National Cancer Institute.
Blood. 2018 Mar 22;131(12):1272-1274. doi: 10.1182/blood-2018-02-826461.
In this issue of , Tholouli et al describe successful in vivo T-cell–depleted allogeneic hematopoietic stem cell transplant (HSCT) in 4 patients with GATA2 deficiency using a reduced-intensity conditioning (RIC) regimen including serotherapy with alemtuzumab. Three immediate questions come to mind when contemplating the first sentence, namely: what is GATA2 deficiency, what is the natural history of the disease that leads to HSCT, and what makes HSCT so special for GATA2 deficiency that it warrants a commentary?
本期 ，Tholouli 等人描述了使用包含阿仑单抗血清疗法的减强度预处理方案，成功地对 4 例 GATA2 缺陷患者进行了 T 细胞耗竭的同种异体造血干细胞移植（HSCT）。在考虑第一句话时，有三个直接的问题，即：什么是 GATA2 缺陷，导致 HSCT 的疾病的自然史是什么，以及是什么使 HSCT 对 GATA2 缺陷如此特别，以至于值得评论？
