两项关键的随机对照试验(RCT)表明,与单独使用ADT相比,醋酸阿比特龙+泼尼松(AAP)联合雄激素剥夺治疗(ADT)可显着延长患有转移性激素敏感性前列腺癌(mHSPC)的男性的生存率。他们的亚组分析表明,对于年轻男性,而不是老年男性,生存益处是显着的。我们的目的是评估RCT的发表是否与不同年龄段的现实世界AAP利用率有关。
使用从美国43个医疗机构收集的TriNetX电子病历数据,我们在2014年6月至2019年6月新诊断为mHSPC的男性中进行了差异事件研究.基于全面公布的算法鉴定合格的受试者。我们分析了年龄在70岁和≥70岁的男性在RCT发布前后的AAP利用率的变化。根据人口统计学因素和临床状况进行调整。
我们的研究包括6,888例新诊断为mHSPC的男性,观察结果为12,738例。其中46%的人年龄为70岁。AAP使用的出版前趋势在各年龄组之间相似,而RCTs的发表与年轻男性的AAP校正摄取率(95%CI,1.2%-5.8%)相对于老年男性高3.5%相关.这一估计反映出,如果年轻男子遵循与老年男子相同的利用趋势,摄取率将比预期高出近3倍。在整个出版后期间,估计数保持一致。
我们的研究表明,RCT的发表与新诊断为mHSPC的年轻男性和老年男性的AAP摄取更快有关。尽管没有针对不同治疗选择的临床指导。这一发现强调了在老年男性中进行验证性研究的重要性,考虑到随机对照试验中亚组分析的不确定性。
Two pivotal randomized controlled trials (RCTs) demonstrate that abiraterone acetate + prednisone (AAP) combined with androgen deprivation therapy (ADT) significantly extends the survival of men with metastatic hormone-sensitive prostate cancer (mHSPC) compared with ADT alone. Their subgroup analyses indicate that the survival benefit is significant for younger men but not older men. We aimed to assess whether publication of the RCTs was associated with differential real-world AAP utilization by age groups.
Using TriNetX electronic medical records data collected from 43 healthcare organizations across the United States, we performed a difference-in-differences event study among men with newly diagnosed mHSPC observed from June 2014 to June 2019. Eligible subjects were identified based on a comprehensive published algorithm. We analyzed the change in utilization rate of AAP before versus after publication of the RCTs among men aged <70 years versus ≥70 years, adjusting for demographic factors and clinical conditions.
Our study included 6,888 men with newly diagnosed mHSPC with 12,738 observations, of whom 46% were aged <70 years. The prepublication trends of AAP utilization were similar between the age groups, whereas publication of the RCTs was associated with a 3.5% higher adjusted uptake rate of AAP among younger men (95% CI, 1.2%-5.8%) relative to older men. This estimate reflects an uptake rate nearly 3 times higher than would have been expected had younger men followed the same utilization trends as older men. The estimates remained consistent throughout the postpublication period.
Our study suggests that publication of the RCTs was associated with faster uptake of AAP among younger versus older men with newly diagnosed mHSPC, despite the absence of clinical guidance for differential treatment selection. This finding highlights the importance of confirmatory studies among older men, considering the uncertainties of subgroup analyses in RCTs.