扩展残疾状态量表和多发性硬化功能综合评分作为多发性硬化临床试验临床终点的评估：定量荟萃分析

Evaluation of the Expanded Disability Status Scale and the Multiple Sclerosis Functional Composite as clinical endpoints in multiple sclerosis clinical trials: quantitative meta-analyses.

作者信息

Bin Sawad Aseel, Seoane-Vazquez Enrique, Rodriguez-Monguio Rosa, Turkistani Fatema

机构信息

a MCPHS University , Boston , MA , USA.

b Umm Al-Qura University , Makkah , Kingdom of Saudi Arabia.

出版信息

Curr Med Res Opin. 2016 Dec;32(12):1969-1974. doi: 10.1080/03007995.2016.1222516. Epub 2016 Sep 7.

DOI:10.1080/03007995.2016.1222516

PMID:27603119

Abstract

OBJECTIVES

This study compared the sensitivity of the Expanded Disability Status Scale (EDSS) and the Multiple Sclerosis Functional Composite (MSFC) as clinical endpoints in multiple sclerosis (MS) clinical trials.

METHODS

Medline (1946 through 12 September 2014) and Embase (1974 through 12 September 2014) databases searches were conducted using keywords and Medical Subject Heading (MeSH) terms related to MS, EDSS, and MSFC. Only studies that used the EDSS and MSFC as endpoints were assessed. All statistical analyses were conducted using comprehensive meta-analysis (CMA). The percentages of the overall changes in EDSS and MSFC were compared. The relative risks were calculated in randomized clinical trials (RCTs).

RESULTS

A total of 123 studies were identified. There were nine studies (6 case series and 3 RCTs) included in the analysis. In the case series, the EDSS change rate in MS patients was 33.5% (95% CI: 12.9-63.2%) and the MSFC change rate was 30.3% (95% CI: 9.2-65.2%). In RCTs, patients who take the drug would be 22.9 times as likely as patients who did not take the drug to experience a change in the EDSS scale (RR = 22.9, 95% CI = 0.996-1.517, p = 0.055). Patients who take the drug would be 48.9 times as likely as patients who did not take the drug to experience a change in the MSFC scale (RR = 48.9, 95% CI = CI = 0.916-2.419, p = 0.108).

LIMITATIONS

This study focused only on MS patient improvement (positive changes) on the EDSS and MSFC. More studies are needed to include patient deterioration (negative changes) on EDSS and MSFC.

CONCLUSIONS

There is controversy about the sensitivity of the EDSS and MSFC in detecting the progression of MS disease. The EDSS and MSFC are effective tools to assess the clinical severity and progression of MS disease. MSFC is more sensitive than EDSS in detecting the progression of MS disease.

摘要

目的

本研究比较了扩展残疾状态量表（EDSS）和多发性硬化功能综合评分（MSFC）作为多发性硬化症（MS）临床试验临床终点的敏感性。

方法

使用与MS、EDSS和MSFC相关的关键词和医学主题词（MeSH）检索Medline（1946年至2014年9月12日）和Embase（1974年至2014年9月12日）数据库。仅评估将EDSS和MSFC用作终点的研究。所有统计分析均使用综合荟萃分析（CMA）进行。比较了EDSS和MSFC总体变化的百分比。在随机临床试验（RCT）中计算相对风险。

结果

共识别出123项研究。分析纳入了9项研究（6项病例系列研究和3项RCT）。在病例系列研究中，MS患者的EDSS变化率为33.5%（95%CI：12.9 - 63.2%），MSFC变化率为30.3%（95%CI：9.2 - 65.2%）。在RCT中，服用药物的患者EDSS量表出现变化的可能性是未服用药物患者的22.9倍（RR = 22.9，95%CI = 0.996 - 1.517，p = 0.055）。服用药物的患者MSFC量表出现变化的可能性是未服用药物患者的48.9倍（RR = 48.9，95%CI = CI = 0.916 - 2.419，p = 0.108）。