文章：

延迟性自体干细胞移植治疗系统性AL淀粉样变性

Deferred autologous stem cell transplantation in systemic AL amyloidosis

原文发布日期：2018-11-05

DOI: 10.1038/s41408-018-0137-9

类型: Article

开放获取: 是

英文摘要：

High-dose melphalan with autologous stem cell transplantation (ASCT) can induce durable haematological and organ responses in systemic AL amyloidosis (AL). Stringent selection criteria have improved safety of ASCT in AL but most patients are transplant-ineligible. We report our experience of deferred ASCT in AL patients who were transplant-ineligible at presentation but had improvements in organ function after induction chemotherapy, enabling them to undergo ASCT. Twenty-two AL patients underwent deferred ASCT from 2011 to 2017. All had serial organ function and clonal response assessment. Organ involvement and responses were defined by amyloidosis consensus criteria. All patients were transplant-ineligible at presentation, predominantly due to advanced cardiac involvement. All received bortezomib-based therapy, with 100% haematologic response (86% complete response (CR)/very good partial response (VGPR)), enabling reversal of ASCT exclusion criteria. Patients underwent deferred ASCT for haematologic progression (45%) or consolidation (55%). There was no transplant-related mortality. Haematologic responses post-ASCT: CR 50%, VGPR 27%, PR 18%, non-response 5%. In all, 85.7% achieved cardiac responses. Median overall survival (OS) was not reached. Median progression-free survival (PFS) was 54 months. This selected cohort achieved excellent haematologic responses, organ responses, PFS and OS with deferred ASCT. If larger studies confirm these findings, this may widen the applicability of ASCT in AL.

摘要翻译： 

大剂量马法兰联合自体干细胞移植（ASCT）可诱导系统性轻链型淀粉样变性（AL）患者获得持久的血液学和器官缓解。严格的筛选标准提高了AL患者接受ASCT的安全性，但多数患者仍不符合移植条件。本文报告了初诊时不符合移植条件、但经诱导化疗后器官功能改善从而能够接受ASCT的AL患者进行延迟移植的经验。2011年至2017年间，22例AL患者接受了延迟ASCT。所有患者均接受系列器官功能和克隆反应评估，器官受累及反应按淀粉样变性共识标准定义。所有患者初诊时均因严重心脏受累等因素不符合移植条件，经硼替佐米为基础的治疗后均获得血液学缓解（完全缓解/非常好的部分缓解率达86%），从而逆转了ASCT排除标准。患者因血液学进展（45%）或巩固治疗（55%）接受延迟ASCT，无移植相关死亡率。移植后血液学缓解率为：完全缓解50%，非常好的部分缓解27%，部分缓解18%，无缓解5%。85.7%患者获得心脏缓解，中位总生存期未达到，中位无进展生存期为54个月。该精选队列通过延迟ASCT获得了优异的血液学缓解、器官缓解、无进展生存和总生存。若更大规模研究证实这些发现，或可拓宽ASCT在AL患者中的适用人群。

原文链接：

Deferred autologous stem cell transplantation in systemic AL amyloidosis