Children with the single-gene disorder neurofibromatosis type 1 (NF1) appear to be at an increased risk for autism spectrum disorder (ASD) and exhibit a unique social-cognitive phenotype compared with children with idiopathic ASD. A complete framework is required to better understand autism in NF1, from neurobiological levels through to behavioural and functional outcomes. The primary aims of this study are to establish the frequency of ASD in children with NF1, examine the social cognitive phenotype, investigate the neuropsychological processes contributing to ASD symptoms and poor social functioning in children with NF1, and to investigate novel structural and functional neurobiological markers of ASD and social dysfunction in NF1. The secondary aim of this study is to compare the neuropsychological and neurobiological features of ASD in children with NF1 to a matched group of patients with idiopathic ASD.
This is an international, multisite, prospective, cross-sectional cohort study of children with NF1, idiopathic ASD and typically developing (TD) controls. Participants will be 200 children with NF1 (3–15 years of age), 70 TD participants (3–15 years) and 35 children with idiopathic ASD (7–15 years). Idiopathic ASD and NF1 cases will be matched on age, sex and intelligence. All participants will complete cognitive testing and parents will rate their child’s behaviour on standardised questionnaires. Neuroimaging will be completed by a subset of participants aged 7 years and older. Children with NF1 that screen at risk for ASD on the parent-rated Social Responsiveness Scale 2nd Edition will be invited back to complete the Autism Diagnostic Observation Scale 2nd Edition and Autism Diagnostic Interview-Revised to determine whether they fulfil ASD diagnostic criteria.
This study has hospital ethics approval and the results will be disseminated through peer-reviewed publications and international conferences.
Cumulative impact zones (CIZs) are a widely implemented local policy intended to restrict alcohol availability in areas proliferated with licensed outlets. Limited previous research has questioned their effectiveness and suggested they may play a more nuanced role in shaping local alcohol environments. This study evaluates the association between CIZ implementation and the number of licence applications made, and the number issued, relative to a control region.
A quantitative observational study.
The inner London Borough of Southwark, which currently enforces three CIZs.
Licence applications received by Southwark Council’s Licensing Authority between 1 April 2006 and 31 March 2017 (n=1254).
Five outlet types were categorised and evaluated: drinking establishments, eateries, takeaways, off sales and other outlets. Primary outcome measures were the number of applications received and the number of licences issued. These were analysed using Poisson regression of counts over time.
Across all CIZs, implementation was associated with greater increases in the number of eateries in CIZ regions (incidence rate ratio (IRR)=1.58, 95% CI: 1.02–2.45, p=0.04) and number of takeaway venues (IRR=3.89, 95% CI: 1.32–11.49, p=0.01), relative to the control area. No discernible association was found for the remaining outlet types. Disaggregating by area indicated a 10-fold relative increase in the number of new eateries in Peckham CIZ (IRR=10.38, 95% CI: 1.39–77.66, p=0.02) and a fourfold relative increase in the number of newly licensed takeaways in Bankside CIZ (IRR=4.38, 95% CI: 1.20–15.91, p=0.03).
CIZs may be useful as policy levers to shape local alcohol environments to support the licensing goals of specific geographical areas and diversify the night-time economy.
To conduct a health economic evaluation of a proposed investment in urban bicycle infrastructure in Stockholm County, Sweden.
A cost-effectiveness analysis is undertaken from a healthcare perspective. Investment costs over a 50-year life cycle are offset by averted healthcare costs and compared with estimated long-term impacts on morbidity, quantified in disability-adjusted life years (DALYs). The results are re-calculated under different assumptions to model the effects of uncertainty.
The Municipality of Stockholm (population 2.27 million) committed funds for bicycle path infrastructure with the aim of achieving a 15% increase in the number of bicycle commuters by 2030. This work is based on a previously constructed scenario, in which individual registry data on home and work address and a transport model allocation to different modes of transport identified 111 487 individuals with the physical capacity to bicycle to work within 30 min but that currently drive a car to work.
Morbidity impacts and healthcare costs attributed to increased physical activity, change in air pollution exposure and accident risk are quantified under the scenario. The largest reduction in healthcare costs is attributed to increased physical activity and the second largest to reduced air pollution exposure among the population of Greater Stockholm. The expected net benefit from the investment is 8.7% of the 2017 Stockholm County healthcare budget, and 3.7% after discounting. The economic evaluation estimates that the intervention is cost-effective and each DALY averted gives a surplus of 9933. The results remained robust under varied assumptions pertaining to reduced numbers of additional bicycle commuters.
Investing in urban infrastructure to increase bicycling as active transport is cost-effective from a healthcare sector perspective.
Commentary on: Lang JE, Bunnell HT, Hossain MJ, et al. Being overweight or obese and the development of asthma. Pediatrics 2018;142. pii: e20182119. doi: 10.1542/peds.2018-2119.
The findings of this study further support the importance of reducing childhood obesity levels to reduce the risk for comorbidities, including asthma. This study also found a more modest increase in the risk of childhood asthma in overweight children, highlighting the importance of interventions focusing on preventing further weight gain for overweight children. Future research can focus on understanding causal pathways and developing effective interventions to manage and prevent childhood obesity and associated comorbidities.
The findings of this study further support the importance of reducing childhood obesity levels to reduce the risk for comorbidities, including asthma.
This study also found a more modest increase in the risk of childhood asthma in overweight children, highlighting the importance of interventions focusing on preventing further weight gain for overweight children.
Future research can focus on understanding causal pathways and developing effective interventions to manage and prevent childhood obesity and associated comorbidities.
There is clear evidence that obesity and asthma both place a significant burden on children, their families and healthcare systems.
Cancer care has expanded from a disease-focused, survival-oriented model to an approach that now considers how survivors can live well in the aftermath of intensive therapy, where they may deal with significant changes to their bodies, mental health or emotional well-being. Research evidence supports the benefit of exercise during and following cancer treatments for cancer-related symptoms, physical functioning and fitness, and health-related quality of life. To move this efficacy evidence into practice, we designed and launched a 5-year study to evaluate the relative benefit from implementing a clinic-to-community-based cancer and exercise model of care.
A hybrid effectiveness and implementation trial design is being used to evaluate the effectiveness of delivery of community-based exercise and to collect data on implementation of the programme. The study opened in January 2017, with estimated completion by January 2022. The programme will be delivered in seven cities across the province of Alberta, Canada, with sites including three academic institutions, six YMCA locations, Wellspring Edmonton and Calgary, and six municipal fitness centres. Participants are adult cancer survivors (n=2500) from all tumour groups and stages and at any time point along their cancer treatment trajectory, up to 3 years post treatment completion. Survivors take part in a minimum of 60 min of mild-to-moderate intensity full body exercise twice weekly for a 12-week period. The primary effectiveness outcome is the proportion of participants meeting or exceeding 150 min of moderate intensity exercise per week at 1-year follow-up. The Reach, Effectiveness, Adoption, Implementation and Maintenance (RE-AIM) framework will be utilised to capture individual-level and organizational-level impact of the exercise programme at 12 and 24 weeks and 1-year follow-up. The cohort of survivors participating in the study will allow for long-term (>5-year) evaluation of rates of cancer recurrence and secondary cancers beyond the funding period.
The study was approved by the Health Research Ethics Board of Alberta. The study is funded by Alberta Innovates and the Alberta Cancer Foundation. The study will help to answer critical questions on the effectiveness of cancer-specific community-based exercise programming in both the short-term and the long-term. Collectively, the findings will help to inform the acceptability, adoption, feasibility, reach and sustainability of community-based exercise.
With several qualified community practitioner nurse prescribers (CPNPs) not prescribing, this research aimed to understand what influences this behaviour.
A qualitative research design.
Semi‐structured interviews, based on the theoretical domains framework (TDF) were conducted with 20 CPNPs. Data collection took place between March‐July 2018 and continued until data saturation was reached.
Nine themes inductively explained prescribing behaviour: 1) ‘Knowledge and experience’; 2) ‘Consultation and communication skills’; 3) ‘Professional confidence and identity’; 4) ‘Wanting the best outcome’; 5) ‘NHS versus patient cost’; 6) ‘Emotion‐led decisions’; 7) ‘Time allocation’; 8) ‘Formulary access’ and 9) ‘Supporting environment for patient‐centred care’. Themes were then deductively mapped to the TDF and COM‐B.
There is an ongoing need to support community practitioner nurse prescribers’ ‘Capability’ to prescribe in terms of knowledge and aquired skills; ‘Opportunity’ to make prescribing easier, such as access to a wider and up to date nurse formulary alongside effective clinical support; and ‘Motivation’ to feel confident in prescribing behaviour, highlighting positive patient outcomes while reducing perceived issues such as cost and non‐adherence.
Findings show that Capability, Opportunity and Motivation all influence the decision to prescribe. Those responsible for professional regulation and training should ensure community practitioner nurse prescribers have access to the relevant knowledge, skills and formulary to facilitate their prescribing behaviour. Professional confidence and identity as a prescriber should be encouraged, with acknowledgment of influences such as cost and emotion. An environment that allows for patient‐centred care and the best outcome should be supported, this may mean increasing time allocated to consultations.
Low back pain is one of the most common and burdensome chronic conditions worldwide. Lifestyle factors, such as excess weight, physical inactivity, poor diet and smoking, are linked to low back pain chronicity and disability. There are few high-quality randomised controlled trials that investigate the effects of targeting lifestyle risk factors in people with chronic low back pain.
The aim of this study is to determine the effectiveness of a Healthy Lifestyle Program (HeLP) for low back pain targeting weight, physical activity, diet and smoking to reduce disability in patients with chronic low back pain compared with usual care. This is a randomised controlled trial, with participants stratified by body mass index, allocated 1:1 to the HeLP intervention or usual physiotherapy care. HeLP involves three main components: (1) clinical consultations with a physiotherapist and dietitian; (2) educational resources; and (3) telephone-based health coaching support for lifestyle risk factors. The primary outcome is disability (Roland Morris Disability Questionnaire) at 26 weeks. Secondary outcomes include pain intensity, weight, quality of life and smoking status. Data will be collected at baseline, and at weeks 6, 12, 26 and 52. Patients with chronic low back pain who have at least one health risk factor (are overweight or obese, are smokers and have inadequate physical activity or fruit and vegetable consumption) will be recruited from primary or secondary care, or the community. Primary outcome data will be analysed by intention to treat using linear mixed-effects regression models. We will conduct three supplementary analyses: causal mediation analysis, complier average causal effects analysis and economic analysis.
This study was approved by the Hunter New England Research Ethics Committee (Approval No 17/02/15/4.05), and the University of Newcastle Human Research Ethics Committee (Ref No H-2017-0222). Outcomes of this trial and supplementary analyses will be disseminated through publications in peer-reviewed journals and conference presentations.
Controlled trials support the efficacy of exercise as a treatment modality for chronic conditions, yet effectiveness of real-world Exercise Physiology services is yet to be determined. This study will investigate the efficacy and cost-effectiveness of services provided by Accredited Exercise Physiologists (AEPs) for clients with type 2 diabetes (T2D) in clinical practice.
A non-randomised, opportunistic control, longitudinal design trial will be conducted at ten Exercise Physiology Clinics. Participants will be individuals with T2D attending one of the Exercise Physiology Clinics for routine AEP services (exercise prescription and counselling) (intervention) or individuals with T2D not receiving AEP services (usual care) (control). The experimental period will be 6 months with measurements performed at baseline and at 6 months. Primary outcome measures will be glycosylated haemoglobin (HbA1c), resting brachial blood pressure (BP), body mass index, waist circumference, 6 min walk test, grip strength, 30 s sit to stand, Medical Outcomes Short-Form 36-Item Health Survey and Active Australia Questionnaire. Secondary outcomes will be medication usage, out-of-pocket expenses, incidental, billable and non-billable health professional encounters and work missed through ill health. Healthcare utilisation will be measured for 12 months prior to, during and 12 months after trial participation using linked data from Medicare Benefits Schedule and Pharmaceutical Benefits Scheme data.
The study is a multicentre trial comprising: University of Tasmania, University of New South Wales Lifestyle Clinic, University of Canberra, Baker Heart and Diabetes Institute (covered under the ethics approval of University of Tasmania Health and Medical Ethics Committee H0015266), Deakin University (Approval number: 2016–187), Australian Catholic University (2016–304R), Queensland University of Technology (1600000049), University of South Australia (0000035306), University of Western Australia (RA/4/1/8282) and Canberra Hospital (ETH.8.17.170). The findings of this clinical trial will be communicated via peer-reviewed journal articles, conference presentations, social media and broadcast media.
Chronic low back pain (CLBP) is the leading cause of disability worldwide. However, there is no consensus in the literature regarding optimal management. Exercise intervention is the most widely used treatment as it likely influences contributing factors such as physical and psychological. Literature evaluating the effects of exercise on CLBP is often generalised, non-specific and employs inconsistent outcome measures. Moreover, the mechanisms behind exercise-related improvements are poorly understood. Recently, research has emerged identifying associations between neuromuscular-biomechanical impairments and CLBP-related disability. This information can be used as the basis for more specific and, potentially more efficacious exercise interventions for CLBP patients.
Ninety-four participants (including both males and females) with CLBP aged 18–65 who present for treatment to a Melbourne-based private physiotherapy practice will be recruited and randomised into one of two treatment groups. Following baseline assessment, participants will be randomly allocated to receive either: (i) strengthening exercises in combination with lumbar force accuracy training exercises or (ii) strengthening exercises alone. Participants will attend exercise sessions twice a week for 12 weeks, with assessments conducted at baseline, midway (ie, 6 weeks into the trial) and at trial completion. All exercise interventions will be supervised by a qualified physiotherapist trained in the intervention protocol. The primary outcome will be functional disability measured using the Oswestry Disability Index. Other psychosocial and mechanistic parameters will also be measured.
This study was given approval by the University of Melbourne Behavioural and Social Sciences Human Ethics Sub-Committee on 8 August 2017, reference number 1 749 845. Results of the randomised controlled trial will be published in peer-reviewed journals.
To examine how observer and self-report measures of shared decision-making (SDM) evaluate the decision-making activities that patients and clinicians undertake in routine consultations.
Multi-method study using observational and self-reported measures of SDM and qualitative analysis.
Breast care and predialysis teams who had already implemented SDM.
Breast care consultants, clinical nurse specialists and patients who were making decisions about treatment for early-stage breast cancer. Predialysis clinical nurse specialists and patients who needed to make dialysis treatment decisions.
Consultations were audio recorded, transcribed and thematically analysed. SDM was measured using Observer OPTION-5 and a dyadic SureScore self-reported measure.
Twenty-two breast and 21 renal consultations were analysed. SureScore indicated that clinicians and patients felt SDM was occurring, but scores showed ceiling effects for most participants, making differentiation difficult. There was mismatch between SureScore and OPTION-5 score data, the latter showing that each consultation lacked at least some elements of SDM. Highest scoring items using OPTION-5 were ‘incorporating patient preferences into decisions’ for the breast team (mean 18.5, range 12.5–20, SD 2.39) and ‘eliciting patient preferences to options’ for the renal team (mean 16.15, range 10–20, SD 3.48). Thematic analysis identified that the SDM encounter is difficult to measure because decision-making is often distributed across encounters and time, with multiple people, it is contextually adapted and can involve multiple decisions.
Self-reported measures can broadly indicate satisfaction with SDM, but do not tell us about the quality of the interaction and are unlikely to capture the multi-staged nature of the SDM process. Observational measures provide an indication of the extent to which elements of SDM are present in the observed consultation, but cannot explain why some elements might not be present or scored lower. Findings are important when considering measuring SDM in practice.
To examine prevalence, types, challenges and the impact of medical/nursing tasks (MNT) on caregivers of older adults with dementia.
Medical/nursing tasks have been perceived as a professional healthcare role; however, research shows that many caregivers of older adults with dementia perform those tasks in the home, such as giving injections, tube feedings or operation of medical equipment. Little is known about the caregivers’ challenges in engaging in these MNT.
Ovid MEDLINE, CINAHL, PsycINFO and Web of Science databases were searched to explore MNT among caregivers of older adults with dementia who lived in a community setting. Four quantitative and nine qualitative studies published between 1980–2018 were included. Overall, process of the review was guided by PRISMA.
About 67% of U.S. caregivers of older adults with dementia performed MNT, including managing multiple medications, wound care and nutritional management. Care recipients' cognitive impairment complicated the provision of those tasks due to their limited cognitive functioning, behavioural changes, comorbidities and complex medication regimen. Insufficient information and training from healthcare professionals as well as caregivers’ age and their own health problems made performance of those tasks even more challenging. As a result, caregivers frequently suffered from emotional distress such as worrying, anxiety and sleep disturbance.
Medical/nursing tasks have become one of the daily tasks of caregivers of older adults with dementia within the home. However, the tasks are difficult and complicated, and inadequate support from healthcare professionals may compromise the caregivers’ well‐being.
Healthcare professionals should provide education and should be aware of caregivers’ needs related to MNT. Structured‐medical information, skill‐based instructions and hands‐on training may be beneficial to decrease the caregivers’ distress from MNT.
To conduct a UK-wide survey of young people who have experienced cancer, carers and professionals, to identify and prioritise research questions to inform decisions of research funders and support the case for research with this unique cancer population.
James Lind Alliance Priority Setting Partnership.
UK health service and community.
A steering group oversaw the initiative and partner organisations were recruited. Unanswered questions were collected in an online survey. Evidence searching verified uncertainties. An interim survey was used to rank questions prior to a final prioritisation workshop.
Young people aged 13–24 years with a current or previous cancer diagnosis, their families, friends, partners and professionals who work with this population.
Two hundred and ninety-two respondents submitted 855 potential questions. Following a refining process and removal of ‘out of scope’ questions, 208 unique questions remained. Systematic evidence checking identified seven answered questions and 16 were the subject of ongoing studies. The interim survey was completed by 174 participants. The top 30 questions were prioritised at a workshop attended by 25 young people, parents and multidisciplinary professionals. The top three priorities are: (1) What psychological support package improves psychological well-being, social functioning and mental health during and after treatment? (2) What interventions, including self-care, can reduce or reverse adverse short-term and long-term effects of cancer treatment? (3) What are the best strategies to improve access to clinical trials? The remaining questions reflect the complete cancer pathway: new therapies, life after cancer, support, education/employment, relapse and end-of-life care.
We have identified shared research priorities for young people with cancer using a rigorous, person-centred approach involving stakeholders typically not involved in setting the research agenda. The breadth of priorities suggest future research should focus on holistic and psychosocial care delivery as well as traditional drug/biology research.
One of the main harms from breast cancer screening is the anxiety caused by false positive results. Various factors may be associated with false-positive anxiety. One modifiable factor may be the method of communication used to deliver results. The aim of this study is to measure the effect on anxiety of receiving benign biopsy results in-person or by telephone.
This is a multi-centre cluster randomised crossover trial in the English National Health Service Breast Screening Programme (NHSBSP) involving repeated survey measures at four time points. Participants will be women of screening age who have a biopsy following a suspicious mammography result, who ultimately receive a benign or normal (B1) result. Centres will trial both telephone and in-person results on a month-by-month basis, being randomised to which communication method will be trialled first. Women will be blinded to the method of communication they will receive. The analysis will compare women who have received telephone results and women who have received in-person results. The primary outcome measure will be anxiety (measured by the Psychological Consequences Questionnaire) after receiving results, while controlling for baseline anxiety. Secondary outcome measures will include anxiety at 3 and 6 months post-results, understanding of results and patient preferences for how results are communicated. Qualitative telephone interviews will also be conducted to further explore women’s reasons for communication preferences. Qualitative and quantitative data will be integrated after initial separate analysis using the pillar integration process.
This study has been approved by the Public Health England Breast Screening Programme Research Advisory Committee, (BSPRAC_0013, ODR1718_040) and the National Health Service Health Research Authority (HRA) West Midlands—Coventry & Warwickshire Research Ethics Committee (17/WM/0313). The findings from this study will be disseminated to key stakeholders within the NHSBSP and via academic publications.
This research is part of a PhD award and is funded by the Economic and Social Research Council Doctoral Training Centre at the University of Warwick and Public Health England. The sponsor for this research is Jane Prewett (email@example.com).
The study purpose is to explore adolescent and adult women's experiences, perceptions, beliefs, knowledge and behaviours related to bladder health across the life course using a socioecological perspective. Lower urinary tract symptoms affect between 20–40% of young adult to middle‐aged women, with symptoms increasing in incidence and severity with aging. There is limited evidence to address bladder health promotion and prevention of dysfunction. This first study of the Prevention of Lower Urinary Tract Symptoms (PLUS) Research Consortium is designed to address gaps in existing qualitative research in this area.
This focus group study will be implemented across seven geographically diverse United States research centres using a semi‐structured focus group guide informed by a conceptual framework based on the socioecological model.
The study was approved in July 2017. A total of 44 focus groups composed of 6–8 participants representing six different age categories (ranging from 11 to over 65 years) will be completed. We aim to recruit participants with diverse demographic and personal characteristics including race, ethnicity, education, socioeconomic status, urban/rural residence, physical/health conditions, and urinary symptom experience. Six of the focus groups will be conducted in Spanish and translated into English. Focus group transcripts will undergo content analysis and data interpretation to identify and classify themes and articulate emerging themes.
This foundational qualitative study seeks to develop an evidence base to inform future research on bladder health promotion in adolescent and adult women.
This study has the potential to provide new insights and understanding into adolescent and adult women's lived experience of bladder health, the experience of lower urinary symptoms and knowledge and beliefs across the life course.
To describe the exercise, physical fitness and musculoskeletal health of nursing students.
Nursing students are prone to musculoskeletal disorders restricting work ability. Physical fitness and leisure‐time exercise may affect responses to workplace exposures and risk for work‐related musculoskeletal disorders.
A cross‐sectional study.
Between August 2013 and April 2015, a convenience sample of 111 nursing students performed submaximal exercise tests. Nursing work, exercise and musculoskeletal health were surveyed and analysed descriptively.
Students’ mean age was 30.0 years, 89.2% were female and 20.0% worked in nursing while studying. Highest annual prevalence of musculoskeletal trouble was in low back (45.6%), neck (32.0%) and shoulder (18.5%) regions. Most exercised regularly but did not meet weekly cardiorespiratory, resistance, neuromotor and flexibility exercise recommendations and had poor to average fitness levels. Approximately 40% were overweight or obese; 26.1% had risk for obesity‐related disease.
Interventions to improve nursing students’ physical condition before entering the nursing workforce appear warranted.
Imbalance between physical work capacity and demanding workloads increases musculoskeletal disorder risk amongst undergraduate nursing students. A large proportion studied reported recent musculoskeletal trouble (particularly low back, neck and shoulder). They exhibited modifiable characteristics of overweight/obese, poor fitness and inadequate leisure‐time exercise, predisposing them to work‐related musculoskeletal disorders. Undergraduate preparation should raise nursing students’ health literacy about physical fitness and ways to achieve it, for their musculoskeletal health and work capacity. Improving nursing students’ fitness may enhance their work preparedness and help them achieve longevity in this physically demanding occupation.
To explore the views of healthcare professionals from cystic fibrosis (CF) multidisciplinary teams (MDT) on physical activity for adolescents with CF, the specific strategies used for physical activity promotion and associated challenges.
In this exploratory study, in-depth qualitative interviews were conducted with 15 healthcare professionals from CF MDTs to explore their views surrounding physical activity promotion for adolescents with CF.
Eleven physiotherapists (nine female), two consultants (both male) and two dieticians (both female) provided written informed consent and participated in the study.
CF clinics in the UK.
While healthcare professionals highlighted the importance of physical activity in the management of CF, they noted that very few patients were motivated solely by (CF or general) health reasons. Healthcare professionals discussed the need for physical activity to be an enjoyable and routine part of their life, undertaken with significant others, outside the clinic whenever possible. Adopted approaches for physical activity promotion focused on providing individualised recommendations that suit the patients’ individual needs and goals and enhance intrinsic motivation for physical activity.
Our research offers valuable information for those seeking to develop interventions to promote physical activity among adolescents with CF. Specifically, intervention developers should focus on developing individualised interventions that focus on enhancing intrinsic motivation and support the integration of physical activity into everyday life.
Small renal masses (SRMs; ≤4 cm) account for two-thirds of new diagnoses of kidney cancer, the majority of which are incidental findings. The natural history of the SRM seems largely indolent. There is an increasing concern regarding surgical overtreatment and the associated health burden in terms of morbidity and economy. Observational data support the safety and efficacy of percutaneous cryoablation but there is an unmet need for high-quality evidence on non-surgical management options and a head-to-head comparison with standard of care is lacking. Historical interventional trial recruitment difficulties demand novel study conduct approaches. We aim to assess if a novel trial design, the cohort embedded randomised controlled trial (RCT), will enable carrying out such a comparison.
Single-centre prospective cohort study of adults diagnosed with SRM (n=200) with an open label embedded interventional RCT comparing nephron sparing interventions. Cohort participants will be managed at patient and clinicians’ discretion and agree with longitudinal clinical data and biological sample collection, with invitation for trial interventions and participation in comparator control groups. Cohort participants with biopsy-proven renal cell carcinoma eligible for both percutaneous cryoablation and partial nephrectomy will be randomly selected (1:1) and invited to consider percutaneous cryoablation (n=25). The comparator group will be robotic partial nephrectomy (n=25). The primary outcome of this feasibility study is participant recruitment. Qualitative research techniques will assess barriers and recruitment improvement opportunities. Secondary outcomes are participant trial retention, health-related quality of life, treatment complications, blood transfusion rate, intensive care unit admission and renal replacement requirement rates, length of hospital stay, time to return to pre-treatment activities, number of work days lost, and health technologies costs.
Ethical approval has been granted (UK HRA REC 19/EM/0004). Study outputs will be presented and published.
Robot-assisted laparoscopic prostatectomy (RALP) offers potential cure for localised prostate cancer but is associated with considerable toxicity. Potency and urinary continence are improved when the neurovascular bundles (NVBs) are spared during a nerve spare (NS) RALP. There is reluctance, however, to perform NS RALP when there are concerns that the cancer extends beyond the capsule of the prostate into the NVB, as NS RALP in this instance increases the risk of a positive surgical margin (PSM). The NeuroSAFE technique involves intraoperative fresh-frozen section analysis of the posterolateral aspect of the prostate margin to assess whether cancer extends beyond the capsule. There is evidence from large observational studies that functional outcomes can be improved and PSM rates reduced when the NeuroSAFE technique is used during RALP. To date, however, there has been no randomised controlled trial (RCT) to substantiate this finding. The NeuroSAFE PROOF feasibility study is designed to assess whether it is feasible to randomise men to NeuroSAFE RALP versus a control arm of ‘standard of practice’ RALP.
NeuroSAFE PROOF feasibility study will be a multicentre, single-blinded RCT with patients randomised 1:1 to either NeuroSAFE RALP (intervention) or standard RALP (control). Treatment allocation will occur after trial entry and consent. The primary outcome will be assessed as the successful accrual of 50 men at three sites over 15 months. Secondary outcomes will be used to aid subsequent power calculations for the definitive full-scale RCT and will include rates of NS; PSM; biochemical recurrence; adjuvant treatments; and patient-reported functional outcomes on potency, continence and quality of life.
NeuroSAFE PROOF has ethical approval (Regional Ethics Committee reference 17/LO/1978). NeuroSAFE PROOF is supported by National Institute for Healthcare Research Research for Patient Benefit funding (NIHR reference PB-PG-1216-20013). Findings will be made available through peer-reviewed publications.