Division of Hematology, Bone Marrow Transplantation, and Hemato-Oncology Center, Chaim Sheba Medical Center, Tel HaShomer, Ramat Gan, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):64-73. doi: 10.1182/hematology.2022000327.
Allogeneic hematopoietic stem cell transplantation is the treatment of choice for high-risk hematological malignancies such as acute myeloid and lymphocytic leukemia, myelodysplastic syndrome, and myeloproliferative disorders. Alternative donor transplantation from either haploidentical (haplo-SCT) or cord blood donor (CBT) is an established therapeutic alternative for patients who need transplants but lack a human leukocyte antigen-matched donor. Although haplo-SCT (mainly non-T-cell-depleted haplo-SCT with posttransplant cyclophosphamide) is increasing while CBT is decreasing worldwide (Figure 1), recent developments in CBT, especially cord blood expansion and other strategies to improve engraftment and immune reconstitution post-CBT, make CBT still a valuable option. This article discusses the 2 options based on the currently available data, focusing on adults, and tries to give some clues to help the transplant physician choose a haploidentical vs a cord blood donor. Given the limited numbers of published or ongoing well-designed randomized controlled trials comparing haplo-SCT to CBT and the overall similar clinical results in the available, mostly registry-based, and single-center studies, with substantial heterogeneity and variability, the decision to perform haplo-SCT or CBT in a given patient depends not only on the patient, disease, and donor characteristics and donor availability (although most if not all patients should have in principle an alternative donor) but also on the transplant physician's discretion and, most importantly, the center's experience and preference and ongoing protocols and strategies.
异基因造血干细胞移植是治疗高危血液系统恶性肿瘤(如急性髓系和淋巴细胞白血病、骨髓增生异常综合征和骨髓增殖性疾病)的首选方法。对于需要移植但缺乏人类白细胞抗原匹配供体的患者,来自半相合(haplo-SCT)或脐带血供体(CBT)的替代供体移植是一种既定的治疗选择。尽管全球范围内 haplo-SCT(主要是非 T 细胞耗竭的 haplo-SCT 联合移植后环磷酰胺)的应用正在增加,而 CBT 的应用正在减少(图 1),但 CBT 的最新发展,特别是脐带血扩增和其他改善移植后植入和免疫重建的策略,使得 CBT 仍然是一种有价值的选择。本文根据现有数据讨论这两种选择方案,重点关注成人,并尝试提供一些线索,帮助移植医生选择半相合供体与脐带血供体。鉴于比较 haplo-SCT 与 CBT 的已发表或正在进行的精心设计的随机对照试验数量有限,以及现有基于登记和单中心的研究中总体相似的临床结果,存在大量异质性和可变性,因此,在给定患者中进行 haplo-SCT 或 CBT 的决定不仅取决于患者、疾病和供体特征以及供体可用性(尽管原则上大多数(如果不是全部)患者都应该有替代供体),还取决于移植医生的酌处权,最重要的是,中心的经验和偏好以及正在进行的方案和策略。