{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T01:14:36Z","timestamp":1792804476231,"version":"4.5.0"},"reference-count":37,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2021,3,10]],"date-time":"2021-03-10T00:00:00Z","timestamp":1615334400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2021,8]]},"abstract":"<jats:sec><jats:title>AIM<\/jats:title><jats:p>To provide insight into outcome and long\u2010term safety and efficacy of deep brain stimulation (DBS), from the perspective of individuals with Lesch\u2013Nyhan disease (LND) and their families.<\/jats:p><\/jats:sec><jats:sec><jats:title>METHOD<\/jats:title><jats:p>We used patient\u2010centered outcome measures to assess long\u2010term outcomes of DBS for 14 individuals (mean [SD] age 10y 10mo [5y 6mo], range 5\u201323y, all males) with LND, after an average duration of 5y 6mo (range 11mo\u201310y 5mo) after surgery. We compared these results with a comprehensive review of previously published cases.<\/jats:p><\/jats:sec><jats:sec><jats:title>RESULTS<\/jats:title><jats:p>Patients and their families reported that DBS of the globus pallidus can be effective both for motor and behavioral disturbances in LND. However, outcome measures were often not significantly changed owing to substantial variability among individuals, and were overall less positive than in previous reports based on clinician assessments. In addition, there was an unexpectedly high rate of adverse events, tempering overall enthusiasm for the procedure.<\/jats:p><\/jats:sec><jats:sec><jats:title>INTERPRETATION<\/jats:title><jats:p>Although DBS might be an effective treatment for LND, more research is needed to understand the reasons for response variability and the unusually high rates of adverse events before DBS can be recommended for these patients.<\/jats:p><\/jats:sec><jats:sec><jats:label\/><jats:p>\n<jats:boxed-text content-type=\"box\" position=\"anchor\"><jats:caption><jats:title>What this paper adds<\/jats:title><\/jats:caption><jats:p>\n<jats:list list-type=\"bullet\">\n\n<jats:list-item><jats:p>Individuals with Lesch\u2013Nyhan disease and their families report variable efficacy of deep brain stimulation.<\/jats:p><\/jats:list-item>\n\n<jats:list-item><jats:p>Long\u2010term outcomes are associated with a high adverse event rate.<\/jats:p><\/jats:list-item>\n<\/jats:list>\n<\/jats:p><\/jats:boxed-text>\n<\/jats:p><\/jats:sec>","DOI":"10.1111\/dmcn.14852","type":"journal-article","created":{"date-parts":[[2021,3,10]],"date-time":"2021-03-10T15:21:37Z","timestamp":1615389697000},"page":"963-968","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Deep brain stimulation in Lesch\u2013Nyhan disease: outcomes from the patient\u2019s perspective"],"prefix":"10.1111","volume":"63","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9896-3416","authenticated-orcid":false,"given":"Jasper E","family":"Visser","sequence":"first","affiliation":[{"name":"Department of Neurology Donders Institute for Brain, Cognition and Behavior, Radboud University Medical Center Nijmegen"},{"name":"Department of Neurology Amphia Hospital Breda the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adam 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