This chapter outlines the aims, objectives and the main methodological approaches used in the study. More detail is provided in subsequent chapters.
Scope of the study
The NIHR commissioning brief requested a focus on ‘young children with neurodisability’. Following Morris et al.,1 we defined neurodisability as any condition that is attributed to impairment of the brain and/or the neuromuscular system and creates functional limitations. We included any non-progressive neurodisability condition that gives rise to physical, non-physical or mixed EDSD. Throughout the research, we gathered information relevant to children with physical and non-physical EDSD separately as we considered that interventions to address these specific types of difficulties may differ. However, as many children with primarily physical EDSD also have non-physical EDSD, we present information relating to children with physical and mixed EDSD together throughout this report.
As there was no prior definition of ‘young children’ specifically relating to EDSD and neurodisability, in our study we defined the age range covered by ‘young children’ during the project. The co-investigators agreed to start the project with a conservative working definition of ‘children as aged up to and including 8 years’. Participants at a consultation group prior to the start of the research agreed that this was an acceptable initial working definition. Broader age ranges were used in some elements of the research to ensure that we captured all of the evidence relating to children aged 0–8 years and enabled all parents and professionals with recent experience to contribute their views. Individual sections of this report state the age range of children with EDSD included in specific elements of the study, and describe why any extensions that were applied were deemed necessary.
The commissioning brief focused on interventions used by parents at home. However, children aged up to 8 years are also fed in education and other care settings using techniques similar to those used at home. Therefore, we included research that focused on children’s eating and drinking at home or at school or nursery. Studies of feeding interventions that were delivered solely on paediatric or neonatal units in a hospital were not included. Enteral feeding, which involves direct feeding by a tube into the stomach, was not included in this study because this intervention is used when oral feeding is not enabling a child to receive sufficient fluid or calories.
Design
To address the study’s aims we used an iterative mixed-methods design. The individual methods comprised systematic reviews (updating three published reviews of interventions and undertaking one review to assess measurement properties of published outcome measures), a mapping review, surveys, focus groups, a Delphi survey and stakeholder consultation workshops ().
Methods used to address each aim of the study. TAU, treatment as usual.
Aim 1: review of research evidence for interventions, outcomes and measures
To address the first aim of the research, we identified the range of interventions that parents could deliver at home to children with EDSD and the outcomes of the interventions that have been evaluated in previous research. We also assessed the extent of the research evidence for the effectiveness of identified interventions. Three relevant systematic reviews4–6 of interventions were published prior to the start of our research. These reviews appraised randomised controlled trials (RCTs) and quasi-experimental designs involving children with cerebral palsy,6 controlled group studies and single-case experimental designs involving children with ASD,5 and RCTs and quasi-experimental designs involving children who have oropharyngeal dysphagia of any neurological origin, including progressive conditions such as Friedreich’s ataxia.4 We updated the first two reviews to include material published from 1 October 2013 and 1 October 2014, respectively.5,6 The lead author of the review on oropharyngeal dysphagia4 updated her review during our study to include research findings from 1 October 2011 and provided us with pre-publication data.
As the three published systematic reviews of interventions appraised only a portion of the evidence on interventions for children with neurodisability and EDSD and their outcomes (i.e. those pertaining to children with the conditions listed above), we conducted a mapping review to find other published studies evaluating any EDSD intervention involving children with any non-progressive neurodisability condition and using any quantitative research design. The mapping review revealed the extent of the evidence for a greater range of types of intervention, in terms of the number of children who participated with physical and mixed EDSD and non-physical EDSD, the research designs used, the outcomes that were evaluated and the measures used to investigate progress in outcome areas.
In a further systematic review, we examined the measurement properties of the most relevant and promising candidate outcome measurement tools for EDSD identified in the systematic or mapping reviews.
Aim 2: defining current provision – interventions, outcomes and measures used in the UK
The second aim of the study was to find out which EDSD interventions HPs in the NHS currently provide or recommend, which interventions parents use at home and how parents and professionals judge whether or not these interventions work. We convened a consultation workshop to present the interventions and outcomes identified from the updates of the three published systematic reviews of interventions to HPs based in the north-east of England. Three speech and language therapists and one dietitian took part. They confirmed the co-investigators’ views that NHS HPs do currently provide or recommend the interventions appraised in the updates of the three published systematic reviews of interventions, sometimes in combination, and that the outcomes measured in the reviews are relevant when working with children with physical, non-physical and mixed EDSD. As all of the interventions and outcomes in the updates of the three published systematic reviews of interventions were deemed by the consultation group to be used with some children with neurodisability, none was removed from consideration in subsequent stages of the research following this consultation. The identified interventions and outcomes informed the development of a topic guide for the first round of focus groups with parents of children with EDSD and HPs working in the NHS. The focus groups, held in the north-east of England, explored whether or not the research-based interventions and outcomes are delivered to specific groups of children and if any interventions or outcomes were missing from our lists.
The interventions and outcomes identified in the updates of the three published systematic reviews of interventions, and discussed in the consultation workshop and focus groups, subsequently informed a UK-wide survey to quantify how frequently each of the parent-delivered interventions were recommended by NHS professionals; which interventions are used by parents; and how parents and professionals evaluate whether or not interventions are successful. We also included education professionals in the survey, to investigate which EDSD interventions are used by staff who feed children with neurodisability at school.
There was a considerable degree of overlap between the two streams of work to meet aims 1 and 2. The interventions and outcomes identified in the updates of the three published systematic reviews of interventions to meet aim 1 informed the topic guides for the focus groups, and were listed in the national survey to meet aim 2. Furthermore, the discussion in the focus groups and the answers to the survey (aim 2) informed searching and data extraction in the mapping review and systematic review of measurement properties (aim 1).
Aim 3: constructing trial frameworks to evaluate eating, drinking and swallowing difficulty interventions for children with neurodisability in the NHS
The third aim of the study was to construct one or more trial frameworks acceptable to children, young people, parents and professionals, or to specify the additional evidence about interventions, outcomes and tools that would be needed to support a future trial. We synthesised evidence gathered throughout the study: linking, building and merging findings to develop full lists of interventions and outcomes that are commonly used by families and/or are supported by research.13,14 Groups of parents and HPs sense checked the summaries in a second round of focus groups held in the north-east, south-west and south-east of England. Following the focus groups, parents and HPs from across the UK rated the importance of each of the identified interventions and outcomes for future research in two rounds of a Delphi survey, which revealed the extent of established consensus on the interventions and outcomes that are essential. The last element of the study involved stakeholder consultation workshops with parents and HPs and separate focus groups with young people with EDSD to inform a framework for future research into EDSD management of children with neurodisability in the NHS.
Delivery of the research
The research team was multidisciplinary and comprised clinical academics, clinicians, health services research methodologists and parents. The team members were from the north-east, south-east and south-west of England. The parent co-investigators (DG and JS) had experience of mixed and non-physical EDSD, were part of UK and international networks of parents of children with neurodisability and had previous experience of working in applied health research. The clinicians and clinical academics provided services to families of children with neurodisability who had EDSD: clinical psychology (HM and HT), community paediatrics (AC), gastroenterology (JT), neurodisability paediatrics (JP, JC and MA) and speech and language therapy (CB, LP and DS). The health services research methodologists had particular interests in childhood disability research (CM), clinical trials and health-related surveys (EM and CM) and evidence synthesis (DC).
The full research team met regularly to monitor the conduct and progress of the study and to consider the findings from each research activity. We discussed decisions on whether or not any interventions, outcomes or measurement tools lacked evidence of use in clinical practice in the UK or supporting research and, therefore, should not be taken forward. In the final months of the study, the research team met three times to discuss the main study findings and to formulate the recommendations for the design of future evaluation studies of interventions.
The research was directly informed by a parent advisory group (PAG) (see Patient and public involvement) that consisted of parents of children with neurodisability and physical, mixed and non-physical EDSD. An external study steering group that included members with clinical and research expertise and links to parent groups provided oversight and advice on the conduct and reporting of the research.
Patient and public involvement
The PAG advised on the methods, procedures, analysis and dissemination of each element of the research design. We recruited parents from the north-east of England to the PAG via social media and local networks known to parent co-investigators Deborah Garland and Johanna Smith. Deborah Garland is a local National Autistic Society (London, UK) representative. Johanna Smith is a parent of a child with mixed EDSD and additional sensory impairment. Five parents expressed an interest in taking part and three or four parents took part in each meeting, with one of those parents contributing their views via e-mail. Equal representation was achieved from parents of children with physical and mixed EDSD (n = 2) and parents of children with non-physical EDSD (n = 2). Their children ranged in age from 6 to 16 years. Meetings took place on four occasions over 10 months in Newcastle. PAG members agreed the dates and venues in advance to ensure convenience and maximise attendance. Any documents that required attention in the meetings were circulated in advance via e-mail to allow PAG members sufficient time to read them. The sessions were led by a minimum of two facilitators (HT, DG and JS) with at least one of the parent co-investigators present. The sessions lasted 2 hours and refreshments were provided. Parents received a £75 shopping voucher to thank them for their contribution.
The first meeting outlined the purpose of the group and agreed how parents would like to receive communication. Members agreed that face-to-face meetings were preferable and that any additional work would be undertaken via e-mail as necessary. The members did not wish to join a closed Facebook group (Facebook, Inc., Menlo Park, CA, USA; www.facebook.com) or use other social media in relation to group tasks. The group members agreed terms of reference for the PAG (see Appendix 1). In the first meeting, the PAG also considered a summary of the findings of the national survey of interventions recommended to and used by parents, and the important outcomes. Members discussed whether or not the findings were consistent with their experiences of supporting their child with EDSD and of service provision.
In the second meeting, the PAG received a summary of all the evidence gathered to date, which included findings from the systematic and mapping reviews, the first round of focus groups and the national survey. The discussion focused on how best to share this information with parents in the second round of focus groups. The PAG also reviewed the wording and layout of the Delphi survey and the associated information sheets. The discussions covered the use of appropriate and accessible language in documents, the presentation of visual information (colours, layout and clarity) and how best to present the information simply, without repetition or unnecessary jargon. The PAG were also presented with three parent-reported tools for outcome measurement that had relatively stronger evidence for supporting robust measurement properties. They commented on their wording, layout and ease of use.
In the third meeting, the PAG reviewed the amended Delphi survey and information sheets that had been modified in response to the discussion in the second round of focus groups. Discussions again focused on keeping information clear and accessible. The PAG also discussed how to order the statements relating to the interventions and outcomes in the Delphi survey and suggested providing examples of each intervention for clarity.
In the fourth meeting, the PAG discussed the findings of the Delphi survey and provided advice on how to present this information to parents at the stakeholder consultation workshops. The PAG reviewed the individual tasks for parents attending the workshops in terms of content, structure and timings. They also advised on how best to present information about the study and key findings in a short presentation, including simplifying language and presenting the study data in a pictorial format. They also suggested creating a document to send to all attendees prior to the workshops to provide a background to the study and clarity on what would happen on the day. The PAG were also asked to give their views via e-mail on the proposed dissemination plan during the final months of the study.
In each chapter of the report, we describe the patient and public involvement (PPI) in the planning and conduct of the individual stages of the study and the interpretation of their findings, along with the strengths and limitations of the individual research methods.
Ethics
The West Midlands and the Black Country Research Ethics Committee approved the study procedures (17/WM/0439). There were four amendments: one non-substantial amendment (to add new sites as participant identification centres), two planned substantial amendments to seek approval for documents developed for the Delphi survey, informed by the findings from earlier study stages, and one further substantial amendment (to change the recruitment target). Newcastle upon Tyne Hospitals NHS Foundation Trust was the research sponsor.
Complete and transparent reporting
The following chapters of the report describe the objectives, methods, results, strengths and limitations of each element of the research, using EQUATOR (Enhancing the Quality and Transparency of Health Research) reporting guidelines (www.equator-network.org/; accessed 11 November 2019), GRIPP2,15 Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA)16 and Strengthening the Reporting of Observational studies in Epidemiology (STROBE).17
