Dipartimento di Ematologia ed Oncologia Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
Pediatr Blood Cancer. 2012 Aug;59(2):372-6. doi: 10.1002/pbc.24177. Epub 2012 Apr 27.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents the only curative treatment for sickle cell disease (SCD), being successful in around 85-90% of patients. Mortality and long-term morbidity (including infertility, gonadal failure, and chronic graft-vs.-host disease) associated with conventional approaches curtail the number of patients who undergo allo-HSCT. Recently, it has been demonstrated that cord blood is as effective as and possibly safer than bone marrow in pediatric patients with SCD. Likewise, transplant strategies based on the use of reduced-intensity regimens and the induction of mixed chimerism have been explored to decrease allo-HSCT short- and long-term complications.
异基因造血干细胞移植（allo-HSCT）是治疗镰状细胞病（SCD）的唯一根治方法，在约 85-90%的患者中取得成功。传统方法相关的死亡率和长期发病率（包括不孕、性腺功能衰竭和慢性移植物抗宿主病）限制了接受 allo-HSCT 的患者数量。最近已经证明，对于 SCD 儿科患者而言，脐带血与骨髓一样有效，而且可能更安全。同样，已经探索了基于使用低强度方案和诱导混合嵌合体的移植策略，以减少 allo-HSCT 的短期和长期并发症。
