摘要
目的探讨单倍型造血干细胞移植(haplo-HSCT)治疗成人原发性噬血细胞综合征(HLH)的疗效。方法对2013年1月至2019年10月期间于首都医科大学附属北京友谊医院接受haplo-HSCT的15例成人原发性HLH患者进行回顾性分析。结果全部15例患者中男10例、女5例,中位年龄21(18~52)岁。家族性噬血细胞综合征2型(FHL-2)8例,FHL-3 4例,Griscelli综合征2(GS-2)、X连锁淋巴组织增生综合征1型(XLP-1)、XLP-2各1例。确诊HLH至接受haplo-HSCT的中位时间为7(2~46)个月。所有患者移植前均接受HLH-94、HLH-2004或DEP方案诱导化疗,8例获得完全应答(CR),7例获得部分应答(PR)。移植预处理:Bu/Cy方案7例,TBI/Cy方案8例。输注单个核细胞12.6(9.2~20.3)×10^8/kg,CD34+细胞4.91(2.51~8.37)×10^6/kg。粒细胞植活中位时间为13(10~23)d,血小板植活中位时间为12(9~36)d。原发、继发性植入失败各1例。Ⅱ~Ⅳ度急性移植物抗宿主病(GVHD)发生率为71.4%(10/14),慢性GVHD发生率为30.8%(4/13)。移植后预期5年总生存(OS)率为65.5%(95%CI 34.9%~73.3%),移植相关死亡率为26.7%(4/15)。一线诱导治疗后立即接受移植(8例)、二线诱导治疗难治/复发患者(7例)的移植后预期5年OS率分别为87.5%(95%CI 38.7%~66.3%)、42.9%(95%CI 8.5%~65.2%)(χ^2=2.387,P=0.122)。移植前CR(8例)、PR(7例)患者的移植后预期5年OS率分别为85.7%(95%CI 50.4%~89.8%)、42.9%(95%CI 6.4%~53.0%)(χ^2=3.185,P=0.074)。结论 haplo-HSCT是成人原发性HLH的有效治疗手段。
Objective This study was designed to evaluate the efficacy of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)for adult-onset primary hemophagocytic lymphohistiocytosis(HLH).Method A retrospective study was carried out to analyze the clinical data of 15 adult patients with primary HLH who received haplo-HSCT from January 2013 to October 2019 in Beijing Friendship Hospital,Capital Medical University,Beijing,China.Results Among the 15 patients included in the study,ten were males and five were females,with a median age of 21 years old(18-52).Eight of the patients had familial hemophagocytic lymphohistiocytosis type 2(FHL-2),four had FHL-3,one had Griscelli syndrome type 2(GS-2),one had X-linked lymphoproliferative disease type 1(XLP-1),and the other had XLP-2.The median time from HLH diagnosis to transplantation was 7 months(2-46 months).Seven patients were treated with Bu/Cy condition regimen prior to transplantation.Meanwhile,the other eight cases were treated with TBI/Cy.The median concentration of mononuclear cell(MNC)infusion was 12.6(9.2-20.3)×10^8/kg and CD34+cells was 4.91(2.51-8.37)×10^6/kg.The median time of leukocyte engraftment was on day 13 following transplantation(10-23 days),and the platelet engraftment was on day 12(9-36).Graft failure(GF)finally occurred in two patients(one primary GF and one secondary GF).The cumulative incidence of acute graft-versus-host-disease(GVHD)grades 2 to 4 was 71.4%(10/14)and chronic GVHD was 30.8%(4/13),respectively.The five-year overall survival(OS)for all 15 cases of primary HLH was 65.5%(95%CI,34.9%-73.3%)and the transplant-related mortality(TRM)was 26.7%(4/15).The five-year OS was 87.5%(95%CI,38.7%-66.3%)in eight patients who received haplo-HSCT subsequent to initial therapy and 42.9%(95%CI,8.5%-65.2%)in patients seven patients who needed salvage therapy prior to haplo-HSCT(χ^2=2.387,P=0.122).The five-year OS was 85.7%(95%CI,50.4%-89.8%)in eight patients who achieved complete response before haplo-HSCT and 42.9%(95%CI,6.4%-53.0%)in seven patients with partial response(χ^2=3.185,P=0.074).Conclusion The results indicated that haplo-HSCT is a promising method for the treatment of primary HLH in adults.
作者
王旖旎
王晶石
魏娜
吴林
金志丽
王昭
Wang Yini;Wang Jingshi;Wei Na;Wu Lin;Jin Zhili;Wang Zhao(Department of Hematology,Beijing Friendship Hospital,Capital Medical University,Beijing 100050,China)
出处
《中华血液学杂志》
CAS
CSCD
北大核心
2020年第6期511-516,共6页
Chinese Journal of Hematology
基金
国家自然科学基金(81871633)
北京市自然科学基金(7181003)
北京市医院管理局扬帆计划临床技术创新项目(XMLX201823)
北京市医院管理局登峰团队人才项目(DFL20180101)。
关键词
原发性噬血细胞综合征
成人
造血干细胞移植
单倍型
Primary hemophagocytic lymphohistiocytosis
Adult onset
Hematopoietic stem cell transplantation
Haploidentical
