Department of Medicine, Division of Hematology/Oncology, Vanderbilt-Meharry Center for Excellence in Sickle Cell Disease, Vanderbilt University Medical Center , Nashville , TN , USA.
College of Medicine, Division of Pediatric Hematology & Oncology, Lagos University Teaching Hospital , Lagos , Nigeria.
Expert Rev Hematol. 2019 Sep;12(9):733-752. doi: 10.1080/17474086.2019.1642103. Epub 2019 Jul 19.
: Severe sickle cell disease is associated with progressive end-organ damage and early mortality in adults. While allogeneic hematopoietic cell transplant from a matched related donor is curative, the vast majority of patients do not have a compatible sibling. Accordingly, platforms using haploidentical donors have been developed, which provide near-universal availability. : This review focuses on the two commonly used approaches for haploidentical hematopoietic transplants, namely T-cell deplete and T-cell replete, each of which is associated with unique benefits and drawbacks. The purpose of this paper is to facilitate individualized decision-making for patients and providers by reviewing the pros- and cons of these differing approaches. : Individuals with sickle cell disease eligible for a hematopoietic cell transplant can be considered based on recent results. Comparable outcomes are seen with T-cell deplete and T-cell replete approaches. The choice depends largely on institutional preference.
严重镰状细胞病与成人进行性终末器官损害和早期死亡有关。虽然异基因造血细胞移植来自匹配的相关供体是治愈性的,但绝大多数患者没有相容的兄弟姐妹。因此,已经开发了使用半相合供体的平台,这提供了近乎普遍的可用性。这篇综述重点介绍了两种常用于半相合造血移植的方法,即 T 细胞耗竭和 T 细胞再填充,每种方法都有其独特的优点和缺点。本文的目的是通过回顾这些不同方法的优缺点,为患者和提供者的个体化决策提供便利。有资格进行造血细胞移植的镰状细胞病患者可以根据最近的结果进行考虑。T 细胞耗竭和 T 细胞再填充方法的结果相当。选择主要取决于机构的偏好。