Wendy Baird

Background: Dose Adjustment For Normal Eating is a 5-day, group-based clinical education programme for adults with type 1 diabetes, training them in the complex self-management skills, enabling them to optimise both their glycaemic management and quality of life. Although Dose Adjustment For Normal Eating is associated with improvements in many aspects of diabetes management, including psychological outcomes, it has been challenging to maintain the biomedical improvements for longer term, which have remained well above the glucose levels recommended by National Institute for Health and Care Excellence. Objectives: We aimed to develop and evaluate Dose Adjustment For Normal Eating plus , an enhanced version of Dose Adjustment For Normal Eating, to enable improvements in glycaemic management (glycated haemoglobin) without compromising the quality of life. The objectives of the programme were to modify the existing Dose Adjustment For Normal Eating curriculum and evaluate it in a cluster randomised controlled trial. Design: This programme comprised four workstreams. The first three workstreams focused on redevelopment of the Dose Adjustment For Normal Eating intervention to become ‘Dose Adjustment For Normal Eating plus ’: involving revision of the curriculum for a 5-day, group-based programme, including relevant behaviour change techniques (workstream 1); development of a new structured, pro-active, follow-up programme to be delivered by Dose Adjustment For Normal Eating plus facilitators post course to support ongoing diabetes self-management (workstream 2); and development of a new technology interface to help glucose pattern recognition to inform improved self-management (workstream 3). We used mixed methods to review, develop and refine the Dose Adjustment For Normal Eating plus intervention (course, individual support and technological aspects). The intervention was piloted in three National Health Service specialist diabetes centres and was reviewed via two iterative waves before finalisation. Workstream 4 was a cluster randomised controlled trial using a pragmatic, parallel group (1 : 1) allocation design (Dose Adjustment For Normal Eating vs. Dose Adjustment For Normal Eating plus ), with concurrent process and health economic evaluations. The primary biomedical outcome was the between-group difference in glycated haemoglobin at 12 months. The primary psychological outcome was the between-group difference in the impact of type 1 diabetes on quality of life (using Audit of Diabetes-Dependent Quality of Life-15) at 12 months. Setting: The workstreams and the randomised controlled trial were undertaken in university and National Health Service specialist centres in England and Scotland. Participants: Four hundred and seventy-one adults aged ≥ 18 years with confirmed diagnosis of type 1 diabetes participated in the cluster randomised controlled trial, which was 144 fewer than planned and approximately 77% of the planned sample size. Intervention: Dose Adjustment For Normal Eating plus is a standardised 5-day educational course (described above), delivered 1 day per week over 5 weeks in groups, meeting face to face, followed by five individual support sessions over 12 months. An online platform supports the upload, visualisation, pattern recognition and review of glycaemic data for participants and clinicians. Trial outcome measures: In the cluster randomised controlled trial, the primary biomedical outcome was glycaemia, defined as glycated haemoglobin, at 12 months. The primary psychological outcome was the impact of diabetes on quality of life (assessed using the widely recognised scale, Audit of Diabetes-Dependent Quality of Life-15). Key secondary biomedical outcomes included rates and proportions of severe hypoglycaemia and diabetic ketoacidosis. Key secondary psychological outcomes included diabetes-specific distress, diabetes-specific positive well-being and fear of hypoglycaemia. Results: In the primary intention-to-treat population (309), mean glycated haemoglobin reduced from 9.1% to 8.2% (75.9–66.5 mmol/mol) in the Dose Adjustment For Normal Eating group and from 9.1% to 8.1% (75.6–65.1 mmol/mol) in the Dose Adjustment For Normal Eating plus group. The cluster randomised controlled trial showed no evidence of a between-group difference at 12 months in glycated haemoglobin {point estimate [95% confidence interval −0.014 (−0.284 to 0.255); p = 0.915]}, or diabetes-specific quality of life {point estimate [95% confidence interval 0.2 (−0.1 to 0.5); p = 0.21]}. At 12 months, significant between-group differences were observed, favouring Dose Adjustment For Normal Eating plus , in secondary psychological outcomes (diabetes-specific distress, diabetes-specific positive well-being and actions taken to avoid hypoglycaemia). Limitations: Due to trial-suspension during coronavirus disease, we recruited 471 adults to the cluster randomised controlled trial, 144 fewer than planned. Although this reduced overall statistical power, we showed that the difference in glycated haemoglobin between the groups was far below that we considered a meaningful clinical difference (0.5%). Recruitment also meant that more participants were using continuous glucose monitoring, both before and during the trial than we anticipated. This may have reduced additional benefit of the Dose Adjustment For Normal Eating plus intervention on glycated haemoglobin. The pandemic probably influenced diabetes self-management during the trial in both groups. Conclusions: Dose Adjustment For Normal Eating plus did not lead to greater falls in glycated haemoglobin at 12 months compared to Dose Adjustment For Normal Eating, but these falls were double those reported in previous Dose Adjustment For Normal Eating randomised controlled trials. Sixty-eight per cent of participants showed clinically relevant improved glucose levels. Those in Dose Adjustment For Normal Eating plus reported significant falls in diabetes distress, positive well-being and qualitative research reported positive experiences of Dose Adjustment For Normal Eating plus among participants and health professionals. Future work: In view of major improvements in diabetes distress and positive experiences in both participants and health professionals, the Dose Adjustment For Normal Eating executives, who co-ordinate the delivery of Dose Adjustment For Normal Eating courses, are planning to include key elements of Dose Adjustment For Normal Eating plus in future courses, particularly structured support to participants following the course and key elements of the curriculum. Study registration: This study is registered as IRAS: 235621 and ISRCTN: 42908016. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Programme Grants for Applied Research Programme (NIHR award ref: RP-PG-0514-20013) and is published in full in Programme Grants for Applied Research ; Vol. 14, No. 21. See the NIHR Funding and Awards website for further award information.

Objectives To evaluate the processes involved in using a novel digitally enabled healthcare system (telehealth in motor neuron disease (TiM)) in people living with motor neuron disease (MND) and their informal carers. We examined TiM implementation, potential mechanisms of impact and contextual factors that might influence TiM implementation or impact. Design An 18-month, single-centre process evaluation within a randomised, pilot and feasibility study. Intervention TiM plus usual care versus usual care alone. Setting A specialist UK MND care centre. Participants 40 patients with MND and 37 primary informal carers. Primary and secondary outcome measures Patient, carer and staff outcomes and experiences using semistructured interviews. Descriptive data on implementation and use of TiM. Results The TiM was acceptable and accessible to patients, carers and staff. Intervention uptake and adherence were good: 14 (70%) patients completed a TiM session at least fortnightly. Barriers to TiM use (such as technology experience and disability) were overcome with well-designed technology and face-to-face training. Reported potential benefits of TiM included improved communication and care coordination, reassurance, identification of complications and the potential for TiM to be an alternative or addition to clinic. Benefits depended on patients’ current level of needs or disability. The main challenges were the large number of alerts that were generated by TiM, how the clinicians responded to these alerts and the mismatch between patient/carer expectations and nurses actions. This could be improved by better communication systems and adjusting the alerts algorithm. Conclusion TiM has the potential to facilitate access to specialist care, but further iterative developments to the intervention and process evaluations of the TiM in different services are required. Trial identifier number ISRCTN26675465.

Objectives Care of patients with motor neuron disease (MND) in a specialist, multidisciplinary clinic is associated with improved survival, but access is not universal. We wanted to pilot and establish the feasibility of a definitive trial of a novel telehealth system (Telehealth in Motor neuron disease, TiM) in patients with MND. Design An 18-month, single-centre, mixed-methods, randomised, controlled pilot and feasibility study. Intervention TiM telehealth plus usual care versus usual care. Setting A specialist MND care centre in the UK. Participants Patients with MND and their primary informal carers. Primary and secondary outcome measures Recruitment, retention and data collection rates, clinical outcomes including participant quality of life and anxiety and depression. Results Recruitment achieved the target of 40 patients and 37 carers. Participant characteristics reflected those attending the specialist clinic and included those with severe disability and those with limited experience of technology. Retention and data collection was good. Eighty per cent of patients and 82% of carer participants reported outcome measures were completed at 6 months. Using a longitudinal analysis with repeated measures of quality of life (QoL), a sample size of 131 per arm is recommended in a definitive trial. The methods and intervention were acceptable to participants who were highly motivated to participate to research. The low burden of participation and accessibility of the intervention meant barriers to participation were minimal. However, the study highlighted difficulties assessing the associated costs of the intervention, the challenge of recruitment in such a rare disease and the difficulties of producing rigorous evidence of impact in such a complex intervention. Conclusion A definitive trial of TiM is feasible but challenging. The complexity of the intervention and heterogeneity of the patient population means that a randomised controlled trial may not be the best way to evaluate the further development and implementation of the TiM. Trial registration number ISRCTN26675465.

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. Methods: We adopted an iterative, user-centered co-design approach, collaborating with those with experience of providing and receiving MND care including patients, carers, clinicians, and technology developers. We explored the unmet needs of those living with MND, how they might be met through service redesign and through the use of digital technologies. We developed a new digital solution and performed initial testing with potential users including clinicians, patients, and carers. Results: We used these findings to develop a telehealth system (TiM) using an Android app into which patients and carers answer a series of questions about their condition on a weekly basis. The questions aim to capture all the physical, emotional, and social difficulties associated with MND. This information is immediately uploaded to the internet for review by the MND team. The data undergoes analysis in order to alert clinicians to any changes in a patient or carer’s condition. Conclusions: We describe the benefits of developing a novel digitally enabled service underpinned by participatory design. Future trials must evaluate the feasibility and acceptability of the TiM system within a clinical environment.

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.Objective: The Diaphragm Pacing in patients with Amyotrophic Lateral Sclerosis (DiPALS) trial evaluated the effect of DP on survival over the study duration in patients with ALS with respiratory failure.Design: The DiPALS trial was a multicentre, parallel-group, open-label, randomised controlled trial incorporating health economic analyses and a qualitative longitudinal substudy.Participants: Eligible participants had a diagnosis of ALS (ALS laboratory-supported probable, clinically probable or clinically definite according to the World Federation of Neurology revised El Escorial criteria), had been stabilised on riluzole for 30 days, were aged ≥ 18 years and were in respiratory failure. We planned to recruit 108 patients from seven UK-based specialist ALS or respiratory centres. Allocation was performed using 1 : 1 non-deterministic minimisation.Interventions: Participants were randomised to either standard care (NIV alone) or standard care (NIV) plus DP using the NeuRX/4 DPS.Main outcome measures: The primary outcome was overall survival, defined as the time from randomisation to death from any cause. Secondary outcomes were patient quality of life [assessed by European Quality of Life-5 Dimensions, three levels (EQ-5D-3L), Short Form questionnaire-36 items and Sleep Apnoea Quality of Life Index questionnaire]; carer quality of life (EQ-5D-3L and Caregiver Burden Inventory); cost–utility analysis and health-care resource use; tolerability and adverse events. Acceptability and attitudes to DP were assessed in a qualitative substudy.Results: In total, 74 participants were randomised into the trial and analysed, 37 participants to NIV plus pacing and 37 to standard care, before the Data Monitoring and Ethics Committee advised initial suspension of recruitment (December 2013) and subsequent discontinuation of pacing (on safety grounds) in all patients (June 2014). Follow-up assessments continued until the planned end of the study in December 2014. The median survival (interquartile range) was 22.5 months (lower quartile 11.8 months; upper quartile not reached) in the NIV arm and 11.0 months (6.7 to 17.0 months) in the NIV plus pacing arm, with an adjusted hazard ratio of 2.27 (95% confidence interval 1.22 to 4.25;p = 0.01).Conclusions: Diaphragmatic pacing should not be used as a routine treatment for patients with ALS in respiratory failure.Future work: It may be that certain population subgroups benefit from DP. We are unable to explain the mechanism behind the excess mortality in the pacing arm, something the small trial size cannot help address. Future research should investigate the mechanism by which harm or benefit occurs further.Trial registration: Current Controlled Trials ISRCTN53817913.Funding: This project was funded by the National Institute for Health Research Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 20, No. 45. See the HTA programme website for further project information. Additional funding was provided by the Motor Neurone Disease Association of England, Wales and Northern Ireland.

The National Institute for Health Research (NIHR) Research Design Service (RDS) for Yorkshire and Humber has been running a public involvement funding scheme since 2008. This scheme awards researchers a small amount of money to help them get involvement from patients and/or the public. Involvement activities take place at the time when researchers are planning studies, and when they are completing application forms to request funding for a proposed research project. After the public involvement activities researchers are asked to write a report for the RDS describing what they did with the public involvement funding. This study analysed those reports using an approach which included members of a public involvement panel in the data analysis process. The aim of the work was to see what the views and experiences of researchers who received funding were, and what might be learned for the future of the scheme. Twenty five reports were analysed. Four main themes were identified, these described: the added value of public involvement; aspects to consider when planning and designing public involvement; different roles of public contributors; and aspects of valuing public member contributions. The group approach to analysis was successful in enabling involvement of a variety of individuals in the process. The findings of the study provide evidence of the value of public involvement during the development of applications for research funding. The results also indicate that researchers recognise the variety in potential roles for the public in research, and acknowledge how involvement adds value to studies. Background A regional Research Design Service, funded by the National Institute for Health Research, introduced a small grant in 2008, to support public involvement (often known as patient and public involvement [PPI]) activities during the development of applications for research funding. Successful applicants are requested to submit a report detailing how the grant money was used, including a description of the aims and outcomes of the public involvement activities. The purpose of this study was to analyse the content of these reports. We aimed to find out what researcher views and experiences of public involvement activities were, and what lessons might be learned. Methods We used an innovative method of data analysis, drawing on group participatory approaches, qualitative content analysis, and Framework Analysis to sort and label the content of the reports. We developed a framework of categories and sub-categories (or themes and sub-themes) from this process. Results Twenty five documents were analysed. Four main themes were identified in the data: the added value of public involvement; planning and designing involvement; the role of public members; and valuing public member contributions. Within these themes, sub-themes related to the timing of involvement (prior to the research study/intended during the research study), and also specific benefits of public involvement such as: validating ideas; ensuring appropriate outcomes; ensuring the acceptability of data collection methods/tools and advice regarding research processes. Other sub-themes related to: finding and approaching public members; timing of events; training/support; the format of sessions; setting up public involvement panels: use of public contributors in analysis and interpretation of data; and using public members to assist with dissemination and translation into practice. Conclusions The analysis of reports submitted by researchers following involvement events provides evidence of the value of public involvement during the development of applications for research funding, and details a method for involving members of the public in data analysis which could be of value to other researchers The findings of the analysis indicate recognition amongst researchers of the variety in potential roles for public members in research, and also an acknowledgement of how involvement adds value to studies.

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. One randomized controlled trial using telehealth in patients with respiratory failure including 22 patients with ALS was identified. While rates of hospitalization were reduced, overall mortality was unchanged and there were too few patients with ALS in the study to detect significant benefit. In conclusion, there is limited evidence to support the use of telemedicine or telehealth in the care of patients with ALS. Future research needs to develop an understanding of the key beneficial aspects of the traditional specialist ALS service and how these factors could be delivered using technology. Successful evaluation and implementation of technologies to facilitate access to specialist care will only be possible if all the relevant impacts of an intervention are understood and measured.

Background: Many adults with type 1 diabetes cannot self-manage their diabetes effectively and die prematurely with diabetic complications as a result of poor glucose control. Following the positive results obtained from a randomised controlled trial (RCT) by the Dose Adjustment For Normal Eating (DAFNE) group, published in 2002, structured training is recommended for all adults with type 1 diabetes in the UK.Aim: With evidence that blood glucose control is not always improved or sustained, we sought to determine factors explaining why some patients benefit from training more than other patients, identifying barriers to successful self-management, while developing other models to make skills training more accessible and effective.Findings: We confirmed that glycaemic outcomes are not always improved or sustained when the DAFNE programme is delivered routinely, although improvements in psychosocial outcomes are maintained. DAFNE courses and follow-up support is needed to help participants instil and habituate key self-management practices such as regular diary/record keeping. DAFNE graduates need structured professional support following training. This is currently either unavailable or provided ad hoc without a supporting evidence base. Demographic and psychosocial characteristics had minimal explanatory power in predicting glycaemic control but good explanatory power in predicting diabetes-specific quality of life over the following year. We developed a DAFNE course delivered for 1 day per week over 5 weeks. There were no major differences in outcomes between this and a standard 1-week DAFNE course; in both arms of a RCT, glycaemic control improved by less than in the original DAFNE trial. We piloted a course delivering both the DAFNE programme and pump training. The pilot demonstrated the feasibility of a full multicentre RCT and resulted in us obtaining subsequent Health Technology Assessment programme funding. In collaboration with the National Institute for Health Research (NIHR) Diabetes Research Programme at King’s College Hospital (RG-PG-0606-1142), London, an intervention for patients with hypoglycaemic problems, DAFNE HART (Dose Adjustment for Normal Eating Hypoglycaemia Awareness Restoration Training), improved impaired hypoglycaemia awareness and is worthy of a formal trial. The health economic work developed a new type 1 diabetes model and confirmed that the DAFNE programme is cost-effective compared with no structured education; indeed, it is cost-saving in the majority of our analyses despite limited glycated haemoglobin benefit. Users made important contributions but this could have been maximised by involving them with grant writing, delaying training until the group was established and funding users’ time off work to maximise attendance. Collecting routine clinical data to conduct continuing evaluated roll-out is possible but to do this effectively requires additional administrator support and/or routine electronic data capture.Conclusions: We propose that, in future work, we should modify the current DAFNE curricula to incorporate emerging understanding of behaviour change principles to instil and habituate key self-management behaviours that include key DAFNE competencies. An assessment of numeracy, critical for insulin dose adjustment, may help to determine whether or not additional input/support is required both before and after training. Models of structured support involving professionals should be developed and evaluated, incorporating technological interventions to help overcome the barriers identified above and enable participants to build effective self-management behaviours into their everyday lives.Trial registration: ClinicalTrials.gov NCT01069393.Funding: The NIHR Programme Grants for Applied Research programme.

Objectives This study explores the experience of gastrostomy insertion from the perspective of the patients and their informal carers. Gastrostomy feeding is commonly used to support motor neurone disease (MND) patients with dysphagia. However, there is lack of information describing patient and carer experiences following gastrostomy insertion. The effect of gastrostomy on quality of life for these patients and their family is currently not well understood. Methods Retrospective qualitative exploration using semistructured interviews with patients and their informal carers to elicit in-depth descriptions of their experiences and views following gastrostomy. Results 27 patients consented to the study; of these, 23 underwent a successful gastrostomy. 10 patients and 8 carers were interviewed, approximately 3 months following a successful gastrostomy. Participants described clinical complications, practical issues, time restrictions imposed by strict feeding regimens and psychological issues, which adversely impacted on quality of life. However, the establishment of a safe alternative route for feeding and medication, and the reduced worry over difficult meals and weight loss, were described by all as outweighing these negative impacts. Participants also described having received education/training on gastrostomy feeding both in hospital and in the community, which helped them to cope during the transition from oral to gastrostomy feeding. Conclusions This study highlights the challenges and benefits of gastrostomy as well as the importance of education and information provision. Emphasis should be given to education before and after insertion along with support and care in the community. While the significant impact of gastrostomy on patients and carers should not be underestimated, the potential benefits were described as outweighing these concerns.