Bachoud-Lévi Anne-Catherine, Ferreira Joaquim, Massart Renaud, Youssov Katia, Rosser Anne, Busse Monica, Craufurd David, Reilmann Ralf, De Michele Giuseppe, Rae Daniela, Squitieri Ferdinando, Seppi Klaus, Perrine Charles, Scherer-Gagou Clarisse, Audrey Olivier, Verny Christophe, Burgunder Jean-Marc
National Centre of Reference for Huntington's Disease, Henri Mondor Hospital, AP-HP, Creteil & NeurATRIS, Créteil, France.
Clinical Pharmacology Unit, Instituto de Medicina Molecular, Lisbon, Portugal.
Front Neurol. 2019 Jul 3;10:710. doi: 10.3389/fneur.2019.00710. eCollection 2019.
The European Huntington's Disease Network (EHDN) commissioned an international task force to provide global evidence-based recommendations for everyday clinical practice for treatment of Huntington's disease (HD). The objectives of such guidelines are to standardize pharmacological, surgical and non-pharmacological treatment regimen and improve care and quality of life of patients. A formalized consensus method, adapted from the French Health Authority recommendations was used. First, national committees (French and English Experts) reviewed all studies published between 1965 and 2015 included dealing with HD symptoms classified in motor, cognitive, psychiatric, and somatic categories. Quality grades were attributed to these studies based on levels of scientific evidence. Provisional recommendations were formulated based on the strength and the accumulation of scientific evidence available. When evidence was not available, recommendations were framed based on professional agreement. A European Steering committee supervised the writing of the final recommendations through a consensus process involving two rounds of online questionnaire completion with international multidisciplinary HD health professionals. Patients' associations were invited to review the guidelines including the HD symptoms. Two hundred and nineteen statements were retained in the final guidelines. We suggest to use this adapted method associating evidence base-medicine and expert consensus to other rare diseases.
欧洲亨廷顿舞蹈症网络（EHDN）委托一个国际特别工作组，为亨廷顿舞蹈症（HD）的日常临床治疗提供基于全球证据的建议。此类指南的目标是规范药物、手术及非药物治疗方案，并改善患者的护理和生活质量。采用了一种根据法国卫生当局建议改编的正式共识方法。首先，国家委员会（法国和英国专家）审查了1965年至2015年间发表的所有涉及HD症状的研究，这些症状分为运动、认知、精神和躯体类别。根据科学证据水平为这些研究赋予质量等级。基于现有科学证据的力度和积累制定临时建议。当缺乏证据时，根据专业共识制定建议。一个欧洲指导委员会通过一个共识过程监督最终建议的撰写，该过程包括两轮在线问卷填写，参与人员为国际多学科HD健康专业人员。邀请患者协会审查包括HD症状在内的指南。最终指南保留了219条声明。我们建议将这种结合循证医学和专家共识的改编方法应用于其他罕见疾病。
