HLA-mismatched/haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for chronic myeloid leukemia:: Improved outcomes in patients in accelerated phase and blast crisis phase
HLA-mismatched/haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for chronic myeloid leukemia:: Improved outcomes in patients in accelerated phase and blast crisis phase
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DOI：
10.1080/07853890801908903
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发表时间：
2008-01-01
影响因子：
4.4
通讯作者：
Lu Dao-Pei
中科院分区：
文献类型：
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作者：
Huang Xiao-Jun;Xu Lan-Ping;Lu Dao-Pei
背景。同种异体造血干细胞移植（Allogeneic hematopoietic stem cell transplantation, alloo - hsct）是目前唯一被证实的治疗慢性髓性白血病（chronic myeloid leukemia， CML）的方法，但缺乏人类白细胞抗原（human leukocyte antigen， HLA）匹配的同胞或非亲属供体限制了其应用。最近，我们开发了一种单倍体同种异体造血干细胞移植的有效方法，其结果与同种hla移植相当。评估接受单倍同种异体造血干细胞移植的CML患者的预后。93例患者采用改良的丁硫丹(BU)/环磷酰胺（CY）2方案治疗，包括抗胸腺细胞球蛋白，然后进行未经处理的血液和骨髓移植。我们的数据显示，急性移植物抗宿主病（GVHD）的累积发病率为64.52%，III-IV级为26.45%，61.79%为慢性移植物抗宿主病，28.93%为广泛慢性移植物抗宿主病。非复发死亡率分别为8.72%（100天）、20.72%（1年）和20.72%（2年）。在慢性期（CP） 1、CP2/CR2、加速期和blast危象患者中，1年和4年无白血病生存的概率相似。4年总生存率分别为76.5% （CP1）、85.7% （CP2/CR2）、73.3%（加速期）和61.5% （blast危象）。多因素分析表明，影响移植结果的因素包括：II-III期急性GVHD和III-IV期急性GVHD患者的HLA-B+DR配错、移植时疾病复发的分期、从诊断到移植的无白血病生存期、总生存期和移植相关死亡率。在我们的方案中，HSCT治疗晚期CML的生存率与稳定期相似。对于缺乏hla相同的亲属供体的患者，单倍体相同的亲属是替代的HSCT供体。
Background. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only proven curative therapy for chronic myeloid leukemia (CML), but lack of human leukocyte antigen (HLA)-matched sibling or unrelated donors has restricted its application. Recently, we developed an effective method for haploidentical allo-HSCT achieving comparable outcomes to HLA-identical transplantation.Aim. To evaluate the outcomes of CML patients who underwent haploidentical allo-HSCT.Methods. Ninety-three patients were treated with a modified busulfan (BU)/cyclophosphamide (CY)2 regimen, including antithymocyte globulin followed by unmanipulated blood and marrow transplantation.Results. Our data showed that the cumulative incidence of acute graft-versus-host disease (GVHD) was 64.52%, and grade III-IV was 26.45%, 61.79% had chronic GVHD, and 28.93% had extensive chronic GVHD. Non-relapse mortality varied at 8.72% (100 days), 20.72% (1 year) and 20.72% (2 years). Probability of 1-year and 4-year leukemia-free survival was similar in chronic phase (CP) 1, CP2/CR2, accelerated phase, and blast crisis patients. Probability of 4-year overall survival varied as 76.5% (CP1), 85.7% (CP2/CR2), 73.3% (accelerated phase), and 61.5% (blast crisis). Multivariate analysis indicated that factors affecting transplantation outcomes were HLA-B+DR mismatches versus others for II-III acute GVHD and III-IV acute GVHD, the stage of disease at transplantation for relapse, and the time from diagnosis to transplantation for leukemia-free survival, overall survival, and transplantation-related mortality. In our protocol, survival of HSCT for advanced CML was similar to stable stage.Conclusions. For patients lacking an HLA-identical related donor, haploidentical relatives are alternative HSCT donors.