Multi-site RCT of Pediatric Constraint-Induced Movement Therapy (CIMT)

描述(由申请人提供)：单侧(偏瘫)脑性瘫痪(CP)在美国约有229,000人受到影响，其中幼儿的CP发生率有所上升。大多数传统的治疗形式被证明是无效的，尽管它们仍然被广泛使用。两篇主要研究综述(参见Huang等人，2010；Sakzewski等人，2009)得出结论，儿童强制诱导运动疗法(CIMT)是最有前途的新疗法之一，尽管其关键特征存在广泛的差异和激烈的辩论。两个亟待解决的问题涉及(A)不同剂量水平的儿科CIMT和(B)不同类型和持续时间的约束的影响。这项研究是一项多点随机对照试验，研究对象为144名患有偏瘫的儿童(3-6岁)。多基线2×2随机对照试验设计将比较两种剂量水平(21天3.0小时/天与21天1.5小时/天)在两种不同的约束条件(全职石膏和部分时间夹板)下的效果。首先在对照条件下进行研究，接受3个月以上的常规治疗，然后交叉随机分配到4个CIMT治疗组中：(1)3.0小时/天剂量，全职铸造；(2)3.0小时/天，部分时间夹板；(3)1.5小时/天，全职铸造；(4)1.5小时/天，部分时间夹板。对于所有群体，父母将接受系统的培训，以帮助练习和扩展他们的孩子新的和改进的上肢技能。儿童将在治疗后3个月进行随访。儿童的神经运动能力将通过一套全面的标准化和创新工具进行评估，由对受试者的控制和治疗条件视而不见的个人在8个月内进行5次。主要的结果测量是辅助手评估(AHA)、儿童运动活动日志(PMAL)和改良的QUEST。视频会议和家长报告的独立评分将补充实验室评估，以及从加速计传输的数据。具体目的1比较两种剂量水平的儿科CIMT的疗效。具体目标2比较了两种约束形式和持续时间的效果，之所以选择这两种形式，是因为关于它们对儿童的影响和大脑皮层重组的假设相互竞争。具体目标3使用关于受试者个体差异的临床诊断和基线数据来探索关于对CIMT的不同反应的假设，包括他们在治疗期间的投入程度，检测到的短期和长期变化的类型和幅度，以及是否记录了神经运动能力以外的其他变化(即“溢出效应”)。这项研究的结果将推动制定严格的、以证据为基础的CIMT实践标准，并有可能大规模实施，为CP儿童带来重大好处。

DESCRIPTION (provided by applicant): Unilateral (hemiparetic) cerebral palsy (CP) affects an estimated 229,000 individuals in the U.S., with CP rates increasing in young children. Most traditional forms of therapy have proven ineffective, although they remain in widespread use. Two major research reviews (cf. Huang et al, 2010; Sakzewski et al, 2009) conclude that pediatric constraint-induced movement therapy (CIMT) is one of the most promising new therapies, despite wide variation in and vigorous debate about its key features. Two urgent, unresolved issues concern the effects associated with (a) different dosage levels of pediatric CIMT and (b) different types and duration of constraint. The proposed study is a multi-site, randomized controlled trial (RCT) of 144 children (3 - 6 yrs old) with hemiparetic CP. The multiple baseline 2 X 2 RCT study design will compare the effects of 2 dosage levels (3.0 hrs/day for 21 days versus 1.5 hrs/day for 21 days) under 2 distinct constraint conditions (Full-time cast versus Part-time splint). Children will first be studied in a control condition, receivin their usual therapy over 3 months, and then crossover to be randomly assigned to one of 4 CIMT treatment groups: (1) 3.0 hrs/day dosage, Full-time cast; (2) 3.0 hrs/day, Part-time splint; (3) 1.5 hrs/day, Full-time cast; and (4) 1.5 hrs/day, Part-time splint. For all groups, parents wil receive systematic training to help practice and extend their children's new and improved upper extremity skills. Children will be followed 3 months post-treatment. Children's neuromotor competence will be assessed via a comprehensive battery of standardized and innovative tools, administered 5 times over 8 months by individuals blinded to the subjects' control and treatment conditions. The primary outcome measures are the Assisted Hand Assessment (AHA), the Pediatric Motor Activity Log (PMAL), and the modified QUEST. Independent scoring of videotaped sessions and parent reports will supplement lab assessments, along with data transmitted from accelerometers. Specific Aim 1 focuses on comparing the effects of 2 dosage levels of pediatric CIMT. Specific Aim 2 compares the effects of 2 forms and duration of constraint, selected because of competing hypotheses about their impact on children and cortical re-organization. Specific Aim 3 uses clinical diagnostic and baseline data about subjects' individual differences to explore hypotheses about differential response to CIMT, including their levels of engagement during treatment, the types and magnitude of short- and longer-term changes detected, and whether other changes are documented beyond the realm of neuromotor competence (i.e., "spillover effects"). The results of this study will advance the development of rigorous, evidence-based standards for the practice of CIMT, with the potential for large-scale implementation to produce major benefits for children with CP.