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原发性系统性 AL 淀粉样变性的自体移植在大型淀粉样变性转诊中心之外是可行的:卡尔加里骨髓移植项目的经验。

Autologous transplantation for primary systemic AL amyloidosis is feasible outside a major amyloidosis referral centre: the Calgary BMT Program experience.

作者信息

Chow L Q M, Bahlis N, Russell J, Chaudhry A, Morris D, Brown C, Stewart D A

机构信息

Departments of Medicine and Oncology, Foothills Hospital, University of Calgary, Calgary, Alberta, Canada.

出版信息

Bone Marrow Transplant. 2005 Oct;36(7):591-6. doi: 10.1038/sj.bmt.1705112.

DOI:10.1038/sj.bmt.1705112
PMID:16062177
Abstract

Recent reports from large amyloidosis referral centers suggest that primary systemic AL amyloidosis patients treated with high-dose melphalan (HDM) and autologous stem cell transplantation (ASCT) survive longer than historical controls treated with less intensive chemotherapy, despite high transplant-related mortality (TRM) rates of >10%. A retrospective review was conducted to determine if the outcome of ASCT for AL amyloidosis at our institution was similar to that reported at major amyloidosis referral centers. Over a 7 year period, we treated a total of 15 AL amyloidosis patients with ASCT, including four with poor prognosis cardiac or multisystem involvement. No TRM was observed. Overall, 10 patients (67%) achieved a complete hematological response and four patients (27%) achieved a complete organ response. The 4-year event-free and overall survival rates were 60% (95% CI 32-89%) and 75% (95% CI 50-100%), respectively. One patient, who presented with cardiac failure and multiorgan involvement with colonic bleeding currently remains in complete remission 62 months post-ASCT. In conclusion, ASCT for primary AL amyloidosis can safely be performed at experienced transplant centers that are not associated with major amyloidosis referral centers, and is feasible for patients who have multisystem involvement, particularly for motivated patients with good performance status.

摘要

大型淀粉样变性转诊中心最近的报告表明,接受大剂量美法仑(HDM)和自体干细胞移植(ASCT)治疗的原发性系统性AL淀粉样变性患者比接受强度较低化疗的历史对照患者存活时间更长,尽管移植相关死亡率(TRM)高于10%。我们进行了一项回顾性研究,以确定我院AL淀粉样变性患者接受ASCT的结果是否与主要淀粉样变性转诊中心报告的结果相似。在7年的时间里,我们共对15例AL淀粉样变性患者进行了ASCT治疗,其中4例患者预后较差,伴有心脏或多系统受累。未观察到TRM。总体而言,10例患者(67%)实现了完全血液学缓解,4例患者(27%)实现了完全器官缓解。4年无事件生存率和总生存率分别为60%(95%CI 32-89%)和75%(95%CI 50-100%)。1例出现心力衰竭、多器官受累并伴有结肠出血的患者在ASCT后62个月仍处于完全缓解状态。总之,原发性AL淀粉样变性的ASCT可以在与主要淀粉样变性转诊中心无关的经验丰富的移植中心安全进行,对于多系统受累的患者,特别是对于身体状况良好且有积极性的患者是可行的。

相似文献

1
Autologous transplantation for primary systemic AL amyloidosis is feasible outside a major amyloidosis referral centre: the Calgary BMT Program experience.原发性系统性 AL 淀粉样变性的自体移植在大型淀粉样变性转诊中心之外是可行的:卡尔加里骨髓移植项目的经验。
Bone Marrow Transplant. 2005 Oct;36(7):591-6. doi: 10.1038/sj.bmt.1705112.
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引用本文的文献

1
Role of high-dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis.大剂量美法仑及自体外周血干细胞移植在 AL 型淀粉样变性中的作用
Am J Blood Res. 2012;2(1):9-17. Epub 2012 Jan 1.
2
Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients.大剂量美法仑和自体干细胞移植后 AL 淀粉样变性的结果:一系列 421 例患者的长期结果。
Blood. 2011 Oct 20;118(16):4346-52. doi: 10.1182/blood-2011-01-330738. Epub 2011 Aug 9.
3
Current treatment in cardiac amyloidosis.
心脏淀粉样变性的当前治疗方法。
Curr Treat Options Cardiovasc Med. 2006 Dec;8(6):468-73. doi: 10.1007/s11936-006-0035-5.