造血干细胞移植及细胞治疗母细胞性浆细胞样树突细胞肿瘤的疗效探讨  

The therapeutic effect of hematopoietic stem cell transplantation and cell therapy for blastic plasmacytoid dendritic cell neoplasm

作　　者：陈妍心 马骁[1] 刘丹 蔡文治[1] 庄娟 柯鹏[1] 吴德沛[1] CHEN Yanxin;MA Xiao;LIU Dan;CAI Wenzhi;ZHUANG Juan;KE Peng;WU Depei(First Affiliated Hospital of Soochow University,Jiangsu Institute of Hematology,Key Laboratory of Thrombosis and Hemostasis under Ministry of Health,Collaborative Innovation Center of Hematology,Institute of Blood and Marrow Transplantation,Soochow University,Suzhou,215006,China;Soochow Hopes Hematology Hospital,Suzhou;Department of Hematology,the Fifth Affiliated Hospital of Sun Yat-sen University)

机构地区：[1]苏州大学附属第一医院、江苏省血液研究所,卫生部血栓与止血重点实验室,血液学协同创新中心,苏州大学造血干细胞移植研究所,江苏苏州215006 [2]苏州弘慈血液病医院 [3]中山大学附属第五医院血液科

出　　处：《临床血液学杂志》2022年第3期174-179,共6页Journal of Clinical Hematology

基　　金：苏州市科技项目基金(No:SLT201911);白求恩公益基金。

摘　　要：目的:探讨不同治疗方式对于母细胞性浆细胞样树突细胞肿瘤(BPDCN)患者的临床疗效及安全性。方法:回顾性分析2015年7月—2021年6月苏州大学附属第一医院收治的6例BPDCN患者的临床资料。结果:所有患者均以皮肤或淋巴结受累起病,部分累及骨髓(5例)、外周血(5例)、脾(1例)、鼻咽部(1例)。所有患者为CD4、CD56双阳性表达,1例出现染色体易位,2例伴分子生物学改变。5例采用化疗桥接异基因造血干细胞移植,其中2例复发;1例给予去甲基化联合BCL-2靶向治疗后复发。3例复发患者中2例采用挽救性嵌合抗原受体T细胞疗法,均因疾病进展死亡;1例给予靶向药物维奈托克联合阿扎胞苷、供者淋巴细胞输注、干扰素注射、局部放疗后缓解。1例死于感染。总生存率50.0%(3/6),中位随访25.5(7.0~72.2)个月,中位无进展生存期8.9个月,中位总生存期25.5个月。结论:BPDCN总体预后不良,建立预后分层模型可能有助于治疗方案选择。化疗桥接异基因造血干细胞移植仍是目前有效的治疗手段且安全性良好,靶向及免疫治疗疗效需进一步验证及改善。Objective:To explore the therapeutic outcomes and safety of different treatments in blastic plasmacytoid dendritic cell neoplasm(BPDCN).Methods:We retrospectively analyzed clinical data at diagnosis and treatment results of 6 patients with BPDCN admitted to the First Affiliated Hospital of Soochow University from July 2015 to June 2021.Results:All six patients had initial skin lesion or lymphadenectasis,with 5 cases involving bone marrow,5 cases involving peripheral blood,1 case involving spleen and 1 case involving nasopharynx.All patients were double positive for CD4 and CD56,and 1 case had chromosomal translocation,2 cases had molecular biological change.Two of five cases relapsed after chemotherapy bridging allogeneic hematopoietic stem cell transplantation(allo-HSCT),and 1 case relapsed after demethylation combined with BCL-2 targeted therapy.Of the 3 patients with recurrence,2 cases treated with salvage chimeric antigen receptor T cell therapy and both died due to disease progression.The other one achieved remission from targeted drug of venetoclax and azacitidine,donor lymphocyte infusion,interferon,local radiotherapy.On the other hand,1 case died of infection.The overall survival rate was 50.0%(3/6).The median follow-up duration was 25.5(7.0-72.2)months,the median progression-free survival was 8.9 months,and the median overall survival was 25.5 months.Conclusion:The overall prognosis of BPDCN is poor.A prognostic stratification model may be helpful for the selection of therapy strategies.Chemotherapy bridging allo-HSCT remains an effective therapy with satisfactory safety at present,and the efficacy of targeted and immunotherapy needs to be further verified and improved.

关 键 词：母细胞性浆细胞样树突细胞肿瘤 异基因造血干细胞移植 嵌合抗原受体T细胞疗法 预后 

分 类 号：R733[医药卫生—肿瘤]