ArticleJournal of patient-reported outcomes2023
Development and early qualitative evidence of two novel patient-reported outcome instruments to assess daily functioning in people with early-stage Parkinson's.
Article in Journal of patient-reported outcomes, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 1 of them a synthesis that pooled it.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
11 citing papers in PubMed, 1 synthesis or guideline pooled it.
- The pharmacodynamics-based prophylactic benefits of GLP-1 receptor agonists and SGLT2 inhibitors on neurodegenerative diseases: evidence from a network meta-analysis.BMC medicine · 2025Pooled it
- A Preliminary Mechanics-Informed Machine Learning Framework for Objective Assessment of Parkinson's Disease and Rehabilitation Outcomes.Diagnostics (Basel, Switzerland) · 2025Article
- Optimizing the MDS-UPDRS Part III for early-stage Parkinson's: early supportive evidence for a limb-related bradykinesia/rigidity sub-score.NPJ Parkinson's disease · 2025Article
- Patient Engagement and Patient Experience Data in Regulatory Review and Health Technology Assessment: Where Are We Today?Therapeutic innovation & regulatory science · 2025Review
- Inclusion in neurological research: empowering people living with neurological diseases.Nature reviews. Neurology · 2025Review
- Neurotrophic factors for Parkinson's disease: Current status, progress, and remaining questions. Conclusions from a 2023 workshop.Journal of Parkinson's disease · 2024Review
- The PompeQoL questionnaire: Development and validation of a new measure for children and adolescents with Pompe disease.Journal of inherited metabolic disease · 2024Article
- An Ethnographic Study of Patient Life Experience in Early-Stage Parkinson's Disease in the United States and Germany.Neurology and therapy · 2024Article
- The value of co-creating a clinical outcome assessment strategy for clinical trial research: process and lessons learnt.Research involvement and engagement · 2023Article
- Improving naturalistic neuroscience with patient engagement strategies.Frontiers in human neuroscience · 2023Article
- Most common, burdensome, and worrisome symptoms experienced by people living with early Parkinson's disease in Germany and the United States-Results from a cross-sectional survey.Frontiers in neurologyArticle
Corrections and comments
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Authors and funding
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPrevious research on concepts that are important to people living with early-stage Parkinson's indicated that 'functional' slowness, fine motor skills, and subtle gait abnormalities are cardinal concepts that are not comprehensively captured by existing patient-reported outcome (PRO) instruments that are used in clinical practice and research to assess symptoms and daily functioning within this patient population. We sought to develop novel PRO instruments to address this unmet need.
methodsPRO instrument development was led by a multidisciplinary research group, including people living with Parkinson's (termed 'patient experts'), as well as patient engagement and involvement, regulatory science, clinical, and outcome measurement experts. A first set of PRO instruments, termed Early Parkinson's Function Slowness (42 items) and Early Parkinson's Mobility (26 items), were drafted to capture 'functional' slowness, fine motor skills, and subtle gait abnormalities. These PRO instruments were used in cognitive debriefing interviews with people living with early-stage Parkinson's (who were not involved with the multidisciplinary research group) to identify issues with relevance, clarity, ease of completion, conceptual overlap, or missing concepts.
resultsSixty people living with early-stage Parkinson's were interviewed, which led to refining the items to 45 for the Early Parkinson's Functional Slowness and 23 for the Early Parkinson's Mobility PRO instruments. Refinement included rewording items to address clarity issues, merging or splitting items to address overlap issues, and adding new items to address missing concepts. The Early Parkinson's Function Slowness PRO instrument resulted in a multidimensional instrument covering upper limb, complex/whole body, general activity, and cognitive functional slowness. The Early Parkinson's Mobility PRO instrument resulted in comprehensive coverage of everyday mobility tasks, with a focus on gait concepts, plus complex/whole body, balance, and lower limb mobility.
conclusionsThe Early Parkinson's Function Slowness and Early Parkinson's Mobility PRO instruments aim to address gaps in existing PRO instruments to measure meaningful symptoms and daily functioning in people living with early-stage Parkinson's. Utilizing a meticulous study design led by a multidisciplinary research group that included patient experts helped to ensure that the PRO instruments were patient-centric, content valid, and meaningful from a clinical and measurement perspective.
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