In the UK, juvenile idiopathic arthritis is the most common inflammatory disorder in childhood, affecting 10 : 100,000 children and young people aged < 16 years each year, with a population ...prevalence of around 1 : 1000. Corticosteroids are commonly used to treat juvenile idiopathic arthritis; however, there is currently a lack of consensus as to which corticosteroid induction regimen should be used with various disease subtypes and severities of juvenile idiopathic arthritis.
The main study objective was to determine the feasibility of conducting a randomised controlled trial to compare the different corticosteroid induction regimens in children and young people with juvenile idiopathic arthritis.
This was a mixed-methods study. Work packages included a literature review; qualitative interviews with children and young people with juvenile idiopathic arthritis and their families; a questionnaire survey and screening log to establish current UK practice; a consensus meeting with health-care professionals, children and young people with juvenile idiopathic arthritis, and their families to establish the primary outcome; a feasibility study to pilot data capture and to collect data for future sample size calculations; and a final consensus meeting to establish the final protocol.
The setting was rheumatology clinics across the UK.
Children, young people and their families who attended clinics and health-care professionals took part in this mixed-methods study.
This study observed methods of prescribing corticosteroids across the UK.
The main study outcomes were the acceptability of a future trial for children, young people, their families and health-care professionals, and the feasibility of delivering such a trial.
Qualitative interviews identified differences in the views of children, young people and their families on a randomised controlled trial and potential barriers to recruitment. A total of 297 participants were screened from 13 centres in just less than 6 months. In practice, all routes of corticosteroid administration were used, and in all subtypes of juvenile idiopathic arthritis. Intra-articular corticosteroid injection was the most common treatment. The questionnaire surveys showed the varying clinical practice across the UK, but established intra-articular corticosteroids as the treatment control for a future trial. The primary outcome of choice for children, young people, their families and health-care professionals was the Juvenile Arthritis Disease Activity Score, 71-joint count. However, results from the feasibility study showed that, owing to missing blood test data, the clinical Juvenile Arthritis Disease Activity Score should be used. The Juvenile Arthritis Disease Activity Score, 71-joint count, and the clinical Juvenile Arthritis Disease Activity Score are composite disease activity scoring systems for juvenile arthritis. Two final trial protocols were established for a future randomised controlled trial.
Fewer clinics were included in this feasibility study than originally planned, limiting the ability to draw strong conclusions about these units to take part in future research.
A definitive randomised controlled trial is likely to be feasible based on the findings from this study; however, important recommendations should be taken into account when planning such a trial.
This mixed-methods study has laid down the foundations to develop the evidence base in this area and conducting a randomised control trial to compare different corticosteroid induction regimens in children and young people with juvenile idiopathic arthritis is likely to be feasible.
Current Controlled Trials ISRCTN16649996.
This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in
; Vol. 24, No. 36. See the NIHR Journals Library website for further project information.
IntroductionNon-ventilator-associated hospital-acquired pneumonia (nv-HAP) is the most common healthcare-associated infection (HCAI), is associated with high mortality and morbidity and places a ...major burden on healthcare systems. Diagnosis currently relies on chest x-rays to confirm pneumonia and sputum cultures to determine the microbiological cause. This approach leads to over-diagnosis of pneumonia, rarely identifies a causative pathogen and perpetuates unnecessary and imprecise antibiotic use. The HAP-FAST study aims to evaluate the feasibility of a randomised trial to evaluate the clinical impact of low-dose, non-contrast-enhanced thoracic CT scans and rapid molecular sputum analysis using the BIOFIRE® FILMARRAY® pneumonia plus panel (FAPP) for patients suspected with nv-HAP.Methods and analysisThe HAP-FAST feasibility study consists of a pilot randomised trial, a qualitative study, a costing analysis and exploratory analyses of clinical samples to investigate the immune-pathophysiology of HAP. Participants are identified and recruited from four acute hospitals in the Northwest of the UK. Using a Research Without Prior Consent model, the pilot trial will recruit 220 adult participants, with or without mental capacity, and with suspected HAP. HAP-FAST is a non-blinded, sequential, multiple assignment, randomised trial with two possible stages of randomisation: first, chest x-ray (CXR) or CT; second, if treated as nv-HAP, FAPP or standard microbiological processing alone (no FAPP). Pathogen-specific antibiotic guidance will be provided for FAPP results. Randomisation uses a web-based platform and followed up for 90 days. The feasibility of a future trial will be determined by assessing trial processes, outcome measures and patient and staff experiences.Ethics and disseminationThis study has undergone combined review by the UK NHS Research Ethics Committee and Health Research Authority. Results will be disseminated via peer-reviewed journals, via the funders’ website and through a range of media to engage the public.Trial registration number NCT05483309.
Currently, the routine treatment for acute appendicitis in the United Kingdom is an appendicectomy. However, there is increasing scientific interest and research into non-operative treatment of ...appendicitis in adults and children. While a number of studies have investigated non-operative treatment of appendicitis in adults, this research cannot be applied to the paediatric population. Ultimately, we aim to perform a UK-based multicentre randomised controlled trial (RCT) to test the clinical and cost effectiveness of non-operative treatment of acute uncomplicated appendicitis in children, as compared with appendicectomy. First, we will undertake a feasibility study to assess the feasibility of performing such a trial.
The study involves a feasibility RCT with a nested qualitative research to optimise recruitment as well as a health economic substudy. Children (aged 4-15 years inclusive) diagnosed with acute uncomplicated appendicitis that would normally be treated with an appendicectomy are eligible for the RCT. Exclusion criteria include clinical/radiological suspicion of perforated appendicitis, appendix mass or previous non-operative treatment of appendicitis. Participants will be randomised into one of two arms. Participants in the intervention arm are treated with antibiotics and regular clinical assessment to ensure clinical improvement. Participants in the control arm will receive appendicectomy. Randomisation will be minimised by age, sex, duration of symptoms and centre. Children and families who are approached for the RCT will be invited to participate in the embedded qualitative substudy, which includes recording of recruitment consultants and subsequent interviews with participants and non-participants and their families and recruiters. Analyses of these will inform interventions to optimise recruitment. The main study outcomes include recruitment rate (primary outcome), identification of strategies to optimise recruitment, performance of trial treatment pathways, clinical outcomes and safety of non-operative treatment. We have involved children, young people and parents in study design and delivery.
In this study we will explore the feasibility of performing a full efficacy RCT comparing non-operative treatment with appendicectomy in children with acute uncomplicated appendicitis. Factors determining success of the present study include recruitment rate, safety of non-operative treatment and adequate interest in the future RCT. Ultimately this feasibility study will form the foundation of the main RCT and reinforce its design.
ISRCTN15830435 . Registered on 8 February 2017.
Abstract
Background
Juvenile idiopathic arthritis (JIA) is an umbrella term for seven relapsing-remitting inflammatory conditions in children and young people (CYP). Early, intensive treatment can ...prevent long-term damage; however, established drugs exhibit a delayed response, prompting the need for rapid-onset treatment in the form of corticosteroids. Given a lack of consensus as to which corticosteroid induction regimen should be used for CYP with JIA, a feasibility trial of different regimens is needed. The aim was to achieve consensus among CYP, families, and healthcare professionals (HCPs) about the primary outcome measures and protocol components to include in a prospective feasibility study.
Methods
A modified Nominal Group Technique was used to achieve consensus on the most appropriate primary outcome measure to be included in a prospective feasibility study, in addition to other protocol components such as inclusion/exclusion criteria. Fifteen participants participated in the process, including a combination of CYP with JIA, families (n = 9) and HCPs (n = 6).
Results
In the first vote, participants agreed that Juvenile Arthritis Disease Activity Score (JADAS) and Physician Global Assessment Score were most meaningful. During sub-group discussions, the need for a composite score which captured the voice of CYP and families was emphasised. In the second vote, JADAS and the JIA Core Set were identified as the most important. Further discussions led to the results of the third vote, agreeing JADAS as the primary outcome measure of choice being measured at 6 weeks after commencement of treatment. The majority of HCPs, CYP and families voted for all JIA sub-types to be included in a prospective feasibility study, with some queries about the inclusion of systemic JIA given its unique presentation. Participants also identified the need for more frequent data collection time points to capture the rapid onset of corticosteroid action, while CYP and families opted for accessible mechanisms for participation, such as digital follow-up strategies.
Conclusion
It is feasible to include CYP, families and HCPs in synthesising complex concepts to agree by consensus the design components of clinical research. The primary outcome measure for inclusion in a prospective feasibility study of corticosteroid regimens in CYP with JIA was co-prioritised, with CYP and families taking a leading role in the ultimate selection of an appropriate outcome measure and other study protocol components. Using consensus methods with CYP, families and HCPs is a systematic and rigorous way in which to select outcome measures that are both meaningful and relevant to everyone involved in the care and treatment of CYP with JIA.
Conflicts of Interest
The authors declare no conflicts of interest.
Objectives
Electronic cigarettes (e‐cigarettes) are promoted as smoking cessation tools, yet they remain unavailable from Stop Smoking Services in England; the debate over their safety and efficacy ...is ongoing. This study was designed to explore perceptions and reasons for use or non‐use of electronic cigarettes as smoking cessation tools, among individuals engaged in Stop Smoking Services.
Methods
Semi‐structured telephone interviews were undertaken with twenty participants engaged in Stop Smoking Services in the north‐west of England. Participants comprised of both individuals who had tried e‐cigarettes (n = 6) and those who had not (n = 14). Interviews were digitally recorded and transcribed verbatim. The transcripts were subject to thematic analysis, which explored participants' beliefs and experiences of e‐cigarettes.
Results
A thematic analysis of transcripts suggested that the following three superordinate themes were prominent: (1) self‐efficacy and beliefs in e‐cigarettes; (2) e‐cigarettes as a smoking cessation aid; and (3) cues for e‐cigarette use. Participants, particularly never users, were especially concerned regarding e‐cigarette efficacy and safety. Overall, participants largely expressed uncertainty regarding e‐cigarette safety and efficacy, with some evidence of misunderstanding.
Conclusions
Evidence of uncertainty and misunderstanding regarding information on e‐cigarettes highlights the importance of providing smokers with concise, up‐to‐date information regarding e‐cigarettes, enabling smokers to make informed treatment decisions. Furthermore, identification of potential predictors of e‐cigarette use can be used to inform Stop Smoking Services provision and future research.
Statement of contribution
What is already known on this subject?
Research suggests that e‐cigarettes may help smokers quit smoking, but further studies are needed.
Electronic cigarette use in Stop Smoking Services has increased substantially in recent years, although e‐cigarettes are currently not regulated.
There is debate within the academic community regarding e‐cigarette efficacy and safety.
What does this study add?
Service users interviewed in the current study felt uncertain regarding e‐cigarette efficacy and safety.
E‐cigarette ever users viewed e‐cigarettes as effective and safe, more often than never users.
Accurate and up‐to‐date education will enable service users to make informed treatment decisions.
ObjectivesThe UK ambulance service is expected to now manage more patients in the community and avoid unnecessary transportations to hospital emergency departments (ED). Most people it attends who ...have experienced seizures have established epilepsy, have experienced uncomplicated seizures and so do not require the full facilities of an ED. Despite this, most are transported there. To understand why, we explored paramedics’ experiences of managing seizures.Design and settingSemistructured interviews were conducted with a purposive sample of paramedics from the English ambulance service. Interviews were transcribed and thematically analysed.ParticipantsA diverse sample of 19 professionals was recruited from 5 different ambulance NHS trusts and the College of Paramedics.ResultsParticipants’ confirmed how most seizure patients attended to do not clinically require an ED. They explained, however, that a number of factors influence their care decisions and create a momentum for these patients to still be taken. Of particular importance was the lack of access paramedics have to background medical information on patients. This, and the limited seizure training paramedics receive, meant paramedics often cannot interpret with confidence the normality of a seizure presentation and so transport patients out of precaution. The restricted time paramedics are expected to spend ‘on scene’ due to the way the ambulance services’ performance is measured and that are few alternative care pathways which can be used for seizure patients also made conveyance likely.ConclusionsParamedics are working within a system that does not currently facilitate non-conveyance of seizure patients. Organisational, structural, professional and educational factors impact care decisions and means transportation to ED remains the default option. Improving paramedics access to medical histories, their seizure management training and developing performance measures for the service that incentivise care that is cost-effective for all of the health service might reduce unnecessary conveyances to ED.
ObjectivesWe explored children’s views on research without prior consent (RWPC) and sought to identify ways of involving children in research discussions.DesignQualitative interview ...study.SettingParticipants were recruited through a UK children’s hospital and online advertising.Participants16 children aged 7–15 years with a diagnosis of asthma (n=14) or anaphylaxis (n=2) with recent (<12 months) experience of emergency care.ResultsChildren were keen to be included in medical research and viewed RWPC as acceptable in emergency situations if trial interventions were judged safe. Children trusted that doctors would know about their trial participation and act in their best interests. All felt that children should be informed about the research following their recovery and involved in discussions with a clinician or their parent(s) about the use of data already collected as well as continued participation in the trial (if applicable). Participants suggested methods to inform children about their trial participation including an animation.ConclusionsChildren supported, and were keen to be involved in, clinical trials in emergency situations. We present guidance and an animation that practitioners and parents might use to involve children in trial discussions following their recovery.
Sodium valproate (VPA) has been associated with a reduced risk of head and neck cancer development. The potential protective mechanism of action is believed to be via inhibition of histone ...deacetylase and subsequent epigenetic reprogramming. SAVER is a phase IIb open-label, randomised control trial of VPA as a chemopreventive agent in patients with high-risk oral epithelial dysplasia (OED). The aim of the trial is to gather preliminary evidence of the clinical and biological effects of VPA upon OED and assess the feasibility and acceptability of such a trial, with a view to inform a future definitive phase III study.
One hundred and ten patients with high-risk OED will be recruited from up to 10 secondary care sites in the UK and randomised into either VPA or observation only for 4 months. Women of childbearing potential will be excluded due to the teratogenic properties of VPA. Tissue and blood samples will be collected prior to randomisation and on the last day of the intervention/observation-only period (end of 4 months). Clinical measurement and additional safety bloods will be taken at multiple time points during the trial. The primary outcome will be a composite, surrogate endpoint of change in lesion size, change in grade of dysplasia and change in LOH profile at 8 key microsatellite regions. Feasibility outcomes will include recruitment targets, compliance with the study protocol and adverse effects. A qualitative sub-study will explore patient experience and perception of the trial.
The current management options for patients with high-risk OED are limited and mostly include surgical resection and clinical surveillance. However, there remains little evidence whether surgery can effectively lead to a notable reduction in the risk of oral cancer development. Similarly, surveillance is associated with concerns regarding delayed diagnosis of OED progressing to malignancy. The SAVER trial provides an opportunity to investigate the effects of a repurposed, inexpensive and well-tolerated medication as a potential chemopreventive strategy for patients with high-risk OED. The clinical and biological findings of SAVER will inform the appropriateness, design and feasibility of a definitive phase III trial.
The trial is registered with the European Clinical Trials Database ( Eudra-CT 2018-000197-30 ). ( http://www.isrctn.com/ISRCTN12448611 ). The trial was prospectively registered on 24/04/2018.
IntroductionThe UK ambulance service often attends to suspected seizures. Most persons attended to will not require the facilities of a hospital emergency department (ED) and so should be managed at ...scene or by using alternative care pathways. Most though are transported to ED. One factor that helps explain this is paramedics can have low confidence in managing seizures.ObjectivesWith a view to ultimately developing additional seizure management training for practicing paramedics, we explored their learning needs, delivery preferences and potential drivers and barriers to uptake and effectiveness.Design and settingSemistructured interviews were conducted with a purposive sample of paramedics from the English ambulance service. Interviews were transcribed and thematically analysed.ParticipantsA diverse sample of 19 professionals was recruited from 5 different ambulance NHS trusts and the College of Paramedics.ResultsParticipants said seizure management was neglected within basic and postregistration paramedic training. Most welcomed additional learning opportunities and identified gaps in knowledge. This included how to differentiate between seizure types and patients that do and do not need ED. Practical, interactive e-learning was deemed the most preferable delivery format. To allow paramedics to fully implement any increase in skill resulting from training, organisational and structural changes were said to be needed. This includes not penalising paramedics for likely spending longer on scene.ConclusionsThis study provides the first evidence on the learning needs and preferences of paramedics regarding seizures. It can be used to inform the development of a bespoke training programme for paramedics. Future research should develop and then assess the benefit such training has on paramedic confidence and on the quality of care they offer to seizure patients.
While non-operative treatment has emerged as an alternative to surgery for the treatment of uncomplicated acute appendicitis in children, comparative patient-centred outcomes are not well documented. ...We investigated these in a feasibility randomised trial. Of 57 randomised participants, data were available for 26. Compared with appendicectomy, children allocated to non-operative treatment reported higher short-term quality of life scores, shorter duration of requiring analgesia, more rapid return to normal activities and shorter parental absence from work. These preliminary data suggest differences exist in recovery profile and quality of life between these treatments that are important to measure in a larger RCT. Trial registration number is ISRCTN15830435.
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