Kamani N, August C S, Douglas S D, Burkey E, Etzioni A, Lischner H W
J Pediatr. 1984 Jul;105(1):42-6. doi: 10.1016/s0022-3476(84)80354-6.
A 5-month-old Amish infant boy with chronic granulomatous disease underwent bone marrow transplantation from his 5-year-old, histocompatible brother after a preconditioning regimen of busulfan 2 mg/kg/day for 4 days, followed by cyclophosphamide 50 mg/kg/day for 4 days. At the time of bone marrow transplantation, he was free of infection, and remained so throughout the course of the transplant. He was engrafted promptly, with complete reversal of the neutrophil function defect and no sign of graft-versus-host disease. This was followed by loss of the erythroid graft and deterioration in neutrophil function over a period of 9 months. Sixteen months after transplantation, he is free of infection and growing normally, with essentially no evidence for neutrophil engraftment.
一名患有慢性肉芽肿病的5个月大阿米什男婴,在接受了预处理方案(白消安2mg/kg/天,共4天,随后环磷酰胺50mg/kg/天,共4天)后,接受了来自其5岁组织相容性兄弟的骨髓移植。在进行骨髓移植时,他没有感染,并且在整个移植过程中一直保持无感染状态。他迅速植入,中性粒细胞功能缺陷完全逆转,且没有移植物抗宿主病的迹象。随后在9个月的时间里,红系移植物丢失,中性粒细胞功能恶化。移植后16个月,他没有感染,生长正常,基本上没有中性粒细胞植入的证据。