Introduction

In 2012, paediatric acquired brain injury (pABI) was identified as one of the leading causes of death in children aged 5 to 19 years1. In the UK, 280 children per 100 000 require at least 24 hours hospitalisation for traumatic brain injury (TBI) each year.2 When considering other aetiologies such as brain tumours, stroke and infection, there are even greater numbers of children surviving pABI.3The long-term, even life-long, effects on social functioning, cognition, emotions and behaviour mean that pABI is a leading cause of disability.4 5

Despite these on-going difficulties, children with pABI do not automatically receive specialist education, often returning to mainstream schools with little or no additional support. Furthermore, although specialist tertiary National Health Service (NHS) services do exist in the UK, there are limited outreach support or community services for ongoing and emerging difficulties.6 Families report struggling to access appropriate treatments despite their child developing significant and complex needs. Families also report significant distress and burden when caring for a child who has survived pABI, leading to an increased risk of mental health difficulties in parents and siblings, and a breakdown in parental relationships.7 Without appropriate neuropsychological interventions, pABI can lead to increased risk of substance misuse,8 mental health difficulties,9 unemployment and criminal behaviour in adulthood.10 Thus, the long-term costs of pABI to the individual, his/her family and society as a whole can be substantial.11

Executive function (EF) difficulties (higher-order cognitive processes that govern goal-directed action and adaptive responses to novel or complex situations) are common following pABI.12 These difficulties can present later in childhood (particularly in early adolescence), sometimes many years after the initial brain injury. EF difficulties can have far reaching effects, including problems with academic achievement,13 social communication,14 emotion and behaviour regulation15 and peer relationships.16 Indeed, families caring for children with pABI often report that EF difficulties significantly contribute to their increased levels of stress.17 Effective interventions targeting child EF and family burden are likely to have significant patient benefit especially as developmental studies show that poor family function can also negatively impact on the child’s development of EF abilities.18

Despite the clear negative impact of EF difficulties following pABI, there is currently a paucity of research examining the effectiveness of interventions to improve function.19 In an attempt to address this gap, Wade and colleagues developed an online web-based problem-solving, communication and self-regulation intervention for adolescents (12 to 17 years) who have survived a TBI, and their families (Teen Online Problem-Solving, TOPS).20 The development of online family problem-solving treatment for paediatric TBI was predicated on evidence of the reciprocity between child recovery and parental psychological well-being and informed by input from survivors of TBI and their families. Problem-solving therapy provides a systematic approach for coping more effectively with life’s challenges by creating a positive mindset and developing, implementing and evaluating solutions to problems.21 Online family problem-solving treatment simplifies the ABCDEs of problem-solving: defining the Aim, Brainstorming, Choosing the best strategy to implement, Doing it (creating a concrete, step-by-step plan) and Evaluating whether it worked. Given the focus on the family rather than the individual, TOPS also focuses on communication skills and collaborative problem-solving. For adolescents with executive function and problem-solving deficits following TBI, the steps of problem-solving also provide an executive function heuristic.22 Additionally, the programme teaches stress management and metacognitive strategies such as stopping to think before acting and self-monitoring that can serve to promote self-regulation. Similar to the face-to-face, multifamily Brain Injury Family Intervention,23 online modules provide didactic information regarding the cognitive and behavioural consequences of TBI, training in problem-solving, communication and self-awareness/regulation and exercises to reinforce understanding. After completing the online modules on their own, families meet with a trained therapist to implement to the problem-solving process around an Aim (goal) that they have identified. Thus, the online problem-solving programme was grounded in the scientific literature, tailored to address the unique neurobehavioral and family consequences of TBI and refined through stakeholder input.20 Problem-solving therapy has also been trialled as a telephone-based intervention for adults with mild TBI and with families of adults with more significant brain injury,24 suggesting its potential utility across age groups and modes of delivery.25

Research to date has been undertaken in the USA, and has focused on children and adolescents who have survived a TBI and their families.20 26–31 These studies have demonstrated improvements in EF, child behaviour, parental depression and family-child conflict when comparing TOPS with an internet-resource comparison control. Families have reported finding the online delivery of the intervention helpful, making the intervention easy to access at a time that is convenient for them. This is an important consideration when providing interventions for children with pABI in the UK and their families, because brain injury services in the UK often cover a large geographical region (many accepting national referrals), and families often have multiple commitments when caring for these teenagers with complex needs.

Despite this emerging evidence base, it is not yet known whether the gains described can also be demonstrated in adolescents with non-traumatic brain injuries (eg, brain tumour, stroke), when the intervention is delivered remotely (ie, no initial home visit to set up the intervention thus keeping delivery costs to a minimum), and if the intervention is cost-effective relative to treatment as usual (TAU).

A British version of the TOPS programme, (TOPS-UK) has been developed. The didactic material is presented with British English spelling and narration, UK-based information including scenarios, video clips and resource links. TOPS-UK has been further modified to include examples of those with paediatric acquired brain injury as well as traumatic brain injury.

The potential of TOPS-UK to have a positive effect on the lives of thousands of adolescents with pABI needs testing in a rigorous multicentre, randomised controlled trial (RCT) in the UK, allowing a definitive test of its clinical and cost-effectiveness. This feasibility study is designed to determine if a large RCT is feasible, with the ultimate aim of the research being to provide an evidence base for effective neuropsychological interventions to be recommended by the Department of Health guidelines (eg, NICE) for survivors of pABI.

A two-arm randomised controlled feasibility phase II study will be conducted.32 The aim is to assess whether a larger, fully powered, definitive RCT and cost-effectiveness analysis can be successfully planned and delivered. In order to gain as much information as possible to inform a fully powered RCT, additional objectives are considered in this feasibility trial, including accessing information related to treatment set-up, delivery costs and resource use (table 1).

Methods and analysis

This is a randomised, controlled, multicentre feasibility study in young patients with pABI aged 12 to 18 years. Twenty-five participants will be randomised (minimised by site and type of brain injury) in a 1:1 ratio to receive TAU or treatment as usual plus Teen Online Problem-Solving (TAU + TOPS UK). Treatment allocation will not be blinded. The protocol (V.4.1, 30 August 2018) is registered on Current Controlled Trials ISRCTN10906069 and follows Standard Protocol Items: Recommendations for Interventional Trial guidelines.33

Intervention

The study has two treatment arms, ‘TAU’ and ‘TAU + TOPS-UK’. Those allocated to the TAU + TOPS UK arm will work through the intervention programme taking 1 to 2 weeks per module. One coach will be trained to deliver the intervention to all of the participants in the intervention arm. The coach will coordinate the timing of the modules so that all families will have completed the intervention at 16 weeks. This will allow time for other commitments and holidays/breaks to be built into the schedule. The length of time taken to complete each module will be recorded to inform timing of follow-up in the main trial.

Qualitative interviews

A single qualitative researcher will conduct semi-structured qualitative process evaluation interviews by telephone or video call with all participants (including those who have not completed follow-up questionnaires but who have not formally withdrawn from the study), research nurses and the study coach. PIS relating to the qualitative interviews will be emailed in advance and consent will be sought at the beginning of the interview by the qualitative researcher. Adolescent and parent participants will be offered the option of being interviewed together or separately. Interviews will be conducted with the help of an agreed topic guide (see online supplementary appendix) and are expected to last between 20 to 60 min. Interviews will be audio-recorded and transcribed verbatim. The transcribed interview data will be fully anonymised and any demographic data about participants will be stored separately. Qualitative interview data will be managed using a computer software package such as Nvivo 11 and thematically analysed.45 The analysis and results will be checked/validated by a second qualitative researcher. The following interviews will be conducted:

1. All participants (both groups). These interviews will allow participants to describe their whole experience of participating in the study including their willingness to be randomised and their experiences and acceptability of outcome measures, what worked well and what less well. This will cover aspects such as clarity of PIS, acceptability and ease of completing the online consent process, ease of completing online questionnaires and the parental support required the adolescent to participate and remain engaged. The interviews will explore adolescent and parent experiences of TAU or TAU + TOPS-UK, including parent’s perceptions of the impact of TAU/TAU + TOPS-UK on their ability to support the adolescent, adolescent’s and parent’s perceptions of the impact of TAU/TAU + TOPS-UK on adolescent’s ability to stay positive, solve problems, be organised, control their emotions and look after themselves (self-care), and participants’ views on potential improvements to TAU/TAU + TOPS-UK. In addition, participants in the TAU + TOPS-UK will be asked about their experience of working with the coach via video calls. This information will be helpful when planning the full RCT and implementation studies. After completion of the interview, each family will be sent a £15 gift voucher as a token of appreciation.

2. Interview with TOPS-UK coach. A single interview will explore the coach’s experiences of supporting the TOPS-UK intervention. The interview will be held by telephone, or face-to-face, once every participant allocated to the intervention arm has completed the programme.

3. Interviews with research nurses. All research nurses involved with the study at the five participating sites will be invited to participate in a single interview to explore their experiences of supporting families through the consent process and any support required to complete baseline or follow-up measures. Feedback on methods of contact with families, number of contact attempts made, how much support was required from adolescents and/or parents, barriers to recruitment and suggestions for future studies will also be sought.

Participants who withdraw from the study, participants who register their interest but do not complete the study consent forms and participants who discontinue the intervention and who do not complete the follow-up questionnaires (ie, non-adherent but not explicitly withdrawn), will be invited to complete an anonymous online feedback survey. The survey will invite participants to comment on any aspects of the study that they found difficult, any aspects of the study that they liked and suggestions on how the study can be improved.

Economic evaluation

This feasibility study will be used to develop a framework for a subsequent, policy-relevant, cost-effectiveness analysis to be undertaken alongside a future RCT. Economic evaluation methods will be developed and assessed regarding the collection of resource use, cost and outcome data. Data on resource use associated with the set-up and delivery of the TOPS treatment will be collected at the participant and coach level for example, coach contact and non-contact time per participant, equipment and consumable costs, training and supervision requirements for the TOPS-UK coach. Data regarding service use will be collected from all participants’ parents using a version of the CSRI specifically modified for this population. This includes use of mental health, community rehabilitation and neuropsychology and educational psychology services and has been previously trailed in this population (Wilson et al, in preparation). In addition to informing the development of the economic evaluation methods, this will provide a profile of the resources and services that constitute TAU. The study will also consider the most appropriate manner in which to capture data on health-related quality of life for use in the estimation of the cost per QALY of the TOPS-UK treatment. The feasibility of participants self-completing the CHU-9D and parents completing the EQ-5D-5L, will be assessed.

In addition to this primary economic outcome, the appropriateness of considering the cost-per unit change on other relevant indicators of health status collected in the baseline and follow-up phases will be explored.

Safety reporting

The risks associated with participating in this study are considered minimal. There is a slight chance for those in the intervention group that raising awareness of injury-related cognitive or behavioural problems through communication and problem-solving might increase family burden and possibly contribute to conflict between family members. However, the purpose of the intervention is ultimately to equip families with skills to handle these difficulties by learning how to change the way they solve problems and talk with one another, and the TOPS-UK coach supporting the online intervention will be trained to handle any emerging problems. Should any issues arise, the coach will have access to a qualified clinical psychologist to provide further support and advice. There is therefore no requirement to report non-serious adverse events in this study, although serious adverse events (SAEs) will be monitored.

SAEs may be reported by clinicians or researchers at site, the TOPS-UK coach, by participants themselves or by any other informant. Adverse event data will be monitored by the Trial Steering Committee (TSC) to ensure safety. The TSC includes an independent statistician, psychologist, paediatrician and patient and public representative and will meet on approximately four occasions. All suspected SAEs will be reported within 24 hours of discovery to the CTU who will notify the Chief Investigator. All SAEs will be followed up until resolution.

Participant and public involvement

A PPI group including patients and the CI was convened at an early stage of grant development and trial design and continued to meet during study set-up. The group gave feedback on the patient-facing materials including age-appropriate PIS, informed consent forms and qualitative interview topic guide content. A lay-representative who is Director of Services and Innovation at the national Child Brain Injury Trust (CBIT) is a co-applicant to the grant, and a member of both the Trial Management Group (TMG) and TSC. During the recruitment period, this lay-representative has been liaising with local CBIT representatives and PICs to identify potentially eligible families. A study twitter account will post trial updates to promote public engagement with the study. The twitter site currently follows and is followed by national brain injury charities disseminating trial updates to a wide audience.

Another PPI representative (parent of a child with pABI) is a member of the TMG, regularly attending meetings and freely contributing to the discussions. Both members provide advice and suggest solutions to problems encountered during the trial, with particular expertise in barriers to recruitment and communication issues within families with a teenager with pABI. This expertise has been cascaded to site staff through a research nurse forum.

The contribution of PPI members within the TMG and TSC will be valuable during analysis, interpretation and dissemination of the study results. Specifically, PPI members will be asked to contribute to data interpretation, assess whether the feasibility study has met its objectives and support different approaches to dissemination of the study. They will also be asked to comment on potential changes for the main trial. The Director of Services and Innovation at CBIT will also support wider PPI in the development of the main trial.

Study management and oversight

The study sponsor organisation is the Royal Devon and Exeter NHS Foundation Trust, Barrack Road, Exeter EX2 5DW. Day-to-day trial management is administered through the UKCRC-registered Peninsula Clinical Trials Unit at Plymouth University. A Trial Management Group including the Chief Investigator, CTU trial managers, trial statistician and other personnel relevant to the study (eg, clinicians, CTU data manager, patient and Sponsor representatives) will meet regularly (usually monthly) throughout the duration of the trial to oversee practical management of the trial.

A TSC, chaired by an independent member, will oversee the conduct and safety of the trial, ensuring that milestones are achieved and general scientific probity is maintained. A Data Monitoring Committee was not required for this feasibility study.

Discussion

In our opinion, TOPS-UK has genuine potential to have a profound and positive effect on the lives of thousands of adolescents with brain injury. The intervention itself already exists and has demonstrated efficacy in the USA, including gains in executive function (planning, problem-solving), reductions in behaviour and mood difficulties and reductions in family burden and stress. TOPS-UK urgently requires testing in a rigorous multicentre randomised trial in the UK, allowing a definitive test of its clinical and cost-effectiveness.

The importance of the current study is to determine if a large RCT is feasible (eg, recruitment, access to resource use data, appropriate outcome measures etc). In addition, although the study is not powered to detect effectiveness, individual patients participating in the study might gain direct benefit from the treatment (TAU + TOPS) in terms of increased executive function skills, reduced behaviour and mood difficulties, and improved quality of life. The feasibility study might also have some implications on clinical and public health practice by raising awareness of pABI, the need for effective treatment, and the variability of TAU (via the dissemination workshop and reports), and improve the measurements of quality of life and health status in adolescents with pABI (via conferences). If the current study demonstrates feasibility, outcomes will be used to inform the development of a fully powered phase III RCT to examine effectiveness and cost-effectiveness of TOPS-UK.

If the phase III RCT is successful, then the clinical and public health practice developments are potentially far reaching. For example, in addition to the direct benefits to the adolescent and his or her family, TOPS-UK might also prevent considerable NHS costs in the future by reducing healthcare service use. Furthermore, given the increased risks of criminal behaviour and poor educational and vocational outcomes following pABI, often associated with poor executive functioning, TOPS-UK might have wider social benefits for these individuals, and society as a whole. In terms of potential impact on local policy-making and improvement in service delivery, TOPS-UK is potentially very cheap to deliver because it is web-based and involves regular but brief professional support that is delivered remotely (via video calls). Furthermore, given its web-based delivery that can be accessed at a time that is convenient for the adolescent and their family, TOPS-UK is less disruptive than clinic appointments, which are often delivered during school-hours. This is an important consideration for an individual with pABI because, typically, the adolescents have already missed a significant amount of school and struggle academically. By enabling free use of the materials to the NHS, clinical services might routinely provide TOPS-UK to adolescents with pABI and their families, meeting the needs of multiple families simultaneously. TOPS-UK might also provide a first-line of treatment for families experiencing distress and, therefore form part of a ‘stepped-care’ model of service delivery such that, families who continue to experience significant difficulties, or who are at high risk, can be referred to specialist services in a timely manner. The ultimate aim of the research programme is to provide an evidence-base for effective neuropsychological interventions to be recommended by the Department of Health guidelines (eg, NICE) for survivors of pABI.