Department of Neurology, University of California, Los Angeles; and California Rehabilitation Institute, Los Angeles (S.C.C.).
Department of Neurology, Stroke Service (D.J.L., S.P.F.), Massachusetts General Hospital, Harvard Medical School, Boston.
Stroke. 2023 Mar;54(3):e86-e90. doi: 10.1161/STROKEAHA.122.042313. Epub 2023 Feb 27.
Different deficits recover to different degrees and with different time courses after stroke, indicating that plasticity differs across the brain's neural systems after stroke. To capture these differences, domain-specific outcome measures have received increased attention. Such measures have potential advantages over global outcome scales, which combine recovery across many domains into a single score and so blur the ability to capture individual measures of stroke recovery. Use of a global end point to rate disability can overlook substantial recovery in specific domains, such as motor or language, and may not differentiate between good and poor recovery for specific neurological domains. In light of these points, a blueprint is proposed for using domain-specific outcome measures in stroke recovery trials. Key steps include selecting a domain in the context of preclinical data, picking a domain-specific clinical trial end point, anchoring inclusion criteria to this end point, scoring this end point both before and after treatment, and then pursuing regulatory approval on the basis of the domain-specific results. This blueprint is intended to foster clinical trials that, by using domain-specific end points, are able to demonstrate favorable results in clinical trials of therapies that promote stroke recovery.
不同的缺陷在中风后以不同的程度和时间进程恢复，这表明中风后大脑的神经系统的可塑性不同。为了捕捉这些差异，特定领域的结果测量受到了越来越多的关注。与将多个领域的恢复综合到一个单一分数的全局结果量表相比，这些措施具有潜在的优势，从而避免了将中风恢复的个体测量模糊化的能力。使用全局终点来评定残疾可能会忽略特定领域（如运动或语言）的大量恢复，并且可能无法区分特定神经领域的良好和不良恢复。有鉴于此，提出了在中风恢复试验中使用特定领域结果测量的蓝图。关键步骤包括在临床前数据的背景下选择一个领域，选择特定领域的临床试验终点，根据该终点确定纳入标准，在治疗前后对该终点进行评分，然后根据特定领域的结果获得监管批准。该蓝图旨在促进临床试验，通过使用特定领域的终点，能够在促进中风恢复的治疗方法的临床试验中展示出有利的结果。
