OMERACT-OARSI Initiative: Osteoarthritis Research Society International set of responder criteria for osteoarthritis clinical trials revisited.
OMERACT-OARSI Initiative: Osteoarthritis Research Society International set of responder criteria for osteoarthritis clinical trials revisited.
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DOI：
10.1016/j.joca.2004.02.001
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发表时间：
2004-05-01
影响因子：
7
通讯作者：
Dougados, M
中科院分区：
文献类型：
--
作者：
Pham, T;van der Heijde, D;Dougados, M
背景资料：OARSI临床试验反应标准倡议常务委员会制定了两套反应标准，将治疗后三个症状领域（疼痛、功能和患者总体评估）的变化结果作为临床试验的单一变量（1）。对于每个域，响应由相对和绝对变化定义，关于药物、给药途径和OA localization.Objective：提出一组简化的响应标准，具有类似的截止值，无论药物、给药途径或OA localization.Methods：Data driven approach：（1）两个数据库被考虑：“细化”数据库和“再访”数据库。(2)评估了六种不同的方案：两种正式的OARSI标准集四种简化标准集的建议方案来自临床随机盲法安慰剂对照试验的数据被用于评估两种正式方案与两种不同数据库（“详细说明”与“修订”）的性能，以及“重新访问”数据库中四种建议的简化方案的性能。安慰剂效应、活性药物效应、治疗效应以及获得观察到的安慰剂效应和活性药物治疗效应所需的样本组大小是针对六种情景中的每一种情景评价的性能。专家意见方法：OMERACTVI会议的与会者对结果进行了讨论，他们投票选择了明确的OMERACT-OARSI标准集结果：数据驱动方法：14项试验共1886例CA患者，15项研究共8164例CA患者，分别在“精心制作”和“重新访问”数据库中进行了评估。在使用不同简化情景时，“重新访问”数据库与使用正式情景时在两个数据库之间观察到的情况类似（“详细说明”与“重新访问”）。每组标准的治疗效果和所需样本量相似。专家意见法：根据专家的意见，这两个先前的表现是最重要的一组最佳反应标准。他们选择了一套标准，同时考虑疼痛和功能作为评价域，需要一个绝对的变化和相对变化从基线定义一个响应，类似的截止无论是药物，管理的途径或CA localization.Conclusion：这种数据驱动和专家的意见的方法是提出一个最佳的简化的一组响应者标准CA临床试验的基础。需要使用其他CA患者集进行其他研究，以进一步验证所提出的OMERACT-OARSI标准集。(C)2004年国际骨关节炎研究学会。由爱思唯尔有限公司出版。保留所有权利。
Background: The OARSI Standing Committee for Clinical Trials Response Criteria Initiative had developed two sets of responder criteria to present the results of changes after treatment in three symptomatic domains (pain, function, and patient's global assessment) as a single variable for clinical trials (1). For each domain, a response was defined by both a relative and an absolute change, with different cut-offs with regard to the drug, the route of administration and the OA localization.Objective: To propose a simplified set of responder criteria with a similar cut-off, whatever the drug, the route or the OA localization.Methods: Data driven approach:(1) Two databases were consideredThe 'elaboration' database with which the formal OARSI sets of responder criteria were elaborated andThe 'revisit' database.(2) Six different scenarios were evaluated:The two formal OARSI sets of criteriaFour proposed scenarios of simplified sets of criteriaData from clinical randomized blinded placebo controlled trials were used to evaluate the performances of the two formal scenarios with two different databases ('elaboration' versus 'revisit') and those of the four proposed simplified scenarios within the 'revisit' database. The placebo effect, active effect, treatment effect, and the required sample arm size to obtain the placebo effect and the active treatment effect observed were the performances evaluated for each of the six scenarios. Experts' opinion approach: Results were discussed among the participants of the OMERACT VI meeting, who voted to select the definite OMERACT-OARSI set of criteria (one of the six evaluated scenarios).Results: Data driven approach: Fourteen trials totaling 1886 CA patients and fifteen studies involving 8164 CA patients were evaluated in the 'elaboration' and the 'revisit' databases respectively.The variability of the performances observed in the 'revisit' database when using the different simplified scenarios was similar to that observed between the two databases ('elaboration' versus 'revisit') when using the formal scenarios. The treatment effect and the required sample arm size were similar for each set of criteria. Experts' opinion approach: According to the experts, these two previous performances were the most important of an optimal set of responder criteria. They chose the set of criteria considering both pain and function as evaluation domain and requiring an absolute change and a relative change from baseline to define a response, with similar cut-offs whatever the drug, the route of administration or the CA localization.Conclusion: This data driven and experts' opinion approach is the basis for proposing an optimal simplified set of responder criteria for CA clinical trials. Other studies, using other sets of CA patients, are required in order to further validate this proposed OMERACT - OARSI set of criteria. (C) 2004 OsteoArthritis Research Society International. Published by Elsevier Ltd. All rights reserved.