Wang Y N, Wang J S, Wei N, Wu L, Jin Z L, Wang Z
Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing 100050, China.
Zhonghua Xue Ye Xue Za Zhi. 2020 Jun 14;41(6):511-516. doi: 10.3760/cma.j.issn.0253-2727.2020.06.014.
This study was designed to evaluate the efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for adult-onset primary hemophagocytic lymphohistiocytosis (HLH) . 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. 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% , 34.9%-73.3%) and the transplant-related mortality (TRM) was 26.7% (4/15) . The five-year OS was 87.5% (95% , 38.7%-66.3%) in eight patients who received haplo-HSCT subsequent to initial therapy and 42.9% (95% , 8.5%-65.2%) in patients seven patients who needed salvage therapy prior to haplo-HSCT ((2)=2.387, =0.122) . The five-year OS was 85.7% (95% , 50.4%-89.8%) in eight patients who achieved complete response before haplo-HSCT and 42.9% (95% , 6.4%-53.0%) in seven patients with partial response ((2)=3.185, =0.074) . The results indicated that haplo-HSCT is a promising method for the treatment of primary HLH in adults.
本研究旨在评估单倍型造血干细胞移植（haplo-HSCT）治疗成人原发性噬血细胞性淋巴组织细胞增生症（HLH）的疗效。进行了一项回顾性研究，分析2013年1月至2019年10月在中国北京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患者的临床资料。纳入研究的15例患者中，男性10例，女性5例，中位年龄21岁（18-52岁）。其中8例患者为2型家族性噬血细胞性淋巴组织细胞增生症（FHL-2），4例为FHL-3，1例为2型格里塞利综合征（GS-2），1例为1型X连锁淋巴增殖性疾病（XLP-1），另1例为XLP-2。从HLH诊断到移植的中位时间为7个月（2-46个月）。7例患者在移植前接受了Bu/Cy预处理方案。同时，另外8例患者接受了TBI/Cy治疗。单核细胞（MNC）输注的中位浓度为12.6（9.2-20.3）×10⁸/kg，CD34⁺细胞为4.91（2.51-8.37）×10⁶/kg。白细胞植入的中位时间为移植后第13天（10-23天），血小板植入为第12天（9-36天）。最终有2例患者发生移植物失败（GF）（1例原发性GF和1例继发性GF）。急性移植物抗宿主病（GVHD）2至4级的累积发生率分别为71.4%（10/14）和慢性GVHD为30.8%（4/13）。15例原发性HLH患者的5年总生存率（OS）为65.5%（95%，34.9%-73.3%），移植相关死亡率（TRM）为26.7%（4/15）。8例在初始治疗后接受haplo-HSCT的患者5年OS为87.5%（95%，38.7%-66.3%），7例在haplo-HSCT前需要挽救治疗的患者5年OS为42.9%（95%，8.5%-65.2%）（χ²=2.387，P=0.122）。8例在haplo-HSCT前达到完全缓解的患者5年OS为85.7%（95%，50.4%-89.8%），7例部分缓解患者5年OS为42.9%（95%，6.4%-53.0%）（χ²=3.185，P=0.074）。结果表明，haplo-HSCT是治疗成人原发性HLH的一种有前景的方法。
