Item Response Theory as an Efficient Tool to Describe a Heterogeneous Clinical Rating Scale in De Novo Idiopathic Parkinson’s Disease Patients

Simon Buatois 1, 2, 3, * Sylvie Retout 1 Nicolas Frey 1 Sebastian Ueckert 4
* Auteur correspondant
1 pRED - Roche Pharma Research and Early Development [Basel]
2 IAME - Infection, Antimicrobiens, Modélisation, Evolution
3 Institut ROCHE [Boulogne-Billancourt]
4 Department of Pharmaceutical Biosciences [Uppsala, Sweden]
Abstract : PURPOSE: 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. METHODS: 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. RESULTS: 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. CONCLUSION: IRT is a powerful tool which enables to capture the heterogeneous nature of the MDS-UPDRS.
Keywords : MDS-UPDRS pharmacometrics item response theory drug effect Parkinson’s disease
Type de document :
Article dans une revue
Pharmaceutical Research, American Association of Pharmaceutical Scientists, 2017, 34 (10), pp.2109-2118. 〈10.1007/s11095-017-2216-1〉
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http://www.hal.inserm.fr/inserm-01563224
Contributeur : Thu Thuy Nguyen <>
Soumis le : lundi 17 juillet 2017 - 14:11:24
Dernière modification le : jeudi 11 octobre 2018 - 14:48:32
Document(s) archivé(s) le : samedi 27 janvier 2018 - 03:36:34

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Simon Buatois, Sylvie Retout, Nicolas Frey, Sebastian Ueckert. Item Response Theory as an Efficient Tool to Describe a Heterogeneous Clinical Rating Scale in De Novo Idiopathic Parkinson’s Disease Patients. Pharmaceutical Research, American Association of Pharmaceutical Scientists, 2017, 34 (10), pp.2109-2118. 〈10.1007/s11095-017-2216-1〉. 〈inserm-01563224〉

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