Allogeneic stem cell transplantation for patients with refractory anaemia with matched related and unrelated donors: delay of the transplant is associated with inferior survival
Allogeneic stem cell transplantation for patients with refractory anaemia with matched related and unrelated donors: delay of the transplant is associated with inferior survival
复制标题
DOI：
10.1111/j.1365-2141.2009.07809.x
复制
发表时间：
2009-09-01
影响因子：
6.5
通讯作者：
Niederwieser, Dietger
中科院分区：
文献类型：
--
作者：
de Witte, Theo;Brand, Ronald;Niederwieser, Dietger
同种异体干细胞移植（allogenic stem cell transplantation, alloSCT）治疗难治性贫血患者可获得50%的无事件生存率，但高非复发死亡率（non-relapse mortality， NRM）阻碍了这种治疗方式的普遍应用。本研究评估了各种移植前变量对结果的影响，包括疾病持续时间、调节方案的强度、供体类型和移植年份。研究人群包括374名患者；244例移植自人类白细胞抗原（HLA）——相同的兄弟姐妹，130例移植自匹配的非亲属供体。平均年龄为39岁。102例患者经减强度调节（RIC）后移植。总体4年生存率为52%。相同的兄弟姐妹供体和匹配的非亲属供体移植患者的4年生存率分别为52%和50%。多因素分析显示，近年来进行移植可提高生存率（P = 0中心点05），降低NRM （P = 0中心点02）。随着年龄的增长，疾病持续时间为1 ~ 12个月，生存率较低。RIC导致相似的生存，尽管复发风险增加（P = 0中心点02）。这种改善的结果允许50岁以上的患者进行同种异体干细胞移植，即使使用匹配的非亲属供体。在仔细分析预后变量后，应优先在诊断后早期进行同种异体细胞移植。
P>Allogeneic stem cell transplantation (alloSCT) for patients with refractory anaemia may result in a 50% event-free survival, but the high non-relapse mortality (NRM) precludes a general application of this therapeutic modality. This study evaluated the impact of various pre-transplant variables, including disease duration, intensity of the conditioning regimen, type of donor and year of transplantation on outcome. The study population consisted of 374 patients; 244 were transplanted from human leucocyte antigen (HLA)-identical siblings and 130 patients from matched unrelated donors. The median age was 39 years. One hundred and two patients were transplanted after reduced intensity conditioning (RIC). The overall 4-year survival was 52%. The 4-year survival of patients transplanted with HLA-identical sibling donors and matched unrelated donors was 52% and 50%, respectively. Multivariate analysis showed an improved survival (P = 0 center dot 05) and a lower NRM (P = 0 center dot 02) when the transplantation was performed in recent years. Increasing age, and disease duration of > 12 months were associated with inferior survival. RIC resulted in a similar survival despite an increased relapse risk (P = 0 center dot 02). This improved outcome permits alloSCT in patients older than 50 years of age, even with the use of matched unrelated donors. AlloSCT should be preferentially performed early after diagnosis after careful analysis of prognostic variables.