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大剂量美法仑与自体外周血干细胞移植治疗AL型淀粉样变性

High dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis.

作者信息

Sanchorawala Vaishali

机构信息

Stem Cell Transplantation Program, Section of Hematology and Oncology, Amyloidosis Center, Boston Medical Center, 820 Harrison Avenue, FGH-1007, Boston, MA 02118, USA.

出版信息

Hematol Oncol Clin North Am. 2014 Dec;28(6):1131-44. doi: 10.1016/j.hoc.2014.08.013. Epub 2014 Sep 30.

Abstract

AL amyloidosis is the most common form of systemic amyloidosis and is associated with an underlying plasma cell dyscrasia. It is often difficult to recognize because of its many manifestations. Recent diagnostic and prognostic advances include the serum-free light chain assay, cardiac MRI, and serologic cardiac biomarkers. Treatment strategies that have evolved during the past decade are prolonging survival and preserving organ function. This article outlines the role of high-dose melphalan and stem cell transplantation. This year marks the 20th anniversary for the first patient who underwent successful stem cell transplantation for this disease at Boston Medical Center.

摘要

AL淀粉样变性是系统性淀粉样变性最常见的形式,与潜在的浆细胞发育异常有关。由于其表现形式多样,往往难以识别。近期诊断和预后方面的进展包括血清游离轻链检测、心脏磁共振成像和血清学心脏生物标志物。过去十年中发展起来的治疗策略正在延长生存期并保护器官功能。本文概述了大剂量美法仑和干细胞移植的作用。今年是波士顿医疗中心首例成功接受该疾病干细胞移植患者的20周年纪念。

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