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dc.contributor.authorGonzalez‐Robles, C
dc.contributor.authorBartlett, M
dc.contributor.authorBurnell, M
dc.contributor.authorClarke, CS
dc.contributor.authorHaar, S
dc.contributor.authorHu, MT
dc.contributor.authorHuxford, B
dc.contributor.authorJha, A
dc.contributor.authorLawton, M
dc.contributor.authorNoyce, A
dc.contributor.authorPiccini, P
dc.contributor.authorPushparatnam, K
dc.contributor.authorRochester, L
dc.contributor.authorSiu, C
dc.contributor.authorvan Wamelen, D
dc.contributor.authorWilliams‐Gray, CH
dc.contributor.authorZeissler, M
dc.contributor.authorZetterberg, H
dc.contributor.authorCarroll, CB
dc.contributor.authorFoltynie, T
dc.contributor.authorWeil, RS
dc.contributor.authorSchrag, A
dc.date.accessioned2024-01-24T12:21:48Z
dc.date.available2024-01-24T12:21:48Z
dc.date.issued2023-12-22
dc.identifier.issn0885-3185
dc.identifier.issn1531-8257
dc.identifier.urihttps://pearl.plymouth.ac.uk/handle/10026.1/21959
dc.description.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.

dc.format.extent433-438
dc.format.mediumPrint-Electronic
dc.languageen
dc.publisherWiley
dc.subjectclinical trials
dc.subjectdisease modification
dc.subjectendpoints
dc.subjectoutcome measures
dc.subjectParkinson disease
dc.subjectPatient and Public Involvement and Engagament (PPIE)
dc.titleEmbedding Patient Input in Outcome Measures for Long‐Term Disease‐Modifying Parkinson Disease Trials
dc.typejournal-article
dc.typeArticle
plymouth.author-urlhttps://www.ncbi.nlm.nih.gov/pubmed/38140767
plymouth.issue2
plymouth.volume39
plymouth.publisher-urlhttp://dx.doi.org/10.1002/mds.29691
plymouth.publication-statusPublished
plymouth.journalMovement Disorders
dc.identifier.doi10.1002/mds.29691
plymouth.organisational-group|Plymouth
plymouth.organisational-group|Plymouth|Research Groups
plymouth.organisational-group|Plymouth|Faculty of Health
plymouth.organisational-group|Plymouth|Research Groups|Institute of Translational and Stratified Medicine (ITSMED)
plymouth.organisational-group|Plymouth|Research Groups|Institute of Translational and Stratified Medicine (ITSMED)|CCT&PS
plymouth.organisational-group|Plymouth|REF 2021 Researchers by UoA
plymouth.organisational-group|Plymouth|Users by role
plymouth.organisational-group|Plymouth|Users by role|Academics
plymouth.organisational-group|Plymouth|REF 2021 Researchers by UoA|UoA03 Allied Health Professions, Dentistry, Nursing and Pharmacy
plymouth.organisational-group|Plymouth|Faculty of Health|Peninsula Medical School
plymouth.organisational-group|Plymouth|Research Groups|FoH - Community and Primary Care
plymouth.organisational-group|Plymouth|Research Groups|FoH - Applied Parkinson's Research
plymouth.organisational-group|Plymouth|Research Groups|Plymouth Institute of Health and Care Research (PIHR)
plymouth.organisational-group|Plymouth|REF 2028 Researchers by UoA
plymouth.organisational-group|Plymouth|REF 2028 Researchers by UoA|UoA03 Allied Health Professions, Dentistry, Nursing and Pharmacy
dc.publisher.placeUnited States
dcterms.dateAccepted2023-11-30
dc.date.updated2024-01-24T12:21:48Z
dc.rights.embargodate2024-1-27
dc.identifier.eissn1531-8257
rioxxterms.versionofrecord10.1002/mds.29691


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