Xu Cheng-Yang, Guan Ai, Zhang Lu, Tian Zhuang, Zhou Dao-Bin, Shen Kai-Ni, Li Jian
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Department of Cardiology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
JACC Asia. 2024 Nov 26;5(1):74-84. doi: 10.1016/j.jacasi.2024.10.005. eCollection 2025 Jan.
The goal of hematological response has been well established in the treatment of systemic light chain amyloidosis. However, the pattern of cardiac response remains unknown.
This study was designed to investigate the cardiac response dynamics in patients with an early and high-quality hematological response.
This retrospective study included newly diagnosed patients who achieved a hematological response of very good partial response or better within 3 months after treatment beginning. Four cardiac response criteria were tested at fixed time points (3, 6, 12, and 24 months after therapy initiation): cardiac complete response, cardiac very good partial response, cardiac partial response (carPR), and cardiac no response.
A total of 201 patients were included, with the median follow-up 37.0 months (Q1-Q3: 18.0-56.5 months). The cardiac response reached a plateau at 24 months, while cardiac complete response, cardiac very good partial response, and carPR were achieved in 21.4% (28 of 131), 38.9% (51 of 131), and 20.6% (27 of 131) of the patients, respectively. At every fixed time point within 12 months after treatment initiation, patients who achieved a carPR or better consistently had better survival than did those who did not. At 3 months, an NT-proBNP concentration ≥3,716 pg/mL was the only factor associated with a decreased likelihood of achieving a carPR within 12 months (OR: 0.269; 95% CI: 0.137-0.512; 0.001).
This study emphasizes the importance of monitoring cardiac response dynamics for disease surveillance. In patients who achieve early and high-quality hematological response, it is important to achieve carPR within 12 months.
血液学缓解目标在系统性轻链淀粉样变治疗中已得到充分确立。然而，心脏缓解模式仍不明确。
本研究旨在调查早期获得高质量血液学缓解患者的心脏缓解动态情况。
这项回顾性研究纳入了新诊断的患者，这些患者在开始治疗后3个月内达到了非常好的部分缓解或更好的血液学缓解。在固定时间点（治疗开始后3、6、12和24个月）测试了四种心脏缓解标准：心脏完全缓解、心脏非常好的部分缓解、心脏部分缓解（carPR）和心脏无缓解。
共纳入201例患者，中位随访时间为37.0个月（第一四分位数-第三四分位数：18.0-56.5个月）。心脏缓解在24个月时达到平台期，分别有21.4%（131例中的28例）、38.9%（131例中的51例）和20.6%（131例中的27例）的患者达到心脏完全缓解、心脏非常好的部分缓解和carPR。在治疗开始后12个月内的每个固定时间点，达到carPR或更好缓解的患者生存率始终高于未达到的患者。在3个月时，NT-proBNP浓度≥3716 pg/mL是与12个月内达到carPR可能性降低相关的唯一因素（比值比：0.269；95%置信区间：0.137-0.512；P<0.001）。
本研究强调了监测心脏缓解动态以进行疾病监测的重要性。在早期获得高质量血液学缓解的患者中，12个月内实现carPR很重要。
