Background

Autologous hematopoietic cell transplantation (HCT) is a first-line therapy for prolonging survival in patients with light-chain (AL) amyloidosis. Cardiac involvement is the most important determinant of survival. However, patients with advanced cardiac involvement have often been excluded from HCT because of high risk for transplantation-related mortality and poor overall survival. Whether baseline left ventricular global longitudinal strain (GLS) can provide additional risk stratification and predict survival after HCT in this high-risk population remains unclear. The aim of this study was to evaluate the prognostic implication of baseline GLS and the added value of GLS beyond circulating cardiac biomarkers for risk stratification in patients with AL amyloidosis undergoing HCT.

Methods

Eighty-two patients with newly diagnosed AL amyloidosis who underwent upfront HCT between January 2007 and April 2014 were included in the study. Clinical, echocardiographic, and serum cardiac biomarker data were collected at baseline and 12 months following HCT. GLS measurements were performed using a vendor-independent offline system. The median follow-up time for survivors was 58 months.

Results

Sixty-four percent of patients were in biomarker-based Mayo stage II or III. GLS, brain natriuretic peptide, troponin, and mitral E/A ratio were identified as the strongest predictors of survival (P < .0001). Other predictors included sex, creatinine, free AL, wall thickness, and ejection fraction. Mayo stage was significantly associated with outcome, with 5-year survival of 93%, 72% and 31% in stage I, II, and III patients, respectively. GLS of 17% was identified as the value that best discriminated survivors from nonsurvivors, and the application of this cutoff value provided further mortality risk stratification within each Mayo stage.

Conclusions

GLS is a strong predictor of survival in patients with AL amyloidosis undergoing HCT, potentially providing incremental value over serum cardiac biomarkers for risk stratification. GLS should be considered as a standard parameter along with serum cardiac biomarkers when evaluating eligibility for HCT or other investigational therapies.

中文翻译：

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二维全球纵向应变对自体造血细胞移植后轻链淀粉样变性患者生存率的预测和附加价值

背景

自体造血细胞移植（HCT）是一线治疗，可延长轻链（AL）淀粉样变性病患者的生存期。心脏受累是生存的最重要决定因素。但是，由于与移植相关的死亡率高风险和较差的总生存率，心脏高级受累患者通常被排除在HCT之外。基线左心室总体纵向张力（GLS）是否可以提供额外的危险分层，并预测高危人群中HCT后的存活率。这项研究的目的是评估基线GLS的预后意义以及超出循环心脏生物标志物的GLS对HCT的AL淀粉样变性患者的危险分层的预后价值。

方法

该研究纳入了2007年1月至2014年4月间接受HCT预先诊断的82例新诊断为AL淀粉样变性的患者。在基线和HCT后12个月收集临床，超声心动图和血清心脏生物标志物数据。GLS测量是使用与供应商无关的离线系统进行的。幸存者的中位随访时间为58个月。

结果

64％的患者处于基于生物标记物的Mayo II或III期。GLS，脑利钠肽，肌钙蛋白和二尖瓣E / A比被确定为存活率的最强预测因子（P  <.0001）。其他预测因素包括性别，肌酐，游离AL，壁厚和射血分数。Mayo分期与预后显着相关，I，II和III期患者的5年生存率分别为93％，72％和31％。17％的GLS被确定为最佳区分幸存者和非幸存者的值，该临界值的应用为每个Mayo阶段的死亡风险分层提供了进一步的依据。

结论

GLS是接受HCT的AL淀粉样变性患者生存的有力预测指标，可能为风险分层提供比血清心脏生物标志物更高的价值。在评估HCT或其他研究疗法的资格时，应将GLS与血清心脏生物标志物一起视为标准参数。