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An MDS Evidence-Based Review on Treatments for Huntington's Disease
JJ. Ferreira, FB. Rodrigues, GS. Duarte, TA. Mestre, AC. Bachoud-Levi, AR. Bentivoglio, JM. Burgunder, F. Cardoso, DO. Claassen, GB. Landwehrmeyer, J. Kulisevsky, MJ. Nirenberg, A. Rosser, J. Roth, K. Seppi, J. Slawek, E. Furr-Stimming, SJ....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, práce podpořená grantem, přehledy
Grantová podpora
200181/Z/15/Z
Wellcome Trust - United Kingdom
MR/L010305/1
Medical Research Council - United Kingdom
RFPPB-16A-1298
Health and Care Research Wales - United Kingdom
PubMed
34842303
DOI
10.1002/mds.28855
Knihovny.cz E-zdroje
- MeSH
- apatie * MeSH
- chorea * MeSH
- Huntingtonova nemoc * farmakoterapie terapie MeSH
- lidé MeSH
- pohybové poruchy * farmakoterapie MeSH
- tetrabenazin terapeutické užití MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- přehledy MeSH
BACKGROUND: Huntington's disease (HD) is a rare neurodegenerative disorder with protean clinical manifestations. Its management is challenging, consisting mainly of off-label treatments. OBJECTIVES: The International Parkinson and Movement Disorder Society commissioned a task force to review and evaluate the evidence of available therapies for HD gene expansion carriers. METHODS: We followed the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach. Eligible randomized controlled trials were identified via an electronic search of the CENTRAL, MEDLINE, and EMBASE databases. All eligible trials that evaluated one or more of 33 predetermined clinical questions were included. Risk of bias was evaluated using the Cochrane Risk of Bias tool. A framework was adapted to allow for efficacy and safety conclusions to be drawn from the balance between the GRADE level of evidence and the importance of the benefit/harm of the intervention. RESULTS: Twenty-two eligible studies involving 17 interventions were included, providing data to address 8 clinical questions. These data supported a likely effect of deutetrabenazine on motor impairment, chorea, and dystonia and of tetrabenazine on chorea. The data did not support a disease-modifying effect for premanifest and manifest HD. There was no eligible evidence to support the use of specific treatments for depression, psychosis, irritability, apathy, or suicidality. Similarly, no evidence was eligible to support the use of physiotherapy, occupational therapy, exercise, dietary, or surgical treatments. CONCLUSIONS: Data for therapeutic interventions in HD are limited and support only the use of VMAT2 inhibitors for specific motor symptoms. © 2021 International Parkinson and Movement Disorder Society.
Centro de Investigación en Red Enfermedades Neurodegenerativas Madrid Spain
Centro Hospitalar Universitário Lisboa Norte Lisbon Portugal
CHDI Management CHDI Foundation Princeton New Jersey USA
CNS Campus Neurológico Torres Vedras Portugal
Department of Neurology Icahn School of Medicine at Mount Sinai New York New York USA
Department of Neurology Medical University Innsbruck Innsbruck Austria
Department of Neurology University Hospitals Leuven Leuven Belgium
Department of Neurology University of Bern Bern Switzerland
Department of Neurology University of Ulm Ulm Germany
Department of Neurosciences KU Leuven Leuven Belgium
Institut d'Investigacions Biomèdiques Sant Pau Barcelona Spain
Instituto de Medicina Molecular João Lobo Antunes Lisbon Portugal
Istituto di Neurologia Università Cattolica del Sacro Cuore Rome Italy
Movement Disorder Unit Fondazione Policlinico Universitario A Gemelli IRCCS Rome Italy
Movement Disorders Unit Neurology Department Hospital de la Santa Creu i Sant Pau Barcelona Spain
Neurology and Stroke Department St Adalbert Hospital Gdansk Poland
Neuropsychologie Interventionelle Lab INSERM U955 E01B PSL University Paris France
Neuroscience and Mental Health Research Institute Cardiff United Kingdom
Swiss Huntington Center Neurozentrum Siloah AG Muri bei Bern Switzerland
UK Dementia Research Institute University College London London United Kingdom
Citace poskytuje Crossref.org
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