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70 至 75 岁免疫球蛋白轻链淀粉样变性患者的自体干细胞移植。

Autologous Stem Cell Transplant for Immunoglobulin Light Chain Amyloidosis Patients Aged 70 to 75.

机构信息

Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota.

Division of Nephrology, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota.

出版信息

Biol Blood Marrow Transplant. 2018 Oct;24(10):2157-2159. doi: 10.1016/j.bbmt.2018.06.017. Epub 2018 Jun 20.

Abstract

Autologous stem cell transplantation (ASCT) has been used in treatment for immunoglobulin light chain (AL) amyloidosis for over 2 decades and is generally reserved for patients younger than 70 years. Herein we report on outcomes of ASCT in a cohort of patients with AL amyloidosis aged 70 years or older. Between August of 2002 and April of 2017, 34 patients aged 70 years or older, with biopsy-proven AL amyloidosis, received an ASCT at the Mayo Clinic Rochester. Seventy percent of patients (n = 24) were transplanted within 6 months of diagnosis, and 74% (n = 25) received reduced-intensity conditioning with melphalan <200 mg/m. Sixty-five percent of patients (n = 22) required hospitalization with a median duration of hospital admission of 8 days. Common reasons for hospitalization included fever or infection (14%), cardiac arrhythmia (14%), nutritional support (24%), and volume overload (19%). Overall response rate was 75%, with a complete response seen in 25% of patients. Overall survival and progression-free survival for the cohort were 66 months and 40 months, respectively. One patient died within 100 days of transplant, representing a 3% 100-day mortality rate. ASCT is safe and efficacious in carefully screened patients aged 70 or above.

摘要

自体干细胞移植 (ASCT) 在免疫球蛋白轻链 (AL) 淀粉样变性的治疗中已经使用了超过 20 年,通常保留给年龄小于 70 岁的患者。在此,我们报告了在梅奥诊所罗切斯特接受 ASCT 的一组年龄在 70 岁或以上的 AL 淀粉样变性患者的结果。在 2002 年 8 月至 2017 年 4 月期间,34 名年龄在 70 岁或以上、经活检证实为 AL 淀粉样变性的患者在梅奥诊所罗切斯特接受了 ASCT。70%的患者(n=24)在诊断后 6 个月内接受移植,74%(n=25)接受了强度低于 200mg/m 的美法仑的减强度预处理。65%的患者(n=22)需要住院治疗,中位住院时间为 8 天。住院的常见原因包括发热或感染(14%)、心律失常(14%)、营养支持(24%)和容量超负荷(19%)。总体缓解率为 75%,25%的患者达到完全缓解。该队列的总生存率和无进展生存率分别为 66 个月和 40 个月。1 名患者在移植后 100 天内死亡,代表 3%的 100 天死亡率。ASCT 在仔细筛选的 70 岁及以上患者中是安全有效的。

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