Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation
Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation
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DOI：
10.1182/blood-2007-03-076679
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发表时间：
2008-01-01
期刊：
影响因子：
20.3
通讯作者：
Fischer, Alain
中科院分区：
文献类型：
--
作者：
Ozsahin, Hulya;Cavazzana-Calvo, Marina;Fischer, Alain
Wiskott-Aldrich综合征（WAS）是一种罕见的x连锁免疫缺陷，伴微血小板减少症、湿疹、复发性感染、自身免疫性疾病和恶性肿瘤，大多数患者可危及生命。在这项长期、回顾性、多中心的研究中，我们分析了96例在1979年至2001年间接受造血干细胞移植（HSCT）后存活至少2年的WAS患者的事件。事件包括慢性移植物抗宿主病（cGVHD）、自身免疫、感染和移植前后并发症的后遗症。3例患者（3%）在移植后2.1 - 21年死亡。总体7年无事件生存率为75%。在不匹配的相关供体受者中，这一比例较低，也与接受HSCT的年龄较大和疾病严重程度有关。最引人注目的发现是观察到20%的患者与混合/分裂嵌合状态密切相关的不依赖cgvhd的自身免疫(P
Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P