Gray Gilstrap Lauren, Niehaus Emily, Malhotra Rajeev, Ton Van-Khue, Watts James, Seldin David C, Madsen Joren C, Semigran Marc J
Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts.
Division of Cardiology, Department of Medicine, Johns Hopkins Medical Institutes, Baltimore, Maryland.
J Heart Lung Transplant. 2014 Feb;33(2):149-56. doi: 10.1016/j.healun.2013.09.004. Epub 2013 Nov 5.
Orthotopic heart transplant (OHT), followed by myeloablative chemotherapy and autologous stem cell transplant (ASCT), has been successful in the treatment of amyloid light-chain (AL) cardiac amyloidosis. The purpose of this study was to identify predictors of survival to OHT in patients with end-stage heart failure due to AL amyloidosis and compare post-OHT survival of cardiac amyloid patients with survival of other cardiomyopathy patients undergoing OHT.
From January 2000 to June 2011, 31 patients with end-stage heart failure secondary to AL amyloidosis were listed for OHT at Massachusetts General Hospital. Univariate and multivariate regression analyses identified predictors of survival to OHT. Kaplan-Meier analysis compared survival between the Massachusetts General Hospital amyloidosis patients and non-amyloid cardiomyopathy patients from the Scientific Registry of Transplant Recipients (SRTR).
Low body mass index was the only predictor of survival to OHT in patients with end-stage heart failure caused by cardiac amyloidosis. Survival of cardiac amyloid patients who died before receiving a donor heart was only 63 ± 45 days after listing. Patients who survived to OHT received a donor organ at 53 ± 48 days after listing. Survival of AL amyloidosis patients on the waiting list was less than patients on the waiting list for all other non-amyloid diagnoses. The long-term survival of amyloid patients who underwent OHT was no different than the survival of non-amyloid, restrictive (p = 0.34), non-amyloid dilated (p = 0.34), or all non-amyloid cardiomyopathy patients (p = 0.22) in the SRTR database.
Amyloid patients who survive to OHT, followed by ASCT, have a survival rate similar to other cardiomyopathy patients undergoing OHT; however, 35% of the patients died awaiting OHT. The only predictor of survival to OHT in AL amyloidosis patients was a low body mass index, which correlated with a shorter time on the waiting list. To optimize the survival of these patients, access to donor organs must be improved.
原位心脏移植（OHT），随后进行清髓性化疗和自体干细胞移植（ASCT），已成功用于治疗轻链（AL）型心脏淀粉样变性。本研究的目的是确定因AL淀粉样变性导致终末期心力衰竭患者接受OHT后的生存预测因素，并比较心脏淀粉样变性患者OHT后的生存率与其他接受OHT的心肌病患者的生存率。
2000年1月至2011年6月，31例因AL淀粉样变性继发终末期心力衰竭的患者在马萨诸塞州总医院被列入OHT名单。单因素和多因素回归分析确定了接受OHT的生存预测因素。Kaplan-Meier分析比较了马萨诸塞州总医院淀粉样变性患者与移植受者科学注册系统（SRTR）中的非淀粉样变性心肌病患者的生存率。
低体重指数是心脏淀粉样变性导致终末期心力衰竭患者接受OHT的唯一生存预测因素。在列入名单后，未接受供体心脏就死亡的心脏淀粉样变性患者的生存期仅为63±45天。存活至接受OHT的患者在列入名单后53±48天接受了供体器官。在等待名单上，AL淀粉样变性患者的生存率低于所有其他非淀粉样变性诊断的患者。在SRTR数据库中，接受OHT的淀粉样变性患者的长期生存率与非淀粉样变性、限制性（p = 0.34）、非淀粉样变性扩张性（p = 0.34）或所有非淀粉样变性心肌病患者（p = 0.22）的生存率没有差异。
存活至接受OHT并随后接受ASCT的淀粉样变性患者的生存率与其他接受OHT的心肌病患者相似；然而，35%的患者在等待OHT期间死亡。AL淀粉样变性患者接受OHT的唯一生存预测因素是低体重指数，这与等待名单上的时间较短相关。为了优化这些患者的生存，必须改善供体器官的获取。
