The Journeying through Dementia psychosocial intervention versus usual care study: a single-blind, parallel group, phase 3 trial.

Background: There is an urgent clinical need for evidence-based psychosocial interventions for people with mild dementia. We aimed to determine the clinical benefits and cost-effectiveness of Journeying through Dementia (JtD), an intervention designed to promote wellbeing and independence in people with mild dementia.

Methods: We did a single-blind, parallel group, individually randomised, phase 3 trial at 13 National Health Service sites across England.

An intervention to promote self-management, independence and self-efficacy in people with early-stage dementia: the Journeying through Dementia RCT.

Background: There are few effective interventions for dementia.

Aim: To determine the clinical effectiveness and cost-effectiveness of an intervention to promote self-management, independence and self-efficacy in people with early-stage dementia.

Objectives: To undertake a randomised controlled trial of the Journeying through Dementia intervention compared with usual care, conduct an internal pilot testing feasibility, assess intervention delivery fidelity and undertake a qualitative exploration of participants' experiences.

Learning from COVID-19 related trial adaptations to inform efficient trial design-a sequential mixed methods study.

Background: Many clinical trial procedures were often undertaken in-person prior to the COVID-19 pandemic, which has resulted in adaptations to these procedures to enable trials to continue. The aim of this study was to understand whether the adaptations made to clinical trials by UK Clinical Trials Units (CTUs) during the pandemic have the potential to improve the efficiency of trials post-pandemic.

Methods: This was a mixed methods study, initially involving an online survey administered to all registered UK CTUs to identify studies that had made adaptations due to the pandemic.

Adjusting for bias in the mean for primary and secondary outcomes when trials are in sequence.

When designing a clinical trial, one key aspect of the design is the sample size calculation. The sample size calculation tends to rely on a target or expected difference. The expected difference can be based on the observed data from previous studies, which results in bias.

Estimating the minimum important difference in the DEMQOL instrument in people with dementia.

Purpose: The Dementia-Related Quality of Life (DEMQOL) measure and the DEMQOL-Utility Score (DEMQOL-U) are validated tools for measuring quality of life (QOL) in people with dementia. What score changes translate to a clinically significant impact on patients' lives was unknown. This study establishes the minimal important differences (MID) for these two instruments.

Journeying through Dementia Randomised Controlled Trial of a Psychosocial Intervention for People Living with Early Dementia: Embedded Qualitative Study with Participants, Carers and Interventionists.

Objective: To identify the barriers and facilitators to the implementation of a complex psychosocial intervention though a study exploring the experiences of participants, carers and interventionists during a trial.

Methods: Individual semi-structured interviews were conducted with participants, their carers, and interventionists from a sample of recruiting sites that took part in the Journeying through Dementia randomized controlled trial (RCT). Interview data were transcribed and analysed using framework analysis.

Assessing fidelity of a community based psychosocial intervention for people with mild dementia within a large randomised controlled trial.

Background: Understanding intervention delivery as intended, particularly in complex interventions, should be underpinned by good quality fidelity assessment. We present the findings from a fidelity assessment embedded as part of a trial of a complex community-based psychosocial intervention, Journeying through Dementia (JtD). The intervention was designed to equip individuals with the knowledge and skills to successfully self-manage, maintain independence, and live well with dementia and involves both group and individual sessions.

What Is the Health and Well-Being Burden for Parents Living With a Child With ADHD in the United Kingdom?

To explore the burden associated with childhood ADHD in a large observational study. We recruited familes with at least one child (6-18 years) with ADHD via 15 NHS trusts in the UK, and collected data from all family members. We made careful adjustments to ensure a like-for-like comparison with two different control groups, and explored the impact of controlling for a positive parental/carer ADHD screen, employment, and relationship status.

MRI in the diagnosis of fetal developmental brain abnormalities: the MERIDIAN diagnostic accuracy study.

Background: Ultrasonography has been the mainstay of antenatal screening programmes in the UK for many years. Technical factors and physical limitations may result in suboptimal images that can lead to incorrect diagnoses and inaccurate counselling and prognostic information being given to parents. Previous studies suggest that the addition of in utero magnetic resonance imaging (iuMRI) may improve diagnostic accuracy for fetal brain abnormalities.

Behavioural activation therapy for post-stroke depression: the BEADS feasibility RCT.

Background: There is currently insufficient evidence for the clinical effectiveness and cost-effectiveness of psychological therapies for post-stroke depression.

Objective: To evaluate the feasibility of undertaking a definitive trial to evaluate the clinical effectiveness and cost-effectiveness of behavioural activation (BA) compared with usual stroke care for treating post-stroke depression.

Design: Parallel-group, feasibility, multicentre, randomised controlled trial with nested qualitative research and a health economic evaluation.

A study of target effect sizes in randomised controlled trials published in the Health Technology Assessment journal.

Background: When designing a randomised controlled trial (RCT), an important consideration is the sample size required. This is calculated from several components; one of which is the target difference. This study aims to review the currently reported methods of elicitation of the target difference as well as to quantify the target differences used in Health Technology Assessment (HTA)-funded trials.

The TiM system: developing a novel telehealth service to improve access to specialist care in motor neurone disease using user-centered design.

Objectives: Attendance at a specialist multidisciplinary motor neurone disease (MND) clinic is associated with improved survival and may also improve quality of life and reduce hospital admissions. However, patients struggle to travel to clinic and may experience difficulties between clinic visits that may not be addressed in a timely manner. We wanted to explore how we could improve access to specialist MND care.

Are pilot trials useful for predicting randomisation and attrition rates in definitive studies: A review of publicly funded trials.

Background/aims: External pilot trials are recommended for testing the feasibility of main or confirmatory trials. However, there is little evidence that progress in external pilot trials actually predicts randomisation and attrition rates in the main trial. To assess the use of external pilot trials in trial design, we compared randomisation and attrition rates in publicly funded randomised controlled trials with rates in their pilots.

PLEASANT: Preventing and Lessening Exacerbations of Asthma in School-age children Associated with a New Term - a cluster randomised controlled trial and economic evaluation.

Background: Asthma episodes and deaths are known to be seasonal. A number of reports have shown peaks in asthma episodes in school-aged children associated with the return to school following the summer vacation. A fall in prescription collection in the month of August has been observed, and was associated with an increase in the number of unscheduled contacts after the return to school in September.

Use of MRI in the diagnosis of fetal brain abnormalities in utero (MERIDIAN): a multicentre, prospective cohort study.

Background: In-utero MRI (iuMRI) has shown promise as an adjunct to ultrasound but the comparative diagnostic performance has been poorly defined. We aimed to assess whether the diagnostic accuracy and confidence of the prenatal diagnosis of fetal brain abnormalities is improved with iuMRI and assess the clinical impact and patient acceptability of iuMRI.

Methods: We did a multicentre, prospective, cohort study in the UK, at 16 fetal medicine centres, of pregnant women aged 16 years or older whose fetus had a brain abnormality detected by ultrasound at a gestational age of 18 weeks or more, had no contraindications to iuMRI, and consented to enter the study.

The impact of ADHD on the health and well-being of ADHD children and their siblings.

Childhood attention-deficit/hyperactivity disorder (ADHD) has been associated with reduced health and well-being of patients and their families. The authors undertook a large UK survey-based observational study of the burden associated with childhood ADHD. The impact of ADHD on both the patient (N = 476) and their siblings (N = 337) on health-related quality of life (HRQoL) and happiness was quantified using multiple standard measures [e.

DiPALS: Diaphragm Pacing in patients with Amyotrophic Lateral Sclerosis - a randomised controlled trial.

Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease resulting in death, usually from respiratory failure, within 2-3 years of symptom onset. Non-invasive ventilation (NIV) is a treatment that when given to patients in respiratory failure leads to improved survival and quality of life. Diaphragm pacing (DP), using the NeuRx/4(®) diaphragm pacing system (DPS)™ (Synapse Biomedical, Oberlin, OH, USA), is a new technique that may offer additional or alternative benefits to patients with ALS who are in respiratory failure.

Using technology to improve access to specialist care in amyotrophic lateral sclerosis: A systematic review.

Our objective was to review the evidence for using technology to improve access to specialist care for patients with amyotrophic lateral sclerosis (ALS) and their carers. Medline, Google Scholar and the Cochrane library were searched for articles describing technology that enabled clinical care of patients with ALS or their carers where the patient/carer and clinician were not in the same location. Two applications were identified: telemedicine to facilitate video conferencing as an alternative to outpatient consultations and telehealth monitoring for patients with respiratory failure.

An Investigation of the Shortcomings of the CONSORT 2010 Statement for the Reporting of Group Sequential Randomised Controlled Trials: A Methodological Systematic Review.

Background: It can be argued that adaptive designs are underused in clinical research. We have explored concerns related to inadequate reporting of such trials, which may influence their uptake. Through a careful examination of the literature, we evaluated the standards of reporting of group sequential (GS) randomised controlled trials, one form of a confirmatory adaptive design.

Saline in acute bronchiolitis RCT and economic evaluation: hypertonic saline in acute bronchiolitis - randomised controlled trial and systematic review.

Background: Acute bronchiolitis is the most common cause of hospitalisation in infancy. Supportive care and oxygen are the cornerstones of management. A Cochrane review concluded that the use of nebulised 3% hypertonic saline (HS) may significantly reduce the duration of hospitalisation.

Estimating the sample size for a pilot randomised trial to minimise the overall trial sample size for the external pilot and main trial for a continuous outcome variable.

Sample size justification is an important consideration when planning a clinical trial, not only for the main trial but also for any preliminary pilot trial. When the outcome is a continuous variable, the sample size calculation requires an accurate estimate of the standard deviation of the outcome measure. A pilot trial can be used to get an estimate of the standard deviation, which could then be used to anticipate what may be observed in the main trial.

A rapid review indicated higher recruitment rates in treatment trials than in prevention trials.

Objectives: To test the hypothesis that the percentage of patients screened that randomize differs between prevention and therapy trials.

Study Design And Setting: Rapid review of randomized controlled trials (RCTs) identified through published systematic reviews in August 2013. Individually randomized, parallel group controlled RCTs were eligible if they evaluated metformin monotherapy or exercise for the prevention or treatment of type 2 diabetes.

SABRE: a multicentre randomised control trial of nebulised hypertonic saline in infants hospitalised with acute bronchiolitis.

Aim: Acute bronchiolitis is the commonest cause for hospitalisation in infancy. Supportive care remains the cornerstone of current management and no other therapy has been shown to influence the course of the disease. It has been suggested that adding nebulised hypertonic saline to usual care may shorten the duration of hospitalisation.

Examining the impact of 11 long-standing health conditions on health-related quality of life using the EQ-5D in a general population sample.

Objectives: Health-related quality of life (HRQoL) measures have been increasingly used in economic evaluations for policy guidance. We investigate the impact of 11 self-reported long-standing health conditions on HRQoL using the EQ-5D in a UK sample.

Methods: We used data from 13,955 patients in the South Yorkshire Cohort study collected between 2010 and 2012 containing the EQ-5D, a preference-based measure.

Exploring the impact of patient and public involvement in a cancer research setting.

An enduring theme in the literature exploring patient and public involvement (PPI) in research has been the focus on evaluating impact, defined usually in terms of participants' practical contribution to enhancing research processes. By contrast, there has been less emphasis on the perspectives and experiences of those involved in PPI. Drawing on qualitative data with people involved in the National Cancer Research Network in the United Kingdom, we report on what motivated participants to get involved and their experiences of involvement in this setting.