无丙二醇型美法仑预处理自体造血干细胞移植治疗系统性轻链型淀粉样变性的疗效与安全性  

Pretreated autologous hematopoietic stem cell transplantation without propylene glycol melphalan for the treatment of systemic light chain amyloidosis

在线阅读下载全文

作  者:李真 陈文萃 郭锦洲 吴晓梅 徐维玮 黄湘华 LI Zhen;CHEN Wencui;GUO Jinzhou;WU Xiaomei;XU Weiwei;HUANG Xianghua(National Clinical Research Center for Kidney Diseases,Jinling Hospital,Affiliated Hospital of Medical School,Nanjing University,Nanjing 210016,China)

机构地区:[1]南京大学医学院附属金陵医院(东部战区总医院)国家肾脏疾病临床医学研究中心,南京210016

出  处:《肾脏病与透析肾移植杂志》2025年第1期8-13,共6页Chinese Journal of Nephrology,Dialysis & Transplantation

基  金:国家自然科学基金面上项目(82270767)。

摘  要:目的:探讨无丙二醇型美法仑在自体造血干细胞移植(ASCT)治疗系统性轻链型(AL型)淀粉样变性患者中的疗效及安全性。方法:回顾性纳入2010—2023年接受ASCT的AL型淀粉样变性患者352例,其中无丙二醇型美法仑预处理患者(A组)127例,丙二醇型美法仑预处理患者(B组)225例,比较两组治疗的安全性及移植后短期血液学缓解率。结果:两组患者的年龄、受累轻链类型、中性粒细胞植入和血小板植入时间无差异,A组患者心脏受累比例更低,但骨髓浆细胞比例更高。A组患者移植后3月血液学总体缓解率(89.4%vs 73.4%,P=0.001)和完全缓解率(39.4%vs 24%,P=0.005)均高于B组,可能与A组有更多患者(79.5%)在移植前接受蛋白酶体抑制剂诱导治疗相关。A组3~4级腹泻(11.8%vs 2.7%,P<0.001)和恶心呕吐(67.7%vs 35.1%,P<0.001)事件发生率更高。两组患者在感染、心肾器官毒性方面未表现出差异。A组患者植入综合征(2.4%vs 7.6%,P=0.043)和黏膜炎(27.6%vs 46.7%,P<0.001)的发生率更低,且未发生3级以上黏膜炎。结论:无丙二醇型美法仑预处理方案在AL型淀粉样变性患者的ASCT治疗中耐受性良好,疗效确切。Objective:To investigate the efficacy and safety of propylene glycol free melphalan in autologous hematopoietic stem cell transplantation(ASCT)for patients with systemic light chain(AL)amyloidosis.Methodology:A total of 352 patients with AL amyloidosis who received ASCT from^(2)010 to 2023 were retrospectively included,including 127 patients with propylene glycol free melphalan pretreatment(group A),225 patients with propylene glycol melphalan pretreatment(group B).The safety of treatment and short-term hematological response rate after transplantation were compared between the two groups.Results:There were no differences in age,type of light chain involved,neutrophil and platelet engraftment time,and group A patients had a lower proportion of cardiac involvement but a higher proportion of bone marrow plasma cells.The overall hematological response rate(89.4%vs 73.4%,P=0.001)and complete response rate(39.4%vs 24%,P=0.005)at 3 months after transplantation in group A were higher than those in group B,which may be related to the fact that more patients in group A(79.5%)received proteasome inhibitor induction therapy before transplantation.Group A had a higher incidence of grade 3~4 diarrhea(11.8%vs 2.7%,P<0.001)and nausea and vomiting(67.7%vs 35.1%,P<0.001).There were no differences in infection,cardiac and renal toxicity between the two groups.The incidences of implantation syndrome(2.4%vs 7.6%,P=0.043)and mucositis(27.6%vs 46.7%,P<0.001)were lower in the group A,and no grade 3 or higher mucositis occurred.Conclusion:The preconditioning regimen of propylene glycol free melphalan is well tolerated and effective in the treatment of ASCT for patients with AL amyloidosis.

关 键 词:无丙二醇型美法仑 系统性轻链型淀粉样变性 疗效 安全性 

分 类 号:R457.7[医药卫生—治疗学] R597.2[医药卫生—临床医学]

 

参考文献:

正在载入数据...

 

二级参考文献:

正在载入数据...

 

耦合文献:

正在载入数据...

 

引证文献:

正在载入数据...

 

二级引证文献:

正在载入数据...

 

同被引文献:

正在载入数据...

 

相关期刊文献:

正在载入数据...

相关的主题
相关的作者对象
相关的机构对象