Bone marrow transplantation for the Wiskott-Aldrich syndrome. Long-term follow-up.
Bone marrow transplantation for the Wiskott-Aldrich syndrome. Long-term follow-up.
复制标题
骨髓移植治疗 Wiskott-Aldrich 综合征。
DOI：
10.1097/00007890-199010000-00018
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发表时间：
1990
期刊：
影响因子：
6.2
通讯作者：
Rappeport,JM
中科院分区：
文献类型：
--
作者：
Rimm,IJ;Rappeport,JM
Wiskott-Aldrich综合征是一种以免疫缺陷和血小板减少为特征的性连锁疾病。对这种疾病的支持性治疗是不够的，据报道，骨髓移植可以获得很好的存活率。本文报道了8例接受骨髓移植的男性患者的长期随访结果。所有患者均接受消融准备治疗，包括抗胸腺细胞血清(ATS)、环磷酰胺、丁硫丹或全身照射(TBI)。骨髓移植自一位人类白细胞抗原相合的捐献者。在这些男性患者中，有7人的移植器官植入良好，现在有足够的淋巴细胞和血小板功能。此外，它们还得到了良好的成长和发展。这表明，对于这种先天性疾病，先进行消融性准备治疗，然后进行早期的骨髓移植是一种非常成功的治疗方法。
Wiskott-Aldrich Syndrome (WAS) is a sex-linked disease characterized by immunodeficiency and thrombocytopenia. Supportive treatment of this disease is inadequate and bone marrow transplantation has been reported to result in excellent survival. The long-term follow-up of 8 male patients who received bone marrow transplantation for the WAS is reported here. All of these patients received ablative preparative treatment consisting of ATS (antithymocyte serum), cytoxan and either busulfan or TBI (total body irradiation). Bone marrow was transplanted from an HLA-matched donor. Seven of eight of these male patients have had excellent engraftment of their transplant and now have adequate lymphocyte and platelet function. In addition, they have had good growth and development. This suggests that ablative preparative treatment followed by early bone marrow transplantation from an HLA-matched donor is a highly successful therapy for this congenital disease.