对于状细胞病的干细胞移植的优异结果
Tanja Vallée1, Irene Schmid1, Lisa Gloning1
1Department of Pediatrics, Dr. Von Hauner Children's Hospital, University Hospital, LMU Munich, Lindwurmstr. 4, 80337, Munich, Germany.
Annals of hematology
|September 19, 2023
概括
使用匹配家庭捐赠者 (MFD),匹配无关捐赠者 (MUD) 或匹配-不匹配家庭捐赠者 (MMFD) 的状细胞病 (SCD) 患者的降低毒性条件导致了优异的生存率和低GVHD. 与busulfan相比,硫调节与更高的移植失败率有关.
科学领域:
- 血液学 血液学 血液学
- 移植免疫学 移植免疫学
- 儿科血液学 儿科血液学
背景情况:
- 状细胞病 (SCD) 患者往往缺乏匹配的家族捐赠者 (MFD) 或匹配的无关捐赠者 (MUD),使得平分异性捐赠者 (MMFD) 成为一个可行的选择.
- 造血干细胞移植 (HSCT) 是SCD的潜在治疗方法,但供体供应仍然是一个挑战.
研究的目的:
- 评估HSCT降低毒性调节方案在不同供体类型 (MFD,MMFD,MUD) 的儿科和年轻成人SCD患者中的疗效和安全性.
- 为了比较结果,包括生存率,移植失败,GVHD和病毒再激活,基于捐赠者类型和调节方案.
主要方法:
- 31名儿科和年轻成人SCD患者的队列接受了MFD (n=15),MMFD (n=10) 或MUD (n=6) 的HSCT.
- 调节包括阿姆图祖马布/ATG,蒂奥特帕,弗鲁达拉宾,以及三硫或向布苏尔,以及移植后的环胺,用于MMFD接受者.
- 随访时间平均为26个月.
主要成果:
- 所有患者都活着,在随访时已关闭免疫抑制.
- 两名MMFD患者 (两人都接受了三硫调节) 经历了二次移植失败.
- 没有观察到急性GVHDIII级或更高或中度/严重的慢性GVHD.
- 没有疾病的,严重的GVHD无生存率为100% (MFD),100% (MUD) 和80% (MMFD).
- 与MFD (40%) 相比,MMFD (100%) 和MUD (83%) 组的病毒重新激活率更高.
- 在2/6 (33%) 患者中发生了移植失败,这些患者接受了treosulfan的条件对比于0/25 (0%) 患者接受了busulfan (p=0.032).
- 90%的患者获得了>=80%的捐赠者合体.
结论:
- 降低毒性骨髓损伤性调节对于儿科和年轻成人SCD患者的HSCT是有效的,在所有供体类型中实现了优异的存活率和低GVHD.
- 向的布苏尔调节似乎优于特雷苏尔,显示了显著较低的移植失败率.
- 与这种降低毒性协议的哈普罗同一性HSCT对于缺乏MFD或MUD的SCD患者来说是一个有前途的选择.
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