Change search
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf
Modeling a Composite Score in Parkinson's Disease Using Item Response Theory
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmaceutical Biosciences.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmaceutical Biosciences. Uppsala Univ, Dept Pharmaceut Biosci, Box 591, S-75124 Uppsala, Sweden..
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmaceutical Biosciences.
2017 (English)In: AAPS Journal, ISSN 1550-7416, E-ISSN 1550-7416, Vol. 19, no 3, 837-845 p.Article in journal (Refereed) Published
Abstract [en]

In the current work, we present the methodology for development of an Item Response Theory model within a non-linear mixed effects framework to characterize the longitudinal changes of the Movement Disorder Society (sponsored revision) of Unified Parkinson's Disease Rating Scale (MDS-UPDRS) endpoint in Parkinson's disease (PD). The data were obtained from Parkinson's Progression Markers Initiative database and included 163,070 observations up to 48 months from 430 subjects belonging to De Novo PD cohort. The probability of obtaining a score, reported for each of the items in the questionnaire, was modeled as a function of the subject's disability. Initially, a single latent variable model was explored to characterize the disease progression over time. However, based on the understanding of the questionnaire set-up and the results of a residuals-based diagnostic tool, a three latent variable model with a mixture implementation was able to adequately describe longitudinal changes not only at the total score level but also at each individual item level. The linear progression rates obtained for the patient-reported items and the non-sided items were similar, each of which roughly take about 50 months for a typical subject to progress linearly from the baseline by one standard deviation. However for the sided items, it was found that the better side deteriorates quicker than the disabled side. This study presents a framework for analyzing MDS-UPDRS data, which can be adapted to more traditional UPDRS data collected in PD clinical trials and result in more efficient designs and analyses of such studies.

Place, publisher, year, edition, pages
SPRINGER , 2017. Vol. 19, no 3, 837-845 p.
Keyword [en]
Parkinson's disease, Item Response Theory, Movement Disorder Society (sponsored revision) Unified Parkinson's Disease Rating Scale, disease progression
National Category
Pharmaceutical Sciences
Identifiers
URN: urn:nbn:se:uu:diva-323036DOI: 10.1208/s12248-017-0058-8ISI: 000400565300024PubMedID: 28247193OAI: oai:DiVA.org:uu-323036DiVA: diva2:1107260
Available from: 2017-06-09 Created: 2017-06-09 Last updated: 2017-06-09Bibliographically approved

Open Access in DiVA

fulltext(1629 kB)51 downloads
File information
File name FULLTEXT01.pdfFile size 1629 kBChecksum SHA-512
3495c77c4012bad66d9807c55a92f6dd17c63267d1260c5b00f06d4946dc1fc2b64905585f7d501dade81c33c002ba66aa7dd53649ab85ffab5816c997105e24
Type fulltextMimetype application/pdf

Other links

Publisher's full textPubMed

Search in DiVA

By author/editor
Karlsson, Mats O.Plan, Elodie L.
By organisation
Department of Pharmaceutical Biosciences
In the same journal
AAPS Journal
Pharmaceutical Sciences

Search outside of DiVA

GoogleGoogle Scholar
Total: 51 downloads
The number of downloads is the sum of all downloads of full texts. It may include eg previous versions that are now no longer available

Altmetric score

Total: 212 hits
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf