuries. Material and methods: A sub-study of a prospective multicentre observational study conducted over 12 months in 11 hospitals of the Spanish Paediatric Emergency Research Group (RiSEUP-SPERG), including children from 0 to 16 years of age consulting for UIs. Epidemiological data, circumstances of the injury, and data on emergency care and discharge destination were recorded on the 13th day of each month. Results: A total of 10,175 episodes were recorded, of which 1,941 were UIs (19.1%), including 1,673, of which 257 (15.4%) were severe. The most frequent complementary test was simple radiography (60.0%), and the most frequent procedure was limb immobilisation (38.6%). A significant relationship was found between presenting with a severe UI and age > 5 years (OR 2.24;95% CI: 1.61-3.16), history of fracture (OR 2.05;95% CI: 1.22-3.43), or sports activity as a mechanism of injury (OR 1.76;95% CI: 1.29-2.38), among others. Conclusion: In Spain, most UIs are not serious. X-rays and immobilisation of extremities are the most frequently performed tests and procedures. Severe UIs were associated with individual factors, such as age > 5 years or history of fracture, and with sports activity as a mechanism associated with severity. It is vital to implement measures to improve the prevention of these injuries and to support the training of caregivers through educational programmes. (c) 2020 Asociacion Espanola de Pediatria. Published by Elsevier Espana, S.L.U. This is an open access article under the CC BY-NC-ND license

摘要： Background Early use of parenteral nutrition in the paediatric intensive care unit (PICU) negatively affects development of executive functions, externalising behaviour, and visual-motor integration 2 years later, compared with omitting parenteral nutrition until PICU day 8 (late parenteral nutrition). The molecular basis of this finding is uncertain. We aimed to test the hypothesis that DNA methylation changes occur during critical illness and that early parenteral nutrition (or a specific macronutrient component hereof) contributes to these changes, which could explain its negative effects on neurocognitive development. Methods This pre-planned secondary analysis of the multicentre PEPaNIC trial (2012-18) included all patients with a last PICU day blood sample (n=825, aged 0-17 years at PICU admission) who were randomly allocated (1:1) to early parenteral nutrition or late parenteral nutrition, as compared with 352 demographically matched healthy children. Investigators were masked to treatment allocation. We used the Infinium Human MethylationEPIC BeadChip to determine the genome-wide peripheral blood leukocyte DNA methylation of 865 859 CpG sites, yielding high-quality results for 403 patients allocated to early parenteral nutrition and for 411 patients allocated to late parenteral nutrition. Applying a false discovery rate of less than 0.05, DNA methylation of patients on the last PICU day was compared with that of healthy children, after excluding all CpG sites differentially methylated upon PICU admission, because these reflected pre-admission conditions and altered leukocyte composition. We used bootstrapped multivariable linear and non-linear regression analyses to assess the effect of early parenteral nutrition versus late parenteral nutrition on illness-induced alterations in DNA methylation and to what extent differentially methylated CpG sites explained impaired neurocognitive development 2 years later. Findings During PICU stay, 159 CpG sites were meth

关键词： capacity   clinical research   competence   Europe   paediatric trials  

摘要： Aim The European Network of Excellence for Paediatric Clinical Research, known as the TEDDY Network, carried out a survey to determine the capacity and competence of paediatric centres to perform research studies. Methods A cross-sectional, web-based pilot survey was conducted from October 2016 to April 2017 with paediatric clinical research centres in 11 countries: Albania, Austria, Belgium, Denmark, Iceland, Ireland, Italy, Norway, Spain, Switzerland and the United Kingdom. All were registered with the TEDDY Network database. Results We approached 107 centres and 63 provided data on their experiences and expertise in paediatric clinical trials. Four groups of performance indicators were identified, referring to scientific experience, trial readiness, trial competence, regulatory issues, ethics and patients. Most centres were actively involved in paediatric clinical research: 53 centres (84.1%) had received funds for more than five paediatric studies in the last 5 years, and 42 (66.7%) had a specific clinical trial unit and dedicated study coordinators. We concluded that the European centres we studied had the capability and capacity to conduct paediatric trials, but there was still room for improvement, including enhanced collaboration. Conclusion This pilot survey demonstrated that there is potential for performing paediatric trials across Europe, but improvements are possible.

关键词： Sickle cell disease   haematopoietic cell transplantation   allogeneic transplantation   transplantation barriers  

摘要： Beginning early in childhood, patients with sickle cell disease [SCD;a group of genetic haemoglobin disorders characterized by the sickle or HbS mutation (HBB E7V)] are at risk of life-threatening and debilitating health events. Despite the high morbidity and mortality of this disease, haematopoietic cell transplantation (HCT), a curative therapy for SCD, remains underutilized. A variety of factors, including the limited availability of suitable donors, play a role in this trend, but do not fully explain the low frequency with which this therapy is employed. The objective of this study was to identify paediatric haematologists' attitudes about HCT as a treatment option for SCD, and to describe the impact of these attitudes on their practices of discussing HCT with families of children affected by this disease. A nationwide survey of paediatric haematologists in the United States was conducted between February and May 2016. Two hundred and eighty-seven surveys were included in the final analysis (response rate 20%). On average, respondents reported informing 42% of families about HCT as a treatment option (N = 248, 95% confidence interval: 38-46). Clinician attitudes about the cost and safety of HCT were associated with practices of discussing this therapy with families. These findings suggest that clinician attitudes and referral practices may play a role in the underutilization of this therapy in the SCD population.