Allogeneic hematopoietic stem cell transplantation for patients with aplastic anemia
Allogeneic hematopoietic stem cell transplantation for patients with aplastic anemia
复制标题
异基因造血干细胞移植治疗再生障碍性贫血
DOI：
10.11406/rinketsu.61.1388
复制
发表时间：
2020
期刊：
影响因子：
--
通讯作者：
賀古 真一
中科院分区：
文献类型：
--
作者：
Kako S;Hayakawa F;Imai K;Tanaka J;Mizuta S;Nishiwaki S;Kanamori H;Mukae J;Ozawa Y;Kondo T;Fukuda T;Ichinohe T;Ota S;Tanaka Y;Murayama T;Kurahashi S;Sakura T;Usui N;Ohtake S;Kiyoi H;Matsumura I;Miyazaki Y;Atsuta Y;賀古 真一;賀古 真一
来自人类白细胞抗原（HLA）匹配的亲属供者的异基因造血干细胞移植（HSCT）是治疗重型再生障碍性贫血（SAA）的有效选择。还报告了来自无关捐献者的HSCT。移植失败和移植物抗宿主病（GVHD）是SAA HSCT的主要障碍，高剂量环磷酰胺（CY）和抗胸腺细胞球蛋白（ATG）的组合已被用作克服这些障碍的预处理方案。然而，由于高剂量CY引起的心脏毒性一直是一个主要的挑战，并且最近认为通过添加氟达拉滨（Flu）来降低CY可以降低毒性。关东细胞治疗研究小组（KSGCT）进行了一项前瞻性研究，使用流感病毒、减少剂量的CY和低剂量的胸腺球蛋白作为预处理方案，取得了令人鼓舞的结果，一年总生存率为96.3%。但是，应该注意的是，使用Flu可能会影响继发性移植物失效的发生。来自替代供体（包括脐带血和单倍体相关供体）的HSCT结果正在改善。因此，我们在SAA的HSCT中有更多选择。
Allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-matched related donor is an effective treatment option for sever aplastic anemia (SAA). HSCT from an unrelated donor has also been reported. Engraftment failure and graft-versus-host disease (GVHD) are major obstacles in HSCT for SAA, and the combination of high-dose cyclophosphamide (CY) and anti-thymocyte globulin (ATG) has been utilized as a conditioning regimen for overcoming these obstacles. However, cardiac toxicity due to high-dose CY has been a major challenge, and the reduction of CY with the addition of fludarabine (Flu) has recently been considered to decrease toxicity. In Kanto Study Group for Cell Therapy (KSGCT), a prospective study using Flu, reduced-dose CY, and low-dose thymoglobulin as a conditioning regimen was performed, which demonstrated a promising result with the overall survival rate of 96.3% at one year. However, it should be noted that the use of Flu may affect the occurrence of secondary graft failure. Outcomes in HSCT from alternative donors, including cord blood and haploidentical-related donors, are improving. Therefore, we have more options in HSCT for SAA.