Hematopoietic Stem Cell Transplantation and Cellular Therapy Program, Division of Hematology/Oncology, Department of Medicine, University of California, Irvine, CA, USA.
Division of Hematology, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Thailand.
Hematology. 2024 Dec;29(1):2366718. doi: 10.1080/16078454.2024.2366718. Epub 2024 Jun 18.
Outcomes of haploidentical hematopoietic stem cell transplantation (haplo-SCT) have improved over time. Graft failure and graft-versus-host disease (GVHD), which were important complications in major human leukocyte antigen (HLA)-disparity stem cell transplantation, have significantly decreased. These improvements have led to an exponential increase in the use of haploidentical donors for transplantation, as well as in the number of publications evaluating haplo-SCT outcomes. Many studies focused on factors important in donor selection, novel conditioning regimens or GVHD prophylaxis, the impact of donor-specific anti-HLA antibodies (DSA), as well as strategies to prevent disease relapse post-transplant. DSA represents an important limitation and multimodality desensitization protocols, including plasma exchange, rituximab, intravenous immunoglobulin and donor buffy coat infusion, can contribute to the successful engraftment in patients with high DSA levels and is currently the standard therapy for highly allosensitized individuals. With regards to donor selection, younger donors are preferred due to lower risk of complications and better transplant outcomes. Moreover, recent studies also showed that younger haploidentical donors may be a better choice than older-matched unrelated donors. Improvement of disease relapse remains a top priority, and several studies have demonstrated that higher natural killer (NK) cell numbers early post-transplant are associated with improved outcomes. Prospective studies have started to assess the role of NK cell administration in decreasing post-transplant relapse. These studies suggest that the incorporation of other cell products post-transplant, including the administration of chimeric antigen receptor T-cells, should be explored in the future.
随着时间的推移，单倍体造血干细胞移植（haplo-SCT）的疗效有所改善。在主要的人类白细胞抗原（HLA）不相容干细胞移植中，移植物失败和移植物抗宿主病（GVHD）是重要的并发症，其发生率已显著降低。这些改进导致了使用单倍体供者进行移植的数量呈指数级增长，同时评估 haplo-SCT 结果的出版物数量也在增加。许多研究集中在供者选择、新型预处理方案或 GVHD 预防、供者特异性抗 HLA 抗体（DSA）的影响以及预防移植后疾病复发的策略等方面。DSA 是一个重要的限制因素，多模态脱敏方案，包括血浆置换、利妥昔单抗、静脉注射免疫球蛋白和供者白细胞悬液输注，可以有助于高 DSA 水平患者的成功植入，目前是高度致敏个体的标准治疗方法。关于供者选择，由于并发症风险较低和移植效果较好，年轻供者更受青睐。此外，最近的研究还表明，年轻的单倍体供者可能比年长的匹配无关供者更好。改善疾病复发仍然是重中之重，多项研究表明，移植后早期 NK 细胞数量较高与改善结局相关。前瞻性研究已经开始评估 NK 细胞输注在降低移植后复发中的作用。这些研究表明，未来应该探索在移植后加入其他细胞产品，包括嵌合抗原受体 T 细胞的给药。
