Neurostimulation for Advanced Parkinson Disease and Quality of Life at 5 Years

Abstract

ImportanceDeep brain stimulation of the subthalamic nucleus (STN-DBS) improves quality of life (QOL) in patients with advanced Parkinson disease (PD). However, controlled studies with more than 3 years of follow-up are lacking.ObjectiveTo investigate the long-term effects of STN-DBS on QOL compared with standard-of-care medication (MED).Design, Setting, and ParticipantsIn this prospective, observational, quasi-experimental, longitudinal nonrandomized controlled trial, 183 patients were screened for eligibility and 167 were enrolled from March 1, 2011, to May 31, 2017, at 3 European university centers. Propensity score matching for demographic and clinical characteristics was applied to 108 patients with PD (62 in the STN-DBS group and 46 in the MED group), resulting in a well-balanced, matched subcohort of 25 patients per group. Data analysis was performed from September 2022 to January 2023.ExposureTreatment for PD of STN-DBS or MED.Main Outcomes and MeasuresAssessments included Parkinson’s Disease Questionnaire 8 (PDQ-8), Unified PD Rating Scale–motor examination, Scales for Outcomes in PD–activities of daily living (ADL) and motor complications, and levodopa-equivalent daily dose. Within-group longitudinal outcome changes, between-group differences, and correlations of change scores were analyzed.ResultsThe study population in the analysis included 108 patients (mean [SD] age, 63.7 [8.3] years; 66 [61.1%] male). At 5-year follow-up, PDQ-8 and ADL worsened only in the MED group (PDQ-8 change, −10.9; 95% CI, −19.0 to −2.7; P = .01; ADL change: −2.0; 95% CI, −3.1 to −0.8; P = .002), whereas both outcomes remained stable in the STN-DBS group (PDQ-8 change, −4.3; 95% CI, −13.2 to 4.7; P = .34; ADL change, −0.8; 95% CI, −2.5 to 1.0; P = .38). Changes in PDQ-8 and ADL correlated moderately (rs = .40, P = .008). Furthermore, STN-DBS outcomes were favorable for motor complications (median difference in change scores between STN-DBS and MED, −2.0; 95% CI, −4.0 to −1.0; P = .003), mobility (−1.0; 95% CI, −2.0 to 0; P = .03), and levodopa-equivalent daily dose reduction (−821.4; 95% CI, −1111.9 to −530.8; P < .001).Conclusions and RelevanceThis study provides evidence of differences in QOL outcomes at 5-year follow-up between STN-DBS (stable) and MED (worsened), mainly driven by the favorable effect of STN-DBS on mobility (class IIb evidence). The association between changes in QOL and ADL, but not motor impairment or complications, highlights the relative importance of ADL outcomes for long-term DBS assessments.Trial RegistrationGerman ClinicalTrials Registry: DRKS00006735