Association between progression-free survival and patients' quality of life in cancer clinical trials
Association between progression-free survival and patients' quality of life in cancer clinical trials
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DOI：
10.1002/ijc.31957
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发表时间：
2019-04-01
影响因子：
6.4
通讯作者：
Gyawali, Bishal
中科院分区：
文献类型：
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作者：
Hwang, Thomas J.;Gyawali, Bishal
生活质量结果为肿瘤治疗中的患者和医生提供了重要信息。然而，无进展生存期（PFS）作为生活质量替代指标的有效性以及临床试验中生活质量终点的纳入和报告尚不清楚。我们对2010年至2015年期间发表的晚期或转移性实体瘤药物的III期临床试验进行了回顾性研究。评估PFS与阳性生活质量之间相关性的相关系数（r）和ROC曲线下面积（AUC）。在纳入的352项III期试验中，190项（54%）包括生活质量终点，其中23%未报告预先规定的生活质量结局;共有125，962例患者入组缺乏或未报告生活质量结局的研究。在报告生活质量结局的147项试验中，99项（67%）报告没有效果，38项（26%）报告了积极效果，10项（7%）报告了治疗对患者整体生活质量的负面影响。PFS与总体生活质量改善之间的相关性较弱（r = 0.34; AUC = 0.72），PFS与任何生活质量领域改善之间的相关性也较弱。总之，PFS获益与患者生活质量的改善没有强相关性，尽管晚期/转移性背景下的治疗具有姑息性目的，但癌症药物临床试验的生活质量数据的可用性较差。
Quality of life outcomes provide essential information for patients and physicians in oncology care. However, the validity of progression-free survival (PFS) as a surrogate for quality of life, and the inclusion and reporting of quality of life endpoints in clinical trials, is unclear. We performed a retrospective study of phase III clinical trials of drugs for advanced or metastatic solid tumors published between 2010 and 2015. Correlation coefficient (r) and area under the ROC curve (AUC) for association between PFS and positive quality of life were evaluated. Of the 352 Phase 3 trials included, 190 (54%) included a quality of life endpoint, of which 23% did not report pre-specified quality of life outcomes; a total of 125,962 patients were enrolled in studies lacking, or not reporting, quality of life outcomes. Among the 147 trials that reported quality of life outcomes, 99 (67%) reported no effect, 38 (26%) reported a positive effect and 10 (7%) reported a negative effect of treatment on patients' global quality of life. The association between PFS and improvement in global quality of life was weak (r = 0.34; AUC = 0.72), as was the association between PFS and improvement in any domain of quality of life. In conclusion, PFS benefit was not strongly correlated with improvements in patients' quality of life, and, despite the palliative intent of treatments in the advanced/metastatic setting, the availability of quality of life data from clinical trials of cancer drugs was poor.