EFFECTIVENESS OF MULTI-DRUG TREATMENTS IN TOURETTE'S

多种药物治疗抽动秽语症的疗效

• 批准号: 6660749
• 负责人: DONALD L GILBERT
• 金额: $ 12.5万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-08-15 至 2006-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6660749
• 关键词: Tourette's syndrome; abnormal involuntary movement; adolescence (12-20); attention deficit disorder; behavioral /social science research tag; blood tests; clinical trials; combination chemotherapy; comorbidity; health care cost /financing; health care service evaluation; health care service utilization; human subject; human therapy evaluation; mental disorder chemotherapy; middle childhood (6-11); neuropharmacologic agent; neuropsychology; obsessive compulsive disorder; outcomes research; patient care management; patient oriented research; pediatrics; pergolide; statistics /biometry; urinalysis

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.

该 K23 提案的目的是让候选人做好成为独立研究者的准备，能够针对同时患有神经系统和精神疾病的儿童设计、获得资金并进行有效的临床试验。 候选人提出了一种独特且整合良好的教育经历组合：1）对精神病学、神经病学和结果研究领域成熟的临床试验研究人员进行指导； 2) 严格的统计学和研究设计硕士培训计划，每月 30 小时的课堂时间，持续 18 个月。 候选人提议将在患有抽动秽语综合症（一种同时出现神经系统和精神症状的典型疾病）的儿童和青少年中进行多种药物治疗的临床有效性试验中吸取的经验教训。抽动秽语综合症是一种神经精神疾病，其特征是一年以上的运动和发声抽搐，造成功能或社交障碍。 儿童和青少年经常同时患有多种神经精神疾病，并接受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单一疗法的疗效研究，其临床相关性可能有限。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，允许同时治疗合并症。 主要假设是培高利特治疗与显着的抽动抑制相关，独立于 OCD 或 ADHD 的同时治疗。 该研究将有能力确定治疗后的共病强迫症或注意力缺陷多动症的存在是否是药物治疗抽动症状变化的显着差异的原因。 拨款的另一个重点将是制定这种疾病的医疗保健利用率和成本的衡量标准，并确定它们与缺乏有效症状管理的关系。