摘要
目的回顾性分析晚期轻链(AL)型淀粉样变患者接受达雷妥尤单抗治疗的疗效和安全性。方法纳入20例于2017年1月至2021年3月在北京协和医院接受达雷妥尤单抗治疗的初治或复发难治晚期AL型淀粉样变患者。收集患者治疗前基线临床资料及治疗随访资料,分析患者的血液学、器官缓解情况,预后及不良反应。结果20例患者的中位年龄为62(45~73)岁,男女比例2.3∶1,9例为初治,11例为复发难治,梅奥2004分期为Ⅱ/Ⅲ期。4例患者于第1个疗程死亡,其余16例患者中位接受3(1~10)个疗程化疗。治疗后1个月、3个月及6个月血液学总缓解率均为80%,≥非常好的部分缓解率分别为45%、60%及60%,中位血液学起效时间为13(6~28)d,3个月、6个月及12个月心脏缓解率分别为20%、30%及40%,中位起效时间为91(30~216)d,1年总生存率为48.4%。至末次随访,9例(45%)患者死亡,1个月内死亡率为25%,Ⅲb期患者1个月内死亡率为40%。3级以上血液学不良反应以淋巴细胞减少最为常见,非血液学不良反应主要为输注反应及感染。结论达雷妥尤单抗治疗初治及复发难治晚期AL型淀粉样变可快速诱导高水平的血液学缓解,但Ⅲb期患者仍有较高的早期死亡率。
Objective The study investigated the efficacy and safety of daratumumab in the treatment of cardiac light chain(AL)amyloidosis.Methods We retrospectively analyzed the clinical characteristics,hematologic response,organ response,long-term survival,and adverse events of 20 patients with newly diagnosed or relapsed/refractory cardiac AL amyloidosis treated with daratumumab in Peking Union Medical College Hospitalo from January 2017 to March 2021.Results The overall median age of 20 patients was 62(range,45-73)yeas,with a male to female ratio of 2.3:1.Nine patients were newly diagnosed,while 11 patients had relapsed or refractory disease.Based on Mayo 2004 cardiac AL staging system,stagesⅡandⅢdiseases were present in 20 patients respectively.Four patients died during the first cycle of daratumumab,and the remaining 16 patients completed a median of 3(range,1-10)cycles of treatment.Overall hematologic response rates were 80%each at 1,3,and 6 months after treatment initiation,and 45%,60%,and 60%of the patients achieved at least a very good partial response at 1,3,and 6 months respectively.The median duration to hematologic response was 13(range,6-28)days.At 3,6,and 12 months,20%,30%,and 40%of the patients respectively achieved a cardiac response,and the median days to response was 91(range,30-216)days.As of the last follow-up,9(45%)patients died.The 1-month mortality rate of all the patients and stage IIIb patients was 25%and 40%,respectively.The 1-year overall survival rate was 48.4%.Lymphocytopenia was the most common hematological adverse event(above grade 3).Non-hematological adverse events were mainly infusion-related reactions and infections.Conclusion Daratumumab could induce deep and rapid hematologic response in newly diagnosed and previously treated cardiac AL amyloidosis patients.However,daratumumab was not effective in preventing the high and early mortality rate in stageⅢb patients.
作者
沈恺妮
苗会蕾
高雅娟
曹欣欣
周道斌
苏薇
李剑
Shen Kaini;Miao Huilei;Gao Yajuan;Cao Xinxin;Zhou Daobin;Su Wei;Li Jian(Department of Hematology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100730,China;Laboratory Department,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100730,China)
出处
《中华血液学杂志》
CAS
CSCD
北大核心
2022年第1期31-34,共4页
Chinese Journal of Hematology
基金
中央高校基本科研业务费专项资金(3332021002)。
