t(11;14)易位对接受硼替佐米治疗的原发性轻链型淀粉样变患者的预后价值  

Prognostic value of translocation t(11;14)in primary light-chain amyloidosis treated with bortezomib-based regimen

作　　者：高雅娟 沈恺妮 冯俊[1] 常龙[1] 李辉[1] 苏薇[2] 曹欣欣[1] 张路[1] 周道斌[1] 李剑[1] Gao Yajuan;Shen Kaini;Feng Jun;Chang Long;Li Hui;Su Wei;Cao Xinxin;Zhang Lu;Zhou Daobin;Li Jian(Department of Hematology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100730,China;Department of Clinical Laboratory,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医学院北京协和医院血液内科,北京100730 [2]中国医学科学院北京协和医学院北京协和医院检验科,北京100730

出　　处：《中华医学杂志》2022年第36期2854-2860,共7页National Medical Journal of China

摘　　要：目的探讨t(11;14)易位在接受硼替佐米方案治疗的初治原发性轻链型淀粉样变患者中的预后价值。方法纳入2015年9月至2021年9月北京协和医院确诊、具有基线t(11;14)信息且接受一线硼替佐米方案化疗的初治原发性轻链型淀粉样变患者,比较t(11;14)阳性组和阴性组之间的基线指标、血液学疗效、器官疗效和预后的差异。结果共纳入152例患者,年龄(59.5±9.1)岁,其中男性93例(61.2%)。依照t(11;14)阳性与否分为阳性和阴性组,其中阳性组46例(30.3%),两组在受累器官比例、2004和2012梅奥分期、重要实验室指标方面差异均无统计学意义(均P>0.05)。两组的首程化疗后血液学≥非常好的部分缓解(VGPR)率差异无统计学意义[28.2%(11/39)比37.4%(34/91),P>0.05];3疗程后两组血液学≥VGPR率差异无统计学意义[35.9%(14/39)比51.1%(46/90),P>0.05];两组的一线方案后最佳血液学疗效达≥VGPR率差异无统计学意义[52.2%(24/46)比64.2%(68/106),P>0.05]。阳性组的3个月、6个月心脏缓解率均低于t(11;14)阴性患者[3个月,15.2%(5/33)比34.6%(28/81),P=0.038;6个月,19.4%(6/31)比50.6%(42/83),P=0.003],但12个月心脏缓解率组间差异无统计学意义[41.7%(10/24)比53.5%(38/71),P>0.05]。另外,阳性组和阴性组的中位总生存期差异无统计学意义(未达到比50.1个月,P>0.05),中位血液学无事件生存期差异无统计学意义(36.2个月比39.9个月,P>0.05)。结论在硼替佐米一线治疗后,t(11;14)阳性患者的心脏缓解率更低,但血液学缓解及生存都与阴性患者相似。Objective To investigate the prognostic value of translocation t(11;14)in newly-diagnosed primary light-chain(AL)amyloidosis patients treated with bortezomib-based regimen.Method Clinical information of newly-diagnosed AL amyloidosis patients in Peking Union Medical College Hospital who had baseline t(11;14)data and accepted bortezomib-combined therapies from September,2015 to September,2021 was collected.The relationships between t(11;14)status and baseline characteristics,hematological response,organ response and prognosis were analyzed.Results A total of 152 patients were included,aged(59.5±9.1)years and 93 cases were male(61.2%).Forty-six patients carried t(11;14)(30.3%).There was no statistical difference in the proportion of organ involved,distribution of Mayo 2004 and 2012 stages and laboratory indexes between patients with and without t(11;14)(all P>0.05).For hematological response,the difference in the rates of≥very good partial response(VGPR)between those with t(11;14)and without after the first cycle[28.2%(11/39)vs 37.4%(34/91),P>0.05]was not statistically significant.After 3 cycles,the difference in the rates of≥VGPR between two groups was not statistically significant[35.9%(14/39)vs 51.1%(46/90),P>0.05].The difference in the ratio of the best hematological response reaching≥VGPR between two groups during the first-line treatment was not statistically significant[52.2%(24/46)vs 64.2%(68/106),P>0.05].But patients with t(11;14)had lower cardiac response rate at 3 months[15.2%(5/33)vs 34.6%(28/81),P=0.038]and 6 months[19.4%(6/31)vs 50.6%(42/83),P=0.003]than those without,but the difference in cardiac response rates at 12 months was not statistically significant[41.7%(10/24)vs 53.5%(38/71),P>0.05].For survival,the differences in overall survival(not reached vs 50.1 months,P>0.05)and hematological event-free survival(36.2 months vs 39.9 months,P>0.05)between patients carrying t(11;14)and those without were not statistically significant.Conclusion Patients with t(11;14)had lower cardiac response rate

关 键 词：淀粉样变性 细胞遗传学 血液学缓解 器官缓解 预后 回顾性队列研究 

分 类 号：R597.2[医药卫生—内科学]