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供者特征预测重型地中海贫血异基因造血干细胞移植的成功率:250例患者的单中心分析

Donor Characteristics Predict the Success of Allogeneic Hematopoietic Stem Cell Transplantation in Thalassemia Major: A Single-Center Analysis of 250 Patients.

作者信息

Raj Revathi, Swaminathan Venkateswaran Vellaichamy, Meena Satishkumar, Varla Harika, Chandar Rumesh, Ramakrishnan Balasubramaniam, Vaikuntham Mythyly, Maljetty Venkadadesikalu, Uppuluri Ramya

机构信息

Department of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Apollo Hospitals, 320, Padma Complex, Anna Salai, Teynampet, Chennai, 600035 India.

Department of Biostatistics, Apollo Hospitals, 320, Padma Complex, Anna Salai, Teynampet, Chennai, 600035 India.

出版信息

Indian J Hematol Blood Transfus. 2022 Apr;38(2):411-415. doi: 10.1007/s12288-021-01487-w. Epub 2021 Sep 7.

DOI:10.1007/s12288-021-01487-w
PMID:35496973
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9001767/
Abstract

We present data on the impact of donor characteristics in a uniform cohort of children who underwent hematopoietic stem cell transplantation (HSCT) for thalassemia major. We performed a retrospective study in children undergoing matched related (MRD) or unrelated (MUD) HSCT from January 2009 to December 2019. We analyzed data on 250 patients (age seven months-19 years), MRD n = 187, MUD n = 63. We documented sex mismatch in 44% of HSCTs. The graft rejection rate was 3.7%; all had a sex mismatched HSCT (P value = 0.001). Graft versus host disease (GVHD) was higher when donors were above two years as compared to less than two years (23%vs.6.5%, P value = 0.006), with higher rates of mixed chimerism when donors were < two years at 33.3%vs.8.3% in > two years (P value = 0.0001). Mortality and GVHD were higher in the MUD group as compared to the MRD group (15%vs.5%, P value = 0.009; 42.9%vs. 23.4%, P value = 0.0001 respectively). Overall survival was 92.8% with a median follow up of 5.4 years, and was superior in MRD versus MUD group (9.4 years versus 4.8 years P = 0.008). The risk of graft rejection was higher with donor-recipient sex mismatch; while initial mortality and chronic GVHD was higher with MUD HSCT.

摘要

我们呈现了关于供者特征对一组接受重型地中海贫血造血干细胞移植(HSCT)的儿童的影响的数据。我们对2009年1月至2019年12月期间接受匹配相关(MRD)或不匹配(MUD)HSCT的儿童进行了一项回顾性研究。我们分析了250例患者(年龄7个月至19岁)的数据,其中MRD组n = 187例,MUD组n = 63例。我们记录到44%的HSCT存在性别不匹配。移植物排斥率为3.7%;所有发生排斥的均为性别不匹配的HSCT(P值 = 0.001)。与供者年龄小于2岁相比,供者年龄大于2岁时移植物抗宿主病(GVHD)发生率更高(23%对6.5%,P值 = 0.006),供者年龄小于2岁时混合嵌合体发生率更高,为33.3%,而供者年龄大于2岁时为8.3%(P值 = 0.0001)。MUD组的死亡率和GVHD高于MRD组(分别为15%对5%,P值 = 0.009;42.9%对23.4%,P值 = 0.0001)。总体生存率为92.8%,中位随访时间为5.4年,MRD组的总体生存率优于MUD组(9.4年对4.8年,P = 0.008)。供者与受者性别不匹配时移植物排斥风险更高;而MUD HSCT时初始死亡率和慢性GVHD更高。

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本文引用的文献

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Front Immunol. 2020 Aug 11;11:1791. doi: 10.3389/fimmu.2020.01791. eCollection 2020.
2
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Int J Hematol Oncol. 2019 Dec 18;8(4):IJH21. doi: 10.2217/ijh-2019-0006.
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