采用常规化疗或大剂量化疗和自体造血干细胞移植治疗系统性轻链 AL 淀粉样变性的单中心患者队列。

A single-centre cohort of patients with systemic light chain AL-amyloidosis treated with conventional chemotherapy or with high-dose chemotherapy and autologous stem cell transplantation.

机构信息

Department of Medical Oncology, University Hospital and University of Berne, Switzerland;

出版信息

Swiss Med Wkly. 2014 Feb 19;144:w13922. doi: 10.4414/smw.2014.13922.

DOI:10.4414/smw.2014.13922

PMID:24554372

Abstract

BACKGROUND

High-dose chemotherapy (HDCT) with autologous stem cell transplantation (ASCT) has been reported to confer better prognosis in systemic light chain AL-amyloidosis as compared with conventional chemotherapy. However, only limited data are available so far on treatment and outcome of AL-amyloidosis patients in Switzerland.

METHODS

Within a single-centre cohort of patients with biopsy confirmed AL-amyloidosis diagnosed between January 1995 and December 2012, we aimed to investigate treatment effects in patients treated with conventional chemotherapy versus HDCT with ASCT.

RESULTS

We identified 50 patients with AL-amyloidosis treated with conventional chemotherapy and 13 patients who received HDCT with ASCT. Clinical characteristics differed between the groups for the age of the patients (59 years for patients with ASCT/HDCT vs 69 years; p= 0.0006) and the troponin-T value (0.015 μg/l vs 0.08 μg/l; p = 0.0279). Patients with ASCT showed a trend towards better overall survival, with median survival not yet reached compared with 53 months in patients on conventional chemotherapy (p = 0.0651).

CONCLUSION

Our results suggest that light chain AL-amyloidosis patients considered fit to undergo HDCT and ASCT may have a better outcome than patients treated exclusively with conventional chemotherapy regimens; however, the better performance status of patients receiving HDCT may have added to this treatment effect.

摘要

背景

与常规化疗相比，大剂量化疗（HDCT）联合自体干细胞移植（ASCT）已被报道可改善系统性轻链 AL 淀粉样变性的预后。然而，迄今为止，瑞士关于 AL 淀粉样变性患者治疗和结局的数据有限。

方法

在 1995 年 1 月至 2012 年 12 月间确诊的经活检证实的 AL 淀粉样变性患者的单中心队列中，我们旨在调查接受常规化疗与 HDCT+ASCT 治疗的患者的治疗效果。

结果

我们确定了 50 例接受常规化疗的 AL 淀粉样变性患者和 13 例接受 HDCT+ASCT 的患者。两组患者的临床特征存在差异，年龄（ASCT/HDCT 组为 59 岁，常规化疗组为 69 岁；p=0.0006）和肌钙蛋白 T 值（0.015μg/l 与 0.08μg/l；p=0.0279）。接受 ASCT 的患者总体生存情况有改善的趋势，中位生存时间尚未达到，而接受常规化疗的患者为 53 个月（p=0.0651）。