沙利度胺联合地塞米松治疗系统性轻链型淀粉样变性的临床观察  

Efficiency and safety of thalidomide combined with dexamethasone in patients with primary systemic amyloidosis

作　　者：赵亮[1] 黄湘华[1] 李婷[2] 任贵生[1] 王庆文[1] 陈文萃[1] 刘志红[1] 

机构地区：[1]南京大学医学院附属金陵医院(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 [2]东南大学医学院

出　　处：《肾脏病与透析肾移植杂志》2016年第5期409-414,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：评估沙利度胺联合地塞米松化疗方案对初次治疗的系统性轻链型（AL）淀粉样变性患者的疗效和安全性。方法：回顾性分析2009年4月至2014年12月在南京军区南京总医院肾脏科确诊并采用沙利度胺联合地塞米松（TD）作为初次治疗方案的AL型淀粉样变性患者的临床及随访资料。应用Kaplan-Meier法计算患者生存时间,COX风险比例模型分析预后危险因素。结果：本研究共纳入患者63例,男女比例为1.52∶1,中位年龄为57岁,中位治疗周期为19月（4~78月）。38例患者（60.3%）心脏受累,38例（60.3%）患者两个或以上的器官受累。37例患者取得血液学反应,总体血液学缓解率为58.7%,中位缓解时间为4月,其中18例（28.6%）达到完全缓解,中位缓解时间为3月,10例（15.9%）达到非常好的部分缓解,中位缓解时间为5.5月,9例（14.3%）达到部分缓解,中位缓解时间为5月。23例（36.5%）患者获得器官缓解,肾脏缓解和心脏缓解分别为23例（36.5%）和10例（15.9%）,中位缓解时间分别为8.5月和18.8月。有3例患者病情复发,中位复发时间为7月（3~19月）。中位随访时间为28月,2年生存率为78.5%,K-M曲线估计的中位生存时间尚未达到。达到血液学缓解的患者组相比未达到血液学缓解的患者组预后更好（3年生存率87.5%vs 36.3%,P〈0.01）心脏受累的患者组相比于无心脏受累患者组预后更差（3年生存率52.4%vs 87.0%,P〈0.01）。常见不良反应包括外周神经病变（23.8%）、胃肠道反应（17.4%）、乏力（11.1%）、皮疹（6.3%）等。9例患者因不良反应和治疗无效停药,12例患者沙利度胺减量,22例患者地塞米松减量。结论：TD方案对初次治疗的AL淀粉样变性患者有一定的疗效,且耐受性较好,长期疗效有待进一步观察。Objective： To evaluate the efficiency and safety of the regiment of thalidomide combined with dexamethasone in untreated primary systemic （AL） amyloidosis patients. Methodology：The clinical data of 63 patients diagnosed AL amyloidosis were retrospectively analyzed from April 2009 to December 2014. All of them were taken the regiment of thalidomide combined with dexamethasone as the first line treatment. Survival curves were constructed according to the Kaplan-Meier method. The relation of clinical factors to all-cause mortality was assessed using proportional hazards regression. Results：They were 38 males and 25 females with a median age of 57 ys. The cycle of treatment was 19 （4 ~ 78）. 38 patients had heart involved, and 38 patients had also two more organs involved. In total, 37 patients （58.7%） achieve different hematological response, median time of response was 4 months; in which 18 patients （28. 6% ） achieve complete response, median time of response was 3 months; 10 patients （15.9%） achieve very good partial response,median time of response was 5.5 months ; and 9 patients （ 14. 3%） achieve partial response, median time of response was 5 months. There were 23 patients （36. 5%） obtaining organ response, in which mainly was renal response, median time to organ response was 8.5 months, heart response rate were 15.9%, median time to organ response was 18. 8 months. 3 patients had a relapse, median time to relapse was 7 months （3 ~ 19 months）.The median follow-up period was 28 months, the survival rate at two years was 78.5%, and the median overall survival time did not reach. Compared to the non- hematological response group, the hematological response group has a better prognosis, prognosis of the heart involvement group was worse comparing to the non-heart involved group. Common adverse reaction was sensory neuropathy （23.8%）, digestive tract reaction （ 17.4% ）, asthenia （ 11.1% ）, rash （ 6.3% ） and so on, without treatment related mortality. 9 patients en

关 键 词：系统性轻链型淀粉样变性 沙利度胺 地塞米松 疗效 毒副作用 

分 类 号：R597.2[医药卫生—内科学]