Neurostimulation for Advanced Parkinson Disease and Quality of Life at 5 Years
Authors: Stefanie T. Jost, Salima Aloui, Julian Evans, Keyoumars Ashkan, Anna Sauerbier, Alexandra Rizos, Jan Niklas Petry-Schmelzer, Alexandra Gronostay, Gereon R. Fink, Veerle Visser-Vandewalle, Angelo Antonini, Monty Silverdale, Lars Timmermann, Pablo Martinez-Martin, K. Ray Chaudhuri, Haidar S. Dafsari, Pablo Martinez-Martin, Carmen Rodriguez-Blazquez , Ray Chaudhuri, Alexandra Rizos, Anna Sauerbier, Miriam Parry, Dhaval Trivedi, Davide Martino, Per Odin, Angelo Antonini, Fabrizio Stocchi, Jacobus J van Hilten, Teus van Laar, Cristian Falup‐Pecurariu, Espen Dietrichs, Kelly E. Lyons, Dan Weintraub, Monica Kurtis, Marcos Serrano-Dueñas, Vanderci Borges, Madhuri Behari, Kalyan Bhattacharya, Hrishikesh Kumar, Bhim Singhal, Roongroj Bhidayasiri, Lim Shen-Yang, Marisol Gallardo, Kazuo Abe, Sevasti Bostantjopoulou, Jose Martin Rabey, Federico Micheli, Mayela Rodriguez‐Violante, Giulio Riboldazzi, Maria Jose Catalan, Cathy Ellis, Lorna Bean, Belinda Kessel, Paul Worth, Gemma Shearing, Rani Sophia, Jagdish Sharma, Monty Silverdale, Suvankar Pal, Keyoumars Ashkan, Charles Adler, Paolo Barone, David J. Brooks, Richard Brown, Marc Cantillon, Camille Carroll, Miguel Coelho, Haidar S. Dafsari, Tove Henriksen, Michele Hu, Peter Jenner, Milica Kramberger, Padma Kumar, Mónica Kurtis, Simon Lewis, Irene Litvan, Kelly Lyons, Mario Masellis, Hideki Mochizuki, James F. Morley, Melissa Nirenberg, Javier Pagonabarraga, Jalesh Panicker, Nicola Pavese, Eero Pekkonen, Ron Postuma, Raymond Rosales, Anthony Schapira, Tanya Simuni, Indu Subramanian, Michele Tagliati, Lars Timmermann, Michele Tinazzi, Jon Toledo, Yoshio Tsuboi, Richard Walker
Journal: JAMA Network Open (2024)

Importance Deep 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. Objective To investigate the long-term effects of STN-DBS on QOL compared with standard-of-care medication (MED). Design, Setting, and Participants In 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. Exposure Treatment for PD of STN-DBS or MED. Main Outcomes and Measures Assessments 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. Results The 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 ( r s = .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 &amp;amp;lt; .001). Conclusions and Relevance This 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 Registration German ClinicalTrials Registry: DRKS00006735