Sugita Junichi
Department of Hematology, Sapporo Hokuyu Hospital.
Rinsho Ketsueki. 2024;65(4):272-281. doi: 10.11406/rinketsu.65.272.
Use of posttransplant cyclophosphamide (PTCy) for the prophylaxis of graft-versus-host disease (GVHD) has revolutionized the field of HLA-haploidentical stem cell transplantation, which was previously considered high-risk and only feasible in specialized centers. The rapid adoption of PTCy is attributed not only to its superior efficacy in suppressing GVHD but also to its affordability and the lack of need for specialized techniques or equipment to administer it. Recently, PTCy has gained attention for its potential effectiveness in GVHD prophylaxis beyond HLA-haploidentical stem cell transplantation. In a phase III trial (BMT CTN 1703 trial) in patients undergoing allogeneic HLA-matched stem cell transplantation with reduced-intensity conditioning, GVHD-free, relapse-free survival at 1 year was significantly better among those who received PTCy-tacrolimus-mycophenolate mofetil than among those who received tacrolimus-methotrexate. In Japan, a phase II clinical trial that investigated PTCy for GVHD prophylaxis following HLA-matched or 1-2 allele mismatched peripheral blood stem cell transplantation confirmed the efficacy and safety of this approach. Effective suppression of GVHD using PTCy is expected to enhance the safety of allogeneic transplantation, potentially improving transplant outcomes and offering hope for better patient care in the field of transplantation.
使用移植后环磷酰胺(PTCy)预防移植物抗宿主病(GVHD)彻底改变了HLA单倍型相合干细胞移植领域,该领域此前被认为是高风险的,且仅在专业中心可行。PTCy的迅速采用不仅归因于其在抑制GVHD方面的卓越疗效,还归因于其可承受性以及给药时无需专门技术或设备。最近,PTCy因其在HLA单倍型相合干细胞移植以外的GVHD预防中的潜在有效性而受到关注。在一项针对接受减低强度预处理的异基因HLA匹配干细胞移植患者的III期试验(BMT CTN 1703试验)中,接受PTCy - 他克莫司 - 霉酚酸酯的患者1年无GVHD、无复发生存率显著高于接受他克莫司 - 甲氨蝶呤的患者。在日本,一项研究PTCy用于HLA匹配或1 - 2个等位基因不匹配的外周血干细胞移植后GVHD预防的II期临床试验证实了该方法的有效性和安全性。使用PTCy有效抑制GVHD有望提高异基因移植的安全性,可能改善移植结果,并为移植领域更好的患者护理带来希望。