Montreal Neurological Institute-Hospital, ALS Center of Excellence, Montreal, Quebec, Canada.
Yale School of Medicine, Section of Movement Disorders, Coeruleus Clinical Sciences LLC, Woodbridge, CT, USA.
Amyotroph Lateral Scler Frontotemporal Degener. 2024 May;25(3-4):382-387. doi: 10.1080/21678421.2024.2320880. Epub 2024 Feb 23.
The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) was developed more than 25 years ago as an instrument to monitor functional change over time in patients with ALS. It has since been revised and extended to meet the needs of high data quality in ALS trials (ALSFRS-R), however a full re-validation of the scale was not completed. Despite this, the scale has remained a primary outcome measure in clinical trials. We convened a group of clinical trialists to discuss and explore opportunities to improve the scale and propose alternative measures. In this meeting report, we present a call to action on the use of the ALSFRS-Revised scale in clinical trials, focusing on the need for (1) harmonization of the ALSFRS-R administration globally, (2) alignment on a set of recommendations for clinical trial design and statistical analysis plans (SAPs), and (3) use of additional outcome measures.
肌萎缩侧索硬化功能评定量表（ALSFRS）是 25 多年前作为一种监测 ALS 患者随时间变化的功能变化的工具而开发的。此后，它经过修订和扩展，以满足 ALS 试验的高数据质量需求（ALSFRS-R），但并未完成全面重新验证。尽管如此，该量表仍然是临床试验中的主要结果测量指标。我们召集了一组临床试验人员，讨论并探讨了改进量表和提出替代措施的机会。在本会议报告中，我们呼吁在临床试验中使用修订后的 ALSFRS-R 量表，重点关注以下三个方面的需求：（1）在全球范围内协调 ALSFRS-R 的管理，（2）在临床试验设计和统计分析计划（SAP）方面达成一致建议，（3）使用额外的结果测量指标。
