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Item Response Theory as an Efficient Tool to Describe a Heterogeneous Clinical Rating Scale in De Novo Idiopathic Parkinson's Disease Patients
F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Clin Pharmacol Roche Innovat Ctr Basel, Grenzacherstr 124, CH-4070 Basel, Switzerland.;Univ Paris Diderot, IAME, UMR 1137, INSERM,Sorbonne Paris Cite, Paris, France.;Roche SAS, Inst Roche, 30,Cours Ile Seguin, F-92650 Boulogne, France..
F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Clin Pharmacol Roche Innovat Ctr Basel, Grenzacherstr 124, CH-4070 Basel, Switzerland.;Roche SAS, Inst Roche, 30,Cours Ile Seguin, F-92650 Boulogne, France..
F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Clin Pharmacol Roche Innovat Ctr Basel, Grenzacherstr 124, CH-4070 Basel, Switzerland..
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmaceutical Biosciences.ORCID iD: 0000-0002-3712-0255
2017 (English)In: Pharmaceutical research, ISSN 0724-8741, E-ISSN 1573-904X, Vol. 34, no 10, 2109-2118 p.Article in journal (Refereed) Published
Abstract [en]

This manuscript aims to precisely describe the natural disease progression of Parkinson's disease (PD) patients and evaluate approaches to increase the drug effect detection power. An item response theory (IRT) longitudinal model was built to describe the natural disease progression of 423 de novo PD patients followed during 48 months while taking into account the heterogeneous nature of the MDS-UPDRS. Clinical trial simulations were then used to compare drug effect detection power from IRT and sum of item scores based analysis under different analysis endpoints and drug effects. The IRT longitudinal model accurately describes the evolution of patients with and without PD medications while estimating different progression rates for the subscales. When comparing analysis methods, the IRT-based one consistently provided the highest power. IRT is a powerful tool which enables to capture the heterogeneous nature of the MDS-UPDRS.

Place, publisher, year, edition, pages
SPRINGER/PLENUM PUBLISHERS , 2017. Vol. 34, no 10, 2109-2118 p.
Keyword [en]
drug effect, item response theory, MDS-UPDRS, Parkinson's disease, pharmacometrics
National Category
Pharmaceutical Sciences
Identifiers
URN: urn:nbn:se:uu:diva-334753DOI: 10.1007/s11095-017-2216-1ISI: 000409050300011OAI: oai:DiVA.org:uu-334753DiVA: diva2:1160594
Available from: 2017-11-27 Created: 2017-11-27 Last updated: 2017-11-27Bibliographically approved

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Ueckert, Sebastian

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