文章：

早期缓解对初治硼替佐米治疗后AL淀粉样变患者预后的影响

Impact of early response on outcomes in AL amyloidosis following treatment with frontline Bortezomib

原文发布日期：2021-06-21

DOI: 10.1038/s41408-021-00510-7

类型: Article

开放获取: 是

英文摘要：

The outcomes in systemic AL amyloidosis are dependent on the depth of haematologic response. However, there is limited data on the impact of the speed of response on outcomes. Here we report the impact of speed of response in a cohort of AL patients treated with upfront Bortezomib. Patients seen from February 2010 until August 2019 are included in the present analysis. 1194 & 1133 patients comprised the ITT and 1-month landmark cohorts. In the landmark cohort, 137 (11.5%), 270 (22.6%), 252 (21.1%) and 352 (31.1%) patients had a CR, VGPR, PR and NR at 1-month. Patients with ≥ VGPR at 1-month had significantly better survival (median not reached; at the end of 1, 2, 5,10 years, 87%/92%, 83%/87%, 68%/72% and 63%/58% of patients in CR/VGPR, respectively, were alive) compared to those with a PR (median OS 60 months) or NR (median OS 32 months) (p < 0.005). At 1-month, patients with CR and iFLC < 20 mg/l had a significantly better survival compared to CR and iFLC > 20 mg/l (p = 0.005). Reaching ≥ VGPR at 1-month significantly improved survival in all Mayo disease stages. In conclusion, patients achieving an early deep haematologic response have a significantly superior survival irrespective of cardiac involvement.
 

摘要翻译： 

系统性AL淀粉样变性的结局取决于血液学缓解的深度。然而，关于缓解速度对预后影响的数据较为有限。本研究报道了接受前线硼替佐米治疗的AL淀粉样变性患者队列中缓解速度的影响。纳入2010年2月至2019年8月期间就诊的患者进行分析。意向治疗分析队列及1个月里程碑分析队列分别包含1194例和1133例患者。在里程碑队列中，137例（11.5%）、270例（22.6%）、252例（21.1%）和352例（31.1%）患者分别在1个月时达到完全缓解、非常好的部分缓解、部分缓解及未缓解。与部分缓解（中位总生存期60个月）或未缓解（中位总生存期32个月）的患者相比，1个月时达到≥非常好的部分缓解的患者生存期显著更优（中位生存期未达到；在1、2、5、10年时，完全缓解/非常好的部分缓解组分别有87%/92%、83%/87%、68%/72%和63%/58%的患者存活）（p < 0.005）。在1个月时，达到完全缓解且受累游离轻链＜20mg/l的患者生存期显著优于完全缓解但受累游离轻链＞20mg/l的患者（p = 0.005）。在所有梅奥疾病分期中，1个月内达到≥非常好的部分缓解均能显著改善生存。综上所述，无论是否存在心脏受累，获得早期深度血液学缓解的患者生存预后均显著更优。

原文链接：

Impact of early response on outcomes in AL amyloidosis following treatment with frontline Bortezomib