Division of Hematology, Children's National Health System, Washington, District of Columbia.
Center for Cellular Therapies and Research, AABB, Bethesda, Maryland.
Biol Blood Marrow Transplant. 2018 Feb;24(2):219-227. doi: 10.1016/j.bbmt.2017.08.034. Epub 2017 Sep 1.
Hematopoietic cell transplantation (HCT) using an HLA-identical sibling donor offers a very high likelihood of cure with good outcomes for patients with sickle cell disease (SCD), and alternative donor HCT for SCD is an area of active clinical research. Thus, HCT is a potential option for a growing number of patients with SCD. This expanded use of HCT has raised several ethical questions. Who is eligible for HCT, in terms of both disease severity and psychosocial factors? Should affected children with matched sibling donors undergo HCT only when they have declared themselves as having significant symptomatology? Regarding donors, special ethical challenges include the use of preimplantation genetic diagnosis to conceive an HLA-identical sibling. In this review, we critically analyze various ethical challenges related to HCT for SCD, and offer recommendations to guide clinical care.
造血干细胞移植（HCT）使用 HLA 相同的同胞供者，为镰状细胞病（SCD）患者提供了非常高的治愈可能性和良好的转归，而异基因供者 HCT 是 SCD 的一个活跃的临床研究领域。因此，HCT 是越来越多 SCD 患者的潜在选择。HCT 的这种广泛应用引发了一些伦理问题。在疾病严重程度和心理社会因素方面，谁有资格进行 HCT？是否应该仅在患有严重症状的匹配同胞供者的患儿中进行 HCT？关于供者，特殊的伦理挑战包括使用胚胎植入前遗传学诊断来孕育 HLA 相同的同胞。在这篇综述中，我们批判性地分析了与 SCD 的 HCT 相关的各种伦理挑战，并提出了建议来指导临床护理。
