Sugita Junichi
Department of Hematology, Hokkaido University Faculty of Medicine.
Rinsho Ketsueki. 2020;61(9):1402-1410. doi: 10.11406/rinketsu.61.1402.
Posttransplant cyclophosphamide (PTCy) was initially developed by the Johns Hopkins group as a graft-versus-host disease (GVHD) prophylaxis for human leukocyte antigen (HLA)-haploidentical stem cell transplantation. Recently, PTCy has been attempted in HLA-matched transplantation. In HLA-haploidentical hematopoietic transplantation, several retrospective studies have compared HLA-matched transplantation with HLA-haploidentical hematopoietic transplantation using PTCy. In these reports, transplantation outcomes have been comparable. Therefore, PTCy- based haploidentical transplantation is a viable alternative to HLA-matched transplantation. Moreover, In HLA-matched transplantation, PTCy alone may be sufficient in the prevention of GVHD in HLA-matched bone marrow transplantation. Although PTCy alone is not enough in HLA-matched peripheral blood stem cell transplantation, PTCy has demonstrated excellent GVHD inhibition when combined with several immunosuppressive agents, such as calcineurin inhibitor and mycophenolate mofetil. Furthermore, PTCy also playing a significant role in other areas, such as aplastic anemia, salvage transplantation for graft failure, and allogeneic transplantation after checkpoint inhibitor use. PTCy can be performed inexpensively and without any special equipment or techniques at any institution. It is expected that PTCy will continue to spread worldwide in the future.
移植后环磷酰胺(PTCy)最初由约翰霍普金斯团队开发,用于预防人类白细胞抗原(HLA)半相合干细胞移植中的移植物抗宿主病(GVHD)。最近,PTCy已被尝试用于HLA匹配的移植。在HLA半相合造血移植中,多项回顾性研究比较了使用PTCy的HLA匹配移植与HLA半相合造血移植。在这些报告中,移植结果具有可比性。因此,基于PTCy的半相合移植是HLA匹配移植的一种可行替代方案。此外,在HLA匹配移植中,单独使用PTCy可能足以预防HLA匹配的骨髓移植中的GVHD。虽然单独使用PTCy在HLA匹配的外周血干细胞移植中不够,但PTCy与几种免疫抑制剂(如钙调神经磷酸酶抑制剂和霉酚酸酯)联合使用时,已显示出优异的GVHD抑制效果。此外,PTCy在其他领域也发挥着重要作用,如再生障碍性贫血、移植失败的挽救性移植以及检查点抑制剂使用后的同种异体移植。PTCy可以在任何机构以低成本进行,无需任何特殊设备或技术。预计未来PTCy将继续在全球范围内推广。