An MDS Evidence-Based Review on Treatments for Huntington's Disease
Por:
Ferreira, JJ, Rodrigues, FB, Duarte, GS, Mestre, TA, Bachoud-Levi, AC, Bentivoglio, AR, Burgunder, JM, Cardoso, F, Claassen, DO, Landwehrmeyer, GB, Kulisevsky, J, Nirenberg, MJ, Rosser, A, Roth, J, Seppi, K, Slawek, J, Furr-Stimming, E, Tabrizi, SJ, Walker, FO, Vandenberghe, W, Costa, J, Sampaio, C
Publicada:
1 ene 2022
Ahead of Print:
1 nov 2021
Resumen:
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. (c) 2021 International Parkinson and Movement Disorder Society
Filiaciones:
Ferreira, JJ:
Univ Lisbon, Fac Med, Lab Clin Pharmacol & Therapeut, Lisbon, Portugal
Inst Med Mol Joao Lobo Antunes, Lisbon, Portugal
CNS, Campus Neurol, Torres Vedras, Portugal
Rodrigues, FB:
Inst Med Mol Joao Lobo Antunes, Lisbon, Portugal
CNS, Campus Neurol, Torres Vedras, Portugal
UCL, UCL Queen Sq Inst Neurol, UCL Huntingtons Dis Ctr, London, England
Duarte, GS:
Univ Lisbon, Fac Med, Lab Clin Pharmacol & Therapeut, Lisbon, Portugal
Inst Med Mol Joao Lobo Antunes, Lisbon, Portugal
Univ Lisbon, Fac Med, Ctr Estudos Med Baseada Evidencia, Lisbon, Portugal
Ctr Hosp Univ Lisboa Norte, Lisbon, Portugal
Mestre, TA:
Univ Ottawa, Ottawa Hosp, Parkinson Dis & Movement Disorders Ctr, Dept Med,Res Inst,Brain & Mind Res Inst,Div Neuro, Ottawa, ON, Canada
Bachoud-Levi, AC:
Henri Mondor Hosp, AP HP, Natl Ctr Reference Huntingtons Dis, Neurol Dept, Creteil, France
PSL Univ, Neuropsychol Intervent Lab, INSERM U955 E01B, Paris, France
Univ Paris Est Creteil, Creteil, France
Bentivoglio, AR:
Univ Cattolica Sacro Cuore, Ist Neurol, Rome, Italy
Fdn Policlin Univ A Gemelli IRCCS, Movement Disorder Unit, Rome, Italy
Burgunder, JM:
Neurozentrum Siloah AG, Swiss Huntington Ctr, Muri Bei Bern, Switzerland
Univ Bern, Dept Neurol, Bern, Switzerland
Cardoso, F:
Univ Fed Minas Gerais, Internal Med Dept, Neurol Serv, Movement Disorders Unit, Belo Horizonte, MG, Brazil
Claassen, DO:
Vanderbilt Univ, Med Ctr, Dept Neurol, Div Behav & Cognit Neurol, Nashville, TN USA
Landwehrmeyer, GB:
Univ Ulm, Dept Neurol, Ulm, Germany
Kulisevsky, J:
Hosp Santa Creu & Sant Pau, Neurol Dept, Movement Disorders Unit, Barcelona, Spain
Univ Autonoma Barcelona UAB, Inst Investigac Biomed St Pau IIB St Pau, Barcelona, Spain
Ctr Invest Red Enfermedades Neurodegenerat CIBERN, Madrid, Spain
Nirenberg, MJ:
Icahn Sch Med Mt Sinai, Dept Neurol, New York, NY 10029 USA
Rosser, A:
Neurosci & Mental Hlth Res Inst, Brain Res & Intracranial Neurotherapeut Unit, Cardiff, Wales
Roth, J:
Charles Univ Prague, Fac Med 1, Dept Neurol, Prague, Czech Republic
Charles Univ Prague, Fac Med 1, Ctr Clin Neurosci, Prague, Czech Republic
Gen Univ Hosp Prague, Prague, Czech Republic
Seppi, K:
Med Univ Innsbruck, Dept Neurol, Innsbruck, Austria
Slawek, J:
Med Univ Gdansk, Fac Hlth Sci, Div Psychiat Neurol Nursing, Gdansk, Poland
St Adalbert Hosp, Neurol & Stroke Dept, Gdansk, Poland
Furr-Stimming, E:
Univ Texas Hlth Sci Ctr Houston, McGovern Med Sch, Dept Neurol, Houston, TX 77030 USA
Tabrizi, SJ:
UCL, UCL Queen Sq Inst Neurol, UCL Huntingtons Dis Ctr, London, England
UCL, Queen Sq Inst Neurol, Dept Neurodegenerat Dis, London, England
UCL, UK Dementia Res Inst, London, England
Walker, FO:
Wake Forest Sch Med, Dept Neurol, Div Neuromuscular Disorders, Winston Salem, NC 27101 USA
Vandenberghe, W:
Univ Hosp Leuven, Dept Neurol, Leuven, Belgium
Katholieke Univ Leuven, Dept Neurosci, Leuven, Belgium
Costa, J:
Univ Lisbon, Fac Med, Lab Clin Pharmacol & Therapeut, Lisbon, Portugal
Inst Med Mol Joao Lobo Antunes, Lisbon, Portugal
Univ Lisbon, Fac Med, Ctr Estudos Med Baseada Evidencia, Lisbon, Portugal
Sampaio, C:
Inst Med Mol Joao Lobo Antunes, Lisbon, Portugal
CHDI Fdn, CHDI Management, Princeton, NJ USA
Open Access
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