Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials.
Mov Disord
; 39(2): 433-438, 2024 Feb.
Article
in En
| MEDLINE
| ID: mdl-38140767
ABSTRACT
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.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Parkinson Disease
Limits:
Humans
Language:
En
Journal:
Mov Disord
Journal subject:
NEUROLOGIA
Year:
2024
Document type:
Article
Affiliation country: