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Expectations of benefit in a trial of a candidate disease-modifying treatment for Parkinson disease

dc.contributor.authorMestre, Tiago
dc.contributor.authorMacklin, Eric A.
dc.contributor.authorAscherio, Alberto
dc.contributor.authorFerreira, Joaquim J
dc.contributor.authorLang, Anthony E.
dc.contributor.authorSchwarzschild, Michael A.
dc.date.accessioned2021-05-12T17:08:52Z
dc.date.available2021-05-12T17:08:52Z
dc.date.issued2021-05-04
dc.description© 2021 International Parkinson and Movement Disorder Societypt_PT
dc.description.abstractBackground: Expectations of benefit have an important therapeutic impact. How well study participants understand the concept of slowing disease progression and how their expectations of benefit are shaped in related clinical trials is not well known. Objective: We aimed to assess expectancy and treatment arm preference of participants in a disease-modification trial in Parkinson disease (PD). Methods: Participant expectations and treatment preference were assessed before treatment randomization in the SURE-PD3 trial (NCT02642393). Results: We included 297 PD patients (0.71 ± 0.67 years after diagnosis). Pre-randomization, 90% of participants expressed a preference for inosine (active treatment) allocation (n = 266/297), and 53% (n = 158) expected to be "somewhat" or "a lot better" in their symptoms over 2 years of treatment with inosine. Conclusions: Participants of a disease-modification trial in PD had likely unrealistic expectations of benefit (ie, improvement in symptoms over years), which may affect clinical trial interpretation and calls for improved education in future disease-modification trials in PD. © 2021 International Parkinson and Movement Disorder Society.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationMov Disord. 2021 May 4pt_PT
dc.identifier.doi10.1002/mds.28630pt_PT
dc.identifier.eissn1531-8257
dc.identifier.issn0885-3185
dc.identifier.urihttp://hdl.handle.net/10451/47813
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherJohn Wiley & Sons, Inc.pt_PT
dc.relation.publisherversionhttps://movementdisorders.onlinelibrary.wiley.com/journal/15318257pt_PT
dc.subjectParkinson diseasept_PT
dc.subjectDisease modificationpt_PT
dc.subjectExpectationpt_PT
dc.subjectClinical trialpt_PT
dc.titleExpectations of benefit in a trial of a candidate disease-modifying treatment for Parkinson diseasept_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.titleMovement Disorderspt_PT
person.familyNameMestre
person.familyNameFerreira
person.givenNameTiago
person.givenNameJoaquim J
person.identifier1624753
person.identifier.ciencia-id7D15-5CD6-6159
person.identifier.orcid0000-0002-6973-7479
person.identifier.orcid0000-0003-3950-5113
person.identifier.scopus-author-id16242276700
person.identifier.scopus-author-id7403252466
rcaap.rightsrestrictedAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublicationca2842d0-4ea8-48c4-9e7a-3bc46801b9c8
relation.isAuthorOfPublication54d84fe7-2cbd-4ddc-bce2-0ff2bd7c4cc9
relation.isAuthorOfPublication.latestForDiscoveryca2842d0-4ea8-48c4-9e7a-3bc46801b9c8

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