McColl M D, Gibson B E
Department of Haematology, Royal Hospital for Sick Children, Yorkhill, Glasgow.
Br J Haematol. 1997 Oct;99(1):58-60. doi: 10.1046/j.1365-2141.1997.3533167.x.
Glanzmann's thrombasthenia is a rare inherited bleeding disorder caused by either quantitative or qualitative abnormalities of the platelet membrane glycoprotein (Gp) IIb/IIIa complex. Bleeding is usually mucocutaneous in origin and may be of a severe nature. We report the use of allegeneic bone marrow transplantation in a 5-year-old child with homozygous type I Glanzmann's thrombasthenia, using the patient's younger brother as marrow donor. Engraftment was successful and has resulted in a resolution of bleeding episodes. We conclude that allogeneic BMT is a potentially curative option for those with Glanzmann's thrombasthenia associated with severe bleeding symptoms.
血小板无力症是一种罕见的遗传性出血性疾病,由血小板膜糖蛋白(Gp)IIb/IIIa复合物的数量或质量异常引起。出血通常起源于黏膜皮肤,可能性质严重。我们报告了对一名患有纯合子I型血小板无力症的5岁儿童进行异基因骨髓移植的情况,使用患者的弟弟作为骨髓供体。移植成功,出血发作得到缓解。我们得出结论,对于伴有严重出血症状的血小板无力症患者,异基因骨髓移植是一种潜在的治愈选择。