National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London, United Kingdom.
Centre for Diagnosis and Treatment of Cardiomyopathies, Cardiovascular Department, Azienda Sanitaria Universitaria Giuliano-Isontina (ASUGI), University of Trieste, Trieste, Italy.
JAMA Cardiol. 2024 Nov 1;9(11):982-989. doi: 10.1001/jamacardio.2024.2555.
Cardiac amyloid infiltration is the key determinant of survival in systemic light-chain (AL) amyloidosis. Current guidelines recommend early switching therapy in patients with a nonoptimal or suboptimal response regardless of the extent of cardiac amyloid infiltration.
To assess the differences between serum biomarkers, echocardiography, and cardiovascular magnetic resonance (CMR) with extracellular volume (ECV) mapping in characterizing cardiac amyloid, the independent prognostic role of these approaches, and the role of ECV mapping to guide treatment strategies.
DESIGN, SETTING, AND PARTICIPANTS: Consecutive patients newly diagnosed with systemic AL amyloidosis (2015-2021) underwent echocardiography, cardiac biomarkers, and CMR with ECV mapping at diagnosis. Data were analyzed from January to June 2024.
The primary outcomes of the study were all-cause mortality and hematological response as defined according to validated criteria: no response (NR), partial response (PR), very good partial response (VGPR), and complete response (CR). Secondary outcomes were the depth and speed of hematological response and overall survival according to ECV.
Of 560 patients with AL amyloidosis, the median (IQR) age was 68 years (59-74 years); 346 patients were male (61.8%) and 214 female (38.2%). Over a median (IQR) 40.5 months 9-58 months), ECV was independently associated with mortality. In the landmark analysis at 1 month, long-term survival was independent of the achieved hematological response in ECV less than 0.30% and ECV of 0.31% to 0.40%, while it was dependent on the depth of the hematological response in ECV greater than 0.40%. In the landmark analysis at 6 months, survival was independent of the achieved hematological response in ECV less than 0.30% and dependent on achieving at least PR in ECV of 0.31% to 0.40%. Survival was dependent on achieving CR in ECV of 0.41% to 0.50% and ECV greater than 0.50%. Achieving a deep hematological response at 1 month was associated with better survival compared with 6 months in patients with ECV greater than 0.40% but not with ECV less than 0.40%.
This study found that ECV mapping, in systemic AL amyloidosis, is an independent predictor of prognosis, can help define the hematological response associated with better long-term outcomes for each patient and potentially inform treatment strategies.
心脏淀粉样物质浸润是系统性轻链（AL）淀粉样变性患者生存的关键决定因素。目前的指南建议无论心脏淀粉样物质浸润的程度如何，对反应不理想或欠佳的患者应尽早转换治疗方案。
评估血清生物标志物、超声心动图和心血管磁共振（CMR）与细胞外容积（ECV）图谱在特征性心脏淀粉样物质、这些方法的独立预后作用以及 ECV 图谱在指导治疗策略中的作用方面的差异。
设计、地点和参与者：连续诊断为系统性 AL 淀粉样变性的患者（2015-2021 年）在诊断时接受了超声心动图、心脏生物标志物和 CMR 与 ECV 图谱检查。数据分析于 2024 年 1 月至 6 月进行。
该研究的主要结局是全因死亡率和血液学反应，根据验证标准定义：无反应（NR）、部分反应（PR）、非常好的部分反应（VGPR）和完全反应（CR）。次要结局是根据 ECV 评估的血液学反应的深度和速度以及总生存率。
在 560 例 AL 淀粉样变性患者中，中位（IQR）年龄为 68 岁（59-74 岁）；346 名男性（61.8%）和 214 名女性（38.2%）。中位（IQR）40.5 个月（9-58 个月）后，ECV 与死亡率独立相关。在 1 个月的标志性分析中，长期生存与 ECV 小于 0.30%和 ECV 为 0.31%至 0.40%时的血液学反应无关，而与 ECV 大于 0.40%时的血液学反应深度有关。在 6 个月的标志性分析中，ECV 小于 0.30%和 ECV 为 0.31%至 0.40%时的生存与达到的血液学反应无关，而与达到至少 PR 的 ECV 有关。在 ECV 为 0.41%至 0.50%和 ECV 大于 0.50%时，生存与达到 CR 有关。在 ECV 大于 0.40%的患者中，与 6 个月相比，1 个月时达到深度血液学反应与更好的生存相关，但在 ECV 小于 0.40%的患者中则没有。
本研究发现，ECV 图谱在系统性 AL 淀粉样变性中是预后的独立预测因子，可帮助确定与每位患者更好的长期结局相关的血液学反应，并可能为治疗策略提供信息。
