Development and early qualitative evidence of two novel patient-reported outcome instruments to assess daily functioning in people with early-stage Parkinson’s

Journal of Patient-Reported Outcomes

卷:7

Research

Ashley F. Slagle¹  Stefan Cano²  Sophie Cleanthous²  Geraldine Blavat³  Casey Gallagher³  Karlin Schroeder³  John Andrejack³  William Brooks³  Natasha Ratcliffe⁴  Paul Burns⁴  Carroll Siu⁴  Lesley Gosden⁴  Bastiaan R. Bloem⁵  Babak Boroojerdi⁶  Milton Biagioni⁷  Thomas Morel⁷  Roger A. Barker⁸ 

[1] Aspen Consulting, LLC, Steamboat Springs, CO, USA;

[2] Modus Outcomes, a Division of Thread, London, UK;

[3] Parkinson’s Foundation, New York, NY, USA;

[4] Parkinson’s UK, London, UK;

[5] Radboud University Medical Centre; Donders Institute for Brain, Cognition and Behaviour; Department of Neurology; Centre of Expertise for Parkinson & Movement Disorders, Nijmegen, The Netherlands;

[6] UCB Biosciences GmbH, Monheim, Germany;

[7] UCB Pharma, Allée de la Recherche 60, 1070, Anderlecht, Brussels, Belgium;

[8] University of Cambridge, Cambridge, UK;

关键词: Parkinson’s;    Early-stage Parkinson’s;    Patient expert;    Patient-reported outcome instrument;    Clinical outcome assessments;    Qualitative evidence;    Slowness;    Mobility;   

DOI  :  10.1186/s41687-023-00577-9

 received in 2022-10-31, accepted in 2023-03-15,  发布年份 2023

来源: Springer

【 摘 要 】

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.

【 授权许可】

CC BY   
© The Author(s) 2023

【 图 表 】

Fig. 11