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: 8827752
• 负责人: Soren Yvette Lowell
• 金额: $ 14.51万
• 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 2017-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8827752
• 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; Health; 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）：特发性震颤是最常见的运动障碍之一，影响近 5% 的 65 岁以上人群。由于预计老年人的增长速度不成比例，预计未来 10 年这种疾病的患病率将会增加人口。特发性震颤的特点是有节奏的、不自主的运动，通常影响上肢，但也可能影响头部、躯干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 的相关通路内。在特发性声音震颤 (EVT) 中，声音相关结构的重复运动会产生语音信号的调制。感知声音影响包括不稳定、声音嘶哑和发声困难增加3, 4，这会对言语清晰度产生负面影响，并产生巨大的心理和身体负担3。目前的 EVT 治疗方法还不够充分，且疗效参差不齐，可能使有限比例的人受益 4-6。酒精可以减轻许多受影响人群的震颤，但其治疗作用因其中毒作用而受到限制。辛酸 (OA) 是一种与酒精相关的食品添加剂，可以减少四肢特发性震颤，同时副作用最小7。辛酸可能直接影响被认为是震颤起源的小脑通路。拟议项目的目标是确定 OA 对 EVT 患者语音特征的影响。这是第一项解决 OA 对声音特定影响的研究，代表着在推进 EVT 循证治疗方面迈出了创新的一步。在随机安慰剂对照交叉设计中，22 名参与者将分别接受 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对条件均不知情。结果将通过比较 OA 和安慰剂条件下声音的声学、感知和功能方面的评估前后测量的变化来确定。具体来说，目标 I 和 II 将确定 OA 对震颤的声学幅度和感知的声音震颤严重程度的影响。目标 III 将确定 OA 对生活质量以及与家人或朋友沟通能力的功能影响。据推测，OA 会影响多个与言语相关的肌肉，与安慰剂相比，OA 会更大程度地减少声音震颤的声学、知觉和功能表现。目标 IV 将确定声音震颤的个体差异和特征，以及它们与治疗反应的关系。拟议的项目将显着推进 EVT 临床治疗的循证决策，为减少这种疾病的衰弱影响迈出重要一步。

项目成果

The Effect of Octanoic Acid on Essential Voice Tremor: A Double-Blind, Placebo-Controlled Study.

辛酸对原发性声音震颤的影响：一项双盲、安慰剂对照研究。

• DOI: 10.1002/lary.27695
• 发表时间: 2019
• 期刊: The Laryngoscope
• 作者: Lowell,SorenY;Kelley,RichardT;Monahan,Marika;Hosbach-Cannon,CarlyJo;Colton,RaymondH;Mihaila,Dragos
• 通讯作者: Mihaila,Dragos

The effect of anchors on reliability of endoscopic tremor ratings.

锚对内窥镜震颤评级可靠性的影响。

• DOI: 10.1002/lary.26034
• 发表时间: 2017
• 期刊: The Laryngoscope
• 作者: Lowell,SorenY;Kelley,RichardT;Busekroos,Lauren;Voleti,Ramani;Hosbach-Cannon,CarlyJ;Colton,RaymondH;Mihaila,Dragos
• 通讯作者: Mihaila,Dragos