轻链型肾淀粉样变的治疗回顾和进展  

Review and Progress in the Treatment of Renal Light Chain Amyloidosis

作　　者：史浩[1] SHI Hao(Department of Nephrology,Ruijin Hospital,Shanghai Jiao Tong University School of Medicine,Shanghai 200025,China)

机构地区：[1]上海交通大学医学院附属瑞金医院肾脏科,上海200025

出　　处：《罕见病研究》2024年第1期30-35,共6页Journal of Rare Diseases

基　　金：上海市临床重点专科建设项目(shslczdzk02502)。

摘　　要：轻链型(AL)肾淀粉样变既往预后差,但其治疗在近10余年取得巨大进展,本文回顾该疾病的传统治疗如烷化剂、蛋白酶体抑制剂等,并对近年新进展如单克隆抗体等进行阐述。硼替佐米有效提高AL淀粉样变患者血液学反应率及生存获益,达雷妥尤单抗联合方案带来更快、更深的血液学缓解,增加心、肾等靶器官反应比率。AL肾淀粉样变的治疗籍此也看到了曙光,部分肾病患者在达雷妥尤单抗治疗后可接近完全缓解。自体造血干细胞移植提高血液学和靶器官反应,符合条件患者可作为首选。随着AL肾淀粉样变患者生存改善,血液学反应良好者的肾脏移植治疗是可行选项。Renal light chain amyloidosis(AL amyloidosis)had poor prognosis before the 21st century.However,the treatment of AL amyloidosis has made great progress in the last decade.We reviewed traditional treatments of AL amyloidosis such as alkylating agents,proteasome inhibitors,and recent advances such as monoclonal antibodies.Bortezomib improved the hematological response and survival effectively of the patients,and the combination of Daratumumab brings faster and deeper hematological response,increasing the response rate of target organs such as the kidneys and heart.The renal response was significant higher in the patients with the therapy of Daratumumab,part of them could achieve very good partial response or better renal response.Autologous hematopoietic stem cell transplantation(auto-HSCT)improves hematological as well as organ re-sponse,and could be the first choice among eligible patients.Kidney transplantation is a feasible option for those with good hematological response.

关 键 词：轻链型肾淀粉样变 硼替佐米 达雷妥尤单抗 自体造血干细胞移植 

分 类 号：R692[医药卫生—泌尿科学]