Department of Biochemistry, University of Pavia, Pavia, Italy.
Curr Opin Oncol. 2011 Mar;23(2):214-20. doi: 10.1097/CCO.0b013e328342b840.
Multiorgan involvement renders patients with AL amyloidosis particularly susceptible to treatment toxicity. The introduction of autologous stem cell transplantation (ASCT) represented a major advancement, but was associated with unacceptable toxicity in high-risk patients. Thus, efforts were made to improve the eligibility criteria for ASCT and to design novel, more effective, conventional-dose regimens. This review focuses on the role of ASCT and conventional-dose therapy in light of advances in risk stratification and patient monitoring.
The possibility of directly measuring the amyloidogenic precursor, the circulating free light chain (FLC), improved monitoring response to therapy. Cardiac biomarkers, N-terminal pro-natriuretic peptide type-B (NT-proBNP) and troponins (cTn) allow the most accurate prognostic stratification and direct the choice of therapy. Serial measurement of NT-proBNP, cTn and FLC are used to rapidly assess treatment efficacy. Bortezomib and immune-modulatory drugs are going to play a major role in conventional-dose therapy and as adjuvant treatment after ASCT.
The choice between ASCT and conventional-dose chemotherapy is based on accurate risk assessment. Tight monitoring of hematologic and cardiac response is the cornerstone of treatment. Upcoming randomized trials will redefine the role of available therapies, assisting in the choice of the growing number of active regimens.
多器官受累使 AL 淀粉样变性患者特别容易发生治疗毒性。自体干细胞移植（ASCT）的引入是一个重大进展，但在高危患者中与不可接受的毒性相关。因此，努力改进 ASCT 的入选标准，并设计新的、更有效、常规剂量的方案。本综述重点讨论了 ASCT 和常规剂量治疗在风险分层和患者监测进展方面的作用。
直接测量淀粉样变性前体，即循环游离轻链（FLC）的可能性，改善了对治疗反应的监测。心脏生物标志物，N 端脑钠肽前体 B 型（NT-proBNP）和肌钙蛋白（cTn）可进行最准确的预后分层，并指导治疗选择。连续测量 NT-proBNP、cTn 和 FLC 可快速评估治疗效果。硼替佐米和免疫调节药物将在常规剂量治疗中以及 ASCT 后作为辅助治疗发挥重要作用。
ASCT 和常规剂量化疗之间的选择基于准确的风险评估。对血液学和心脏反应的严格监测是治疗的基石。即将进行的随机试验将重新定义现有治疗方法的作用，有助于选择越来越多的有效方案。
