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Treosulfan-based conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with non-malignant diseases.

作者信息

Suh Jin Kyung, Im Ho Joon, Kang Sung Han, Kim Hyery, Choi Eun Seok, Koh Kyung-Nam

机构信息

Department of Pediatrics, Korea Cancer Center Hospital, Korea Institute of Radiological & Medical Sciences, Seoul, Korea.

Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.

出版信息

Bone Marrow Transplant. 2022 Apr;57(4):681-684. doi: 10.1038/s41409-021-01556-8. Epub 2022 Feb 7.

DOI:10.1038/s41409-021-01556-8
PMID:35132202
Abstract
摘要

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Treosulfan-thiotepa-fludarabine-based conditioning regimen for allogeneic transplantation in patients with thalassemia major: a single-center experience from north India.基于三硫鸟嘌呤-噻替派-氟达拉滨的预处理方案用于重型地中海贫血患者的异基因移植:来自印度北部的单中心经验。
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Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major.以白消安-氟达拉滨或三氧化二砷-氟达拉滨为基础的骨髓清除性预处理方案治疗重型地中海贫血患儿。
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Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context.成人血友病和血红蛋白病的治疗方法:全球背景下的免疫、基因和干细胞治疗途径
Biomedicines. 2025 Aug 19;13(8):2022. doi: 10.3390/biomedicines13082022.
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Reduced toxicity matched sibling bone marrow transplant results in excellent outcomes for severe congenital neutropenia.毒性降低的匹配同胞骨髓移植可为严重先天性中性粒细胞减少症带来优异的治疗效果。
Front Immunol. 2024 Feb 26;15:1369243. doi: 10.3389/fimmu.2024.1369243. eCollection 2024.
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Is Treosulfan-Based Conditioning Attractive as a Reduced-Intensity Conditioning Regimen in Korea?

本文引用的文献

1
Incorporation of thiotepa in a reduced intensity conditioning regimen may improve engraftment after transplant for HLH.在减低强度预处理方案中加入噻替派可能会改善噬血细胞性淋巴组织细胞增生症(HLH)移植后的植入情况。
Br J Haematol. 2020 Mar;188(6):e84-e87. doi: 10.1111/bjh.16370. Epub 2020 Jan 27.
2
Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies.HLH 和原发性免疫缺陷病的造血细胞移植的低强度预处理。
Blood. 2018 Sep 27;132(13):1438-1451. doi: 10.1182/blood-2018-01-828277. Epub 2018 Jul 11.
3
Hematopoietic Stem Cell Transplantation for XIAP Deficiency in Japan.
基于三氧化二砷的预处理方案在韩国作为一种低强度预处理方案是否具有吸引力?
J Korean Med Sci. 2023 Sep 11;38(36):e281. doi: 10.3346/jkms.2023.38.e281.
日本针对XIAP缺陷的造血干细胞移植
J Clin Immunol. 2017 Jan;37(1):85-91. doi: 10.1007/s10875-016-0348-4. Epub 2016 Nov 4.
4
Bone marrow transplantation for non-malignant diseases using treosulfan-based conditioning.使用基于曲奥舒凡的预处理方案进行非恶性疾病的骨髓移植。
Pediatr Blood Cancer. 2015 Feb;62(2):299-304. doi: 10.1002/pbc.25267. Epub 2014 Oct 4.
5
Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes.XIAP 缺陷的异基因造血细胞移植:国际调查显示预后不良。
Blood. 2013 Feb 7;121(6):877-83. doi: 10.1182/blood-2012-06-432500. Epub 2012 Nov 6.
6
Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities.含曲奥沙仑的方案可实现高植入率,相关移植发病率和死亡率低,适用于患有非恶性疾病和重大合并症的儿童。
Br J Haematol. 2008 Jun;142(2):257-62. doi: 10.1111/j.1365-2141.2008.07064.x. Epub 2008 May 19.