自体外周血造血干细胞移植与硼替佐米联合地塞米松治疗原发性轻链型淀粉样变性的疗效和安全性比较  

Comparison analysis of outcomes in primary light chain amyloidosis patients treated by auto peripheral blood stem cell transplantation or bortezomib plus dexamethasone

作　　者：赵茜[1] 王利平[1] 宋萍[1] 李锋[1] 周小钢[1] 于亚平[1] 安志明[1] 王旭丽[1] 翟勇平[1] 

机构地区：[1]南京军区南京总医院血液科,210002

出　　处：《中华血液学杂志》2016年第4期283-287,共5页Chinese Journal of Hematology

基　　金：南京军区医学科技创新项目（10MA096）

摘　　要：目的比较大剂量马法兰预处理的自体外周血造血干细胞移植（auto-PBSCT）与硼替佐米联合地塞米松（VD方案）治疗原发性轻链型淀粉样变性的疗效和安全性。方法回顾性分析2004年9月至2012年9月38例原发性轻链型淀粉样变性患者的临床资料，其中auto-PBSCT组15例，VD组23例。结果所有患者的中位随访时间为34（1～112）个月，auto-PBSCT组为38（5～112）个月，VD组为31（1～108）个月。38例患者中15例（39.5%）获得器官缓解，auto-PBSCT组和VD组器官缓解率差异无统计学意义[33.3%（5/15）对43.5%（10/23），P=0.532]，达到器官缓解的中位时间差异有统计学意义[6（3～10）个月对3（1～6）个月，P=0.032]。两组患者的3年总生存率分别为72.0%和66.9%，预期总生存期分别为84.7个月和75.9个月（P=0.683）。auto-PBSCT组Ⅲ/Ⅳ级骨髓抑制（P〈0.001）、发热（P〈0.001）和恶心、呕吐（P=0.006）的发生率高于VD组，而肺感染（P=0.069）、血流感染（P=0.059）的发生率差异无统计学意义（P〉0.05）。结论auto-PBSCT和VD方案治疗原发性轻链型淀粉样变性患者的器官缓解率相当，生存时间无明显差异，但auto-PBSCT组不良反应更多见。Objective To explore the feature of primary light chain amyloidosis patients treated with high-dose melphalan with auto peripheral blood stem cell transplantation （auto-PBSCT） and bortezomib plus dexamethasone （VD）. Methods Thirty-eight patients diagnosed from September 2004 to September 2012 were analyzed retrospectively, including 15 cases received auto-PBSCT, 23 cases exposed with VD. Results The median follow-up duration for the patients was 34 months （range, 1-112 months）, including auto-PBSCT group of 38 months （range, 5-112 months） and VD group of 31 months （range, 1-108 months）. The organ response rate in all the patients was 39.5% （15/38）, and the organ response rate between these two groups has no significant difference [33.3% （5/15） vs 43.5% （10/23）, P= 0.532]. However, the median time of organ response was significant difference [6 （3-10） months vs 3 （ 1- 6） months, respectively （P=0.032）]. The 3-year overall survival （OS） rates in the two groups were 72.0% and 66.9%, and their average survival were 84.7 months and 75.9 months, respectively （P=0.683）. In the patients with auto-PBSCT, the occurrence of Ⅲ-Ⅳ grade of bone marrow suppression （P 〈 0.001 ）, fever （P 〈 0.001 ）, nausea and infection （P=0.006） were obviously higher than those with VD, hut there was no statistically significant difference in pulmonary infection （P=0.069） and bloodstream infection （P=0.059）. Conclusions The preliminary results have presented that primary light chain amyloidosis patients treated with auto-PBSCT or VD had similar organ response rate and survival. However, more adverse events occurred in the group of auto-PBSCT.

关 键 词：淀粉样变性 免疫球蛋白轻链 造血干细胞移植 回顾性研究 硼替佐米 

分 类 号：R597.2[医药卫生—内科学]