Castrillo-Viguera Carmen, Grasso Daniela L, Simpson Elizabeth, Shefner Jeremy, Cudkowicz Merit E
Massachusetts General Hospital, Charlestown, 02129, USA.
Amyotroph Lateral Scler. 2010;11(1-2):178-80. doi: 10.3109/17482960903093710.
Our objective was to survey ALS clinicians and researchers regarding what percentage reduction in the ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised) slope they would consider clinically meaningful. A nine-question survey was provided to 65 members of the Northeast ALS Consortium (NEALS). They were asked to rate the clinical relevance of 10-50% changes in decline of the ALSFRS-R slope on a seven-point scale (1-7), where 1='not at all clinically meaningful', 4='somewhat clinically meaningful', and 7='very clinically meaningful'. Ninety per cent of participants rated a 20% change in the decline of the ALSFRS-R score as the percentage in which a somewhat clinically significant change starts to be noted (i.e. score of 4 or higher). All participants endorsed a 25% or higher change in the ALSFRS-R score as at least somewhat clinically meaningful (score of 4 or higher). Ninety-three per cent of the participants viewed a 50% change in decline as very clinically meaningful (score of 7). This survey demonstrated that the majority of clinicians and clinical researchers surveyed believe that a therapy that resulted in a change of 20% or greater in the slope of the ALSFRS-R would be clinically meaningful.
我们的目标是就肌萎缩侧索硬化功能评定量表修订版（ALSFRS-R）斜率降低多少百分比他们会认为具有临床意义，对肌萎缩侧索硬化症临床医生和研究人员进行调查。向东北肌萎缩侧索硬化症联盟（NEALS）的65名成员提供了一份包含九个问题的调查问卷。要求他们按照七点量表（1 - 7）对ALSFRS-R斜率下降10% - 50%的临床相关性进行评分，其中1 =“完全没有临床意义”，4 =“有点临床意义”，7 =“非常有临床意义”。90%的参与者将ALSFRS-R评分下降20%评定为开始出现一定临床显著变化的百分比（即评分4或更高）。所有参与者都认可ALSFRS-R评分25%或更高的变化至少具有一定临床意义（评分4或更高）。93%的参与者认为下降50%具有非常显著的临床意义（评分7）。这项调查表明，接受调查的大多数临床医生和临床研究人员认为，导致ALSFRS-R斜率变化20%或更大的疗法将具有临床意义。
