Hoppe C C, Walters M C
Department of Hematology/Oncology, Children's Hospital Oakland, Oakland, California 96409, USA.
Curr Opin Oncol. 2001 Mar;13(2):85-90. doi: 10.1097/00001622-200103000-00001.
Hematopoietic cell transplantation (HCT) is a treatment with curative potential for sickle cell disease (SCD). The experience of HCT for persons with beta-thalassemia major has been successfully extended to SCD. Currently, the event-free survival rate after allogeneic-matched sibling HCT for SCD is 82%. However, short-term and long-term transplant-related complications remain substantial barriers to HCT, particularly in older patients with life-long complications of SCD. Novel conditioning regimens that minimize transplant-associated toxicity have been developed and show promise for wider application of HCT. Alternative stem cell sources may also expand the availability of HCT for selected patients with SCD.
造血细胞移植（HCT）是一种对镰状细胞病（SCD）具有治愈潜力的治疗方法。重型β地中海贫血患者的HCT经验已成功扩展至SCD。目前，SCD患者接受异基因匹配同胞HCT后的无事件生存率为82%。然而，短期和长期的移植相关并发症仍然是HCT的重大障碍，尤其是在患有SCD终身并发症的老年患者中。已开发出可将移植相关毒性降至最低的新型预处理方案，并显示出HCT更广泛应用的前景。替代干细胞来源也可能扩大HCT对选定SCD患者的可及性。
