Multisite RCT of 3 Neurorehabilitation Therapies for Infants with Asymmetrical CP

不对称脑瘫婴儿的 3 种神经康复治疗的多部位随机对照试验

• 批准号: 9040231
• 负责人: STEPHANIE C. DELUCA
• 金额: $ 71.81万
• 依托单位: VIRGINIA POLYTECHNIC INST AND ST UNIV
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-03-01 至 2018-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9040231
• 关键词: 7 year old; Adverse effects; Adverse event; Affect; Aftercare; Articular Range of Motion; Awareness; Behavior; Behavioral; Bilateral; Blinded; Brain; Cerebral Palsy; Child; Childhood; Congenital Abnormality; Data; Data Analyses; Development; Documentation; Elements; Enrollment; Future; Hand; Handedness; Health; Home environment; Hydrocortisone; Impairment; Infant; Laws; Lead; Life; Limb structure; Link; Long-Term Effects; Measures; Modeling; Monitor; Motor; Near-Infrared Spectroscopy; Neurorehabilitation; Occupational Therapy; Outcome; Parents; Patient Self-Report; Physical therapy; Physically Handicapped; Play; Productivity; Protocols documentation; Quality of life; Randomized; Randomized Controlled Trials; Reporting; Research Design; Rest; Risk; Safety; Salivary; Sampling; Sensory; Series; Shapes; Side; Skin; Splint Device; Stress; Testing; Time; Touch sensation; Training; Upper Extremity; arm; base; behavior observation; clinical practice; constraint induced movement therapy; cost; disability; dosage; effective therapy; evidence base; hemiparesis; human subject protection; improved; indexing; light weight; meetings; neuroimaging; novel; novel therapeutics; primary outcome; prospective; response; secondary outcome; skills; social learning; theories; tool; trial comparing; trial design

DESCRIPTION (provided by applicant): Cerebral palsy (CP) is the most common class of childhood neuromotor disabilities, resulting in lifelong impairments in productivity and health as well as high costs. To date, no form of infant therapy has proven efficacious via an adequately powered RCT; further, usual and customary therapies often fail to produce clinically meaningful benefits. This revised application builds on new findings from 2 independent preliminary studies showing benefits for infants who received alternative forms of constraint-induced movement therapy (CIMT). Using a multisite RCT design, 72 infants (6 - 18 mos old) with unilateral or asymmetrical CP will be randomly assigned to receive one of 3 manualized forms of multi-component therapies. Primary outcomes include objective assessments (at baseline then 1 wk, 6 mos, and 12 mos post-therapy) of affected upper extremity (UE) skills in unilateral and bilateral activities, as well as changes in brain lateralization based on a novel fNIRS protocol fo infants. The specific aims are: 1) to compare the effects of 3 promising forms of Infant-CIMT that are identical in dosage (3 hr/day X 21 days) and their key therapy elements (shaping, massed practice, home-based, embedded in play and everyday activities, provided by a trained therapist in partnership with parents) but that differ in their use of constraint - continuous casting, part-time splint, or no constraint; and 2) to assess stress levels and safety related to constraint condition (continuous or part-time) compared to no constraint. Stress will be assessed using multiple salivary cortisol samples from infants and parents, parent self-report, and observations. Safety monitoring will include range of motion, skin integrity, sensory awareness, functional use of the casted arm and hand, as well as changes in laterality scores involving the casted side. Even though prior studies affirm the general safety and acceptability of using constraint with infants, there are strongly competing hypotheses about its potential benefits versus risks. Impact: if one or more of the tested infant therapies produces large magnitude benefits lasting up to 12 mos later, then these findings would have a high impact on future clinical practice. Moreover, highly effective therapy very early in life may lead to much higher levels of independence, learning, social engagement, and quality of life for children with unilateral and asymmetrical CP.

描述(申请人提供)：脑性瘫痪(CP)是最常见的儿童神经运动障碍，导致终生生产力和健康障碍以及高昂的费用。到目前为止，还没有一种形式的婴儿疗法通过足够强大的RCT被证明是有效的；此外，常规和常规疗法往往无法产生临床上有意义的好处。这项修订后的申请建立在两项独立初步研究的新发现基础上，这些研究表明，接受替代形式的强制诱导运动疗法(CIMT)的婴儿有好处。采用多点随机对照试验设计，72名患有单侧或不对称性脑性瘫痪的婴儿(6-18个月大)将被随机分配接受3种手册形式的多组分疗法之一。主要结果包括客观评估(基线时，治疗后1周、6个月和12个月)单侧和双侧活动中受影响的上肢(UE)技能，以及基于新的婴儿fNIRS方案的大脑偏侧化的变化。具体目标是：1)比较3种有希望的婴儿-CIMT的效果，这些形式的剂量相同(3小时/天×21天)及其关键治疗要素(整形、集中练习、居家、游戏和日常活动，由训练有素的治疗师与父母合作提供)，但在使用约束-连续铸造、兼职夹板或无约束方面存在差异；以及2)评估约束条件(连续或兼职)与无约束条件下相关的应激水平和安全性。压力将使用来自婴儿和父母的多个唾液皮质醇样本、父母自我报告和观察来评估。安全监测将包括活动范围、皮肤完整性、感官知觉、固定臂和手的功能使用，以及涉及固定侧的偏侧性评分的变化。尽管先前的研究肯定了对婴儿使用强制措施的总体安全性和可接受性，但关于其潜在好处与风险存在强烈竞争的假说。影响：如果一个或多个被测试的婴儿疗法在12个月后产生了巨大的益处，那么这些发现将对未来的临床实践产生很大影响。此外，对于患有单侧和不对称性脑瘫的儿童，在生命早期进行高效的治疗可能会导致更高水平的独立性、学习能力、社会参与度和生活质量。