uu.seUppsala University Publications
Change search
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association
  • 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
A Novel Method for Analysing Frequent Observations from Questionnaires in Order to Model Patient-Reported Outcomes: Application to EXACT (R) Daily Diary Data from COPD Patients
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmaceutical Biosciences.ORCID iD: 0000-0003-2935-0090
GlaxoSmithKline, Clin Pharmacol Modelling & Simulat, London, England.
GlaxoSmithKline, Clin Pharmacol Modelling & Simulat, London, England.
GlaxoSmithKline, Clin Pharmacol Modelling & Simulat, London, England.
Show others and affiliations
2019 (English)In: AAPS Journal, ISSN 1550-7416, E-ISSN 1550-7416, Vol. 21, no 4, article id UNSP 60Article in journal (Refereed) Published
Abstract [en]

Chronic obstructive pulmonary disease (COPD) is a progressive lung disease with approximately 174 million cases worldwide. Electronic questionnaires are increasingly used for collecting patient-reported-outcome (PRO) data about disease symptoms. Our aim was to leverage PRO data, collected to record COPD disease symptoms, in a general modelling framework to enable interpretation of PRO observations in relation to disease progression and potential to predict exacerbations. The data were collected daily over a year, in a prospective, observational study. The e-questionnaire, the EXAcerbations of COPD Tool (EXACT (R)) included 14 items (i.e. questions) with 4 or 5 ordered categorical response options. An item response theory (IRT) model was used to relate the responses from each item to the underlying latent variable (which we refer to as disease severity), and on each item level, Markov models (MM) with 4 or 5 categories were applied to describe the dependence between consecutive observations. Minimal continuous time MMs were used and parameterised using ordinary differential equations. One hundred twenty-seven COPD patients were included (median age 67years, 54% male, 39% current smokers), providing approximately 40,000 observations per EXACT (R) item. The final model suggested that, with time, patients more often reported the same scores as the previous day, i.e. the scores were more stable. The modelled COPD disease severity change over time varied markedly between subjects, but was small in the typical individual. This is the first IRT model with Markovian properties; our analysis proved them necessary for predicting symptom-defined exacerbations.

Place, publisher, year, edition, pages
2019. Vol. 21, no 4, article id UNSP 60
Keywords [en]
EXACT questionnaire, IRT, Markov model, NONMEM, PRO
National Category
Pharmacology and Toxicology
Identifiers
URN: urn:nbn:se:uu:diva-383495DOI: 10.1208/s12248-019-0319-9ISI: 000466176800001PubMedID: 31028495OAI: oai:DiVA.org:uu-383495DiVA, id: diva2:1316449
Available from: 2019-05-17 Created: 2019-05-17 Last updated: 2019-05-17Bibliographically approved

Open Access in DiVA

fulltext(1455 kB)24 downloads
File information
File name FULLTEXT01.pdfFile size 1455 kBChecksum SHA-512
7cdb2856f2912630feb4718fe582a37334913b655ece5eb5fb0359f01b17e9914863ee1904e6856444fe52ee93610dc5b651afeae1d6ad3ef27c7e9418c6181a
Type fulltextMimetype application/pdf

Other links

Publisher's full textPubMed

Authority records BETA

Germovsek, EvaKarlsson, Mats O.Plan, Elodie L.

Search in DiVA

By author/editor
Germovsek, EvaKarlsson, Mats O.Plan, Elodie L.
By organisation
Department of Pharmaceutical Biosciences
In the same journal
AAPS Journal
Pharmacology and Toxicology

Search outside of DiVA

GoogleGoogle Scholar
Total: 24 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

doi
pubmed
urn-nbn

Altmetric score

doi
pubmed
urn-nbn
Total: 64 hits
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association
  • 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