Zesiewicz Theresa, Vega Joshua, Gooch Clifton, Ghanekar Shaila, Huang Yangxin, Bezchlibnyk Yarema, Staffetti Joseph, Kingsbury Chase
Department of Neurology, USF Ataxia Research Center University of South Florida (USF) Tampa Florida USA.
Department of Neurology, James A. Haley Veteran's Hospital Tampa Florida USA.
Mov Disord Clin Pract. 2022 Jul 5;9(6):728-734. doi: 10.1002/mdc3.13492. eCollection 2022 Aug.
Essential tremor (ET) is one of the most common tremor disorders in the world. Despite this, only one medication, propranolol, is approved by the Food and Drug Administration to treat it.
We analyzed controlled clinical trials in ET, spanning the last 50 years, to identify potential shortcomings in the therapeutic clinical pipeline.
Outcomes reviewed included demographics (specifically gender and race), therapeutic modalities, funding information, location of research, and trends over time. Clinical trials published in English were identified in scientific databases (Pubmed, SCOPUS, Cochrane Central Register of Controlled Trials, ClinicalTrials.gov, and the World Health Organization International Clinical Trials Registry Platform from 1970 through December 2021. Included trials were prospective, either single- or double-blinded (including blinded video assessments for surgical trials), with change in limb, head, or voice tremor as the primary outcome measure.
One hundred and eighty-six controlled clinical trials were accepted for extraction, including 4207 patients. Of the 145 trials that included gender, males comprised 59% of the patient population. Only 6.4% of studies provided racial demographics; in these studies, 70.5% of patients were Caucasian. The most common therapeutic modality over the past 50 years was "pharmaceutical" (56%), and the most common pharmaceutical studied was propranolol (32%). 41% of clinical trials reported no specific funding.
Future efforts should focus on increasing funding for clinical trial research in ET worldwide, and trials should be designed to be more inclusive of disadvantaged minorities.
特发性震颤(ET)是世界上最常见的震颤疾病之一。尽管如此,美国食品药品监督管理局仅批准了一种药物——普萘洛尔用于治疗该疾病。
我们分析了过去50年中关于ET的对照临床试验,以确定治疗临床流程中潜在的不足之处。
审查的结果包括人口统计学信息(特别是性别和种族)、治疗方式、资金信息、研究地点以及随时间的趋势。通过科学数据库(1970年至2021年12月期间的PubMed、SCOPUS、Cochrane对照试验中央注册库、ClinicalTrials.gov和世界卫生组织国际临床试验注册平台)检索以英文发表的临床试验。纳入的试验为前瞻性试验,采用单盲或双盲设计(包括手术试验的盲法视频评估),以肢体、头部或声音震颤的变化作为主要结局指标。
186项对照临床试验被纳入提取分析,涉及4207名患者。在145项纳入性别的试验中,男性占患者总数的59%。只有6.4%的研究提供了种族人口统计学信息;在这些研究中,70.5%的患者为白种人。过去50年中最常见的治疗方式是“药物治疗”(56%),研究最多的药物是普萘洛尔(32%)。41%的临床试验未报告具体资金来源。
未来的工作应集中于增加全球范围内ET临床试验研究的资金投入,并且试验设计应更具包容性,纳入弱势群体。