title: Guidelines for clinical pharmacological practices in Huntington's disease.
abstract: OBJECTIVE
  Evidence-based medicine is a difficult goal to achieve in rare diseases where randomized controlled trials are lacking. This report provides guidelines that capitalize on both the literature and expertise of the French National Huntington Disease Reference Centre to optimalize pharmacological therapeutic interventions for Huntington's disease (HD).
  
  
  METHODS
  HD experts conducted a systematic analysis of the literature from 1965 to 2013, using a scoring procedure established by the French National Authority for Health. These experts offered their views when evidence was missing to set up provisional guidelines for care in HD. These guidelines were then scored and amended through two subsequent online questionnaires (using SurveyMonkey®scoring), and one face-to-face meeting with an external multidisciplinary working group as a step towards validation.
  
  
  RESULTS
  Except for the beneficial effects of tetrabenazine in chorea, none of the published recommendations were grounded on established scientific evidence. Second-generation antipsychotics are nevertheless the first choice for patients with psychiatric manifestations (low level of evidence). All other guidelines are based on low-level evidence and little professional agreement.
  
  
  CONCLUSIONS
  Patients' care has greatly improved over the last few years despite the lack of high-level evidence standards. Guidelines are based on the expertise of trained specialists from the French National Plan for Rare Diseases. This strategy should now be extended internationally to promote future studies and to harmonize worldwide care of HD.
authors: Faculté de médecine, université Paris Est, 94010 Créteil, France; Inserm, U955, équipe 01, neuropsychologie interventionnelle, 94010 Créteil, France; École normale supérieure, département d'études cognitives, 75005 Paris, France; Neurologie cognitive, centre de référence maladie de Huntington, hôpital H.-Mondor-A.-Chenevier, AP-HP, 94010 Créteil, France.
authors: G
authors: G
authors: Désaméricq
authors: Faculté de médecine, université Paris Est, 94010 Créteil, France; Inserm, U955, équipe 01, neuropsychologie interventionnelle, 94010 Créteil, France; École normale supérieure, département d'études cognitives, 75005 Paris, France; Neurologie cognitive, centre de référence maladie de Huntington, hôpital H.-Mondor-A.-Chenevier, AP-HP, 94010 Créteil, France.
authors: K
authors: K
authors: Youssov
authors: Département de génétique, centre de référence maladie de Huntington, groupe hospitalier Pitié-Salpêtrière, AP-HP, 75013 Paris, France.
authors: P
authors: P
authors: Charles
authors: Faculté de médecine, université Paris Est, 94010 Créteil, France; Inserm, U955, équipe 01, neuropsychologie interventionnelle, 94010 Créteil, France; École normale supérieure, département d'études cognitives, 75005 Paris, France; Neurologie cognitive, centre de référence maladie de Huntington, hôpital H.-Mondor-A.-Chenevier, AP-HP, 94010 Créteil, France.
authors: N
authors: N
authors: Saleh
authors: Service de neurologie, Centre national de référence des maladies neurogénétiques, CHU d'Angers, 49933 Angers, France.
authors: A
authors: A
authors: Olivier
authors: Service de neurologie, Centre national de référence des maladies neurogénétiques, CHU d'Angers, 49933 Angers, France.
authors: C
authors: C
authors: Sherer-Gagou
authors: Service de neurologie, Centre national de référence des maladies neurogénétiques, CHU d'Angers, 49933 Angers, France; UMR Inserm 1083/CNRS 6214, université d'Angers, 49933 Angers, France.
authors: C
authors: C
authors: Verny
authors: Faculté de médecine, université Paris Est, 94010 Créteil, France; Inserm, U955, équipe 01, neuropsychologie interventionnelle, 94010 Créteil, France; École normale supérieure, département d'études cognitives, 75005 Paris, France; Neurologie cognitive, centre de référence maladie de Huntington, hôpital H.-Mondor-A.-Chenevier, AP-HP, 94010 Créteil, France. Electronic address: bachoud@gmail.com.
authors: A-C
authors: AC
authors: Bachoud-Lévi
mesh_terms: D014150:Antipsychotic Agents / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D019317:Evidence-Based Medicine; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy*; D017410:Practice Guidelines as Topic
chemical_list: D014150:Antipsychotic Agents
keywords: Drug trials; Evidence-based practice; Guidelines; Huntington's disease; Symptomatic treatment