Institut Universitaire d'Hémato-Oncologie et de Thérapie Cellulaire, Hôpital Maisonneuve-Rosemont, CIUSSS de l'Est de l'Île de Montréal, Montréal, QC, Canada.
Department of Medicine, Faculty of Medicine, Université de Montréal, Montréal, QC, Canada.
Blood Adv. 2023 Oct 10;7(19):5717-5726. doi: 10.1182/bloodadvances.2023010599.
Cord blood (CB) transplantation is hampered by low cell dose and high nonrelapse mortality (NRM). A phase 1-2 trial of UM171-expanded CB transplants demonstrated safety and favorable preliminary efficacy. The aim of the current analysis was to retrospectively compare results of the phase 1-2 trial with those after unmanipulated CB and matched-unrelated donor (MUD) transplants. Data from recipients of CB and MUD transplants were obtained from the Center for International Blood and Marrow Transplant Research (CIBMTR) database. Patients were directly matched for the number of previous allogeneic hematopoietic stem cell transplants (alloHCT), disease and refined Disease Risk Index. Patients were further matched by propensity score for age, comorbidity index, and performance status. Primary end points included NRM, progression-free survival (PFS), overall survival (OS), and graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) at 1 and 2 years after alloHCT. Overall, 137 patients from CIBMTR (67 CB, 70 MUD) and 22 with UM171-expanded CB were included. NRM at 1 and 2 years was lower, PFS and GRFS at 2 years and OS at 1 year were improved for UM171-expanded CBs compared with CB controls. Compared with MUD controls, UM171 recipients had lower 1- and 2-year NRM, higher 2-year PFS, and higher 1- and 2-year GRFS. Furthermore, UM171-expanded CB recipients experienced less grades 3-4 acute GVHD and chronic GVHD compared with MUD graft recipients. Compared with real-world evidence with CB and MUD alloHCT, this study suggests that UM171-expanded CB recipients may benefit from lower NRM and higher GRFS. This trial was registered at www.clinicaltrials.gov as #NCT02668315.
脐带血 (CB) 移植受到细胞剂量低和非复发死亡率 (NRM) 高的阻碍。UM171 扩增 CB 移植的 1-2 期试验证明了其安全性和初步疗效。本分析的目的是回顾性比较 1-2 期试验结果与未处理 CB 和匹配无关供体 (MUD) 移植的结果。从国际血液和骨髓移植研究中心 (CIBMTR) 数据库中获得了 CB 和 MUD 移植受者的数据。患者根据既往异基因造血干细胞移植 (alloHCT) 的数量、疾病和细化疾病风险指数进行直接匹配。患者根据年龄、合并症指数和表现状态的倾向评分进一步匹配。主要终点包括 alloHCT 后 1 年和 2 年的 NRM、无进展生存期 (PFS)、总生存期 (OS) 和移植物抗宿主病 (GVHD) 无复发存活率 (GRFS)。总体而言，CIBMTR 中有 137 名患者（67 名 CB，70 名 MUD）和 22 名接受 UM171 扩增 CB 的患者符合纳入标准。与 CB 对照组相比，UM171 扩增 CB 的 1 年和 2 年 NRM 较低，2 年 PFS 和 1 年 OS 较高。与 MUD 对照组相比，UM171 组的 1 年和 2 年 NRM 较低，2 年 PFS 较高，1 年和 2 年 GRFS 较高。此外，与 MUD 移植物受者相比，UM171 扩增 CB 受者发生 3-4 级急性 GVHD 和慢性 GVHD 的情况较少。与 CB 和 MUD alloHCT 的真实世界证据相比，本研究表明，UM171 扩增 CB 受者可能受益于较低的 NRM 和较高的 GRFS。该试验在 www.clinicaltrials.gov 上注册，编号为 #NCT02668315。
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