PANDA: A case-study examining a successful Audiology and Otology Patient and Public Involvement and Engagement research group

There has been increasing involvement of patients and members of the public in research; however, case studies describing patient research groups with hearing loss are non-existent. Such case studies will be valuable, enabling evidence-based dialogue and promoting best practice in the engagement of patients, the public and researchers. This paper aims to discuss this practice. The absence of such dialogue may hinder initial efforts by researchers to realise the potential of Patient and Public Involvement. The objective of this study was to set up and run a patient and public involvement and engagement group in audiology research, use the lessons learnt to provide a guide to others in a similar situation, and prompt the dialogue referred to above. A successful group with over 70 members has been set up, with an average attendance for meetings of between 15 and 20 participants. Feedback from the group indicates that members are happy with, and benefit from, their involvement and particularly appreciate the concern of those managing the group better to accommodate sensory impairments. Additionally, the group has improved research output for specific grant applications. We conclude that although this case study contains elements unique to the setting (a large NHS Trust in the Midlands), it also provides transferable observations and resources that can be adapted and utilised by researchers working with patients and the public with hearing loss. Experience Framework This article is associated with the Patient, Family & Community Engagement lens of The Beryl Institute Experience Framework. (http://bit.ly/ExperienceFramework) Access other PXJ articles related to this lens. Access other resources related to this lens

The Physiotherapy eSkills Training Online resource improves performance of practical skills: A controlled trial

Background: E-learning is a common and popular mode of educational delivery, but little is known about its effectiveness in teaching practical skills. The aim of this study was to determine whether the Physiotherapy eSkills Training Online resource in addition to usual teaching improved the performance of practical skills in physiotherapy students. Method: This study was a non-randomised controlled trial. The participants were graduate entry physiotherapy students enrolled in consecutive semesters of a neurological physiotherapy unit of study. The experimental group received the Physiotherapy eSkills Training Online resource as well as usual teaching. The Physiotherapy eSkills Training Online resource is an online resource incorporating (i) video-clips of patient-therapist simulations; (ii) supportive text describing the aim, rationale, equipment, key points, common errors and methods of progression; and (iii) a downloadable PDF document incorporating the online text information and a still image of the video-clip for each practical skill. The control group received usual teaching only. The primary outcomes were the overall performance of practical skills as well as their individual components, measured using a practical examination. Results: The implementation of the Physiotherapy eSkills Training Online resource resulted in an increase of 1.6 out of 25 (95% CI -0.1 to 3.3) in the experimental group compared with the control group. In addition, the experimental group scored 0.5 points out of 4 (95% CI 0 to 1.1) higher than the control group for 'effectiveness of the practical skill' and 0.6 points out of 4 (95% CI 0.1 to 1.1) higher for 'rationale for the practical skill'. Conclusion: There was improvement in performance of practical skills in students who had access to the Physiotherapy eSkills Training Online resource in addition to usual teaching. Students considered the resource to be very useful for learning.7 page(s

Precision medicine in laryngeal cancer: protocol of the laryngeal cancer cohort (LARCH).

IntroductionLaryngeal cancer disproportionately affects socioeconomically disadvantaged patients. Treatment can render a patient nil by mouth or in need of a permanent tracheostomy. In the past 30 years, survival has remained at best static and at worst it has declined. Currently, there is no method of prognosticating how a patient will respond to treatment.The LARyngeal Cancer coHort (LARCH) aims to establish how survival and quality-of-life outcomes compare between surgery and (chemo)radiotherapy in early and advanced laryngeal cancer and how the presenting features of laryngeal cancer influence oncological, functional and quality-of-life outcome.Methods and analysisThis study is the first enhanced laryngeal cancer disease cohort. In the initial phase, we aim to deliver a prospective cohort study of 150 patients in 8 centres over a 3-year period.Patient, tumour, quality-of-life and laryngeal functional data will be collected from patients with squamous cell carcinoma of the larynx at baseline, 6, 12 and 24 months. Multiple logistic regression analyses will be used to quantify locoregional control and identify factors associated with control overall and by treatment modality and identify factors associated with quality of life overall and by treatment modality.Ethics and disseminationMost interventions take place as part of routine care, with LARCH providing a mechanism for recording this data centrally. When successfully recruiting in the North of England, we plan to roll out LARCH nationwide; in the future, LARCH can be used as a trial platform in the disease. The results will be submitted for publication in high-impact international peer-reviewed journals and presented to scientific meetings. Access to the anonymised LARCH dataset by other researchers will be publicised and promoted.Trial registration numberISRCTN27819867

Effects of a high-dose 24-h infusion of tranexamic acid on death and thromboembolic events in patients with acute gastrointestinal bleeding (HALT-IT): an international randomised, double-blind, placebo-controlled trial

Background: Tranexamic acid reduces surgical bleeding and reduces death due to bleeding in patients with trauma. Meta-analyses of small trials show that tranexamic acid might decrease deaths from gastrointestinal bleeding. We aimed to assess the effects of tranexamic acid in patients with gastrointestinal bleeding. Methods: We did an international, multicentre, randomised, placebo-controlled trial in 164 hospitals in 15 countries. Patients were enrolled if the responsible clinician was uncertain whether to use tranexamic acid, were aged above the minimum age considered an adult in their country (either aged 16 years and older or aged 18 years and older), and had significant (defined as at risk of bleeding to death) upper or lower gastrointestinal bleeding. Patients were randomly assigned by selection of a numbered treatment pack from a box containing eight packs that were identical apart from the pack number. Patients received either a loading dose of 1 g tranexamic acid, which was added to 100 mL infusion bag of 0·9% sodium chloride and infused by slow intravenous injection over 10 min, followed by a maintenance dose of 3 g tranexamic acid added to 1 L of any isotonic intravenous solution and infused at 125 mg/h for 24 h, or placebo (sodium chloride 0·9%). Patients, caregivers, and those assessing outcomes were masked to allocation. The primary outcome was death due to bleeding within 5 days of randomisation; analysis excluded patients who received neither dose of the allocated treatment and those for whom outcome data on death were unavailable. This trial was registered with Current Controlled Trials, ISRCTN11225767, and ClinicalTrials.gov, NCT01658124. Findings: Between July 4, 2013, and June 21, 2019, we randomly allocated 12 009 patients to receive tranexamic acid (5994, 49·9%) or matching placebo (6015, 50·1%), of whom 11 952 (99·5%) received the first dose of the allocated treatment. Death due to bleeding within 5 days of randomisation occurred in 222 (4%) of 5956 patients in the tranexamic acid group and in 226 (4%) of 5981 patients in the placebo group (risk ratio [RR] 0·99, 95% CI 0·82–1·18). Arterial thromboembolic events (myocardial infarction or stroke) were similar in the tranexamic acid group and placebo group (42 [0·7%] of 5952 vs 46 [0·8%] of 5977; 0·92; 0·60 to 1·39). Venous thromboembolic events (deep vein thrombosis or pulmonary embolism) were higher in tranexamic acid group than in the placebo group (48 [0·8%] of 5952 vs 26 [0·4%] of 5977; RR 1·85; 95% CI 1·15 to 2·98). Interpretation: We found that tranexamic acid did not reduce death from gastrointestinal bleeding. On the basis of our results, tranexamic acid should not be used for the treatment of gastrointestinal bleeding outside the context of a randomised trial

The contribution of X-linked coding variation to severe developmental disorders

Abstract: Over 130 X-linked genes have been robustly associated with developmental disorders, and X-linked causes have been hypothesised to underlie the higher developmental disorder rates in males. Here, we evaluate the burden of X-linked coding variation in 11,044 developmental disorder patients, and find a similar rate of X-linked causes in males and females (6.0% and 6.9%, respectively), indicating that such variants do not account for the 1.4-fold male bias. We develop an improved strategy to detect X-linked developmental disorders and identify 23 significant genes, all of which were previously known, consistent with our inference that the vast majority of the X-linked burden is in known developmental disorder-associated genes. Importantly, we estimate that, in male probands, only 13% of inherited rare missense variants in known developmental disorder-associated genes are likely to be pathogenic. Our results demonstrate that statistical analysis of large datasets can refine our understanding of modes of inheritance for individual X-linked disorders

The IDENTIFY study: the investigation and detection of urological neoplasia in patients referred with suspected urinary tract cancer - a multicentre observational study

Objective To evaluate the contemporary prevalence of urinary tract cancer (bladder cancer, upper tract urothelial cancer [UTUC] and renal cancer) in patients referred to secondary care with haematuria, adjusted for established patient risk markers and geographical variation. Patients and Methods This was an international multicentre prospective observational study. We included patients aged ≥16 years, referred to secondary care with suspected urinary tract cancer. Patients with a known or previous urological malignancy were excluded. We estimated the prevalence of bladder cancer, UTUC, renal cancer and prostate cancer; stratified by age, type of haematuria, sex, and smoking. We used a multivariable mixed-effects logistic regression to adjust cancer prevalence for age, type of haematuria, sex, smoking, hospitals, and countries. Results Of the 11 059 patients assessed for eligibility, 10 896 were included from 110 hospitals across 26 countries. The overall adjusted cancer prevalence (n = 2257) was 28.2% (95% confidence interval [CI] 22.3–34.1), bladder cancer (n = 1951) 24.7% (95% CI 19.1–30.2), UTUC (n = 128) 1.14% (95% CI 0.77–1.52), renal cancer (n = 107) 1.05% (95% CI 0.80–1.29), and prostate cancer (n = 124) 1.75% (95% CI 1.32–2.18). The odds ratios for patient risk markers in the model for all cancers were: age 1.04 (95% CI 1.03–1.05; P < 0.001), visible haematuria 3.47 (95% CI 2.90–4.15; P < 0.001), male sex 1.30 (95% CI 1.14–1.50; P < 0.001), and smoking 2.70 (95% CI 2.30–3.18; P < 0.001). Conclusions A better understanding of cancer prevalence across an international population is required to inform clinical guidelines. We are the first to report urinary tract cancer prevalence across an international population in patients referred to secondary care, adjusted for patient risk markers and geographical variation. Bladder cancer was the most prevalent disease. Visible haematuria was the strongest predictor for urinary tract cancer

Using a prognostic medical device for early identification of pressure ulcers: protocol for study design.

Background:An objective, physiological measurement taken using a medical device may reduce the incidence of pressure ulcers through earlier detection of problems signs before visual signs appear. Research in this field is hampered by variations in clinical practice and patient-level confounders.Aim:The authors outline key considerations for designing a protocol for a study to assess the efficacy and safety of a prognostic medical device in reducing pressure ulcer incidence in a hospital, including comparators, randomisation, sample size, ethics and practical issues.Method:Key issues relating to methodology and ethics are considered alongside a theoretical protocol, which could support future researchers in wound care trials.Results:A prospective, three-armed, multi-centre, stratified cluster-randomised controlled trial is proposed. The third arm is recommended as it is expected that patients will need to be moved for the medical device to be used and repositioning is a preventive strategy. A minimum of 16 200 patients in 33 wards would needed to be recruited to achieve statistical significance. Ethical considerations in terms of consent or assent need to be considered.Conclusion:The hypothetical study designed to evaluate the effectiveness of a diagnostic or prognostic medical device in reducing pressure ulcer incidence in secondary care, while accounting for biases, would require large sample sizes and involves risks of inter-operator and inter-device reliability, heterogeneity of users and the vague clinical interpretation of device results. Robust research in this field has the potential to influence or change policy and practice relating to the prevention of pressure ulcers in secondary care

Using a prognostic medical device for early identification of pressure ulcers: protocol for study design

Item is not available in this repository.Rosalyn Parker - ORCID: 0000-0002-0873-6878 https://orcid.org/0000-0002-0873-6878Background: An objective, physiological measurement taken using a medical device may reduce the incidence of pressure ulcers through earlier detection of problems signs before visual signs appear. Research in this field is hampered by variations in clinical practice and patient-level confounders. Aim: The authors outline key considerations for designing a protocol for a study to assess the efficacy and safety of a prognostic medical device in reducing pressure ulcer incidence in a hospital, including comparators, randomisation, sample size, ethics and practical issues. Method: Key issues relating to methodology and ethics are considered alongside a theoretical protocol, which could support future researchers in wound care trials. Results: A prospective, three-armed, multi-centre, stratified cluster-randomised controlled trial is proposed. The third arm is recommended as it is expected that patients will need to be moved for the medical device to be used and repositioning is a preventive strategy. A minimum of 16200 patients in 33 wards would needed to be recruited to achieve statistical significance. Ethical considerations in terms of consent or assent need to be considered. Conclusion: The hypothetical study designed to evaluate the effectiveness of a diagnostic or prognostic medical device in reducing pressure ulcer incidence in secondary care, while accounting for biases, would require large sample sizes and involves risks of inter-operator and inter-device reliability, heterogeneity of users and the vague clinical interpretation of device results. Robust research in this field has the potential to influence or change policy and practice relating to the prevention of pressure ulcers in secondary care. © 2024 MA Healthcare Ltd.https://doi.org/10.12968/bjon.2024.015833pubpub1