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

描述（由申请人提供）：单侧（轻偏瘫）脑性麻痹（CP）影响美国估计229，000人，幼儿的脑瘫率在上升。大多数传统形式的治疗已被证明是无效的，尽管它们仍然被广泛使用。两个主要的研究综述（参见。Huang等人，2010; Sakzewski等人，2009）得出结论，尽管其关键特征存在广泛差异和激烈争论，但儿科约束诱导运动疗法（CIMT）是最有前途的新疗法之一。两个紧急的，未解决的问题涉及的影响（a）不同剂量水平的儿科CIMT和（B）不同类型和持续时间的限制。该研究是一项多中心随机对照试验（RCT），纳入144名轻偏瘫CP儿童（3 - 6岁）。多基线2 X 2 RCT研究设计将比较2种剂量水平（3.0小时/天，持续21天与1.5小时/天，持续21天）在2种不同限制条件（全时石膏与部分时间夹板）下的效果。儿童将首先在对照条件下进行研究，接受他们的常规治疗超过3个月，然后交叉随机分配到4个CIMT治疗组之一：（1）3.0小时/天剂量，全时石膏;（2）3.0小时/天，部分时间夹板;（3）1.5小时/天，全时石膏;和（4）1.5小时/天，部分时间夹板。对于所有团体，家长将接受系统的培训，以帮助实践和扩展他们的孩子的新的和改进的上肢技能。将在治疗后3个月对儿童进行随访。儿童的神经运动能力将通过一组全面的标准化和创新工具进行评估，由对受试者的对照和治疗条件不知情的个体在8个月内进行5次给药。主要结果指标是辅助手部评估（AHA）、儿科运动活动日志（PMAL）和改良的QUEST。独立评分的录像会议和家长报告将补充实验室评估，沿着数据传输从加速计。具体目标1侧重于比较2种剂量水平的儿科CIMT的效果。具体目标2比较了2种形式和持续时间的约束，选择，因为他们对儿童和皮质重组的影响竞争的假设的影响。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的差异反应的假设，包括他们在治疗期间的参与水平，检测到的短期和长期变化的类型和幅度，以及是否记录了超出神经运动能力范围的其他变化（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儿童带来重大益处。

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.