A combination of distribution- and anchor-based approaches determined minimally important differences (MIDs) for four endpoints in a breast cancer scale
A combination of distribution- and anchor-based approaches determined minimally important differences (MIDs) for four endpoints in a breast cancer scale
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DOI：
10.1016/j.jclinepi.2004.01.012
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发表时间：
2004-09-01
影响因子：
7.2
通讯作者：
Wood, WC
中科院分区：
文献类型：
--
作者：
Eton, DT;Cella, D;Wood, WC
目的：确定癌症治疗功能评估-乳腺（FACT-B）中4个评分的基于分布和锚点的最小重要差异（MID）估计值：乳腺癌子量表（BCS）、试验结局指数（TOI）、FACT-G研究设计和设置：我们使用了转移性乳腺癌III期临床试验的数据（ECOG研究1193; n = 739）和转移性乳腺癌疼痛的前瞻性观察性研究（n = 129）。三分之一和一半的标准偏差和I测量标准误差被用作基于分布的标准。用于确定锚为基础的差异的临床指标包括ECOG体能状态，目前的疼痛，和response to treatment.Results：FACT-B评分响应性能状态和疼痛锚，但不治疗反应。将基于分布和基于锚点的方法的结果相结合，得到了MID的估计值：BCS = 2-3点，TOI = 5 -6点，FACT-G = 5-6点，FACT-B = 7-8点。这些估计值可与其他疗效指标结合使用，以确定有意义的获益，并为临床试验中的样本量估计提供基础。(C)2004年爱思唯尔公司All rights reserved.
Objective: To determine distribution- and anchor-based minimal important difference (MID) estimates for four scores from the Functional Assessment of Cancer Therapy-Breast (FACT-B): the breast cancer subscale (BCS), Trial Outcome Index (TOI), FACT-G (the general version), and FACT-B.Study Design and Setting: We used data from a Phase III clinical trial in metastatic breast cancer (ECOG study 1193; n = 739) and a prospective observational study of pain in metastatic breast cancer (n = 129). One third and one half of the standard deviation and I standard error of measurement were used as distribution-based criteria. Clinical indicators used to determine anchor-based differences included ECOG performance status, current pain, and response to treatment.Results: FACT-B scores were responsive to performance status and pain anchors, but not to treatment response. By combining the results of distribution- and anchor-based methods, MID estimates were obtained: BCS = 2-3 points, TOI = 5-6 points, FACT-G = 5-6 points, and FACT-B = 7-8 points.Conclusion: Distribution- and anchor-based estimates of the MID do show convergence. These estimates can be used in combination with other measures of efficacy to determine meaningful benefit and provide a basis for sample size estimation in clinical trials. (C) 2004 Elsevier Inc. All rights reserved.