期刊
EXPERT REVIEW OF NEUROTHERAPEUTICS
卷 22, 期 9, 页码 781-788出版社
TAYLOR & FRANCIS LTD
DOI: 10.1080/14737175.2022.2091988
关键词
Huntington's disease; fronto-striatal circuits; chorea; parkinsonism; dystonia; globus pallidus externus/internus; deep-brain stimulation; striatum; brain grafts; unified Huntington's disease rating scale (UHDRS); chorea; safety; efficacy; quality of life
Huntington's Disease is a progressive neurological disorder with limited response to established therapeutics. Pallidal deep brain stimulation and neurorestorative strategies have shown potential for the treatment of refractory Huntington's disease. However, clinical trials have produced inconsistent results and these therapies remain experimental and not ready for clinical use.
Introduction: Huntington's Disease as progressive neurological disorders associated with motor, behavioral, and cognitive impairment poses a therapeutic challenge in case of limited responsiveness to established therapeutics. Pallidal deep brain stimulation and neurorestorative strategies (brain grafts) scoping to modulate fronto-striatal circuits have gained increased recognition for the treatment of refractory Huntington's disease (HD). Areas covered: A review (2000-2022) was performed in PubMed, Embase, and Cochrane Library covering clinical trials conceptualized to determine the efficacy and safety of invasive, stereotactic-guided deep-brain stimulation and intracranial brain-graft injection targeting the globus pallidus and adjunct structures (striatum). Expert opinion: Stereotactic brain-grafting strategies were performed in few HD patients with inconsistent findings and mild-to-moderate clinical responsiveness with a recently published large, randomized-controlled trial (NCT 00190450) yielding negative results. We identified 19 in-human DBS trials (uncontrolled) targeting the globus pallidus internus/externus along with randomized-controlled trial pending report (NCT 02535884). We did not detect any significant changes in the UHDRS total score after restorative injections, while in contrast, the use of deep-brain stimulation resulted in a significant reduction of chorea. GPi-DBS should be considered in cases where selective chorea is present. However, both invasive therapies remain experimental and are not ready for the implementation in clinical use.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据