Superior outcome from frontline cord blood transfusion combined HLA-full-matched unrelated donor hematopoietic stem cell transplantation for aplastic anemia

作者：Xinru Jia, Tonglin Hu, Chuanao Xin, Qi Liu, Ying‐Ying Shen, Yaonan Hong, Shan Liu, Zexing Sun, Rudan Zheng, Xiawan Yang, Wenbin Liu, Qinghong Yu, Jianping Shen, Shengyun Lin, Yuhong Zhou, Baodong Ye, Dijiong Wu · 发表于：Blood · 年份：2025 · DOI：10.1182/blood-2025-2512 · 被引用次数：1 · 研究领域：Hematopoietic Stem Cell Transplantation、Mesenchymal stem cell research、Organ and Tissue Transplantation Research

Abstract Aplastic anemia (AA) is a bone marrow failure syndrome caused by aberrant immune responses and is associated with high mortality and poor prognosis, particularly in patients with severe AA (SAA) or transfusion dependent non severe AA (TD-NSAA). Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been increasingly adopted in the treatment of AA and has yielded encouraging results. However, fewer than 30% of patients requiring allo-HSCT are able to identify a suitable matched sibling donor (MSD), thereby limiting the broader application of MSD-HSCT. In such cases, alternative donor sources, including matched unrelated donor (MUD) and haploidentical donor (HID), are considered viable options. While there has been reported a higher frequency of chronic graft-versus-host disease (GVHD) with MUD-HSCT and increased rates of poor graft function in haploidentical HSCT (Haplo-HSCT). Additionally, specific human leukocyte antigen (HLA) mismatches in MUD-HSCT have been implicated in heightened risks of severe acute GVHD and mortality. Thus, further clinical practice is required to optimize the strategy. Umbilical cord blood (UCB) has been shown to decrease mixed chimerism (MC) and relapse rates, enhancing the success of combination transplantation strategies. Based on this rationale, our center implemented a novel approach combining UCB transfusion with MUD-HSCT (MUD-cord-HSCT), a strategy that has received limited investigation both domestically and international...