研究动态
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自体干细胞移植治疗 AL 淀粉样变性:浑水。

Autologous stem cell transplantation in AL amyloidosis: Muddy waters.

发表日期:2024 Aug 10
作者: Patrick Hagen, Anita D'Souza
来源: BLOOD REVIEWS

摘要:

免疫球蛋白轻链 (AL) 淀粉样变性是一种恶性浆细胞恶液质,导致多器官发病。高剂量马法兰和自体干细胞移植(ASCT)是首选的巩固方法,并且随着患者选择标准的改进而安全。随着基于硼替佐米和达雷妥尤单抗的诱导治疗的出现,几乎所有患者都能实现深度血液学反应,但基于达雷妥尤单抗的诱导治疗的随访时间很短。因此,诱导后进行 ASCT 的传统方法受到质疑。鉴于 AL 涉及多器官,除反应深度和无血液学进展生存期 (PFS) 之外的终点非常重要。主要器官功能障碍 PFS (MOD-PFS) 增加了 PFS,并且是 PFS、肾脏和心脏器官进展以及总体生存率的复合终点。目前尚不清楚哪种巩固方法(ASCT 或非 ASCT)会在 MOD-PFS 范围内产生改善的结果,最近开放的 S2213 试验将试图回答这个问题。版权所有 © 2024 Elsevier Ltd。保留所有权利。
Immunoglobulin light chain (AL) amyloidosis is a malignant plasma cell dyscrasia causing multi-organ morbidity. High dose melphalan and autologous stem cell transplantation (ASCT) is a preferred consolidation approach and is safe with improved patient selection criteria. With the advent of bortezomib and daratumumab based induction therapy, nearly all patients can achieve deep hematological responses but follow up for daratumumab based induction is short. Consequently, the traditional approach of induction followed by ASCT is called into question. Given the multi-organ involvement of AL, endpoints beyond depth of response and hematological progression free survival (PFS) are important. Major organ dysfunction PFS (MOD-PFS) adds to PFS and is a composite endpoint of PFS, renal and cardiac organ progression, and overall survival. It is currently unknown which consolidative approach (ASCT or non-ASCT) will generate improved outcomes across the MOD-PFS spectrum a question the recently opened S2213 trial will attempt to answer.Copyright © 2024 Elsevier Ltd. All rights reserved.