Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial
Abstract 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 w...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2020-03-01
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| Series: | Health and Quality of Life Outcomes |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s12955-020-01299-y |
| _version_ | 1811206098915426304 |
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| author | Mehdi Zahra Isabelle Durand-Zaleski Michal Górecki Silke Walleser Autiero Gillian Barnett W. M. Michael Schüpbach |
| author_facet | Mehdi Zahra Isabelle Durand-Zaleski Michal Górecki Silke Walleser Autiero Gillian Barnett W. M. Michael Schüpbach |
| author_sort | Mehdi Zahra |
| collection | DOAJ |
| description | Abstract 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 < 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. 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. |
| first_indexed | 2024-04-12T03:42:07Z |
| format | Article |
| id | doaj.art-79ea07493ff944feadd179a8b65280a4 |
| institution | Directory Open Access Journal |
| issn | 1477-7525 |
| language | English |
| last_indexed | 2024-04-12T03:42:07Z |
| publishDate | 2020-03-01 |
| publisher | BMC |
| record_format | Article |
| series | Health and Quality of Life Outcomes |
| spelling | doaj.art-79ea07493ff944feadd179a8b65280a42022-12-22T03:49:15ZengBMCHealth and Quality of Life Outcomes1477-75252020-03-011811810.1186/s12955-020-01299-yParkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trialMehdi Zahra0Isabelle Durand-Zaleski1Michal Górecki2Silke Walleser Autiero3Gillian Barnett4W. M. Michael Schüpbach5Medtronic International Trading SarlHôpital de l’Hotel-DieuHealth Technology Assessment ConsultingMedtronic International Trading SarlGillian Barnett and Associates LimitedAssistance Publique Hôpitaux de Paris, Institut National de Santé et en Recherche Médicale, Institut du Cerveau et de la Moelle Epinière, Centre d’Investigation Clinique 1422, Département de Neurologie, Hôpital Pitié-SalpêtrièreAbstract 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 < 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. 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.http://link.springer.com/article/10.1186/s12955-020-01299-yUtilityQuality of lifeParkinson’s diseaseDeep brain stimulation |
| spellingShingle | Mehdi Zahra Isabelle Durand-Zaleski Michal Górecki Silke Walleser Autiero Gillian Barnett W. M. Michael Schüpbach Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial Health and Quality of Life Outcomes Utility Quality of life Parkinson’s disease Deep brain stimulation |
| title | Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial |
| title_full | Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial |
| title_fullStr | Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial |
| title_full_unstemmed | Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial |
| title_short | Parkinson’s disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial |
| title_sort | parkinson s disease with early motor complications predicting eq 5d 3l utilities from pdq 39 data in the earlystim trial |
| topic | Utility Quality of life Parkinson’s disease Deep brain stimulation |
| url | http://link.springer.com/article/10.1186/s12955-020-01299-y |
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