自体外周血干细胞移植治疗原发性系统性淀粉样变性的中远期疗效观察  

Mid-term outcome of autologous stem cell transplantation in patients with systemic amyloidosis

作　　者：陈文萃[1] 黄湘华[1] 张瑾[1] 任贵生[1] 龚德华[1] 任强[1] 刘志红[1] 王庆文[1] 

机构地区：[1]南京军区南京总医院肾脏科国家肾脏疾病临床医学研究中心全军肾脏病研究所,南京210016

出　　处：《肾脏病与透析肾移植杂志》2016年第4期319-323,313,共6页Chinese Journal of Nephrology,Dialysis & Transplantation

基　　金：国家科技支撑计划课题(2013BAI09B04;2015BAI12B05);江苏省临床医学中心(BL2012007)

摘　　要：目的:探讨自体外周血干细胞移植(AHSCT)治疗原发性系统性淀粉样变性(简称AL淀粉样变性)的中远期疗效。方法:回顾分析2010年7月至2014年3月在南京军区南京总医院肾脏科接受AHSCT治疗的AL淀粉样变性患者共87例。结果:所有患者均移植成功,移植期间常见并发症为消化道症状,3级及以上并发症发生率低。移植后12月、24月、36月的总体生存率分别为94.3%、83.9%及78.9%;无进展生存(PFS)率分别为85.1%、83.5%及83.5%;肾存活率分别为91.7%、82.6%、77.1%。移植相关死亡率(TRM)为3.4%。多因素分析提示脑钠肽(BNP)、受累及未受累游离轻链的差值(d FLC)>58.07 mg/L是影响生存的独立危险因素,尿NAG酶是影响肾存活的独立危险因素。结论:对于严格选择的AL淀粉样变性患者,AHSCT是一种安全有效的治疗手段,血液学及器官反应率高,术后总体生存率及肾存活率均较高。Objective： To investigate the mid-term curative effect of autologous hematopoietic stem cell transplantation（ AHSCT） for patients with primary systemic amyloidosis（ AL）. Methodology： A total of eighty seven patients diagnosed as AL and received AHSCT were enrolled in this study from July 2010 to March 2014. They were 46 males and 41 females with an average age of 51. 9 ± 7. 80 years old. The mean time of follow up was 21 months ranged from 12 to 33 months,and their mid-term curative effects of AHSCT were analyzed. Results： All patients had achieved in AHSCT,the common complication were digestive tract symptoms during AHSCT,the incidence of grade 3 and above complications was low. At 12 months,24 months and 36 months after AHSCT,the OS rate was 94. 3%,83. 9% and 78. 9%; PFS rate was85. 1%,83. 5% and 83. 5%; Kidney survival rate was 91. 7%,82. 6% and 77. 1%,respectively. Transplantation related mortality（ TRM） was 3. 4%. Multivariate analysis showed that brain natriuretic peptide（ BNP） and difference free light chain（ d FLC） 〉58. 07 mg / L were independently associated with patient survival. Urinary N- Acetyl beta Glucosidase-D（ NAG） was independently associated with kidney survival. Conclusion： AHSCT was effective and safe for selected AL patients,the hematologic and organ response rate was high,and the OS and kidney survival rate were also exciting.

关 键 词：原发性系统性淀粉样变性 自体外周血干细胞移植 疗效 

分 类 号：R597.2[医药卫生—内科学]