Effects of octanoic acid for treatment of essential voice tremor

辛酸治疗原发性声音震颤的疗效

• 批准号: 8500511
• 负责人: Soren Yvette Lowell
• 金额: $ 14.82万
• 依托单位: SYRACUSE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-04-10 至 2016-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8500511
• 关键词: Acoustics; Address; Affect; Aftercare; Alcohols; American; Animals; Auditory; Blinded; Body Regions; Botulinum Toxins; Calcium Channel; Cerebellum; Characteristics; Clinical Treatment; Crossover Design; Data; Data Analyses; Data Collection; Demographic Aging; Disabled Persons; Disease; Double-Blind Method; Drug Targeting; Effectiveness; Elderly; Emotional; Essential Tremor; Ethanol; Evidence based treatment; Family; Fatty Acids; Food Additives; Frequencies; Friends; Goals; Growth; Head; Hoarseness; Human; Individual; Inferior; Injection of therapeutic agent; Intake; Intervention; Intoxication; Investigation; Involuntary Movements; Judgment; Larynx; Limb Tremors; Limb structure; Measures; Movement; Movement Disorders; Muscle; Neuraxis; Neurologic; Octanoic Acids; Octanols; Older Population; Oral; Outcome; Parents; Paresis; Participant; Pathway interactions; Pharmaceutical Preparations; Pharmacological Treatment; Phase; Physical Function; Placebo Control; Placebos; Prevalence; Quality of life; Randomized; Reducing Agents; Reporting; Research; Research Personnel; Severities; Side; Signal Transduction; Source; Speech; Speech Intelligibility; Structure; System; Time; Treatment Efficacy; Treatment outcome; Tremor; United States Food and Drug Administration; United States National Institutes of Health; Upper Extremity; Voice; Voice Disorders; Voice Quality; disability; evidence base; experience; handicapping condition; improved; innovation; muscular system; nervous system disorder; novel; psychosocial; public health relevance; relating to nervous system; social; treatment response

DESCRIPTION (provided by applicant): Essential tremor is among the most common movement disorders, affecting nearly 5% of people over the age of 65. Increased prevalence of this disorder is expected in the next 10 years due to the projected disproportionate growth of the older population. Essential tremor is characterized by rhythmic, involuntary movements that often affect the upper limbs but can also affect the head, trunk and voice-related structures. The origin of the tremor is thought to be within the cerebellum and related pathways of the central nervous system (CNS)1, 2. In essential voice tremor (EVT), repetitive movements of voice-related structures produce modulations of the speech signal. Perceptual voice effects include increased instability, hoarseness, and vocal effort3, 4, which negatively impact speech intelligibility and produce substantial psychosocial and physical burden3. Current treatment for EVT is inadequate, with variable effectiveness that may benefit a limited proportion of people4-6. Alcohol reduces tremor in many affected people, but its usefulness for treatment is limited by its intoxicating effects. Octanoic acid (OA) is a food additive that is related to alcohol, and produces a reduction in essential tremor of the limbs with minimal side effects7. Octanoic acid may directly affect the cerebellar pathways where tremor is thought to originate. The goal of the proposed project is to determine the effects of OA on the voice characteristics of people with EVT. This represents an innovative step toward advancing the evidence-based treatment of EVT by being the first study to address the voice-specific effects of OA. In a randomized, placebo controlled cross-over design, 22 participants will each receive the OA and placebo conditions, with both participants and experimenters blinded to condition during data collection and analysis. Outcomes will be determined by comparing changes in pre- to post-assessment measures of acoustic, perceptual and functional aspects of voice between the OA and placebo conditions. Specifically, Aims I and II will determine the effects of OA on the acoustic magnitude of tremor and perceived voice tremor severity. Aim III will determine the functional impact of OA on quality of life and ability to communicate with family or friends. It is hypothesized that OA, which can affect multiple speech-related muscles, will produce a greater reduction in acoustic, perceptual and functional manifestations of voice tremor than the placebo condition. Aim IV will determine the individual variability and characteristics of voice tremor, and how these relate to treatment response. The proposed project will significantly advance evidence-based decisions for the clinical treatment of EVT, providing a vital step toward reducing the debilitating effects f this disorder.

描述（由申请人提供）：基本震颤是最常见的运动障碍之一，影响了65岁以上的近5％的人。由于预计老年人群的不成比例增长，预计这种疾病的患病率会增加。基本震颤的特征是有节奏的非自愿运动，通常会影响上肢，但也会影响头部，后备箱和与声音相关的结构。震颤的起源被认为是在小脑和中枢神经系统（CNS）1，2的相关途径内。知觉语音效应包括增加的不稳定性，嘶哑和声音努力3，4，它们对语音清晰度产生负面影响并产生大量的社会心理和身体负担3。当前对EVT的治疗不足，有效性可变，可能会受益于有限的人4-6。酒精减少了许多受影响的人的震颤，但其治疗的有用性受到其陶醉的影响的限制。辛酸（OA）是一种与酒精相关的食物添加剂，并产生肢体的必要震颤，而副作用最小。辛酸可能直接影响震颤起源的小脑途径。拟议项目的目的是确定OA对EVT人士语音特征的影响。这是朝着推进基于证据的EVT处理的创新步骤，它是第一个解决OA语音特定效应的研究。在随机，安慰剂控制的跨界设计中，22名参与者将获得OA和安慰剂条件，参与者和实验者在数据收集和分析过程中均蒙蔽了条件。结果将通过比较OA和安慰剂条件之间声音，感知和功能方面的评估后的变化与评估后的变化。具体而言，目标I和II将确定OA对震颤和感知震颤严重程度的声学幅度的影响。 AIM III将确定OA对生活质量和与家人或朋友交流的能力的功能影响。假设可以影响多种语音相关的肌肉的OA将比安慰剂条件更大的声音，感知和功能表现。 AIM IV将确定配音震颤的个人变异性和特征，以及这些与治疗反应的关系。拟议的项目将显着推进EVT临床治疗的基于证据的决策，从而为减少这种疾病的衰弱效应迈出了至关重要的一步。