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轻链淀粉样变性 2012:新纪元。

Light chain amyloidosis 2012: a new era.

机构信息

Department of Haematology, Hadassah Hebrew University Medical Centre, Jerusalem, Israel.

出版信息

Br J Haematol. 2013 Mar;160(5):582-98. doi: 10.1111/bjh.12191. Epub 2013 Jan 7.

Abstract

AL amyloidosis patients with multi-organ and particularly cardiac involvement have historically been considered to have a bad prognosis. The introduction of autologous stem cell transplantation was associated with unacceptable toxicity in high-risk patients, but responding patients have prolonged overall survival. Toxicities can be decreased by careful patient selection, but this reduces the applicability of this treatment modality to a limited number of patients. Efforts are therefore needed to design novel more effective regimens, with the use of new medications, such as thalidomide, lenalidomide and bortezomib, next generation immunomodulatory drugs and proteasome inhibitors. Their combination with dexamethasone and alkylating agents show promising results, allowing a high percentage of remission and subsequent event-free and overall survival, even in a significant proportion of high risk, poor prognosis populations. This review includes the state-of-the-art treatment for AL amyloidosis patients as of 2012, in light of the progress in management of this disease during recent years.

摘要

AL 淀粉样变患者多器官受累,特别是心脏受累,既往认为预后不良。自体干细胞移植的应用与高危患者不可接受的毒性相关,但反应良好的患者总生存时间延长。通过仔细的患者选择可降低毒性,但这将减少这种治疗方式的适用性,使其仅适用于少数患者。因此,需要努力设计新的更有效的方案,使用新的药物,如沙利度胺、来那度胺和硼替佐米、新一代免疫调节剂和蛋白酶体抑制剂。它们与地塞米松和烷化剂联合使用显示出有前景的结果,即使在很大比例的高危、预后不良人群中,也能实现高缓解率以及无事件和总生存。本文综述了 2012 年 AL 淀粉样变患者的最新治疗方法,考虑到近年来该疾病管理方面的进展。

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