Sickle Cell Branch, National Heart, Lung, and Blood Institute, Bethesda, MD; Department of Hematology, Washington Hospital Center/Georgetown University, Washington, DC.
Division of Blood and Marrow Transplantation, Children's National Health System, Washington, DC.
Semin Hematol. 2018 Apr;55(2):94-101. doi: 10.1053/j.seminhematol.2018.04.013. Epub 2018 May 3.
Many patients with sickle cell disease experience severe morbidity and early mortality. The only curative option remains hematopoietic stem cell transplantation. Although HLA-matched sibling transplantation has been very successful for adults and children, the vast majority of patients with sickle cell disease do not have an HLA-matched sibling. Alternative donor options include haploidentical, unrelated umbilical cord blood, and matched unrelated donor transplantation. This report summarizes major alternative donor transplantation studies reported to date and ongoing and upcoming clinical trials. We conclude that when there is no HLA-match, all these approaches should be systematically considered before ruling out the option of hematopoietic stem cell transplantation.
许多镰状细胞病患者经历严重的发病和早期死亡。唯一的根治方法仍然是造血干细胞移植。虽然 HLA 匹配的同胞供体移植对成人和儿童非常成功,但绝大多数镰状细胞病患者没有 HLA 匹配的同胞。其他供体选择包括半相合、非相关脐带血和匹配的非相关供体移植。本报告总结了迄今为止报告的主要替代供体移植研究以及正在进行和即将进行的临床试验。我们的结论是,当没有 HLA 匹配时,在排除造血干细胞移植的可能性之前,所有这些方法都应系统地考虑。