Department of Pathology, St. Jude Children's Research Hospital, MS 342, 262 Danny Thomas Place, Memphis, TN 38105, USA.
Department of Pediatrics, The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Abramson Research Center Room 316D, Philadelphia, PA 19010, USA.
Clin Lab Med. 2021 Mar;41(1):101-119. doi: 10.1016/j.cll.2020.10.007. Epub 2020 Dec 24.
Red blood cell (RBC) transfusion is critical in managing acute and chronic complications of sickle cell disease. Alloimmunization and iron overload remain significant complications of transfusion therapy and are minimized with prophylactic Rh and K antigen RBC matching and iron chelation. Matched sibling donor hematopoietic stem cell transplant (HSCT) is a curative therapeutic option. Autologous hematopoietic stem cell (HSC)-based gene therapy has recently shown great promise, for which obtaining sufficient HSCs is essential for success. This article discusses RBC transfusion indications and complications, transfusion support during HSCT, and HSC mobilization and collection for autologous HSCT with gene therapy.
红细胞（RBC）输血对于治疗镰状细胞病的急性和慢性并发症至关重要。同种免疫和铁过载仍然是输血治疗的严重并发症，通过预防性 Rh 和 K 抗原 RBC 匹配和铁螯合可以将其最小化。同胞供体造血干细胞移植（HSCT）是一种有治愈希望的治疗选择。自体造血干细胞（HSC）为基础的基因治疗最近显示出巨大的潜力，为此成功的关键是获得足够的 HSCs。本文讨论了 RBC 输血的适应证和并发症、HSCT 期间的输血支持，以及用于自体 HSCT 与基因治疗的 HSC 动员和采集。
