文章：

Blinatumomab与成人复发/难治性急性淋巴细胞白血病历史标准治疗的比较

Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia

原文发布日期：2016-09-23

DOI: 10.1038/bcj.2016.84

类型: Original Article

开放获取: 是

英文摘要：

We compared outcomes from a single-arm study of blinatumomab in adult patients with B-precursor Ph-negative relapsed/refractory acute lymphoblastic leukemia (R/R ALL) with a historical data set from Europe and the United States. Estimates of complete remission (CR) and overall survival (OS) were weighted by the frequency distribution of prognostic factors in the blinatumomab trial. Outcomes were also compared between the trial and historical data using propensity score methods. The historical cohort included 694 patients with CR data and 1112 patients with OS data compared with 189 patients with CR and survival data in the blinatumomab trial. The weighted analysis revealed a CR rate of 24% (95% CI: 20–27%) and a median OS of 3.3 months (95% CI: 2.8–3.6) in the historical cohort compared with a CR/CRh rate of 43% (95% CI: 36–50%) and a median OS of 6.1 months (95% CI: 4.2–7.5) in the blinatumomab trial. Propensity score analysis estimated increased odds of CR/CRh (OR=2.68, 95% CI: 1.67–4.31) and improved OS (HR=0.536, 95% CI: 0.394–0.730) with blinatumomab. The analysis demonstrates the application of different study designs and statistical methods to compare novel therapies for R/R ALL with historical data.

摘要翻译： 

我们将一项针对成人B前体费城染色体阴性复发/难治性急性淋巴细胞白血病（R/R ALL）患者的blinatumomab单臂研究结果与来自欧洲和美国的既往数据集进行了比较。通过blinatumomab试验中预后因素的频率分布对完全缓解（CR）和总生存期（OS）的估计值进行加权分析，并采用倾向评分方法对试验数据与历史数据进行比较。历史队列包含694例具有CR数据的患者和1112例具有OS数据的患者，而blinatumomab试验中则有189例具有CR和生存数据的患者。加权分析显示，历史队列的CR率为24%（95%置信区间：20%-27%），中位OS为3.3个月（95%置信区间：2.8-3.6），而blinatumomab试验的CR/CRh率为43%（95%置信区间：36%-50%），中位OS为6.1个月（95%置信区间：4.2-7.5）。倾向评分分析表明，blinatumomab可显著提高CR/CRh几率（比值比=2.68，95%置信区间：1.67-4.31）并改善OS（风险比=0.536，95%置信区间：0.394-0.730）。本分析展示了应用不同研究设计和统计方法将R/R ALL新型疗法与历史数据进行比较的过程。

原文链接：

Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia