How effective are disease-modifying drugs in delaying progression in relapsing-onset MS?
How effective are disease-modifying drugs in delaying progression in relapsing-onset MS?
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DOI：
10.1212/01.wnl.0000271884.11129.f3
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发表时间：
2007-10-09
期刊：
影响因子：
9.9
通讯作者：
Sketris, I. S.
中科院分区：
文献类型：
--
作者：
Brown, M. G.;Kirby, S.;Sketris, I. S.
目的：我们的目的是评估在“真实世界”条件下，疾病缓解药物 (DMD) 在延缓复发性 (R-onset) 确诊 MS 患者的多发性硬化症 (MS) 残疾进展方面的有效性。方法：对于 DMD 作为一类，治疗效果大小以绝对值和相对于 MS 自然史的相对值进行估计。基本模型估计了治疗前后扩展残疾状态量表 (EDSS) 的年度变化。扩展模型估计了治疗前年份、首次药物治疗年份、换药后治疗年份以及治疗停止后年份的年度 EDSS 变化。模型填充了 1980 年至 2004 年的临床数据，包括 1988 年至 2004 年所有接受 DMD 治疗的新斯科舍人的数据。对复发缓解型 MS (RRMS)、继发进展型 MS (SPMS) 和 R 发病组进行了估计。结果：估计治疗前每年 EDSS 增加约为 RRMS 组 1 个 EDSS 点的 0.10，SPMS 组为 0.31，R 发病组为 0.16。对于 RRMS 组 (-0.103, 0.000)、SPMS 组 (- 0.065, 0.011) 和 R 发作组 (-0.162, 0.000)，第一种药物每个治疗年避免的 EDSS 增加的估计值显着；相对效应大小估计值分别为 112%、21% 和 105%。估计 EDSS 进展在药物转换和治疗停止后数年内会更快。结论：在“真实世界”临床实践的背景下，我们对疾病缓解药物 (DMD) 相对治疗效果大小的估计与关键试验中的 DMD 治疗效果估计相似，尽管我们的研究结果具有统计学意义。 DMD 作为一类药物，可有效延缓复发性多发性硬化症 (MS) 患者 (90%) 的扩展残疾状态量表进展，尽管对于复发缓解型 MS 的有效性比继发进展性 MS 组要好得多。
Objective: Our objective was to estimate the effectiveness of disease-modifying drugs ( DMDs) in delaying multiple sclerosis ( MS) disability progression in relapsing-onset ( R-onset) definite MS patients under "real-world" conditions.Methods: Treatment effect size, for DMDs as a class, was estimated in absolute terms and relative to MS natural history. A basic model estimated annual Expanded Disability Status Scale ( EDSS) change before and after treatment. An expanded model estimated annual EDSS change in pretreatment years, treatment years on first drug, treatment years after drugs were switched, and in years after treatment stopped. Models were populated with 1980 through 2004 clinical data, including 1988 through 2004 data for all Nova Scotians treated with DMDs. Estimates were made for relapsing-remitting MS ( RRMS), secondary progressive MS ( SPMS), and R-onset groups.Results: Estimated pretreatment annual EDSS increases were approximately 0.10 of one EDSS point for the RRMS group, 0.31 for the SPMS group, and 0.16 for the R-onset group. Estimates of EDSS increase avoided per treatment year on the first drug were significant for the RRMS group ( -0.103, 0.000), the SPMS group ( - 0.065, 0.011), and the R-onset group ( -0.162, 0.000); relative effect size estimates were 112%, 21%, and 105%. Estimated EDSS progression was faster in years after drug switches and treatment stops.Conclusions: Our estimates of disease-modifying drug ( DMD) relative treatment effect size, in the context of "real- world" clinical practice, are similar to DMD treatment efficacy estimates in pivotal trials, though our findings attained statistical significance. DMDs, as a class, are effective in delaying Expanded Disability Status Scale progression in patients with relapsing-onset definite multiple sclerosis ( MS) ( 90%), although effectiveness is much better for relapsing - remitting MS than for secondary progressive MS groups.