原发性系统性淀粉样变性的预后及危险因素分析

Outcomes and risk factors in patients with idiopathic AL amyloidosis

目的:分析我国汉族人群原发性系统性(AL)淀粉样变性患者的临床顶后特点及相关危险因素。方法:回顾性分析2003年3月至2011年8月期间南京军区南京总医院全军肾脏病研究所诊断的AL淀粉样变性患者的临床预后特点,利用Kaplan-Meier法计算患者的累计生存率,利用COX回归模型分析相关危险因素。结果:本研究共纳入患肯245例,中位年龄56岁,其中男性153例,以乏力及水肿起病,除肾脏外,最常见的受累器官是肠道(55.9%)和心脏(46.9%)。中位病程为7月,主要表现为大量蛋白尿(尿蛋白定量4.86±4.32g/24h),其中62.45%患者尿蛋白>3.5g/d。25%的患者诊断时合并肾功能不全。M蛋白主要为λIgG(36.06%)和λIgA(18.75%)。中位随访20月,患者中位生存时间33.6月(1～98月),1年、2年、3年和5年的生存率分别为68.3%、52.7%、47.8%和30.7%。亚组分析发现心脏受累、肝脏受累、低血压、肾功能不全患者生存时间缩短,经特殊治疗(化疗或干细胞移植)后患者预后明显改善。COX多因素分析表明年龄、肝脏受累、心脏受累是患者预后的独立危险因素,而特殊治疗是患昔预后独立保护因素。生存分析表明肾脏中位生存时间为45月(1～98月),1年、2年、3年及5年肾存活率分别为78.6%、69.6%、63.2%和36.9%。多因素分析表明诊断时肌酐升高和低血压是进展至ESRD的独立危险因素。结论:AL淀粉样变性患者常合并肠道和心脏受累,长期预后较差,其中合并心脏和肝脏受累、低血压及肾功能不全者预后更差,而接受特殊治疗可明显提高患者生存率;诊断时肌酐升高及低血压提示肾脏预后不良。

Objective： To investigate the clinical features and outcomes in AL amyloidosis patients with renal involvement. Methodology：Two hundred and forty five patients with biopsy-proven AL amyloidosis in Jinling hospital from March 2003 to August 2011 were studied. They were 153 males and 92 females with the median age of 56 years old at diagnosis. All patients had renal involvement. The clinical and laboratory information were collected from diagnosis to death or until the last available clinical control. The patients＇ survival and renal outcomes were analyzed, and the relationships between clinical parameters were also assessed. Results：The most common affect organs were intestinal tract （55.9%） and heart （46. 7% ） besides renal. Proteinuria was the major clinical feature of these patients, and the mean level of proteinuria was 4. 86 ＋ 4. 32 g/24h. 25% patients had renal insufficiency at diagnosis. The mean time of follow-up was 20 months （range from 1 -98）. 111 patients were dead,the median survival time of all patients was 33.6 months,the patients with heart involvement,hepatic involvement,hypotension and renal insufficiency had a worse survival, and specific therapy can improve the survival. The 1,2,3 and 5-year cumulative survival rate, calculated by Kaplan-Meier method, were 68.3 % , 52. 7 % ,47.8 % and 30. 7% respectively. Multivariate COX analysis showed the age, hepatic involvement, heart involvement and specific therapy can significantly influence survival in these patients. The median time of renal survival was 45 months,the 1,2,3 and 5-year renal survival rate were 78.6% ,69.6% ,63.2% and 36. 9% respectively, Multivariate COX analysis showed the levels of serum creatinine and degree of hypotension were the important risk factors of renal failure. Conehtsion：Our data demonstrated the clinical features and survival of Chinese patients with renal AL amyloidosis. These patients were usually combined with intestinal tract and heart involvement, the survival of our cohort was poor, and the heart involvement, hepatic involvement, hypotension, renal insufficiency and specific therapy can significantly influence survival. The high serum creatinine level and hypotension at diagnosis implied the bad renal outcome.