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关键词： Paediatric oncology   Acute myeloid leukaemia   Paediatric Strategy Forum   Drug development   Cancer therapeutics  

摘要： Purpose: The current standard-of-care for front-line therapy for acute myeloid leukaemia (AML) results in short-term and long-term toxicity, but still approximately 40% of children relapse. Therefore, there is a major need to accelerate the evaluation of innovative medicines, yet drug development continues to be adult-focused. Furthermore, the large number of competing agents in rare patient populations requires coordinated prioritisation, within the global regulatory framework and cooperative group initiatives. Methods: The fourth multi-stakeholder Paediatric Strategy Forum focused on AML in children and adolescents. Results: CD123 is a high priority target and the paediatric development should be accelerated as a proof-of-concept. Efforts must be coordinated, however, as there are a limited number of studies that can be delivered. Studies of FLT3 inhibitors in agreed paediatric investigation plans present challenges to be completed because they require enrolment of a larger number of patients than actually exist. A consensus was developed by industry and academia of optimised clinical trials. For AML with rare mutations that are more frequent in adolescents than in children, adult trials should enrol adolescents and when scientifically justified, efficacy data could be extrapolated. Methodologies and definitions of minimal residual disease need to be standardised internationally and validated as a new response criterion. Industry supported, academic sponsored platform trials could identify products to be further developed. The Leukaemia and Lymphoma Society PedAL/EUpAL initiative has the potential to be a major advance in the field. Conclusion: These initiatives continue to accelerate drug development for children with AML and ultimately improve clinical outcomes. (C) 2020 Elsevier Ltd. All rights reserved.

摘要： Therapeutic hypothermia is among the adjuvant therapies suggested for refractory or super-refractory status epilepticus (R/SR-SE) in paediatric patients. Experimental evidence of neuroprotective and antiseizure effects provides a strong rationale for using therapeutic hypothermia in patients with status epilepticus. Thus, hypothermia between 20 degrees C and 33 degrees C in animals with status epilepticus is associated not only with significantly less neuronal damage, predominantly in the hippocampal CA1, CA2, and CA3 areas, but also with increased seizure latency and decreased seizure frequency and duration. Therapeutic hypothermia has rarely been used in paediatric R/SR-SE. In the few reported cases, seizure control was markedly improved but nearly half the patients experienced recurrences after rewarming. Studies are needed to clarify the modalities and indications of therapeutic hypothermia in paediatric patients with R/SR-SE. What this paper adds Hypothermia at 20 degrees C to 33 degrees C is neuroprotective and has antiseizure effects in experimental status epilepticus. In children, antiseizure effects are marked but recurrences after rewarming are common.

关键词： Hydrocephalus   Slit ventricle syndrome   Over-drainage   Fixed pressure gravitational valves   Miethke  

摘要： Objective The management of paediatric hydrocephalous remains challenging with the complication and revision rates being consistent in the literature. We hypothesise that the use of a fixed pressure gravitational valve for all de novo shunt insertions decreases the rate of functional revisions and that by implementing the routine use of gravitational valves in children, we would see a reduction in over-drainage and slit ventricle syndrome. Methods Retrospective data collection in a single centre, between February 2010 and August 2018. All patients undergoing fixed pressure gravitational Miethke valve insertion were included. We collected data on patients' demographics, reason for shunt insertion, type of valve and time to and reason for first revision. Data analysis was done with SPSS. Results A total of 235 patients were included in our study (124 males, 111 females), aged from 0 to 18.6 years (median 0.28). A total of 99 shunt revisions were documented, 30 of which secondary to ventricular catheter malfunction and 28 secondary to infection. The overall mechanical valve survival rates were 88.5%, 86.4% and 85.5% at 1, 2 and 5 years, respectively. Shunt revision due to over-drainage was documented in only 3 cases (1.3%). Conclusion Our results are in agreement with existing literature regarding shunt failures secondary to all extrinsic factors to the valve (infection and mechanical failure). We have shown that the use of a Miethke fixed pressure valve for all de novo shunt insertions in paediatric hydrocephalus decreases the need for functional revisions with valve survival rates being superior to the ones described for other types.

关键词： handover   medical education   paediatric  

摘要： Aim Handovers have been established as a valuable educational tool;nevertheless, a paucity of literature exists evaluating contributors to the educational experience. Our study aimed to investigate participants' educational experiences based on a paediatric handover, and the facilitators and barriers towards teaching and learning during this process. Methods A case study was conducted using semi-structured interviews. Data were collected exploring participants' perspectives of their educational experiences within a handover. An inductive, thematic content analysis was performed to identify key themes, assisted by nVivo software. Results Four key themes contributing to the educational experience within a handover were identified related to the organisation of a handover, team dynamics, teaching and learning moments and tensions between the clinical tasks and education. A model is proposed aimed at optimising education within this context. Conclusions Our study identified contributing factors towards the educational experiences at handovers and provides strategies to optimise these.

摘要： This commentary is on the original article by Fairhurst et al. on pages 1031–1039 of this issue.

摘要： After finding the first cases in Italy, the Government declared a state of emergency. In The Lancet, Stefano Spina and colleagues (1) reported the strategy of the Emergency Medical System (EMS) of the metropolitan area of Milan to contrast the recent COVID-19 outbreak in Italy. The EMS instituted a team that manages the patients and their flow to local hospitals. This response is critical, given the public health problem and the need to avoid the Emergency Department overcrowding.