Deep brain stimulation in Lesch-Nyhan disease: outcomes from the patient's perspective.

Details

Serval ID
serval:BIB_0862198EDB01
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Deep brain stimulation in Lesch-Nyhan disease: outcomes from the patient's perspective.
Journal
Developmental medicine and child neurology
Author(s)
Visser J.E., Cotton A.C., Schretlen D.J., Bloch J., Tedroff K., Schechtmann G., Radu Djurfeldt D., Gonzalez V., Cif L., Jinnah H.A.
ISSN
1469-8749 (Electronic)
ISSN-L
0012-1622
Publication state
Published
Issued date
08/2021
Peer-reviewed
Oui
Volume
63
Number
8
Pages
963-968
Language
english
Notes
Publication types: Journal Article
Publication Status: ppublish
Abstract
To provide insight into outcome and long-term safety and efficacy of deep brain stimulation (DBS), from the perspective of individuals with Lesch-Nyhan disease (LND) and their families.
We used patient-centered outcome measures to assess long-term outcomes of DBS for 14 individuals (mean [SD] age 10y 10mo [5y 6mo], range 5-23y, all males) with LND, after an average duration of 5y 6mo (range 11mo-10y 5mo) after surgery. We compared these results with a comprehensive review of previously published cases.
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.
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. What this paper adds Individuals with Lesch-Nyhan disease and their families report variable efficacy of deep brain stimulation. Long-term outcomes are associated with a high adverse event rate.
Pubmed
Web of science
Open Access
Yes
Create date
15/03/2021 14:44
Last modification date
27/07/2021 6:36
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