Krishnamurti Lakshmanan
Aflac Cancer and Blood Disorders Center, Emory University, Children's Healthcare of Atlanta, Atlanta, GA, USA.
Hematol Oncol Stem Cell Ther. 2020 Jun;13(2):53-57. doi: 10.1016/j.hemonc.2019.12.008. Epub 2020 Mar 12.
Availability of an HLA-identical sibling donor raises the question, "should young children with SCD, and an available HLA identical sibling donor be considered for hematopoietic cell transplantation (HCT) even before they manifest severe clinical presentations of sickle cell disease (SCD)?" The overall survival (OS) and event free survival (EFS) following HCT from an HLA identical sibling is excellent in young children, and worsen with increasing age at HCT. SCD related complications, organ dysfunction, quality of life, and risk for premature mortality all worsen with age. The ethical principles of non-maleficence, beneficence, autonomy and justice all support the consideration of this life, quality of life, and organ saving therapy at a young age.
有一个 HLA 匹配的同胞供者就引发了这样一个问题：“患有镰状细胞病（SCD）且有可用的 HLA 匹配同胞供者的幼儿，即使在他们尚未出现镰状细胞病的严重临床表现之前，是否就应考虑进行造血细胞移植（HCT）？” 来自 HLA 匹配同胞的 HCT 后，幼儿的总生存期（OS）和无事件生存期（EFS）非常好，且会随着 HCT 时年龄的增加而变差。与 SCD 相关的并发症、器官功能障碍、生活质量以及过早死亡风险均会随着年龄增长而恶化。不伤害、有益、自主和公正等伦理原则均支持在幼儿期考虑这种挽救生命、提高生活质量和保护器官的治疗方法。
