近日，南方医科大学南方医院儿童造血干细胞移植中心一项有关治疗地中海贫血的临床研究论着发表在国际顶级血液学期刊《血液》（Blood）杂志上。国际权威专家评论说：“该成果代表了当前国际上造血干细胞移植治疗地贫的显着进步，标志着中国在该领域的临床研究已达国际领先水平。”
文章的第一作者和通讯作者是亚洲儿童骨髓移植临床研究委员会副主席、南方医科大学南方医院儿科主任李春富教授。李春富曾于1999年成功地为一例重型Ⅲβ-地中海贫血男孩，完成了母亲供髓HLA不全相合骨髓移植，这是全球首例成功病例。
DOI:10.1182/blood-2012-03-417998
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A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation
Chunfu Li1,*, Xuedong Wu1, Xiaoqing Feng1, Yuelin He1, Huaying Liu1, Fuyu Pei1, Jianyu Liao1, Lan He1, Lei Shi1, Na Li1, Qiujun Liu1, Shiting Liu2, Geyu Chen1, Qingxia Su1, Yuqiong Ren1, Yanhua Wang1, and Wanxia Tan1
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 peripheral 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.
