A prospectively registered observational study was conducted to assess the significance of allogeneic hematopoietic stem cell transplantation from highly HLA-matched unrelated donors (UD) and cord blood (CB) on outcomes in adult acute leukemia (AL) and myelodysplastic syndrome (MDS). Between 2007 and 2015, 231 transplant-eligible patients were registered for a phase 2 study of alternative donor transplantation. After registration, a sufficient time period was given to find appropriate UD. Patients received CB transplantation (CBT) if an appropriate UD was unavailable. In total, 119 patients received CBT (106 AL and 13 MDS) and 91 patients received UD transplantation (UDT) (86 AL and 5 MDS). The median age was 39 years in both groups. The primary objective was overall survival (OS); secondary objectives included cumulative incidences of non-relapse mortality (NRM) and relapse, and disease-free survival. Diagnosis, disease status at transplantation, refined disease risk index, and hematopoietic cell transplant-specific comorbidity index did not differ between UDT and CBT. In multivariate analyses, graft source was not a significant risk factor for all objectives. In adjusted analyses, UDT and CBT showed similar OS, NRM, and relapse in this prospective study. CB can be a comparable alternative stem cell source to UD by achieving a timely transplant.

进行了一项前瞻性注册观察性研究，以评估高度HLA匹配的无关供体（UD）和脐带血（CB）的同种异体造血干细胞移植对成人急性白血病（AL）和骨髓增生异常综合征（MDS）结局的重要性。在2007年至2015年之间，有231名符合移植条件的患者被注册进行替代供体移植的2期研究。注册后，给予足够的时间来找到合适的UD。如果没有合适的UD，患者应接受CB移植（CBT）。总共119例患者接受了CBT（106 AL和13 MDS），91例接受了UD移植（UDT）（86 AL和5 MDS）。两组的中位年龄均为39岁。主要目标是整体生存率（OS）；次要目标包括非复发死亡率（NRM）和复发的累积发生率以及无病生存期。在UDT和CBT之间，诊断，移植时的疾病状态，精确的疾病风险指数和造血细胞移植特异性合并症指数没有差异。在多变量分析中，移植物来源并不是所有目标的重要危险因素。在调整的分析中，UDT和CBT在该前瞻性研究中显示出相似的OS，NRM和复发。通过及时移植，CB可以作为UD的可替代干细胞来源。移植物来源并不是所有目标的重要危险因素。在调整后的分析中，UDT和CBT在该前瞻性研究中显示出相似的OS，NRM和复发。通过及时移植，CB可以作为UD的可替代干细胞来源。移植物来源并不是所有目标的重要危险因素。在调整后的分析中，UDT和CBT在该前瞻性研究中显示出相似的OS，NRM和复发。通过及时移植，CB可以作为UD的可替代干细胞来源。