Blood:造血细胞移植治疗Wiskott-Aldrich综合征的预后

2020-04-19 QQY MedSci原创

年龄与总存活率(OS)有显著的相关性,HCT时年龄不足5岁的患者的OS更好。 供体髓细胞移植<50%与HCT后的血小板恢复不良有关。

奥尔德里奇(Wiskott-Aldrich)综合征(WAS)是一种X染色体连锁病,是由WAS基因突变引起的,可导致血小板减少、湿疹、复发性感染、自身免疫性疾病和恶性肿瘤。造血细胞移植(HCT)是主要的治疗方法,其目的是纠正潜在的免疫缺陷和血小板减少。

随着时间的推移,HCT的预后有所改善,特别是对于有与HLA匹配的兄弟姐妹和无相关的捐赠者的患者。本研究报道了2005年-2015年在29个原发性免疫缺陷治疗联合中心采用HCT治疗的129位WAS患者的预后。

HCT时的中位年龄是1.2岁。大部分患者(65%)进行了骨髓清除白消安为基础的调理。中位随访4.5年,5年总体存活率(OS)达91%。与满5岁时进行HCT的患者相比,不满5岁时进行HCT的5年OS明显更好(94% vs 66%)。

即使在脐带血受体中,无论供体类型如何,OS都是非常好(90%)。调节强度不影响OS,但与HCT后供体T细胞和髓细胞移植相关。特别是接受氟达拉滨/美法仑为基础的低强度治疗方案的患者更有可能在HCT后早期出现供体骨髓嵌合<50%。

此外,与低比例(5%-49%)髓细胞移植的受体相比,进行全(>95%)髓细胞移植的受体的血小板计数更高。

总而言之,自2005年以来,WAS的HCT预后较既往报道的有所改善。高比例的供体髓细胞移植对血小板重建至关重要。

原始出处:

Lauri Burroughs, et al. Excellent Outcomes Following Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: A PIDTC Report. Blood. April 8, 2020.

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    2020-04-20 diushouji
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    2020-04-20 zhangjingnwpu
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    2020-04-20 俅侠
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