化疗联合Venetoclax桥接异基因造血干细胞移植治疗母细胞性浆细胞样树突细胞肿瘤  

Chemotherapy Combined with Venetoclax Followed by AlloHematopoietic Stem Cell Transplantation for Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm

作　　者：程平[1] 王兰兰 王秋香 关军[1] 周英[1] 胡彬[2] 冯燕[3] 邹亮[1] 程辉[1] CHENG Ping;WANG Lan-Lan;WANG Qiu-Xiang;GUAN Jun;ZHOU Ying;HU Bin;FENG Yan;ZOU Liang;CHENG Hui(Department of Hematology,Wuhan First Hospital,Wuhan 430022,Hubei Province,China;Department of Dermatology,Wuhan First Hospital,Wuhan 430022,Hubei Province,China;Department of Pathology,Wuhan First Hospital,Wuhan 430022,Hubei Province,China)

机构地区：[1]武汉市第一医院血液科,湖北武汉430022 [2]武汉市第一医院皮肤科,湖北武汉430022 [3]武汉市第一医院病理科,湖北武汉430022

出　　处：《中国实验血液学杂志》2023年第5期1531-1536,共6页Journal of Experimental Hematology

摘　　要：目的:探讨化疗联合Venetoclax桥接异基因造血干细胞移植(allo-HSCT)治疗母细胞性浆细胞样树突细胞肿瘤的疗效及安全性。方法:回顾性分析2017年7月至2021年12月在武汉市第一医院血液科行allo-HSCT治疗的3例母细胞性浆细胞样树突细胞肿瘤患者的资料。结果:3例患者中男性1例,女性2例,年龄27-52岁。首诊时有典型的皮肤病变,也有以急性白血病为主要特征累及全身。肿瘤细胞特征性的分子标记物CD4、CD56、CD123及CD303均为阳性,均表达Bcl-2。3例患者在初始诱导化疗(1例)或复发、进展(2例)后的化疗中联用Venetoclax。2例行allo-HSCT前获得完全缓解,1例部分缓解。预处理均采用不含全身放射治疗的清髓性预处理方案。移植物抗宿主病的预防方案为抗胸腺细胞球蛋白^(+)酶酚酸酯^(+)环孢素/他克莫司±甲氨蝶呤。回输单个核细胞数为(16.73-18.35)×10~8/kg,CD34^(+)细胞数(3.57-4.65)×10~6/kg。Allo-HSCT后(^(+)21至^(+)28 d)3例患者均获得完全缓解,供受者嵌合率100%,无Ⅲ-Ⅳ度移植物抗宿主病。1例于移植后^(+)50 d死亡,2例随访时间分别为28和15个月,仍无病生存。结论:母细胞性浆细胞样树突细胞肿瘤为高度侵袭性恶性肿瘤,总体预后差。化疗联合Venetoclax桥接allo-HSCT可能使患者获得长期无病生存甚至治愈。移植后的维持治疗目前仍不清楚。Objective:To investigate the efficacy and safety of chemotherapy combined with venetoclax followed by allogeneic hematopoietic stem cell transplantation(allo-HSCT)for the treatment of blastic plasmacytoid dendritic cell neoplasm(BPDCN).Methods:The clinical data of 3 patients with BPDCN undergoing allo-HSCT in Department of Hematology,Wuhan First Hospital from July 2017 to November 2021 were collected and retrospectively analyzed.Results:Among the 3 patients,there were 1 male and 2 females,aged 27-52 years old.Skin lesions were observed during initial diagnosis,and it could also be characterized by acute leukemia.Characteristic molecular markers of tumor cells,such as CD4,CD56,CD123,and CD303 were positive.In addition,the expression detection of Bcl-2 in 3 patients were positive.Chemotherapy combined with venetoclax in the initial induction of chemotherapy(1 case)or disease recurrence and progress(2 cases)was performed.There were 2 cases evaluated as complete remission(CR)and 1 case as partial remission(PR)before allo-HSCT.The patients all received a nonmyeloablative conditioning without total body irradiation(TBI).The prevention programme of graft-versus-host disease(GVHD)was antithymocyte globulin+mycophenolate mof etil+cyclosporin A/FK506±methotrexate.The number of mononuclear cell(MNC)count was(16.73-18.35)×108/kg,and CD34+cell count was(3.57-4.65)×106/kg.The 3 patients were evaluated as CR after allo-HSCT(+21 to+28 d),the donor-recipient chimerism rate was 100%,andⅢ-ⅣGVHD was not observed.One patient died at+50 d after transplantation,two patients were followed up for 28 months and 15 months,respectively,and achieved disease-free survival(DFS).Conclusions:BPDCN is a highly aggressive malignant tumor with poor prognosis.Chemotherapy combined with venetoclax followed by allo-HSCT may lead to long-term DFS or even cure.Posttransplant maintenance is still unclear.

关 键 词：母细胞性浆细胞样树状突细胞肿瘤 化疗 Venetoclax 异基因造血干细胞移植 

分 类 号：R733[医药卫生—肿瘤]