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近日，南方醫科大學(xué)南方醫院兒童造血干細胞移植中心一項有關(guān)治療地中海貧血的臨床研究論著(zhù)發(fā)表在國際頂級血液學(xué)期刊《血液》（Blood）雜志上。國際權威專(zhuān)家評論說(shuō)：“該成果代表了當前國際上造血干細胞移植治療地貧的顯著(zhù)進(jìn)步，標志著(zhù)中國在該領(lǐng)域的臨床研究已達國際領(lǐng)先水平。”

文章的第一作者和通訊作者是亞洲兒童骨髓移植臨床研究委員會(huì )副主席、南方醫科大學(xué)南方醫院兒科主任李春富教授。李春富曾于1999年成功地為一例重型Ⅲβ-地中海貧血男孩，完成了母親供髓HLA不全相合骨髓移植，這是全球首例成功病例。

We used a novel NF-08-TM transplant protocol based on intravenous busulfan, cyclophosphamide, fludarabine, and thiotepa in 82 consecutive patients with β-thalassemia major (TM), including 52 with allogeneic pe**heral blood stem cell transplantation (PBSCT) from unrelated donors (UD) with well-matched human leukocyte antigens and 30 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSD). The median age at transplantation was 6.0 years (range: 0.6-15.0), and the ratio of male to female patients was 56:26. The median follow-up time was 24 months (range: 12-39 months). The estimated 3-year overall survival and TM-free survival were 92.3% and 90.4% in the UD-PBSCT group and 90.0% and 83.3% in the MSD-HSCT group. The cumulative incidences of graft rejection and grade III-IV acute graft-versus-host disease were 1.9% and 9.6%, respectively, in the UD-PBSCT group and 6.9% and 3.6%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality was 7.7% in the UD-PBSCT group and 10.0% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat β thalassemia patients in the absence of MSD.