The use of human leukocyte antigen (HLA)–haploidentical haematopoietic stem cell transplantation (HSCT) with post-transplant cyclophosphamide (PTCY), which markedly reduces the risk of graft-versus-host disease, has rapidly increased worldwide, even in children. It was initially developed for post-transplant relapse or non-remission at transplant for patients with high-risk haematologic malignancies. However, this strategy is currently used more frequently for standard-risk, transplant-eligible paediatric haematological malignancies. It has recently been recognised in adults that the transplant outcomes after PTCY-based HLA–haploidentical HSCT are comparable with those achieved after HLA-matched HSCT. Therefore, even in children, parental donors who are HLA–haploidentical donors and cord blood are currently considered the next donor candidates when an HLA-matched related or unrelated donor is unavailable. This review addresses the current status of the use of haplo-HSCT with PTCY for paediatric haematologic malignancies and future directions for donor selection (sex, age, ABO blood type, and HLA disparity), donor source, the dose of infused CD34+cells, optimal conditioning, the concomitant graft-versus-host disease prophylaxis other than PTCY, and the pharmacokinetic study of CY and CY metabolites. These aspects present key solutions for further improvements in the outcomes of haplo-HSCT with PTCY for paediatric haematological malignancies.
采用人白细胞抗原（HLA）单倍体相合造血干细胞移植（HSCT）联合移植后环磷酰胺（PTCY）方案，可显著降低移植物抗宿主病风险，目前已在全球范围内迅速推广，儿童患者中的应用亦日益增多。该方案最初是为移植后复发或移植时未缓解的高危血液系统恶性肿瘤患者开发的。然而，当前该策略更频繁地应用于符合移植条件的标危儿童血液系统恶性肿瘤。近期成人研究表明，基于PTCY的HLA单倍体相合HSCT的移植效果与HLA全相合HSCT相当。因此，即使在儿童患者中，当缺乏HLA全相合相关或无关供者时，作为HLA单倍体相合供者的父母供者及脐带血目前已被视为次选供体来源。本综述探讨了PTCY方案单倍体HSCT在儿童血液系统恶性肿瘤中的应用现状，以及供者选择（性别、年龄、ABO血型、HLA差异）、供体来源、CD34+细胞输注剂量、最佳预处理方案、除PTCY外的移植物抗宿主病联合预防策略、环磷酰胺及其代谢物药代动力学研究等未来发展方向。这些方面为进一步改善PTCY方案单倍体HSCT治疗儿童血液系统恶性肿瘤的疗效提供了关键解决方案。
肿瘤(癌症)患者之家