Department of Neurology, MS Center Amsterdam, Amsterdam University Medical Centers, Amsterdam, The Netherlands.
Multiple Sclerosis Center, Swedish Neuroscience Institute, Seattle, WA, USA.
Mult Scler. 2022 Sep;28(10):1606-1619. doi: 10.1177/13524585221105465.
Patient-reported outcome measures (PROMs) are often used in clinical research, but little is known about their performance as longitudinal outcomes.
We used data from ASCEND, a large SPMS trial ( = 889), to investigate changes on the Short Form Health Survey 36 (SF-36 v2) and the Multiple Sclerosis Impact Scale (MSIS-29) over 2 years of follow-up.
PROM scores changed little over the 2 years of follow-up. In contrast to physical disability measures, there was no consistent trend in PROM change: significant worsening occurred about as often as improvement. Using a 6-month confirmation reduced the number of both worsening and improvement events without altering their relative balance. There was no clear difference in worsening events in groups based on population characteristics, nor was there a noticeable effect using different thresholds for clinically significant change.
We found little consistent change in MSIS-29 and SF-36 over 2 years of follow-up in people with SPMS. Our findings show a disconnect between disability worsening and PROM change in this population. Our findings raise caution about the use of these PROMs as primary outcome measures in SPMS trials and call for a critical reappraisal of the longitudinal use of these measures in SPMS trials.
患者报告的结局测量（PROMs）常用于临床研究，但对于其作为纵向结局的表现知之甚少。
我们使用 ASCEND 数据，这是一项大型 SPMS 试验（ = 889），调查了在 2 年的随访中，简短健康调查 36 项量表（SF-36 v2）和多发性硬化影响量表（MSIS-29）的变化。
在 2 年的随访中，PROM 评分变化不大。与身体残疾测量相比，PROM 变化没有一致的趋势：恶化和改善同样常见。使用 6 个月的确认可以减少恶化和改善事件的数量，而不会改变它们的相对平衡。根据人群特征，恶化事件在不同组之间没有明显差异，使用不同的临床显著变化阈值也没有明显效果。
我们发现 SPMS 患者在 2 年的随访中，MSIS-29 和 SF-36 的变化很小。我们的研究结果表明，在该人群中，残疾恶化与 PROM 变化之间存在脱节。我们的研究结果对在 SPMS 试验中使用这些 PROMs 作为主要结局测量提出了谨慎，呼吁对 SPMS 试验中这些测量的纵向使用进行批判性重新评估。
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