Objective: To analyze the clinical characteristics of adult patients with hemophagocytic lymphohistiocytosis (HLH) receiving haploidentical donor hematopoietic stem cell transplantation (HID HSCT). Method: We retrospectively reviewed 20 adult patients with HLH from August 2009 to August 2014.The clinical features and outcome were analyzed. Results: Conditioning regimens consisted of total body irradiation/etoposide/cyclophosphamide (TBI/VP-16/CTX) and busulfan (Bu)/VP-16/CTX in HLH with anti-thymocyte globulin (ATG) 8 mg/kg.The stem cells were mobilized from donors' peripheral blood.Median time to white blood cell engraftment was 13 (9-27) days.Median time to platelet engraftment was 14 (10-28) days.Mixed chimerism after transplantation developed in 4 patients and no patient presented graft failure.Eight patients developed grade Ⅱ to Ⅲ acute graft-versus-host disease (GVHD), while as chronic GVHD occurred in 9 patients.Among 12 patients with EB virus(EBV) reactivation, 2 patients developed post-transplant lymphoproliferative disorder (PTLD), 7 were suspected as PTLD and 3 were considered as relapse of primary disease.With a median follow-up of 20 months (range: 0.5-108 months) after transplantation, the estimated 2-year overall survival (OS) rate was (60.0±11.0)% in all patients.During the follow-up, 12 patients survived, 8 died including 5 within 100 days after HSCT.Among 5 non-remission patients before HSCT, 4 patients died within 100 days after HCT. Conclusions: HID HSCT is an effective treatment for adult patients with HLH to achieve remission and long-term survival. High proportion of mixed chimerism has been seen at early stage after transplantation.EBV reactivation and early transplant-related mortality are common.
目的： 探讨亲缘单倍体造血干细胞移植(HID HSCT)治疗成人噬血细胞性淋巴组织细胞增生症(HLH)的临床特征。 方法： 回顾性分析首都医科大学附属北京友谊医院2009年8月至2014年8月HID HSCT治疗的20例成人HLH的疗效和预后。 结果： 预处理方案包括全身照射/依托泊苷/环磷酰胺和马利兰/依托泊苷/环磷酰胺，抗人胸腺细胞球蛋白剂量为8 mg/kg。干细胞来源均为外周血。中位白细胞植活时间13 (9～27) d，中位血小板植活时间14 (10～28) d。移植后出现混合嵌合状态4例，无病例植入失败。发生Ⅱ～Ⅳ度急性移植物抗宿主病8例，慢性移植物抗宿主病9例。EB病毒(EBV)复燃12例，复燃中位时间33(15～80) d，其中确诊移植后淋巴组织增殖性疾病(PTLD) 2例，疑似PTLD 7例，原发病复发3例。移植后中位随访20 (0.5～108)个月，2年总生存率为(60.0±11.0)%。截至随访结束，存活12例，死亡8例，死于移植后100 d内5例。移植前未获得缓解者5例，4例死亡，均死于移植后100 d内。 结论： HID HSCT治疗成人HLH在移植早期容易发生混合嵌合，EBV复燃发生显著，移植早期相关死亡表现突出。.
Keywords: Haploidentical donor; Hematopoietic stem cell transplantation; Lymphohistiocytosis, hemophagocytic; Mixed chimerism.