Comparison of disease activity in SPMS and PPMS in the context of multicenter clinical trials.
Comparison of disease activity in SPMS and PPMS in the context of multicenter clinical trials.
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DOI：
10.1371/journal.pone.0045409
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发表时间：
2012
期刊：
影响因子：
3.7
通讯作者：
Cadavid D
中科院分区：
文献类型：
--
作者：
Orbach R;Zhao Z;Wang YC;O'Neill G;Cadavid D
回顾性单中心自然史研究表明，达到残疾里程碑的时间和达到残疾里程碑的年龄在原发性（PPMS）和继发性（SPMS）进行性多发性硬化症中相似，表明它们可能是同一疾病的表型变异。在此，我们在国际多中心临床试验的背景下比较了SPMS和PPMS的纵向疾病活动。我们分析了过去10年中在一项SPMS和一项PPMS临床试验中随机分配至安慰剂组的所有受试者在2年内系统收集的所有客观结局指标。使用临床疾病活动性的常规和探索性定义。疾病活动分为3个不同类别：间歇性活动、进展和改善。常规MRI测量和1例患者报告的生活质量结局测量（可用于比较）。绘制所有结果的热图，然后进行分层聚类。206例SPMS受试者有101个结局变量，135例PPMS受试者有79个结局变量。比较显示，SPMS和PPMS受试者在2年内的所有变量中表现出相似的疾病活动性，但两个变量除外，EDSS感觉系统的常见恶化在PPMS中更常见，而9孔PEG的恶化在SPMS中更常见。间歇活动是SPMS和PPMS中最常见的疾病活动模式。两者的临床恶化和改善频率相似。在我们研究的两项多中心国际临床试验中，SPMS和PPMS受试者的纵向疾病活动几乎相同。
Retrospective single center natural history studies have shown that times to reach disability milestones and ages at which they are reached are similar in primary (PPMS) and secondary (SPMS) progressive multiple sclerosis suggesting that they may be phenotypic variations of the same disease. Here we compared longitudinal disease activity in SPMS and PPMS in the context of international multicenter clinical trials. We analyzed all objective outcome measures that were systematically collected over 2 years for all subjects randomized to placebo arms in one SPMS and one PPMS clinical trial over the last decade. Conventional and exploratory definitions of clinical disease activity were used. Disease activity was analyzed in 3 different categories intermittent activity, progression, and improvement. Conventional MRI measures and one patient reported outcome measure of quality of life were included when available for comparison. Heat maps were drawn for all results followed by hierarchical clustering. There were 101 outcome variables from 206 SPMS subjects and 79 outcome variables from 135 PPMS subjects. The comparison revealed that SPMS and PPMS subjects exhibited similar disease activity over 2 years in all but two of the variables in common worsening in the EDSS sensory system was more common in PPMS while worsening on the 9 hole PEG was more common in SPMS. Intermittent activity was the most common pattern of disease activity in SPMS and PPMS. Clinical worsening and improvement occurred at similar frequency in both. Longitudinal disease activity was nearly identical in SPMS and PPMS subjects in the context of the two multicenter international clinical trials we examined.
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