Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center.
Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center.
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来自单一中心的 26 名 Wiskott-Aldrich 综合征患者的骨髓移植。
DOI：
10.1016/s0022-3476(96)70248-2
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发表时间：
1996
期刊：
影响因子：
--
通讯作者：
A. Fischer
中科院分区：
文献类型：
--
作者：
H. Ozsahin;F. Deist;M. Benkerrou;M. Cavazzana‐Calvo;L. Gomez;C. Griscelli;S. Blanche;A. Fischer
我们回顾性分析了一个中心26例Wiskott- Aldrich综合征（WAS）患者骨髓移植（BMT）的结果。共进行28例移植手术。10名未被选中的患者接受了来自基因相同的人类白细胞抗原（HLA）供体的未经处理的骨髓。8例患者治愈，术后存活1.5 ~ 16.5年。一名患者成功地从一名匹配的非亲属捐赠者那里接受了t细胞耗尽的骨髓。16例患者因WAS发生危及生命的并发症（即难治性血小板减少症、自身免疫包括血管炎和败血症）而接受HLA部分不相容的BMT治疗。除一人外，所有患者在接受了丁硫丹和环磷酰胺治疗后，都接受了t细胞枯竭的骨髓。一名患者有两个bmt。17次移植中有12次移植成功。添加淋巴细胞功能相关抗原-1和CD2分子的单克隆抗体似乎可以改善移植。6例患者长期存活，其余患者死于病毒感染（n = 7），其中以eb病毒诱导的b淋巴细胞增生性疾病为主。T细胞和b细胞功能发育迟缓可导致严重的感染并发症。这些结果证实了HLA基因相同的BMT在WAS中的良好结果，而HLA部分不相容供者的BMT应严格限制在有严重WAS并发症的患者中。（j pediatrics; 1996;129:238-44）
We retrospectively analyzed the outcome of bone marrow transplantation (BMT) performed in 26 patients with Wiskott- Aldrich syndrome (WAS) in one center. Twenty-eight transplantation procedures were performed. Ten unselected patients received unmanipulated marrow from a donor with genetically identical human leukocyte antigen (HLA). Eight patients were cured and survive 1.5 to 16.5 years after BMT. One patient successfully received a T-cell-depleted marrow from a matched unrelated donor. Sixteen patients were selected to receive a related HLA partially incompatible BMT because of the occurrence of life-threatening complications from the WAS (i.e., refractory thrombocytopenia, autoimmunity including vasculitis and sepsis). All but one received T-cell-depleted marrow after a conditioning regimen of busulfan and cyclophosphamide. One patient had two BMTs. Engraftment occurred in 12 of 17 attempts. The addition of monoclonal antibodies to lymphocyte function-associated antigen-1 and CD2 molecules appeared to improve engraftment. Six patients were long-term survivors, whereas others died of viral infections (n = 7), among which Epstein-Barr virus-induced B-lymphocyte proliferative disorder was predominant. Delay in development of full T- and B-cell functions accounted for severe infectious complications. These results confirm the excellent outcome of HLA genetically identical BMT in WAS, whereas BMT from HLA partially incompatible donors should be strictly restricted to patients with severe complications of WAS. (J PEDIATR 1996;129:238-44)
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DOI：
10.1016/0955-3886(91)90119-n
发表时间：
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期刊：
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影响因子：
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DOI：
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DOI：
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发表时间：
1990
期刊：
Immunodeficiency reviews
影响因子：
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作者：
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影响因子：
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影响因子：
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作者：
Rimm,IJ;Rappeport,JM
通讯作者：
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