Tuzovic Mirela, Yang Eric H, Baas Arnold S, Depasquale Eugene C, Deng Mario C, Cruz Daniel, Vorobiof Gabriel
Division of Cardiology, Department of Medicine, Ronald Reagan UCLA Medical Center, Los Angeles, CA, USA.
Ahmanson-UCLA Cardiomyopathy Center, Department of Medicine, Ronald Reagan UCLA Medical Center, Los Angeles, CA, USA.
Curr Oncol Rep. 2017 Jul;19(7):46. doi: 10.1007/s11912-017-0607-4.
Cardiac amyloidosis in the United States is most often due to myocardial infiltration by immunoglobulin protein, such as in AL amyloidosis, or by the protein transthyretin, such as in hereditary and senile amyloidosis. Cardiac amyloidosis often portends a poor prognosis especially in patients with systemic AL amyloidosis. Despite better understanding of the pathophysiology of amyloid, many patients are still diagnosed late in the disease course. This review investigates the current understanding and new research on the diagnosis and treatment strategies in patients with cardiac amyloidosis. Myocardial amyloid infiltration distribution occurs in a variety of patterns. Structural and functional changes on echocardiography can suggest presence of amyloid, but CMR and nuclear imaging provide important complementary information on amyloid burden and the amyloid subtype, respectively. While for AL amyloid, treatment success largely depends on early diagnosis, for ATTR amyloid, new investigational agents that reduce production of transthyretin protein may have significant impact on clinical outcomes. Advancements in the non-invasive diagnostic detection and improvements in early disease recognition will undoubtedly facilitate a larger proportion of patients to receive early therapy when it is most effective.
在美国,心脏淀粉样变性最常见的原因是免疫球蛋白蛋白浸润心肌,如在AL淀粉样变性中,或转甲状腺素蛋白浸润心肌,如在遗传性和老年性淀粉样变性中。心脏淀粉样变性往往预示着预后不良,尤其是在系统性AL淀粉样变性患者中。尽管对淀粉样变性的病理生理学有了更好的理解,但许多患者在疾病进程中仍被诊断较晚。本综述探讨了目前对心脏淀粉样变性患者诊断和治疗策略的认识以及新研究。心肌淀粉样浸润分布有多种模式。超声心动图上的结构和功能变化可提示淀粉样变性的存在,但心脏磁共振成像(CMR)和核成像分别提供了关于淀粉样蛋白负荷和淀粉样蛋白亚型的重要补充信息。对于AL淀粉样变性,治疗成功很大程度上取决于早期诊断,而对于ATTR淀粉样变性,减少转甲状腺素蛋白产生的新型研究药物可能会对临床结果产生重大影响。无创诊断检测的进展以及早期疾病识别的改善无疑将促使更大比例的患者在最有效的时候接受早期治疗。