COMPLETE CORRECTION OF GLANZMANNS THROMBASTHENIA BY ALLOGENEIC BONE-MARROW TRANSPLANTATION
COMPLETE CORRECTION OF GLANZMANNS THROMBASTHENIA BY ALLOGENEIC BONE-MARROW TRANSPLANTATION
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DOI：
10.1111/j.1365-2141.1985.tb07358.x
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发表时间：
1985-01-01
影响因子：
6.5
通讯作者：
BOIRON, M
中科院分区：
文献类型：
--
作者：
BELLUCCI, S;DEVERGIE, A;BOIRON, M
同种异体骨髓移植（BMT）成功纠正了一名 4 岁男孩的 I 型血小板无力症。捐献者是他 14 岁的 HLA-A、B 和 D 完全相同的兄弟，他是血小板无力症杂合子。用环磷酰胺和胸腹照射调理后的第一次移植被拒绝，但在准备方案中使用 CCNU [洛莫司汀]、环磷酰胺、丙卡巴肼和马抗人胸腺细胞球蛋白的第二次移植成功。血小板膜抗原、PLA1 和糖蛋白 IIb/IIIa 复合物的研究以及血小板功能研究证实了植入。出血症状完全消失；血小板膜标志物和凝块回缩迅速恢复至正常值；和血小板聚集测试更慢。二十四个月。骨髓移植后，患者病情良好，患有轻度慢性肝移植抗宿主病。 BMT 似乎是治疗严重遗传性血小板疾病的一种可能方法。
Allogeneic bone marrow transplantation (BMT) successfully corrected type I thrombasthenia in a 4-yr-old boy. The donor was his HLA-A, B and D identical 14-yr-old brother who was heterozygous for thrombasthenia. A 1st transplant after conditioning with cyclophosphamide and thoracoabdominal irradiation was rejected, but a 2nd transplant using CCNU [lomustine], cyclophosphamide, procarbazine and horse antihuman thymocyte globulin in the preparative regimen was successful. Engraftment was proven by studies of platelet membrane antigens, PLA1 and glycoprotein IIb/IIIa complex and by platelet function studies. Hemorrhagic manifestations completely disappeared; platelet membrane markers and clot retraction returned promptly to normal values; and platelet aggregation tests more slowly. Twenty-four mo. after bone-marrow transplant, the patient was well with mild chronic hepatic graft vs. host disease. BMT appears to be a possible treatment for severe inherited platelet disorders.