轻链淀粉样变性的诊治更新及存在的问题  

Diagnosis and treatment update of light chain amyloidosis and the existing problems

作　　者：黄仲夏[1] 胡婉莉 

机构地区：[1]首都医科大学附属北京朝阳医院西院血液与肿瘤科,100043

出　　处：《白血病．淋巴瘤》2018年第2期76-79,共4页Journal of Leukemia & Lymphoma

基　　金：北京市石景山区医学重点扶持专科建设项目

摘　　要：轻链淀粉样变性（AL）主要是免疫球蛋白轻链错误折叠形成的淀粉样物质,引起人体多脏器损害的一种克隆性浆细胞病,其发生机制不清楚.临床上通常以患者心、肾等某一脏器衰竭为突出表现.治疗主要为美法仑联合地塞米松或硼替佐米等靶向新药化疗或进行自体干细胞移植治疗.疗效的判断不仅以血清游离轻链及M蛋白水平判断血液学反应,更要以氨基末端B型脑钠肽前体和肌钙蛋白I判断心脏等重要脏器功能改善情况.在新药时代,心脏受累是生存和预后的决定因素.AL的现代治疗,不仅要给予抗浆细胞的新药靶向治疗,更要结合抗淀粉样变治疗,以清除AL的伴侣蛋白.尽管治疗上有很多可喜进展,但也有不少问题亟待解决.Light chain amyloidosis （AL） is a clonal plasma cell disease in which multiple organs of human body are damaged by amyloid misfolded by light chain of immunoglobulin. Its mechanism is still unclear. Clinically, heart failure, renal insufficiency and other organs failure are seen as prominent symptoms for the majority of patients. The main treatments include melphalan combined with dexamethasone or bortezomib or other target new drugs or autologous stem cell transplantation. Efficacy assessment depends on not only the serum free light chain and M protein levels to judge the hematological response, but also pro-brain natriuretic peptide and troponin I as biomarker to assess the heart and other vital organs function for better outcomes. In the era of new drugs, cardiac involvement is a determinant of survival and prognosis of AL patients. The modern AL treatment usually takes new drug targeted therapy against plasma cells, and combines with anti-amyloidosis treatment to clear the accumulation of amyloidosis chaperonin. Despite the promising advances in treatment, many problems need to be resolved.

关 键 词：轻链 淀粉样变性 预后 抗浆细胞疗法 抗淀粉样变疗法 

分 类 号：R597.2[医药卫生—内科学]