摘要
目的观察脂质体阿霉素联合依托泊苷和大剂量甲泼尼龙方案(DEP方案)挽救治疗难治性巨噬细胞活化综合征(MAS)的有效性。方法回顾性收集2016年1月至2022年1月北京友谊医院诊治的38例难治性MAS患者的临床特点、DEP方案挽救治疗前后的实验室检查结果,参照美国中西部协作组制订的标准疗效评价,每2周疗效评价。相关样本之间采用非参数检验进行统计学分析。结果38例患者中,男女比例1:3.75,中位年龄30(15,69)岁。原发病为AOSD 29例,SLE 6例,RA 1例,未分化CTD 2例。初始治疗2周后,38例患者总反应率为95%(36/38),其中完全缓解率为24%(9/38),部分缓解率为71%(27/38);治疗4周后(部分患者数据丢失)35例患者总反应率为97%(34/35),其中完全缓解率为46%(16/35),部分缓解率为51%(18/35例);治疗6周后(部分患者数据丢失)35例患者总反应率为97%(34/35例),其中完全缓解率为49%(17/35),部分缓解率为49%(17/35)。达到部分缓解或完全缓解的患者在诱导后积极治疗自身免疫疾病,病情均得到持续缓解。结论DEP方案可能是一种有效的用于难治性CTD相关噬血细胞性淋巴组织细胞增条症挽救治疗方案。
Objective To investigate the efficacy of liposomal doxombicin combined with etoposide and high dose methylprednisolone(DEP)as a salvage therapy for refractory macrophage activation syndrome(MAS).Methods Totally 38 patients with refractory MAS were enrolled in this study from January 2016 to January 2022 in Beijing Friendship Hospital,including clinical characteristics and laboratory test results before and after DEP treatment,were retrospectively collected.The efficacy was evaluated every 2 weeks according to the United States Midwest Cooperative HLH Group.Relevant samples were statistically analyzed using non-parametric tests.Results Of 38 refractory MAS patients,8 males and 30 females were included into this study.The median age was 30(15-69)years old.The underlying disease were adult onset Still's disease in 29 cases,Systemic lupus erythematosus in 6 cases,Rheumatoid arthritis in 1 case and Undifferentiated Connective-Tissue disease in 2.The overall response rate was 95%(36/38),including 9 patients(24%)achieved complete remission and 27 patients(71%)achieved partial remission after 2 weeks of treatment.The overall response rate was 97%(34/35),including 16(46%)complete remission and 18(51%)partial remission after 4 weeks of treatment(due to lack of data in some patients).The overall response rate was 97%(34/35),including 17(49%)complete remission and 17(49%)partial remission after 6 weeks of treatment.Patients who achieved partial remission or complete remission were actively treated for the underlying diseases after induction,and their conditions were in persistent remission.Conclusion The DEP regimen may be an effective salvage therapy for the treatment of refractory MAS.
作者
尹冬飞
王晶石
吴林
宋德利
王昭
Yin Dongfei;Wang Jingshi;Wu Lin;Song Deli;Wang Zhao(Department of Hematology,Beijing Friendship Hospital,Beijing 100050,China)
出处
《中华风湿病学杂志》
CAS
CSCD
北大核心
2023年第2期85-90,共6页
Chinese Journal of Rheumatology
基金
国家自然科学基金(8187163)
北京市医管局"登峰"人才培养计划(DFL20180101)。
