Division of Pediatric Hematology/Oncology, Washington University, St Louis Children's Hospital, St Louis, MO 63110, USA.
Hematology Am Soc Hematol Educ Program. 2011;2011:273-9. doi: 10.1182/asheducation-2011.1.273.
Allogeneic HSCT controls sickle cell disease (SCD)-related organ damage and is currently the only curative therapy available. Over the last 2 decades, HSCT has been limited largely to myeloablative matched sibling donor (MSD) procedures that are feasible only in a minority of patients. As the natural history of the disease has evolved, it is clear that subsets of patients with severe disease are at risk for sudden death, devastating CNS and pulmonary complications, and debilitating vasoocclusive crises. For these patients, the benefits of transplantation can outweigh the risks if HSCT can be safely and successfully performed with low early and late toxicities. This review describes advances and ongoing investigation of HSCT for SCD from the perspectives of recipient age and presentation, donor stem cell source, intensity of conditioning, family and medical perspectives, and other variables that influence outcome. Ultimately, HSCT should be viewed as a viable treatment option for SCD on par with other therapies for select patients who can benefit from the procedure.
异基因造血干细胞移植（HSCT）可控制镰状细胞病（SCD）相关器官损伤，是目前唯一可用的根治性疗法。在过去的 20 年中，HSCT 主要局限于清髓性匹配同胞供者（MSD）程序，而该程序仅适用于少数患者。随着疾病自然史的发展，很明显，一些严重疾病的患者存在猝死、严重中枢神经系统和肺部并发症以及使人虚弱的血管阻塞性危象的风险。对于这些患者，如果 HSCT 可以安全且成功地进行，并且早期和晚期毒性较低，则移植的益处可能超过风险。本综述从受者年龄和表现、供者干细胞来源、预处理强度、家庭和医疗观点以及影响结果的其他变量等方面描述了 SCD 患者 HSCT 的进展和正在进行的研究。最终，应将 HSCT 视为 SCD 的一种可行治疗选择，与其他可从该程序中受益的特定患者的疗法相媲美。
