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. 2020 Aug 21;15(8):e0237498.
doi: 10.1371/journal.pone.0237498. eCollection 2020.

Interpretation of health-related quality of life outcomes in Parkinson's disease from the EARLYSTIM Study

Pablo Martinez-Martin  1 , Guenther Deuschl  2 , Lisa Tonder  3 , Alfons Schnitzler  4 , Jean-Luc Houeto  5 , Lars Timmermann  6 , Joern Rau  7 , Carmen Schade-Brittinger  7 , Valerie Stoker  3 , Marie Vidailhet  8 , Paul Krack  9 ; EARLYSTIM Study Group
Affiliations

Interpretation of health-related quality of life outcomes in Parkinson's disease from the EARLYSTIM Study

Pablo Martinez-Martin et al. PLoS One. .

Abstract

The EARLYSTIM Study compared deep brain stimulation (DBS) with best medical treatment (BMT) over 2-years, showing a between-group difference of 8.0 from baseline in favor of DBS in health-related quality of life (HRQoL), measured with the PDQ-39 SI (summary index). This study obtained complementary information about the importance of the change in HRQoL as measured by the PDQ-39, using anchor-based (Patient Global Impression of Change, PGIC) and distribution-based techniques (magnitude of change, effect size, thresholds, distribution of benefit) applied to the EARLYSTIM study data. Anchor-based techniques showed a difference follow-up-baseline for patients who reported "minimal improvement" of -5.8 [-9.9, -1.6] (mean [95%CI]) in the DBS group vs -2.9 [-9.0, 3.1] in the BMT group. As the vast majority (80.8%) of DBS patients reported "much or very much improvement", this difference was explored for the latter group and amounted to -8.7 for the DBS group and -6.5 in the BMT group. Distribution-based techniques that analyzed the relative change and treatment effect size showed a moderate benefit of the DBS on the HRQoL, whereas a slight worsening was observed in the BMT group. The change in the DBS group (-7.8) was higher than the MIC (Minimally Important Change) estimated value (-5.8 by the anchor; -6.3 by triangulation of thresholds), but not in the BMT (0.2 vs. -3.0 to -5.4, respectively). Almost 90% of the patients in the DBS group declared some improvement (58.3% and 56.7% beyond the estimated MIC), which was significantly different from the BMT group whose proportions were 32.0% and 30.3%, respectively. The number needed to treat to improve ≥1 MIC by DBS vs BMT was 3.8. Change in depression, disability and pain influenced the improvement of the DBS group. DBS improved HRQoL in a high proportion of patients to a significant and moderate degree, at 2 years follow-up.

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Conflict of interest statement

The following authors have declared no competing interests: J Rau, C Schade- Brittinger, M Vidailhet These authors have declared the following: P. Martinez-Martin has received honoraria from National School of Public Health (ISCIII), Britannia, and Editorial Viguera for lecturing in courses; International Parkinson and Movement Disorder Society (MDS) for management of the Program on Rating Scales; Bial, and Zambon for advice in clinical-epidemiological studies. Financial support by MDS for attending the MDS International Congress 2019. Grant for Research: International Parkinson and Movement Disorder Society, for development and validation of the MDS-NMS. G. Deuschl has received lecture fees from Boston Scientific and has been serving as a consultant for Boston Scientific, Cavion, Functional Neuromodulation. He received royalties from Thieme publishers. He is a government employee and receives through his institution funding for his research from the German Research Council, the German Ministry of Education and Research and Medtronic. L. Tonder is employed by Medtronic Inc. A. Schnitzler AS received financial support from University of Kiel, during the study; has served as a consultant for Medtronic, Boston Scientific, St Jude Medical, and Grünenthal; and has received lecture fees from Abbvie, Boston Scientific, St Jude Medical, Medtronic, UCB, MEDA Pharma, Teva Pharma, and GlaxoSmithKline. J-L Houeto has received research grant from Agence National de la Recherche, Association France Parkinson, and AbbVie and fees for lectures and consultancies from Medtronic, Zambon, AbbVie, and Lundbeck. L. Timmermann has received in the past year payments as a consultant for Boston Scientific, and honoraria as a speaker on symposia sponsored by UCB, Desitin, Boston Scientific, Abbott. The institution of L.T., not L.T. personally received funding by the German Research Foundation, the German Ministry of Education and Research and the Deutsche Parkinson Vereinigung V Stoker is employed by Medtronic, Inc P. Krack reports grants from Swiss National Science Foundation, Roger de Spoelberch Foundation, Bertarelli Foundation, Michael J Fox Foundation grants and personal fees from Boston Scientific, grants from Annemarie Opprecht Foundation Parkinson Schweiz, and Michael J Fox Foundation outside the submitted work. This competing interest statement does not alter our adherence to PLOS ONE policies on sharing data and materials.

Figures

Fig 1
Fig 1. Cumulative distribution curves of PDQ-39 change from baseline to 24 months (1-cumulative %).
See this image and copyright information in PMC

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