原发型心肌淀粉样变的治疗进展

程仙; 张刘燕; 许菲; 徐东杰

doi:10.13201/j.issn.1001-1439.2017.05.003

原发型心肌淀粉样变的治疗进展

- 南京医科大学第一附属医院心内科(南京, 210026)
基金项目:
江苏高校优势学科建设工程一期项目[No：XK05200903（NG09）]

详细信息

通讯作者: 徐东杰,E-mail:djxu@njmu.edu.cn

中图分类号: R542.2

收稿日期: 2016-11-14

修回日期: 2017-01-03

An update for treatment of primary cardiac amyloidosis

- Department of Cardiology, The First Affiliated Hospital With Nanjing Medical University, Nanjing, 210026, China

More Information

Corresponding author: XU Dongjie, E-mail: djxu@njmu.edu.cn

Received Date: 14 November 2016

Revised Date: 03 January 2017

摘要

摘要: 原发型即免疫球蛋白轻链型(AL型)淀粉样变是最常见的心肌淀粉样变类型,早期诊断技术及治疗方案的进展,极大改善了原发型心肌淀粉样变患者的预后。自体外周造血干细胞移植可显著延长患者的生存期限,但干细胞移植仅适用于一部分患者。目前马法兰联合地塞米松方案因其良好的耐受性,已成为不适合移植患者的一线方案。近来,蛋白酶体抑制剂及免疫调节剂为复发难治或无法移植的原发型心肌淀粉样变患者带来了新的希望。
- 心肌淀粉样变 /
- 干细胞移植 /
- 马法兰 /
- 地塞米松
Abstract: Primary cardiac amyloidosis is the most common type of cardiac amyloidosis. The development of early diagnosis and therapy greatly improve the prognosis of primary cardiac amyloidosis.Autologous hematopoietic stem cell transplantation can obviously prolong the survival time of patients,but is suitable for a part of patients.Because of its well tolerance, the combination of melphalan and dexamethasone has been the first-line treatment for patients who are not suitable for stem cell transplantation. In addition, proteasome inhibitors and immune regulator play an important role in treatment of refractory primary cardiac amyloidosis.
- cardiac amyloidosis /
- stem cell transplantation /
- melphalan /
- dexamethasone

HTML全文

参考文献(27)

[1]	徐东杰,周芳,张海峰,等.关注心肌淀粉样变的诊断线索[J].临床心血管病杂志,2014,30(2):93-94.
[2]	毛歆歆,崔全才.心肌淀粉样变性的研究及进展[J].诊断病理学杂志,2011,18(6):468-471.
[3]	DISPENZIERI A, GERTZ M A, KYLE R A, et al.Serum cardiac troponins and N-terminal pro-brain natriuretic peptide:a staging system for primary systemic amyloidosis[J].J Clin Oncol,2004,22:3751-3757.
[4]	NUVOLONE M,MERLINI G.Systemic amyloidosis:novel therapies and role of biomarkers[J].Nephrol Dial Transplant,2016[Epub ahead of print]
[5]	KUMAR S, DISPENZIERI A, LACY M Q, et al.Revised prognostic staging system for light chain amyloidosis incorporating cardiac biomarkers and serum free light chain measurements[J].J Clin Oncol,2012,30:989-995.
[6]	PALLADINI G, FOLI A, MILANI P, et al.Best use of cardiac biomarkers in patients with AL amyloidosis and renal failure[J].Am J Hematol,2012,87:465-471.
[7]	GOODMAN H J, GILLMORE J D, LACHMANN H J, et al.Outcome of autologous stem cell transplantation for AL amyloidosis in the UK[J].Br J Haematol,2006,134:417-425.
[8]	黄湘华.自体干细胞移植治疗AL淀粉样变性及新型诱导方案的研究[D].南京大学,2012.
[9]	DISPENZIERI A, GERTZ M A,BUADI F.What do I need to know about immunoglobulin light chain (AL) amyloidosis?[J].Blood Rev,2012,26:137-154.
[10]	沈恺妮,李剑.原发性轻链型淀粉样变的治疗进展[J].中国实验血液学杂志,2015,23(3):910-914.
[11]	HWA Y L, KUMAR S K, GERTZ M A, et al.Induction therapy pre-autologous stem cell transplantation in immunoglobulin light chain amyloidosis:a retrospective evaluation[J].Am J Hematol,2016,91:984-988.
[12]	章友康,姚英.重视AL型肾淀粉样变的规范治疗[J].中华肾病研究电子杂志,2014,3(2):60-64.
[13]	PALLADINI G, RUSSO P, NUVOLONE M, et al.Treatment with oral melphalan plus dexamethasone produces long-term remissions in AL amyloidosis[J]. Blood,2007,110:787-788.
[14]	PALLADINI G, RUSSO P, LAVATELLI F, et al.Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide[J].Ann Hematol,2009,88:347-350.
[15]	SANCHORAWALA V, SUN F, QUILLEN K, et al.Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem-cell transplantation[J].Blood,2007,110:3561-3563.
[16]	SHIMOJIMA Y,MATSUDA M,ISHII W, et al. High-dose melphalan followed by autologous stem cell support in primary systemical amyloidosis with multiple organ involvement[J].Intern Med,2005,44:484-489.
[17]	WECHALEKAR A D, LACHMANN H J, OFFER M, et al. Efficacy of bortezomib in systemic AL amyloidosis with relapsed/refractory clonal disease[J]. Haematologica,2008,93:295-298.
[18]	LANDAU H, HASSOUN H, ROSENZWEIG M A, et al.Bortezomib and dexamethasone consolidation following risk-adapted melphalan and stem cell transplantation for patients with newly diagnosed light-chain amyloidosis[J]. Leukemia,2013,27:823-828.
[19]	KASTRITIS E, WECHALEKAR A D, DIMOPOULOS M A, et al.Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis[J].J Clin Oncol,2010,28:1031-1037.
[20]	陈文萃.AL型淀粉样变性的治疗.[J].肾脏病与透析肾移植杂志,2012,21(6):561-567.
[21]	PATEL K S,HAWKINS P N. Cardiac amyloidosis:where are we today?[J].J Intern Med,2015,278:126-144.
[22]	VENNER C P, LANE T, FOARD D, et al. Cyclophosphamide, bortezomib, and dexamethasone therapy inALamyloidosis is associated with high clonal response rates and prolonged progression-free survival[J].Blood,2012,119:4387-4390.
[23]	OFFIDANI M, CORVATTA L, CARAFFA P, et al.An evidence-based review of ixazomib citrate and its potential in the treatment of newly diagnosed multiple myeloma[J].Onco Targets Ther,2014,7:1793-1800.
[24]	DISPENZIERI A, LACY M Q,ZELDENRUST S R,et al.The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis[J].Blood,2007,109:465-470.
[25]	SANCHORAWALA V, WRIGHT D G, ROSENZWEIG M,et al. Lenalidomide and dexamethasone in the treatment of ALamyloidosis:results of a phase 2 trial[J].Blood,2007,109:492-496.
[26]	DISPENZIERI A,BUADI F,LAUMANN K,et al. Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis[J].Blood,2012,119:5397-5404.
[27]	PALLADINI G, DISPENZIERI A, GERTZ M A, et al.New criteria for response to treatment in immunoglobulin light chain amyloidosis based on free light chain measurement and cardiac biomarkers:impact on survival outcomes[J].J Clin Oncol,2012,30:4541-4549.