EFFECTIVENESS OF MULTI-DRUG TREATMENTS IN TOURETTE'S

该K23提案的目的是准备候选人成为独立的研究者，能够设计，获得资金，并在儿童中进行有效的临床试验，同时发生神经和精神疾病。 候选人提出了一个独特的和良好的教育经验的整合组合：1）与精神病学，神经病学和成果研究方面的临床试验研究人员的指导; 2）在统计和研究设计方面严格的硕士培训计划，每月30小时的课堂时间超过18个月。 候选人建议实施在患有图雷特综合征的儿童和青少年的多种药物治疗的临床有效性试验中吸取的经验教训，图雷特综合征是一种共同发生的神经和精神症状的范式障碍。图雷特综合征是一种神经精神障碍，其特征在于超过一年的运动和发声抽搐，其在功能上或社会上令人不安。 儿童和青少年经常有多个共同发生的神经精神病诊断，并与从未一起测试过的药物组合治疗。目前的临床研究通常涉及可能具有有限临床相关性的单药治疗的疗效研究。该K奖的研究提案包括一项多巴胺激动剂培高利特的随机对照临床有效性研究，允许同时治疗共病疾病。 主要假设是培高利特治疗与显著的抽搐抑制相关，与同时治疗OCD或ADHD无关。 这项研究将有能力确定是否存在治疗的共病强迫症或多动症解释药物治疗的抽搐症状变化的显着差异。 补助金的另一个重点将是制定这种疾病的医疗保健利用和费用的措施，并确定它们与缺乏有效症状管理的关系。

The purpose of this K23 proposal is to prepare the candidate to become an independent investigator, able to design, acquire funding for, and perform valid clinical trials in children with co-occurring neurologic and psychiatric disorders. The candidate proposes a unique and well- integrated combination of educational experiences: 1) mentorship with established clinical trials researchers in psychiatry, neurology, and outcomes research; and 2) a rigorous master's training program in statistics and research design, with 30 hours of classroom time per month over 18 months. The candidate proposes to implement the lessons learned in a clinical effectiveness trial of multi-drug treatments in children and adolescents with Tourette's Syndrome, a paradigm disorder of co-occurring neurologic and psychiatric symptoms. Tourette's Syndrome is a neuropsychiatric disorder characterized by greater than one year of motor and vocal tics that are functionally or socially disturbing. Children and adolescents frequently have multiple co-occurring neuropsychiatric diagnoses and are treated with combinations of medications that have never been tested together. Current clinical research typically involves efficacy studies of monotherapy that may have limited clinical relevance. The research proposal of this K award includes a randomized controlled clinical effectiveness study of a dopamine agonist, pergolide, allowing for concurrent treatment of comorbid disorders. The primary hypothesis is that treatment with pergolide will be associated with significant tic suppression, independent of concurrent treatment for OCD or ADHD. The study will have the power to determine whether the presence of treated co-morbid OCD or ADHD accounts for significant variance in the change in tic symptoms on medication. An additional focus of the grant will be developing measures of health care utilization and costs in this disorder and determining their relationship to the presence of absence of effective symptom management.