Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials

التفاصيل البيبلوغرافية
العنوان: Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials
المؤلفون: Gonzalez-Robles, Cristina, Bartlett, Michèle, Burnell, Matthew, Clarke, Caroline S., Haar, Shlomi, Hu, Michele T., Huxford, Brook, Jha, Ashwani, Lawton, Michael, Noyce, Alastair, Piccini, Paola, Pushparatnam, Kuhan, Rochester, Lynn, Siu, Carroll, van Wamelen, Daniel, Williams-Gray, Caroline H., Zeissler, Marie Louise, Zetterberg, Henrik, 1973, Carroll, Camille B., Foltynie, Thomas, Weil, Rimona S., Schrag, Anette
المصدر: Movement Disorders. 39(2):433-438
مصطلحات موضوعية: Neurosciences, Neurovetenskaper, clinical trials, disease modification, endpoints, outcome measures, Parkinson disease, Patient and Public Involvement and Engagament (PPIE)
الوصف: Background: Clinical trials of disease-modifying therapies in PD require valid and responsive primary outcome measures that are relevant to patients. Objectives: The objective is to select a patient-centered primary outcome measure for disease-modification trials over three or more years. Methods: Experts in Parkinson's disease (PD), statistics, and health economics and patient and public involvement and engagement (PPIE) representatives reviewed and discussed potential outcome measures. A larger PPIE group provided input on their key considerations for such an endpoint. Feasibility, clinimetric properties, and relevance to patients were assessed and synthesized. Results: Although initial considerations favored the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III in Off, feasibility, PPIE input, and clinimetric properties supported the MDS-UPDRS Part II. However, PPIE input also highlighted the importance of nonmotor symptoms, especially in the longer term, leading to the selection of the MDS-UPDRS Parts I + II sum score. Conclusions: The MDS-UPDRS Parts I + II sum score was chosen as the primary outcome for large 3-year disease-modification trials. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
الوصول الحر: https://gup.ub.gu.se/publication/332727Test
قاعدة البيانات: SwePub
الوصف
تدمد:08853185
15318257
DOI:10.1002/mds.29691