An MDS Evidence-Based Review on Treatments for Huntington's Disease.

Melissa J Nirenberg, Cristina Sampaio, João Costa, Wim Vandenberghe, Francis O Walker, Sarah J Tabrizi, Erin Furr-Stimming, Jaroslaw Slawek, Klaus Seppi, Jan Roth, Anne Rosser, Joaquim J Ferreira, Jaime Kulisevsky, G Bernard Landwehrmeyer, Daniel O Claassen, Francisco Cardoso, Jean-Marc Burgunder, Anna Rita Bentivoglio, Anne-Catherine Bachoud-Levi, Tiago A Mestre, Gonçalo S Duarte, Filipe B Rodrigues

Journal: Movement disorders : official journal of the Movement Disorder Society 2022;37(1):25-35

PMID: 34842303

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Abstract

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

Address: Laboratory of Clinical Pharmacology and Therapeutics, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.; Instituto de Medicina Molecular João Lobo Antunes, Lisbon, Portugal.; CNS - Campus Neurológico, Torres Vedras, Portugal.; UCL Huntington's Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom.; Centro de Estudos de Medicina Baseada na Evidência, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.; Centro Hospitalar Universitário Lisboa Norte, Lisbon, Portugal.; Parkinson disease and Movement Disorders Centre, Division of Neurology, Department of Medicine, The Ottawa Hospital Research Institute, The University of Ottawa Brain and Mind Research Institute, Ottawa, Ontario, Canada.; National Centre of Reference for Huntington's Disease, Neurology Department, Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, Créteil, France.; Neuropsychologie Interventionelle Lab, INSERM U955 E01B, PSL University, Paris, France.; Université Paris Est Créteil, Créteil, France.; Istituto di Neurologia, Università Cattolica del Sacro Cuore, Rome, Italy.; Movement Disorder Unit, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.; Swiss Huntington Center, Neurozentrum Siloah AG, Muri bei Bern, Switzerland.; Department of Neurology, University of Bern, Bern, Switzerland.; Movement Disorders Unit, Neurology Service, Internal Medicine Department of the Federal University of Minas Gerais, Belo Horizonte, Brazil.; Department of Neurology, Division of Behavioral and Cognitive Neurology, Vanderbilt University Medical Center, Nashville, Tennessee, USA.; Department of Neurology, University of Ulm, Ulm, Germany.; Movement Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.; Institut d'Investigacions Biomèdiques Sant Pau (IIB-Sant Pau), Universitat Autònoma de Barcelona (UAB), Barcelona, Spain.; Centro de Investigación en Red Enfermedades Neurodegenerativas (CIBERNED), Madrid, Spain.; Department of Neurology, Icahn School of Medicine at Mount Sinai, New York, New York, USA.; Neuroscience and Mental Health Research Institute (Brain Research And Intracranial Neurotherapeutics Unit), Cardiff, United Kingdom.; Department of Neurology and Center of Clinical Neuroscience, 1st Faculty of Medicine, Charles University and General University Hospital in Prague, Prague, Czech Republic.; Department of Neurology, Medical University Innsbruck, Innsbruck, Austria.; Division of Psychiatric-Neurological Nursing, Faculty of Health Sciences, Medical University of Gdansk, Gdansk, Poland.; Neurology and Stroke Department, St. Adalbert Hospital, Gdansk, Poland.; Department of Neurology, University of Texas Health Science Center at Houston, McGovern Medical School, Houston, Texas, USA.; Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London, United Kingdom.; UK Dementia Research Institute, University College London, London, United Kingdom.; Division of Neuromuscular Disorders, Department of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.; Department of Neurology, University Hospitals Leuven, Leuven, Belgium.; Department of Neurosciences, KU Leuven, Leuven, Belgium.; CHDI Management/CHDI Foundation, Princeton, New Jersey, USA.

DOI: 10.1002/mds.28855