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近日，南方醫(yī)科大學(xué)南方醫(yī)院兒童造血干細(xì)胞移植中心一項(xiàng)有關(guān)治療地中海貧血的臨床研究論著發(fā)表在國際頂級血液學(xué)期刊《血液》（Blood）雜志上。國際權(quán)威專家評論說：“該成果代表了當(dāng)前國際上造血干細(xì)胞移植治療地貧的顯著進(jìn)步，標(biāo)志著中國在該領(lǐng)域的臨床研究已達(dá)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副主席、南方醫(yī)科大學(xué)南方醫(yī)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.