Development and early qualitative evidence of two novel patient-reported outcome instruments to assess daily functioning in people with early-stage Parkinson’s
Background Previous 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. Methods PRO 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. Results Sixty 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. Conclusions The 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..
Plain English Summary Clinical outcome assessment tools can be used to show how a disease impacts a person’s daily life. These outcomes are assessed with tools like surveys or questionnaires. In Parkinson’s, most of the tools currently available do not reflect the things that are important to people living with early-stage Parkinson’s. Instead, these tools were designed to assess people in later stages of the disease. In this study, the research team created two new tools to assess daily functioning in early-stage Parkinson’s. People with early-stage Parkinson’s were interviewed to test the new tools. The language and questions in the tools were changed based on feedback from the interviews. The tools were reviewed by a large team, including patient experts and healthcare professionals involved in Parkinson’s care. Experts in outcome measures and clinical trials also reviewed the tools. The review step ensured that the tools are easy to understand and appropriate for people living with early-stage Parkinson’s. The new tools focus on the symptoms and experiences that are most important to people living with early-stage Parkinson’s. We hope that the tools will more clearly show the impact of early-stage Parkinson’s and how it progresses. Further testing of these new tools is needed. The aim is to use them in future clinical trials to measure the experience of early-stage Parkinson’s more accurately..
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:7 |
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| Enthalten in: |
Journal of patient-reported outcomes - 7(2023), 1 vom: 20. Apr. |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Morel, Thomas [VerfasserIn] |
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| Links: |
Volltext [kostenfrei] |
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| Themen: |
Clinical outcome assessments |
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| Anmerkungen: |
© The Author(s) 2023 |
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| doi: |
10.1186/s41687-023-00577-9 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 520 | |a Background Previous 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. Methods PRO 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. Results Sixty 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. Conclusions The 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. | ||
| 520 | |a Plain English Summary Clinical outcome assessment tools can be used to show how a disease impacts a person’s daily life. These outcomes are assessed with tools like surveys or questionnaires. In Parkinson’s, most of the tools currently available do not reflect the things that are important to people living with early-stage Parkinson’s. Instead, these tools were designed to assess people in later stages of the disease. In this study, the research team created two new tools to assess daily functioning in early-stage Parkinson’s. People with early-stage Parkinson’s were interviewed to test the new tools. The language and questions in the tools were changed based on feedback from the interviews. The tools were reviewed by a large team, including patient experts and healthcare professionals involved in Parkinson’s care. Experts in outcome measures and clinical trials also reviewed the tools. The review step ensured that the tools are easy to understand and appropriate for people living with early-stage Parkinson’s. The new tools focus on the symptoms and experiences that are most important to people living with early-stage Parkinson’s. We hope that the tools will more clearly show the impact of early-stage Parkinson’s and how it progresses. Further testing of these new tools is needed. The aim is to use them in future clinical trials to measure the experience of early-stage Parkinson’s more accurately. | ||
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