文章：

新型药物时代下原发性浆细胞白血病的真实世界预后数据：希腊骨髓瘤研究组的多中心全国性研究

Real-world data on prognosis and outcome of primary plasma cell leukemia in the era of novel agents: a multicenter national study by the Greek Myeloma Study Group

原文发布日期：2018-03-09

DOI: 10.1038/s41408-018-0059-6

类型: Article

开放获取: 是

英文摘要：

We have studied the efficacy and the prognostic impact of novel agents in 50 primary plasma cell leukemia (pPCL) patients registered in our database. Eighty percent of patients were treated upfront with novel agent-based combinations; 40% underwent autologous stem cell transplantation (ASCT). Objective response rate was 76; 38% achieved at least very good partial response (≥vgPR) and this correlated significantly with bortezomib-based therapy plus ASCT. At the time of evaluation, 40 patients had died. Early mortality rate (≤1 month) was 6%. Median progression-free survival (PFS) and overall survival (OS) were 12 months and 18 months respectively, both significantly longer in patients treated with bortezomib-based therapy + ASCT vs. others (PFS: 18 vs. 9 months; p = 0.004, OS: 48 vs. 14 months; p = 0.007). Bortezomib-based therapy + ASCT predicted for OS in univariate analysis. In multivariate analysis, achievement of ≥vgPR and LDH ≥ 300 U/L were significant predictors for OS. These real-world data, based on one of the largest reported national multicenter series of pPCL patients treated mostly with novel agents support that, among the currently approved induction therapies, bortezomib-based regimens are highly effective and reduce the rate of early mortality whereas in combination with ASCT consolidation they prolong OS.

摘要翻译： 

我们对数据库中登记的50例原发性浆细胞白血病（pPCL）患者进行了新型药物疗效及预后影响的研究。其中80%的患者前期接受了基于新型药物的联合治疗方案；40%接受了自体干细胞移植（ASCT）。客观缓解率为76%；38%的患者达到至少非常好的部分缓解（≥vgPR），且该结果与基于硼替佐米的疗法联合ASCT显著相关。截至评估时，已有40例患者死亡。早期死亡率（≤1个月）为6%。中位无进展生存期（PFS）和总生存期（OS）分别为12个月和18个月；接受基于硼替佐米的疗法联合ASCT治疗的患者，其PFS和OS均显著优于其他方案（PFS：18个月 vs 9个月，p=0.004；OS：48个月 vs 14个月，p=0.007）。单变量分析显示，基于硼替佐米的疗法联合ASCT是OS的预测因素。多变量分析中，实现≥vgPR和乳酸脱氢酶（LDH）≥300 U/L是OS的显著预测因子。这些真实世界数据源于全球已报道的最大规模国家级多中心pPCL病例系列，其中大多数患者接受了新型药物治疗，结果证实：在目前获批的诱导治疗方案中，基于硼替佐米的方案疗效显著，可降低早期死亡率，而与ASCT巩固治疗联用更能显著延长OS。

原文链接：

Real-world data on prognosis and outcome of primary plasma cell leukemia in the era of novel agents: a multicenter national study by the Greek Myeloma Study Group