Peer-reviewed article
Improving Measurement and Priorities for Assessment in Clinical Trials of childhood-onset Dystonia International Core Outcome Set (Dystonia IMPACT-COS): A protocol.
Gimeno H, Scott H, Edwards K, Redding EP, Kul-Want ET et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This is a protocol document describing plans to establish international consensus on outcome measurement standards for clinical trials of childhood-onset dystonia. The study will use a three-phase mixed-methods approach including scoping reviews, qualitative interviews, surveys, and Delphi consensus procedures involving health professionals, families, and children. The goal is to standardize how dystonia-related motor and non-motor outcomes are measured in research and clinical practice.
Key points
- Childhood-onset non-degenerative dystonia causes substantial lifelong disability and functional impairment
- Currently there is no consensus on which outcomes to measure or how to measure them in dystonia research
- The study involves three phases: scoping review and stakeholder engagement, international e-Delphi consensus, and identification of available outcome measures
- The protocol includes participation from children, families, and professionals across health, social care, and education sectors
- Study is being co-produced with advisory groups and will disseminate findings through peer-reviewed publications and engagement events
Limitations
This is a protocol document describing planned research rather than completed findings, so it cannot report actual results or evidence about intervention effectiveness. The document does not evaluate any intervention for improving child well-being outcomes.
Abstract
Introduction Dystonia is a movement disorder characterised by abnormal movements and/or postures, initiated or worsened by voluntary action. Non-degenerative childhood dystonia is common, represents a substantial source of lifelong disability, and is often associated with other motor and non-motor phenotypes that can contribute to functional impairments. However, there is no consensus on how to measure motor and non-motor outcomes associated with dystonia, nor is there consensus on what outcomes to measure. This prevents adequate evaluation and development of evidence-based interventions and decision-making in clinical practice. Here, we present the protocol for establishing global expert consensus on the Dystonia Impact Core Outcome Set (DI-COS) to describe what dystonia-associated outcomes to measure and how to measure them. Methods and analysis The DI-COS study includes a mixed-methods programme with three phases. Phase 1 includes; (i) scoping review, (ii) qualitative interview study with children and families, and (iii) international survey of health, social care, and education-based professionals. Phase 2 is a three round international e-Delphi study informed by Phase 1, and expert consensus from: (i) a professional panel and (ii) experts with lived experience (parents and children). Content and thematic analysis, and descriptive statistics will be used to analyse qualitative and quantitative data, respectively. Results will be triangulated to inform development of the Delphi survey in Phase 2. Phase 3 will identify the outcome measures available and recommend assessment outcomes agreed as part of Phase 2 Consensus. Ethics and dissemination Ethical approval has been obtained from Camden and King's Cross Research Ethics Committee (25/LO/0150) and Queen Mary, University of London Research Ethics Committee. All dissemination activities (including papers submitted to peer-reviewed journals, abstracts submitted to national/international conferences, visual outputs and engagement events), will be co-produced, co-designed and co-presented/authored with members of the study's advisory groups and shared via project's networks.