Haematopoietic stem cell transplantation for patients with myelodysplastic syndromes and secondary acute myeloid leukaemias: a report on behalf of the Chronic Leukaemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT)
Haematopoietic stem cell transplantation for patients with myelodysplastic syndromes and secondary acute myeloid leukaemias: a report on behalf of the Chronic Leukaemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT)
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DOI：
10.1046/j.1365-2141.2000.02200.x
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发表时间：
2000-09-01
影响因子：
6.5
通讯作者：
Apperley, J
中科院分区：
文献类型：
--
作者：
de Witte, T;Hermans, J;Apperley, J
来自hla相同的兄弟姐妹供体的同种异体干细胞移植是治疗受年龄和缺乏兄弟姐妹供体限制的骨髓增生异常综合征年轻患者的一种治疗选择。替代干细胞来源最近被广泛使用，如无血缘关系的供体、不相同的家庭成员或自体移植。欧洲血液和骨髓移植组织（EBMT）报告了1378例移植的分析，根据已知的危险因素解决了不同手术的结果。在885例移植了来自配对兄弟姐妹的干细胞的患者中，估计的无病生存期（DFS）和估计的3年复发风险均为36%。在多变量分析中，年龄和疾病分期对DFS、生存和治疗相关死亡率具有独立的预后意义。在疾病早期移植的患者复发的风险明显低于晚期移植的患者。198例自愿非亲属供体患者3年的估计DFS为25%，91例替代家庭供体患者为28%，126例首次完全缓解的自体移植患者为33%。非亲属供体患者的非复发死亡率为58%，非同一家族供体患者为66%，自体移植患者为25%。不同家族供体患者的复发率为18%，非亲属供体患者的复发率为41%，自体干细胞移植患者的复发率为55%。同种异体和自体干细胞移植已成为骨髓增生异常综合征患者的治疗选择。hla相同的同胞供体移植是首选的治疗方案。没有兄弟姐妹供体的患者可以接受自体干细胞移植或替代供体移植。年龄小于20岁的患者可采用非亲属供体移植治疗。年龄超过40岁的患者，也可能是年龄在20到40岁之间的患者，可能从自体干细胞移植中获益最多。
Allogeneic stem cell transplantation from an HLA-identical sibling donor is a curative treatment option for a young patient with myelodysplastic syndrome, limited by age and lack of sibling donors. Alternative stem cell sources have been used more recently, such as unrelated donors, non-identical family members or autologous transplants. This analysis of 1378 transplants reported to the European Group for Blood and Marrow Transplantation (EBMT) addresses the outcome of the varying procedures according to the known risk factors. The estimated disease-free survival (DFS) and estimated relapse risk at 3 years were both 36% for 885 patients transplanted with stem cells from matched siblings. In the multivariate analysis, age and stage of disease had independent prognostic significance for DFS, survival and treatment-related mortality. Patients transplanted at an early stage of disease had a significantly lower risk of relapse than patients transplanted at more advanced stages. The estimated DFS at 3 years was 25% for the 198 patients with voluntary unrelated donors, 28% for the 91 patients with alternative family donors and 33% for the 126 patients autografted in first complete remission. The non-relapse mortality was 58% for patients with unrelated donors, 66% for patients with non-identical family donors and 25% for autografted patients. The relapse rate of 18% was relatively low for patients with non-identical family donors, 41% for patients with unrelated donors and 55% for patients treated with autologous stem cell transplantation, Both allogeneic and autologous stem cell transplantation have emerged as treatment options for patients with myelodysplastic syndromes. Transplantation with an HLA-identical sibling donor is the preferred treatment option. Patients without an sibling donor may be treated with either autologous stem cell transplantation or an alternative donor transplantation. Patients: younger than 20 years may be treated with an unrelated donor transplantation. Patients older than 40 years, and probably also patients between 20 and 40 years, may benefit most from an autologous stem cell transplantation.