Methodologic lessons learned from hot flash studies
Methodologic lessons learned from hot flash studies
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DOI：
10.1200/jco.2001.19.23.4280
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发表时间：
2001-12-01
影响因子：
45.3
通讯作者：
Windschitl, H
中科院分区：
文献类型：
--
作者：
Sloan, JA;Loprinzi, CL;Windschitl, H
目的：在进行一系列前瞻性临床试验的过程中，致力于确定癌症幸存者潮热的新治疗机会，在相关的方法学问题上获得了相当多的经验。这篇文章是为了回应对这一方法的许多质疑而写的。患者和方法：这项工作使用了一系列涉及968名患者的7个不同的临床试验。潮热强度的可靠和有效定义是从患者报告的描述中发展出来的。对患者完成的日记进行伴随的有效性和可靠性评估，以比较热闪光数据与毒性和生活质量(QOL)终点，并使用变异性分析和相关程序检查患者组之间的一致性。使用Meta分析的参数数据来检验II期和III期临床试验设计的相对权威性。结果：这些研究中使用的日常日记具有一致性和可靠性，几乎没有遗漏数据。潮热发作频率和潮热发作评分(频率乘以平均严重程度)变量产生的终点结果几乎相同。对于III期安慰剂对照研究，每个治疗组50名患者似乎适合提供足够的功率规格，以检测临床上有意义的潮热活动变化。对于II期试验，每个试验的25名患者似乎提供了对最终潮热疗效的合理估计，以筛选潜在的药物进行更明确的测试。结论：根据这些经验获得的数据，我们可以计划和进行更有效的试验，以确定减少潮热活动的有效药物。J Clin Oncol19：4280-4290。(C)2001年，由美国临床肿瘤学会主办。
Purpose : In the course of conducting a series of prospective clinical trials devoted to defining new treatment opportunities for hot flashes in cancer survivors, considerable experience has been acquired with related methodologic issues. This article has been written in response to many queries regarding this methodology.Patients and Methods: A series of seven different clinical trials that involved 968 patients was used for this work. Reliable and valid definitions of hot flash intensity were developed from patient-reported descriptions. Concomitant validity and reliability assessment of patient-completed diaries was undertaken to compare hot flash data with toxicity and quality-of-life (QOL) end points and to examine consistency across patient groups using variability analysis and correlation procedures. Parametric data from this meta-analysis was used to examine relative power considerations for the design of phase II and phase III clinical trials.Results: Daily diaries used in these studies exhibited consistency and reliability and had few missing data. Hot flash frequency and hot flash score (frequency multiplied by average severity) variables produced almost identical end point results. For phase III placebo-controlled studies, 50 patients per treatment arm seem appropriate to provide sufficient power specifications to detect a clinically meaningful change in hot flash activity. For phase II trials, 25 patients per trial seem to provide reasonable estimates of eventual hot flash efficacy to screen potential agents for more definitive testing.Conclusion: Given the data gained from these experiences, we can plan and carry out more efficient trials to identify efficacious agents for the reduction of hot flash activity. J Clin Oncol 19:4280-4290. (C) 2001 by American Society of Clinical Oncology.