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在淀粉样变性患者中,通过优化患者选择以降低自体干细胞移植治疗相关死亡率。

Refinement in patient selection to reduce treatment-related mortality from autologous stem cell transplantation in amyloidosis.

机构信息

Division of Hematology, Mayo Clinic, Rochester, MN 55905, USA.

出版信息

Bone Marrow Transplant. 2013 Apr;48(4):557-61. doi: 10.1038/bmt.2012.170. Epub 2012 Sep 10.

Abstract

This study sought to develop selection guidelines to determine the eligibility for SCT of patients with light-chain amyloidosis. Patients with biopsy-confirmed lightchain amyloidosis who underwent SCT between 8 March 1996 and 31 December 2011 were reviewed in two cohorts by date of transplantation: between 8 March 1996 and 30 June 2009 (n=410) and between 1 July 2009 and 31 December 2011 (n=89). Also evaluated were patients who died before post-transplant day 100 to determine the features predictive of early death. After 1 July 2009, fewer transplant recipients had Mayo stage III cardiac involvement. Mortality before post-transplant day 100 was 10.5% (43/410) in the earlier group and 1.1% (1/89) in the later group. In the earlier group, one-quarter of transplant recipients with N-terminal pro-brain natriuretic peptide (NT-proBNP) >5000 pg/mL died by 10.3 months. When serum troponin T was >0.06 ng/mL, 25% died at 3.7 months. The Mayo staging system is predictive for OS but not useful for selecting transplant recipients. Patients with serum troponin T >0.06 ng/mL or NT-proBNP >5000 pg/mL (not on dialysis) should not be considered candidates for SCT because of early mortality.

摘要

本研究旨在制定筛选指南,以确定轻链淀粉样变性患者接受 SCT 的资格。对 1996 年 3 月 8 日至 2011 年 12 月 31 日期间接受 SCT 的经活检证实为轻链淀粉样变性的患者进行了两个队列的回顾性研究,根据移植日期分为两组:1996 年 3 月 8 日至 2009 年 6 月 30 日(n=410)和 2009 年 7 月 1 日至 2011 年 12 月 31 日(n=89)。还评估了在移植后第 100 天之前死亡的患者,以确定预测早期死亡的特征。在 2009 年 7 月 1 日之后,接受移植的患者中,梅奥 III 期心脏受累的比例较低。在较早的一组中,有 10.5%(43/410)的移植受者在移植后第 100 天之前死亡,而在较晚的一组中,有 1.1%(1/89)的患者死亡。在较早的一组中,四分之一的 N 末端脑钠肽前体(NT-proBNP)>5000 pg/mL 的移植受者在 10.3 个月时死亡。当血清肌钙蛋白 T >0.06ng/mL 时,25%的患者在 3.7 个月时死亡。梅奥分期系统可预测 OS,但对选择移植受者没有帮助。对于血清肌钙蛋白 T >0.06ng/mL 或 NT-proBNP >5000pg/mL(未透析)的患者,由于早期死亡率高,不应考虑作为 SCT 的候选者。

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