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心脏淀粉样变性患者心脏移植后延迟自体干细胞移植的经验。

Delayed autologous stem cell transplantation following cardiac transplantation experience in patients with cardiac amyloidosis.

机构信息

Division of Cardiology, JC Walter Jr. Transplant Center, Houston Methodist Hospital, Houston, Texas.

Center for Cell and Gene Therapy, Baylor College of Medicine and Houston Methodist Hospital, Houston, Texas.

出版信息

Am J Transplant. 2019 Oct;19(10):2900-2909. doi: 10.1111/ajt.15487. Epub 2019 Jul 1.

Abstract

This study sought to retrospectively investigate the outcomes of patients with light-chain amyloidosis (AL) with advanced cardiac involvement who were treated with a strategy of heart transplantation (HT) followed by delayed autologous stem cell transplantation (ASCT) at 1-year posttransplant. Patients with AL amyloidosis with substantial cardiac involvement have traditionally had very poor survival (eg, several months). A few select centers have reported their outcomes for HT followed by a strategy of early ASCT (ie, 6 months) for CA. The outcomes of patients undergoing a delayed strategy have not been reported. All patients with AL amyloidosis at a single institution undergoing evaluation for HT from 2004-2018 were included. Retrospective analyses were performed. Sixteen patients underwent HT (including two combined heart-kidney transplant) for AL amyloidosis. ASCT was performed in a total of nine patients to date at a median 13.5 months (12.8-32.9 months) post-HT. Survival was 87.5% at 1 year and 76.6% at 5 years, comparable to institutional outcomes for nonamyloid HT recipients. In addition to these 16 patients, two patients underwent combined heart-lung transplantation. A strategy of delayed ASCT 1-year post-HT for patients with AL amyloidosis is feasible, safe, and associated with comparable outcomes to those undergoing an earlier ASCT strategy.

摘要

本研究旨在回顾性调查接受心脏移植(HT)后延迟自体干细胞移植(ASCT)治疗的伴有晚期心脏受累的轻链淀粉样变(AL)患者的结局。心脏受累明显的 AL 淀粉样变患者的传统生存率非常低(例如,数月)。少数精选中心报告了 HT 后早期 ASCT(即 6 个月)治疗 CA 的结果。接受延迟策略的患者的结果尚未报道。对 2004 年至 2018 年期间在单一机构接受 HT 评估的所有 AL 淀粉样变患者进行了回顾性分析。共对 16 例 AL 淀粉样变患者进行了 HT(包括 2 例心脏-肾脏联合移植)。迄今为止,共有 9 例患者共进行了 ASCT,中位时间为 HT 后 13.5 个月(12.8-32.9 个月)。1 年生存率为 87.5%,5 年生存率为 76.6%,与非淀粉样 HT 受者的机构结局相当。除了这 16 例患者,还有 2 例患者接受了心脏-肺联合移植。HT 后 1 年进行延迟 ASCT 治疗 AL 淀粉样变是可行、安全的,与早期 ASCT 策略相比,其结局相当。

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