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