文章：

AL淀粉样变性的综合综述：一些实用建议

Comprehensive Review of AL amyloidosis: some practical recommendations

原文发布日期：2021-05-18

DOI: 10.1038/s41408-021-00486-4

类型: Review Article

开放获取: 是

英文摘要：

Amyloid light chain (AL) amyloidosis is among the more common and more severe of the amyloidoses usually involving the slow proliferation of a bone-marrow-residing plasma cell (PC) clone and the secretion of unstable immunoglobulin-free light chains (FLC) that infiltrate peripheral tissues and result in detrimental end-organ damage. Disease presentation is rather vague, and the hallmark of treatment is early diagnosis before irreversible end-organ damage. Once diagnosed, treatment decision is transplant-driven whereby ~20% of patients are eligible for autologous stem cell transplantation (ASCT) with or without bortezomib-based induction. In the setting of ASCT-ineligibility, bortezomib plays a central role in upfront treatment with the recent addition of daratumumab to the current emerging standard of care. In general, management of AL amyloidosis is aimed at achieving deep, durable responses with very close monitoring for early detection of relapse/refractory disease. This article provides a comprehensive review of the management of patients with AL amyloidosis including goals of therapy, current treatment guidelines in the setting of both ASCT-eligibility and ineligibility, treatment response monitoring recommendations, toxicity management, and treatment of relapse/refractory disease.
 

摘要翻译： 

淀粉样轻链（AL）淀粉样变性是较常见且较严重的淀粉样变性类型，通常涉及骨髓中浆细胞克隆的缓慢增殖，以及不稳定的免疫球蛋白游离轻链的分泌。这些游离轻链浸润外周组织，导致有害的终末器官损伤。该疾病的临床表现较为隐匿，治疗的关键在于在不可逆的终末器官损伤发生前实现早期诊断。一旦确诊，治疗决策以移植为导向，约20%的患者适合接受自体干细胞移植（ASCT），并可能联合或不联合基于硼替佐米的诱导治疗。对于不适合ASCT的患者，硼替佐米在前线治疗中占据核心地位，且近期达雷妥尤单抗已被纳入当前新兴的标准治疗方案。总体而言，AL淀粉样变性的治疗目标在于获得深度且持久的缓解，并通过密切监测早期发现复发或难治性疾病。本文全面综述了AL淀粉样变性的患者管理，包括治疗目标、适合与不适合ASCT的当前治疗指南、治疗反应监测建议、毒性管理以及复发或难治性疾病的治疗策略。

原文链接：

Comprehensive Review of AL amyloidosis: some practical recommendations