Abstract
BackgroundA 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.MethodsThis 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.ResultsThe change from baseline in predicted mean utilities was statistically significant at all visits up to 24 months for the DBS group (p < 0.001) with both algorithms, and statistically significant at 12 months only (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 < 0.001). Based on the Hoehn and Yahr scale, predicted utilities deteriorated with increasing disease severity.ConclusionsAmong 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.
Highlights
A utility value is a health-related quality of life metric (HRQoL) metric used in a cost-effectiveness analysis
In patients with advanced Parkinson’s disease (PD) implanted with deep brain stimulation (DBS), two studies have reported a positive impact of DBS on utilities, as measured using the Euroqol 5 dimensions (EQ-5D)-3L [7, 8]
Dimension i corresponds to getting a response to an EQ5D-3L question (i.e. Mobility 2 indicates the response “some problems” in the Mobility dimension). β is the vector of the regression coefficients. x is the matrix of the PDQ-39 scores of the subscales
Summary
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. Recognition of the multidimensional impact of Parkinson’s disease (PD) on patients’ health-related quality of life (HRQoL) led to the development of the PD-specific 39 item Questionnaire (PDQ-39) [1], the most commonly used HRQoL instrument in clinical studies in PD [2], with superior clinimetric and psychometric properties compared with other PD-specific instruments [3]. Utilities represent the strength of an individual’s preference for specific health-related outcomes (“preference weights”) and are used for calculating quality–adjusted life years, a combination of survival and utilities generated by healthcare interventions, the metric used in cost-utility analyses. To our knowledge, utilities for patients with PD and early motor complications have, far, not been reported
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