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(guó)際頂級(jí)血液學(xué)期刊《血液》（Blood）雜志上。國(guó)際權(quán)威專(zhuān)家評(píng)論說(shuō)：“該成果代表了當(dāng)前國(guó)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(guó)在該領(lǐng)域的臨床研究已達(dá)國(guó)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(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.