Limerick Emily, Abraham Allistair
Cellular and Molecular Therapeutics Branch, National Health, Lung, and Blood Institute, Bethesda, MD 20892, USA.
Division of Blood and Marrow Transplantation, Children's National Hospital, Washington, DC 20010, USA.
J Clin Med. 2022 Jul 3;11(13):3856. doi: 10.3390/jcm11133856.
One out of every five hundred African American children in the United States has sickle cell disease (SCD). While multiple disease-modifying therapies are available, hematopoietic cell transplantation (HCT) remains the only curative option for children with SCD. HLA-matched sibling HCT has demonstrated excellent efficacy, but its availability remains limited; alternative donor strategies are increasingly explored. While Busulfan-Cyclophosphamide has become the most widespread conditioning regimen employed in HCT for pediatric SCD, many other regimens have been examined. This review explores different conditioning regimens across the intensity spectrum: from myeloablative to non-myeloablative. We describe survival and organ function outcomes in pediatric SCD patients who have received HCT and discuss the strengths and weaknesses of the various conditioning intensities. Finally, we posit novel directions in allogeneic HCT for SCD.
在美国，每五百名非裔美国儿童中就有一人患有镰状细胞病（SCD）。虽然有多种疾病改善疗法可供选择，但造血细胞移植（HCT）仍然是患有SCD的儿童的唯一治愈选择。人类白细胞抗原（HLA）匹配的同胞造血细胞移植已显示出卓越的疗效，但其可用性仍然有限；人们越来越多地探索替代供体策略。虽然白消安-环磷酰胺已成为小儿SCD造血细胞移植中使用最广泛的预处理方案，但也研究了许多其他方案。本综述探讨了不同强度范围内的预处理方案：从清髓性到非清髓性。我们描述了接受造血细胞移植的小儿SCD患者的生存和器官功能结果，并讨论了各种预处理强度的优缺点。最后，我们提出了SCD异基因造血细胞移植的新方向。
