COMPARING TREATMENTS USING QUALITY-ADJUSTED SURVIVAL - THE Q-TWIST METHOD
COMPARING TREATMENTS USING QUALITY-ADJUSTED SURVIVAL - THE Q-TWIST METHOD
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DOI：
10.2307/2684631
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发表时间：
1995-05-01
影响因子：
1.8
通讯作者：
GOLDHIRSCH, A
中科院分区：
文献类型：
--
作者：
GELBER, RD;COLE, BF;GOLDHIRSCH, A
患者的生活质量是治疗评估的重要组成部分。我们概述了一种称为 Q-TWiST（无症状和毒性的质量调整时间）的统计方法，该方法将生活质量考虑因素纳入治疗比较中。多变量删失生存数据用于将总生存时间划分为处于一组渐进临床健康状态的时间段，这些状态的生活质量可能有所不同。平均健康状态持续时间仅限于临床试验的随访限制，是从数据中得出的，并与价值权重相结合来估计质量调整生存期。该方法强调基于阈值效用分析的治疗比较，强调不同健康状态持续时间之间的权衡；它并不旨在提供结合生活质量和数量的独特结果。我们还描述了该方法的三个最新扩展：可以使用比例风险和加速故障时间回归模型包含协变量，可以使用参数模型将有限估计预测到超出后续限制，并且可以结合生活质量维度进行荟萃分析。基本方法通过对临床试验数据的分析来证明，该临床试验比较了治疗乳腺癌的长辅助化疗方案与短持续时间辅助化疗方案。临床健康状态由以下三个结果定义：（1）治疗结束毒性，（2）疾病复发，以及（3）死亡。结果允许人们评估与长期治疗相关的毒性作用增加和无复发间隔增加之间的权衡。
The quality of life of patients is an important component of evaluation of therapies. We present an overview of a statistical method called Q-TWiST (Quality-Adjusted Time Without Symptoms and Toxicity) which incorporates quality-of-life considerations into treatment comparisons. Multivariate censored survival data are used to partition the overall survival time into periods of time spent in a set of progressive clinical health states which may differ in quality of life. Mean health state durations, restricted to the follow-up limits of the clinical trial, are derived from the data and combined with value weights to estimate quality-adjusted survival. The methodology emphasizes treatment comparisons based on threshold utility analyses that highlight trade-offs between different health state durations; it is not intended to provide a unique result combining quality and quantity of life. We also describe three recent extensions of the methodology: covariates can be included using proportional hazards and accelerated failure time regression models, restricted estimates can be projected beyond follow-up limits using parametric models, and meta-analyses can be performed incorporating quality-of-life dimensions. The basic methods are demonstrated in an analysis of data from a clinical trial comparing long versus short duration adjuvant chemotherapy regimens for the treatment of breast cancer. The clinical health states are defined by the following three outcomes: (1) end of treatment toxicity, (2) disease recurrence, and (3) death. The results allow one to evaluate the trade-off between the increased toxic effects and the increased recurrence-free interval associated with the long duration treatment.