文章：

评估一项随机3期临床试验中使用地舒单抗或唑来膦酸治疗的多发性骨髓瘤患者亚组的结果

Evaluating results from the multiple myeloma patient subset treated with denosumab or zoledronic acid in a randomized phase 3 trial

原文发布日期：2016-01-08

DOI: 10.1038/bcj.2015.96

类型: Original Article

开放获取: 是

英文摘要：

In a phase 3 trial of denosumab vs zoledronic acid in patients (n=1776) with bone metastases and solid tumors or multiple myeloma, denosumab was superior to zoledronic acid for the primary end point of prevention of skeletal-related events. There was no difference in overall survival between the two groups; however, an ad hoc overall survival analysis in the multiple myeloma subset of patients (n=180) favored zoledronic acid (hazard ratio (HR) 2.26; 95% confidence interval (CI) 1.13–4.50; P=0.014). In the present analysis, we found imbalances between the groups with respect to baseline risk characteristics. HRs with two-sided 95% CIs were estimated using the Cox model. After adjustment in a covariate analysis, the CI crossed unity (HR 1.86; 95% CI 0.90–3.84; P=0.0954). Furthermore, we found a higher rate of early withdrawals for the reasons of lost to follow-up and withdrawal of consent in the zoledronic acid group; after accounting for these, the HR was 1.31 (95% CI 0.80–2.15; P=0.278). In conclusion, the survival results in multiple myeloma patients in this trial were confounded and will eventually be resolved by an ongoing phase 3 trial.

摘要翻译： 

在一项针对骨转移及实体瘤或多发性骨髓瘤患者（n=1776）的地舒单抗与唑来膦酸的3期试验中，地舒单抗在预防骨骼相关事件的主要终点方面优于唑来膦酸。两组总生存期无差异；然而，在多发性骨髓瘤亚组患者（n=180）中的一项事后总生存分析显示唑来膦酸更具优势（风险比[HR] 2.26；95%置信区间[CI] 1.13-4.50；P=0.014）。本次分析发现组间基线风险特征存在不平衡。采用Cox模型计算双侧95%CI的风险比。经协变量分析调整后，置信区间跨越1（HR 1.86；95%CI 0.90-3.84；P=0.0954）。此外，唑来膦酸组因失访和撤销知情同意导致的早期退出率更高；校正这些因素后，风险比为1.31（95%CI 0.80-2.15；P=0.278）。最终结论：本试验中多发性骨髓瘤患者的生存结果存在混杂因素，有待正在进行的3期试验最终阐明。

原文链接：

Evaluating results from the multiple myeloma patient subset treated with denosumab or zoledronic acid in a randomized phase 3 trial