Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence.
Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence.
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DOI：
10.1182/bloodadvances.2023010599
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发表时间：
2023-10-10
期刊：
影响因子：
7.5
通讯作者：
中科院分区：
文献类型：
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与标准CB和MUD移植相比，UM 171 CB移植导致较低的非复发死亡率。与MUD移植相比，UM 171脐带移植具有更高的无进展和无GVHD复发生存率。低细胞剂量和高非复发死亡率（NRM）阻碍了脐带血（CB）移植。UM 171扩增CB移植的1-2期试验证明了安全性和有利的初步疗效。目前分析的目的是回顾性比较1-2期试验与未经操作的CB和匹配的非亲缘供者（MUD）移植后的结果。从国际血液和骨髓移植研究中心（CIBMTR）数据库获得CB和MUD移植受者的数据。患者的既往异基因造血干细胞移植（alloHCT）次数、疾病和精确疾病风险指数直接匹配。通过年龄、合并症指数和体能状态的倾向评分进一步匹配患者。主要终点包括NRM、无进展生存期（PFS）、总生存期（OS）和alloHCT后1年和2年的无移植物抗宿主病（GVHD）无复发生存期（GRFS）。总体而言，纳入了来自CIBMTR的137例患者（67例CB，70例MUD）和22例UM 171扩增CB患者。与CB对照相比，UM 171扩增CB的1年和2年NRM较低，2年PFS和GRFS以及1年OS改善。与MUD对照组相比，UM 171受体的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移植物受体相比，UM 171扩增的CB受体经历较少的3-4级急性GVHD和慢性GVHD。与CB和MUD alloHCT的真实世界证据相比，本研究表明，UM 171扩增的CB受体可能受益于较低的NRM和较高的GRFS。本试验在www.clinicaltrials.gov上注册为#NCT02668315。
A UM171 CB transplant leads to lower nonrelapse mortality compared with a standard CB and MUD transplant. A UM171 cord transplant has a higher progression-free and GVHD-free relapse-free survival compared with a MUD transplant. 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.
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DOI：
10.1056/nejmoa1602074
发表时间：
2016-09-08
期刊：
The New England journal of medicine
影响因子：
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作者：
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影响因子：
4.3
作者：
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通讯作者：
Barker, Juliet N.
影响因子：
20.3
作者：
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DOI：
10.1126/science.1256337
发表时间：
2014-09-19
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Fares I;Chagraoui J;Gareau Y;Gingras S;Ruel R;Mayotte N;Csaszar E;Knapp DJ;Miller P;Ngom M;Imren S;Roy DC;Watts KL;Kiem HP;Herrington R;Iscove NN;Humphries RK;Eaves CJ;Cohen S;Marinier A;Zandstra PW;Sauvageau G
通讯作者：
Sauvageau G