Gillmore Julian D, Goodman Hugh J, Lachmann Helen J, Offer Mark, Wechalekar Ashutosh D, Joshi Jayshree, Pepys Mark B, Hawkins Philip N
National Amyloidosis Centre, Department of Medicine, Royal Free and University College Medical School, London, United Kingdom.
Blood. 2006 Feb 1;107(3):1227-9. doi: 10.1182/blood-2005-08-3253. Epub 2005 Oct 6.
Extensive cardiac amyloid deposition in systemic AL amyloidosis is associated with a grave prognosis. Heart transplantation is rarely performed because of the systemic and progressive nature of the disease. Patients with severe cardiac amyloid infiltration are ineligible for the preferred treatment of melphalan chemotherapy with stem cell transplantation (SCT) rescue because of the high risk for treatment-related mortality. Heart transplantation followed by SCT was performed in 5 patients with AL amyloidosis and predominant cardiomyopathy. Patients were followed up for a median of 95 months (range, 37-118 months) from diagnosis. At censor, 3 of 5 patients were well without evidence of intracardiac or extracardiac amyloid accumulation, and median overall survival by Kaplan-Meier estimate was not reached. Two patients died of progressive amyloidosis 33 and 90 months after heart transplantation after relapse of their underlying plasma cell dyscrasia. Heart transplantation followed by SCT is feasible in selected patients with cardiac AL amyloidosis and may confer substantial survival benefit.
系统性轻链（AL）淀粉样变性中广泛的心脏淀粉样蛋白沉积与严重预后相关。由于该疾病的全身性和进行性，心脏移植很少进行。严重心脏淀粉样蛋白浸润的患者因治疗相关死亡率高，不符合美法仑化疗联合干细胞移植（SCT）挽救的首选治疗条件。5例以心肌病为主的AL淀粉样变性患者接受了心脏移植后行SCT。从诊断开始，患者的中位随访时间为95个月（范围37 - 118个月）。在随访结束时，5例患者中有3例情况良好，无心脏内或心脏外淀粉样蛋白蓄积证据，根据Kaplan-Meier估计，中位总生存期未达到。2例患者在心脏移植后33个月和90个月因潜在浆细胞异常增生复发死于进行性淀粉样变性。对于选定的心脏AL淀粉样变性患者，心脏移植后行SCT是可行的，且可能带来显著的生存获益。
