Children with hyperkinetic movement disorders like dystonia are being asked to draw and photograph their experiences to help researchers finally agree on what counts as a meaningful treatment outcome. These conditions cause involuntary muscle contractions that twist the body into abnormal postures, making everyday tasks—writing, eating, playing—extremely difficult. The problem is that no one has agreed on a standard way to measure whether a treatment actually works. Without this, large clinical trials cannot happen, and care varies wildly from one clinic to the next. This project tackles that gap head-on. If successful, the research will produce a core outcome set—a shortlist of what to measure in every future trial—so that studies can be compared and combined. It will also test whether a problem-solving therapy called CO-OP, where children set their own goals and work through a "plan-do-check" cycle, is feasible to study in a full-scale trial. The immediate impact is on clinical research infrastructure: better trials mean better evidence, which means clinicians and families can make informed decisions about treatment.
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Background: Hyperkinetic movement disorders, including dystonia, are characterised by excessive and involuntary muscle contractions, leading to abnormal postures and repetitive movements that worsen during voluntary actions. These movements cause significant disability and interfere with social life, education, and participation, impacting health and economic outcomes. Dystonia is the third movement disorder after Parkinson's and tremor (underestimated prevalence 15-30/100.000 people). In childhood, dystonia is often associated with cerebral palsy, the most common lifelong physical disability affecting 17 million people globally, but many are rare disorders. The heterogeneity of symptoms, diagnoses, and variations in motor severity and functional ability make evaluating and managing these conditions challenging. The unpredictable and variable nature of involuntary movements further complicates the assessment of treatment effects. Additionally, the lack of outcome measurement consensus and limited validated measures results in a critically under-served population with no large trials available, variable usual care and no agreed best practice. One promising treatment is Cognitive Orientation to Occupational Performance (CO-OP). Children/young people set their goals and are guided to discover personalised strategies through 'goal-plan-do-check' (a problem-solving approach). Once successful strategies are identified, individuals practice achieving real-life tasks/goals. Learning is transferred to untrained goals enabling individuals to solve their own problems. Aims: To develop a core outcome set for studies/trials in childhood-onset hyperkinetic movement disorders and determine the acceptability and feasibility of conducting a full-scale CO-OP randomised controlled trial. Methods: Work package (WP)-1: Core Outcome Set Development of Delphi questionnaire Scoping review: mapping outcomes to the International Classification of Functioning Qualitative interviews with children/young people and parents/carers: open-ended questioning and creative methods (i.e., photography, drawing) (face-to-face/online) International online survey with professionals. Three-round international online Delphi questionnaire with children/young people, parents/carers, and professionals, informed by the scoping review, qualitative interviews and professional survey. Establish core outcome set after reviewing Delphi findings through two expert panels (i)children/young people and family/carers, (ii)professionals. WP2: Randomised feasibility study of CO-OP with embedded process and feasibility economic evaluations (following MRC-complex intervention guidelines). Feasibility: Progression criteria (green/amber/red) for key parameters, e.g., recruitment and retention rates, adherence, acceptability, and standard deviation of primary outcome measure. Fifty participants will be randomised to immediate (CO-OP+ usual care) or waiting-list control (usual care) across five sites. Acceptability: Post-intervention questionnaires and semi-structured interviews with staff, children/young people, and parents/carers using the Theoretical Framework of Acceptability. Convergent method to synthesise quantitative and qualitative data. Feasibility of an economic evaluation: Resource use data and quality of life on changes of CO-OP versus usual care. Impact: Outputs will support an HTA-stage 1 application and help establish my research career. A core outcome set will provide a standardised approach for assessing treatment effects. Understanding what matters to individuals with lived experience can improve future studies/trials. Findings will inform the design of a CO-OP trial, allowing precise implementation and evaluation. Dissemination: Research interns with lived experience will help tailor outputs and support dissemination planning, i.e., comics, lay summaries, peer-reviewed papers, or conferences. Broad dissemination will maximise the research findings' impact through existing partnerships with charities, clinicians, researchers, faith, and community-leaders.
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