Department of Hematology, Christian Medical College, Vellore, India.
Hematology and Stem Cell Transplantation Section, Division of Hematology/Oncology, Department of Medicine, Vanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center and Veterans Affairs Medical Center, Nashville, TN, USA.
Bone Marrow Transplant. 2014 May;49(5):607-10. doi: 10.1038/bmt.2013.216. Epub 2014 Jan 20.
Allogeneic hematopoietic SCT remains the only treatment that can correct the hematological manifestations in patients with thalassemia major. Improving the clinical outcomes of high-risk, heavily transfused patients with liver fibrosis and inadequate iron chelation remains a challenge. Because of the relatively high probability of graft rejection and regimen-related toxicity in many patients receiving SCT for advanced thalassemia major, further development of new treatment regimens is warranted. This review addresses the reported clinical studies in patients with advanced thalassemia major and we have summarized our suggested conditioning approach to improve the outcome after SCT.
异基因造血干细胞移植仍然是唯一可以纠正重型地中海贫血患者血液学表现的治疗方法。改善伴有肝纤维化和铁螯合不足的高危、大量输血重型地中海贫血患者的临床结局仍然是一个挑战。由于许多接受晚期重型地中海贫血 SCT 的患者发生移植物排斥和与方案相关毒性的概率相对较高，因此需要进一步开发新的治疗方案。本综述讨论了晚期重型地中海贫血患者的报道临床研究，我们总结了我们建议的预处理方法，以改善 SCT 后的结局。
