BACKGROUND:
The identification of suitable outcome measures will improve the evaluation of integrated NHS care for the large number of children affected by neurodisability, and has the potential to encourage the provision of more appropriate and effective health care. This research sought to appraise the potential of patient-reported outcome measures (PROMs) for children and young people with neurodisability.
AIM:
This research aimed (i) to identify key outcomes of health care for children with neurodisability, eyond morbidity and mortality, from the perspectives of children, parents and professionals; (ii) to critically appraise existing generic multidimensional PROMs; and (iii) to examine whether or not the key outcomes might be measured by existing PROMs. We also sought agreement on a definition of neurodisability.
METHODS:
Data were gathered in three main ways, (i) a systematic review identified eligible generic multidimensional PROMs and peer-reviewed studies evaluating psychometric performance using English-language questionnaires. Studies were appraised for methodological quality and psychometric performance was appraised using standard criteria. (ii) Focus groups and interviews with children and young people with neurodisability, and separately with parents, sought to identify important outcomes of NHS care, and their feedback on example PROM questionnaires. (iii) An online Delphi survey was conducted with a multidisciplinary sample of health professionals to seek agreement on appropriate NHS outcomes. In addition, we convened a consensus meeting with a small nominal group of young people, parents and professionals; the group sought agreement on a core set of important health outcomes.
RESULTS:
From the systematic review, we identified 126 papers that reported eligible evidence regarding the psychometric performance of 25 PROMs. Evidence of psychometric robustness was more favourable for a small number of PROMs: KIDSCREEN (generic), DISABKIDS (chronic-generic) and Child Health Utility 9D (preference-based measure). The Pediatric Quality of Life Inventory and KINDL offer both self-report and a proxy report version for a range of age bands, but evidence of their psychometric performance was weaker. Evidence was lacking in one or more respects for all candidate PROMs, in both general populations and those with neurodisability. Proxy reporting was found generally to be poorly correlated with self-report. Focus groups and interviews included 54 children and young people, and 53 parents. The more important health outcomes were felt to be communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, parents of children with intellectual impairment identified behaviour, toileting and safety as important outcomes. Participants suggested problems with the face validity of example PROM questionnaires for measuring NHS care. In the Delphi survey, 276 clinicians from a wide range of professions contributed to at least one of four rounds. Professionals rated pain, hearing, seeing, sleep, toileting, mobility and communication as key goals for the NHS but also identified treating neurological symptoms as important. Professionals in the Delphi survey and parents working with the research team agreed a proposed definition for neurodisability. The consensus meeting confirmed overlap between the outcomes identified as important by young people, parents and professionals, but not complete agreement.
CONCLUSIONS:
There was agreement between young people, parents and professionals regarding a core suite of more important health outcomes: communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, behaviour, toileting and safety were identified as important by parents. This research suggests that it would be appropriate to measure these constructs using PROMs to assess health care. None of the candidate PROMs in the review adequately captures all of the identified constructs, and there is inadequate evidence that candidate PROMs are psychometrically robust for use across children with neurodisability. Further consultation with young people, families and professionals is warranted to support the use of PROMs to measure NHS outcomes. Research to test potential PROMs with different age groups and conditions would be valuable.
PROTOCOL:
This research will identify which PROMs would be the most appropriate to assess the effectiveness of NHS care for children with neurodisability. There are a number of PROMs that could be used with children. At the moment, there is no research that has looked specifically at how well these questionnaires work for young people with neurodisability. The proposed research will investigate this issue by addressing the question - what outcomes of NHS care should be measured for children with neurodisability?
To answer the question we will use a range of structured approaches to understand the perspectives of children with neurodisability, parents and professionals and review relevant evidence. The research is planned in stages so that by the end a shared a shared vision can emerge of the specific health outcomes that should be used for this group of disabled children. The methods include a structured review and appraisal of the psychometric performance of PROMs when used with children with neurodisability, qualitative research with disabled children and parents, and Delphi survey with professionals.
The proposed research directly addresses the needs of the NHS within the new Outcomes Framework and key recommendations from the Kennedy review of barriers to improving NHS services for children and young people. Getting this right will benefit both the NHS and disabled young people and their families by improving the evaluation of integrated NHS care, and potentially influencing the provision of effective and efficient health and social interventions.
Morris C, Janssens A, Allard A, Thompson-Coon J, Shilling V, Tomlinson R, Williams J, Fellowes A, Rogers M, Allen K, Beresford B, Green C, Jenkinson
Disease Category: Child health, Developmental, psychosocial, & learning problems, Neurology
Disease Name: Neurodisability
Age Range: 0 - 18
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Patient perspectives
- Systematic review of outcome measures/measurement instruments
- Recommendations made
- Consensus meeting
- Delphi process
- Focus group(s)
- Interview
- Systematic review