重型地贫危害极大，造血干细胞移植（HSCT）是根治的唯一方法。我们前期应用HSCT治疗地贫154例，取得了较好的疗效，无地贫生存（TFS）率达92%，但我们发现主要来源脐带血移植（CBT）的疗效明显比骨髓移植（BMT）差，TFS分别为86.3%和97.5%（P=0.0011）。我们推测这结果与CBT移植后造血和免疫重建延迟有关。本课题拟以不同干细胞来源（脐带和骨髓）HSCT地贫患者移植前和移植后不同时点的外周血和骨髓细胞等为对象，采用流式细胞技术、RT-PCR、Genescan分析、免疫磁珠分选、酶联免疫斑点法、混合淋巴细胞培养、造血祖细胞集落培养等实验方法，前瞻性研究和比较不同干细胞来源HSCT地贫患者移植后造血和免疫重建状况，特别是T细胞各亚群的数量及功能重建状况，探讨影响其造血和免疫重建的相关因素。为进一步提高地贫HSCT（特别是CBT）的临床疗效提供实验依据。

Thalassemia major is a severe hereditary disease. Hematopoietic stem cell transplantation(HSCT) is the only therapeutic option that can cure thalassemia disease.154 patients with thalassemia major had accepted HSCT in our hospital.142 (92%)patients were thalassemia-free survival(TFS).The outcome of the patients with cord blood transplantation(CBT) was inferior than that with bone marrow transplantation(BMT).TFS in CBT and BMT groups were 86.3% and 97.5% ,respectively(P=0.011).We suppose the inferior outcome of CBT is associated with delayed immunohematologic reconstitution after transplatation compared with BMT. In this study, we prospectively investigate the immunohematologic reconstitution in thalassemia major after HSCT with different stem cell source（CB and BM ）. We will study the blood and bone marrow samples of thalassemia patients before HSCT and 1,3,6,12 months posttransplatation. Flow-cytometric analysis, RT-PCR, MACS Cell Separation, Genescan analysis, Enzyme-Linked Immunospot assay, Mixed lymphocyte culture,Colony-forming cell (CFC) assay will be used. The results will help us understanding the character and the impact factor of immunohematologic reconstitution in thalassemia patient after HSCT with different stem cell sources. The study will help to improve the outcome of HSCT(particularly of CBT)for thalassemia in the future.