文章：

维奈托克在t(11;14)复发/难治性AL淀粉样变性中诱导深度血液学缓解

Venetoclax induces deep hematologic remissions in t(11;14) relapsed/refractory AL amyloidosis

原文发布日期：2021-01-11

DOI: 10.1038/s41408-020-00397-w

类型: Article

开放获取: 是

英文摘要：

Venetoclax is efficacious in relapsed/refractory t(11;14) multiple myeloma, thus warranting investigation in light-chain amyloidosis (AL). This retrospective cohort includes 43 patients with previously treated AL, from 14 centers in the US and Europe. Thirty-one patients harbored t(11;14), 11 did not, and one t(11;14) status was unknown. Patients received a venetoclax-containing regimen for at least one 21- or 28-day cycle; the median prior treatments was three. The hematologic response rate for all patients was 68%; 63% achieved VGPR/CR. t(11;14) patients had higher hematologic response (81% vs. 40%) and higher VGPR/CR rate (78% vs. 30%, odds ratio: 0.12, 95% CI 0.02–0.62) than non-t(11;14) patients. For the unsegregated cohort, median progression-free survival (PFS) was 31.0 months and median OS was not reached (NR). For t(11;14), median PFS was NR and for non-t(11;14) median PFS was 6.7 months (HR: 0.14, 95% CI 0.04–0.53). Multivariate analysis incorporating age, sex, prior lines of therapy, and disease stage suggested a risk reduction for progression or death in t(11;14) patients. Median OS was NR for either subgroup. The organ response rate was 38%; most responders harbored t(11;14). Grade 3 or higher adverse events occurred in 19% with 7% due to infections. These promising results require confirmation in a randomized clinical trial.
 

摘要翻译： 

维奈克拉在复发/难治性t(11;14)多发性骨髓瘤中表现显著疗效，因此值得在轻链淀粉样变性病（AL）中开展研究。本回顾性队列纳入来自美国和欧洲14个中心的43例既往接受过治疗的AL患者，其中31例携带t(11;14)，11例未携带，1例t(11;14)状态未知。患者接受至少一个周期（21天或28天）的含维奈克拉方案治疗；既往中位治疗线数为三线。全体患者的血液学缓解率为68%；63%达到非常好的部分缓解或完全缓解。与非t(11;14)患者相比，t(11;14)患者血液学缓解率更高（81% vs. 40%），非常好的部分缓解或完全缓解率也更高（78% vs. 30%，比值比：0.12，95%置信区间0.02–0.62）。整体队列中位无进展生存期为31.0个月，中位总生存期尚未达到。t(11;14)患者中位无进展生存期尚未达到，非t(11;14)患者为6.7个月（风险比：0.14，95%置信区间0.04–0.53）。结合年龄、性别、既往治疗线数和疾病分期的多变量分析显示，t(11;14)患者的疾病进展或死亡风险降低。两组中位总生存期均未达到。器官缓解率为38%；多数缓解者携带t(11;14)。19%患者发生3级及以上不良事件，其中7%为感染相关。这些积极结果仍需随机临床试验进一步验证。

原文链接：

Venetoclax induces deep hematologic remissions in t(11;14) relapsed/refractory AL amyloidosis