单倍体造血干细胞移植治疗母细胞性浆细胞样树突状细胞肿瘤的临床研究  

Clinical study of haploidentical hematopoietic stem cell transplantation for treatment of blastic plasmacytoid dendritic cell neoplasm

作　　者：沈耀耀 张旭辉[1] 陈佳[1] 赵晔[1] 陈淳 仇惠英[1] 吴德沛[1] 

机构地区：[1]苏州大学附属第一医院,江苏省血液研究所,苏州大学造血干细胞移植研究所,国家卫生计生委血栓与止血重点实验室,血液学协同创新中心,215006

出　　处：《国际输血及血液学杂志》2017年第6期476-481,共6页International Journal of Blood Transfusion and Hematology

基　　金：国家重点研发计划（2016YFC0902803）;江苏省创新能力建设专项（BM2015004）;国家自然科学基金面上项目（81730003、81470346）

摘　　要：目的探讨单倍体造血干细胞移植（haplo-HSCT）治疗母细胞性浆细胞样树突状细胞肿瘤（BPDCN）的方案及疗效。方法选择2016年5月至2017年6月，于苏州大学附属第一医院住院的2例经化疗获得缓解，并且成功接受haplo-HSCT的BPDCN患者为研究对象。回顾性分析这2例患者的临床表现、实验室检查结果、治疗策略及预后情况，并结合国内外相关文献，探讨该病的诊断及治疗研究进展。 结果①患者1，因＂无痛性皮肤结节＂起病，病变累及鼻咽部及骨髓。其免疫组织化学检查结果显示：CD123＋、CD56＋、CD4＋、Ki67＋（30%-40%）、CD31＋、CD33weak。该例患者接受6个疗程的全身化疗达缓解后，成功接受父供女haplo-HSCT。该例患者haplo-HSCT过程中，共计输注单个核细胞（MNC）11.58×108/kg，CD34＋细胞5.00×106/kg。其接受haplo-HSCT后，第15天获得造血功能重建。截至2017年9月10日，该例患者已无病生存15个月。②患者2，因＂全身皮肤散在结节＂起病，病变累及骨髓。其免疫组织化学结果显示：CD34-、CD68＋＋、CD3-、CD20-、CD4weak、CD8-、S100＋＋、CK-、CD56weak，EB病毒编码小RNA（EBER）原位杂交结果呈阴性、髓过氧化物酶（MPO）呈阴性、末端脱氧核苷酸转移酶（TdT）呈阴性、Melan-A-、CD1a-、Ki67＋（75%-80%）、CD123-、CD56＋。该例患者接受2个疗程化疗达缓解后，接受父供子haplo-HSCT。该例患者haplo-HSCT过程中，共计输注MNC 13.57×108/kg，CD34＋细胞2.69×106/kg。其接受haplo-HSCT后，第11天获得造血功能重建。截至2017年9月10日，该例患者已无病生存3＋个月。结论BPDCN是一种具有高度侵袭性的血液系统恶性肿瘤，CD4＋及CD56＋为其典型的免疫表型。放化疗可使BPDCN患者获得暂时缓解，异基因造血干细胞移植（allo-HSCT）可显著改善该病预后，使患者获得长期生存。haplo-HSCT更因其供体易获得、疗效可靠�Objective To investigate the treatment protocols and therapeutic effects of haploidentical hematopoietic stem cell transplantation （haplo-HSCT） in treatment of patients with blastic plasmacytoid dendritic cell neoplasm （BPDCN）. Methods From May 2016 to June 2017, two patients hospitalized at the First Affiliated Hospital of Soochow University who were relieved by chemotherapy and were successfully treated with haplo-HSCT were enrolled in this study. The clinical manifestations, laboratory characteristics, treatment protocols and prognosis of these two patients were retrospectively analyzed, and the diagnosis and treatment progress of BPDCN were discussed combined with relevant literatures at home and abroad.Results ① Case one presented with painless skin nodules. The immunohistochemistry results of tumors involving the nasopharynx and bone marrow were CD123＋ , CD56＋ , CD4＋ , Ki67＋ （30%-40%）, CD31＋ and CD33Week. This patient who achieved disease remission with 6 courses of systemic chemotherapy received haplo-HSCT successfully, with 13. 57 ）〈 108/kg of MNC and 5. 00×10^6/kg of CD34＋ cells. Her hematopoietic reconstitution was on the 15th day after transplantation, which resulting a long-lasting remission for 15 months up to 10 September 2017. ② Case two presented with generalized and discrete skin nodules. The immunohistochemistry results of tumors involving the bone marrow were CD34- , CD68＋＋ , CD3-, CD20-, CD4weak, CD8-, S100＋＋, CK , CD56weak, Epstein-Barr virus-encoded mall RNA （EBER）-, myeloperoxidase （MPO）-, terminal, deoxynucleotidyl transferase （TdT） , Melan-A-, CDla , Ki67＋ （75%-80%）, CD123- and CD56＋. This patient who achieved disease remission with 2 courses of systemic chemotherapy received haplo-HSCT successfully, with 11.58 ×10^8/kg of MNC and 2.69×10^6/kg of CD34＋ cells. His hematopoietic reconstitution was on the llth day after transplantation, which resulting a remission for more than 3 months up to 10 September 2017. Conclusions BPDC

关 键 词：肿瘤 树突细胞 浆细胞 造血干细胞移植 药物疗法 

分 类 号：R733[医药卫生—肿瘤]