Lungu Codrin, Hirtz Deborah, Damiano Diane, Gross Paul, Mink Jonathan W
From the Office of the Clinical Director (C.L.) and Office of Clinical Research (D.H.), National Institute of Neurological Disorders and Stroke, NIH, Bethesda; Functional and Applied Biomechanics Section (D.D.), Clinical Center, NIH, Bethesda, MD; Cerebral Palsy Research Network (P.G.), Woodinville, WA; and Departments of Neurology, Neuroscience, and Pediatrics (J.W.M.), University of Rochester, NY.
Neurology. 2016 Sep 20;87(12):1293-8. doi: 10.1212/WNL.0000000000003116. Epub 2016 Aug 24.
Cerebral palsy (CP) is heterogeneous in etiology and manifestations, making research into relevant therapies difficult and limiting the generalizability of the results. We report here on the NIH CP symposium, where stakeholders from academic, clinical, regulatory, and advocacy backgrounds discussed the major challenges and needs for moving forward with clinical research in CP, and outlined priorities and action items. New information is constantly generated through research into pathogenesis and etiology. Clinical research and new therapeutic approaches need to keep pace, through large data registry integration and new research designs. Development of standardized data collection, increasing academic focus on CP research, and iterative approaches to treatment throughout the patients' lives, have all been identified as areas of focus. The workshop identified critical gaps and areas of focus to increase the evidence base for therapeutic approaches to determine which treatments work best for which patients in the near future. These include consolidation and optimization of databases and registries, updates to the research methodology, and better integration of resources and stakeholders.
脑瘫（CP）在病因和表现上具有异质性，这使得相关治疗研究困难重重，也限制了研究结果的普遍适用性。我们在此报告美国国立卫生研究院（NIH）的脑瘫研讨会，来自学术、临床、监管和宣传领域的利益相关者讨论了推进脑瘫临床研究的主要挑战和需求，并概述了优先事项和行动项目。通过对发病机制和病因的研究，新信息不断涌现。临床研究和新的治疗方法需要跟上步伐，通过整合大型数据登记库和采用新的研究设计来实现。标准化数据收集的发展、学术界对脑瘫研究关注度的提高以及贯穿患者一生的迭代治疗方法，都已被确定为重点关注领域。该研讨会确定了关键差距和重点关注领域，以增加治疗方法的证据基础，从而在不久的将来确定哪种治疗方法对哪些患者最有效。这些包括数据库和登记库的整合与优化、研究方法的更新，以及资源和利益相关者的更好整合。
