Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial

Autor: W. M. Michael Schüpbach, Gillian Barnett, Isabelle Durand-Zaleski, Mehdi Zahra, Silke Walleser Autiero, Michal Górecki
Přispěvatelé: Medtronic International Trading Sarl [Tolochenaz], Hôpital Hôtel-Dieu [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Service de santé publique [Mondor], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Health Technology Assessment Consulting [Kraków], Gillian Barnett and Associates [Dunfanaghy], Institut du Cerveau et de la Moëlle Epinière = Brain and Spine Institute (ICM), Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Service de neurologie 1 [CHU Pitié-Salpétrière], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Pitié-Salpêtrière [AP-HP], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Bern University Hospital [Berne] (Inselspital), Universität Bern [Bern], Centre d'investigation clinique Neurosciences [CHU Pitié Salpêtrière] (CIC Neurosciences), CHU Pitié-Salpêtrière [AP-HP], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Institut du Cerveau = Paris Brain Institute (ICM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Service de Neurologie [CHU Pitié-Salpêtrière], IFR70-CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Universität Bern [Bern] (UNIBE), Gestionnaire, Hal Sorbonne Université, Centre d'investigation clinique pluridisciplinaire [CHU Pitié Salpêtrière] (CIC-P 1421), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP]
Jazyk: angličtina
Rok vydání: 2020
Předmět:
Male
Quality of life
medicine.medical_specialty
Parkinson's disease
Deep brain stimulation
medicine.medical_treatment
Cost-Benefit Analysis
lcsh:Computer applications to medicine. Medical informatics
03 medical and health sciences
0302 clinical medicine
Utility
EQ-5D
Surveys and Questionnaires
Post-hoc analysis
medicine
Humans
030212 general & internal medicine
[SDV.NEU] Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]
Multinomial logistic regression
[SDV.IB] Life Sciences [q-bio]/Bioengineering
business.industry
030503 health policy & services
Research
Public Health
Environmental and Occupational Health
Parkinson Disease
General Medicine
Middle Aged
Physical Functional Performance
medicine.disease
[SDV.SPEE] Life Sciences [q-bio]/Santé publique et épidémiologie
Physical therapy
Parkinson’s disease
lcsh:R858-859.7
Female
[SDV.NEU]Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]
[SDV.IB]Life Sciences [q-bio]/Bioengineering
[SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie
Analysis of variance
Metric (unit)
0305 other medical science
business
Algorithms
Zdroj: Health and Quality of Life Outcomes
Health and Quality of Life Outcomes, BioMed Central, 2020, 18 (1), pp.49. ⟨10.1186/s12955-020-01299-y⟩
Health and Quality of Life Outcomes, 2020, 18 (1), pp.49. ⟨10.1186/s12955-020-01299-y⟩
Health and Quality of Life Outcomes, Vol 18, Iss 1, Pp 1-8 (2020)
ISSN: 1477-7525
DOI: 10.1186/s12955-020-01299-y⟩
Popis: Background A utility value is a health-related quality of life metric (HRQoL) metric used in a cost-effectiveness analysis. While utilities as outcomes in the treatment of advanced Parkinson’s disease (PD) with deep brain stimulation (DBS) are available, they do not currently exist for PD with early motor complications. The objectives of this study were to predict utilities from observed disease-specific HRQoL data using two mapping algorithms, and investigate their performance in terms of longitudinal changes within and between treatment groups, and distribution by PD severity. Methods This is a post hoc analysis of data from the EARLYSTIM trial of DBS compared with best medical therapy (BMT) in PD patients with early motor complications We used two published algorithms comprising ordinal and multinomial regression models to map EQ-5D-3L utilities from observed PD-specific 39 item Questionnaire (PDQ-39) scores in EARLYSTIM. Utilities were calculated using the predicted functioning levels of EQ-5D-3L dimensions and the established EQ-5D-3L UK tariffs. Statistical analyses (analysis of variance, two-tailed Student’s t-test) were used to test the change from baseline within groups and difference in change from baseline between groups in utilities. Boxplots were developed to investigate the distribution of predicted utilities by PD severity, measured using the Hoehn and Yahr scale. Results The change from baseline in predicted mean utilities was statistically significant at all visits up to 24 months for the DBS group (p p = 0.04) for the BMT group with one algorithm. With both algorithms, the between-groups difference in change from baseline in predicted mean utilities favored DBS at all follow-up visits (p Conclusions Among PD patients with early motor complications, utilities predicted by both mapping algorithms using PDQ-39 data demonstrated a statistically and clinically meaningful improvement with DBS compared with BMT. It was not possible to conclude if one algorithm was more responsive than other. In the absence of utilities collected directly from patients, mapping is an acceptable option permitting economic evaluations to be undertaken.
Databáze: OpenAIRE
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