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Recent results of hematopoietic stem cell transplantation for thalassemia with busulfan-based conditioning regimen in France: improved thalassemia free survival despite frequent mixed chimerism. A retrospective study from the Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC).

作者信息

Rossi Marica, Szepetowski Sarah, Yakouben Karima, Paillard Catherine, Sirvent Anne, Castelle Martin, Pegon Charline, Piguet Christophe, Grain Audrey, Angoso Marie, Robin Marie, Dhedin Nathalie, Pondarré Corinne, Dumesnil de Maricourt Cécile, Berceanu Ana, Simon Pauline, Marcais Ambroise, Poirée Maryline, Gandemer Virginie, Plantaz Dominique, Nguyen Stéphanie, Michel Gérard, Loundou Anderson, Dalle Jean-Hugues, Thuret Isabelle

机构信息

Department of Pediatric Hematology, Robert Debré Hospital, GHU APHP Nord, Université Paris Cité, Paris, France.

Department of Pediatric Hematology and Oncology, Rare Disease Center for Thalassemia, La Timone Hospital, Marseille, France.

出版信息

Bone Marrow Transplant. 2023 Nov;58(11):1254-1256. doi: 10.1038/s41409-023-02079-0. Epub 2023 Aug 4.

DOI:10.1038/s41409-023-02079-0
PMID:37542188
Abstract
摘要

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1
Recent results of hematopoietic stem cell transplantation for thalassemia with busulfan-based conditioning regimen in France: improved thalassemia free survival despite frequent mixed chimerism. A retrospective study from the Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC).法国采用基于白消安的预处理方案进行造血干细胞移植治疗地中海贫血的近期结果：尽管频繁出现混合嵌合体，但无地中海贫血生存率有所提高。来自法语国家干细胞移植与细胞治疗协会（SFGM-TC）的一项回顾性研究。
Bone Marrow Transplant. 2023 Nov;58(11):1254-1256. doi: 10.1038/s41409-023-02079-0. Epub 2023 Aug 4.
2
Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience.使用定制的基于白消安的预处理方案进行先天性血红蛋白病的异基因干细胞移植：单中心经验
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3
Impact of T cell chimerism on clinical outcome in 117 patients who underwent allogeneic stem cell transplantation with a busulfan-containing reduced-intensity conditioning regimen.117例接受含白消安的减低强度预处理方案的异基因干细胞移植患者中T细胞嵌合状态对临床结局的影响。
Biol Blood Marrow Transplant. 2008 Oct;14(10):1148-1155. doi: 10.1016/j.bbmt.2008.07.013.
4
Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression.在降低毒性的造血干细胞移植治疗地中海贫血中找到平衡：输注的 CD3+细胞计数和免疫抑制的作用。
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A novel conditioning regimen with pre-transplantation immunosuppression reduces the complication rates in hematopoietic stem cell transplantation in transfusion-dependent β-thalassemia.一种采用移植前免疫抑制的新型预处理方案可降低输血依赖型β地中海贫血患者造血干细胞移植的并发症发生率。
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Pretransplant immunosuppression followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control.高危地中海贫血患者采用移植前免疫抑制、低毒预处理和干细胞移植：一种安全的疾病控制方法。
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Good engraftment after reduced intensity targeted busulfan-based conditioning and matched related donor hematopoietic cell transplantation in hemoglobinopathies.在接受降低强度靶向白消安为基础的预处理和匹配相关供者造血细胞移植后，血红蛋白病的移植物植入情况良好。
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Younger children with nonmalignant disease have increased incidence of mixed myeloid chimerism after allogeneic hematopoietic cell transplantation with busulfan-based conditioning.接受基于白消安预处理的异基因造血细胞移植后，患有非恶性疾病的年幼儿童混合髓系嵌合现象的发生率增加。
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本文引用的文献

1
Busulfan and cyclophosphamide-based conditioning regimen still holds the promise of being a safe and efficacious regimen for allogeneic transplantation in patients with transfusion-dependent thalassemia, even in high risk.基于白消安和环磷酰胺的预处理方案仍然有望成为输血依赖型地中海贫血患者异基因移植的一种安全有效的方案，即使是高危患者。
Eur J Haematol. 2022 Nov;109(5):447-457. doi: 10.1111/ejh.13825. Epub 2022 Jul 26.
2
Thalassaemia.地中海贫血症。
Lancet. 2022 Jun 18;399(10343):2310-2324. doi: 10.1016/S0140-6736(22)00536-0. Epub 2022 Jun 9.
3
Goal-Oriented Monitoring of Cyclosporine Is Effective for Graft-versus-Host Disease Prevention after Hematopoietic Stem Cell Transplantation in Sickle Cell Disease and Thalassemia Major.
目的导向监测环孢素在镰状细胞病和重型地中海贫血造血干细胞移植后预防移植物抗宿主病的有效性。
Biol Blood Marrow Transplant. 2020 Dec;26(12):2285-2291. doi: 10.1016/j.bbmt.2020.01.016. Epub 2020 Jan 31.
4
Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France.法国234例年龄小于30岁的镰状细胞贫血患者在清髓性预处理和同胞匹配移植后的长期无事件生存率、嵌合状态及生育结局
Haematologica. 2020 Jan;105(1):91-101. doi: 10.3324/haematol.2018.213207. Epub 2019 May 16.
5
Long-term outcome of mixed chimerism after stem cell transplantation for thalassemia major conditioned with busulfan and cyclophosphamide.重型地中海贫血患者经白消安和环磷酰胺预处理行干细胞移植后嵌合体状态的长期转归。
Bone Marrow Transplant. 2018 Feb;53(2):169-174. doi: 10.1038/bmt.2017.231. Epub 2017 Oct 16.
6
Population pharmacokinetic modeling of Thymoglobulin(®) in children receiving allogeneic-hematopoietic cell transplantation: towards improved survival through individualized dosing.接受异基因造血细胞移植的儿童中兔抗人胸腺细胞球蛋白（Thymoglobulin®）的群体药代动力学建模：通过个体化给药提高生存率
Clin Pharmacokinet. 2015 Apr;54(4):435-46. doi: 10.1007/s40262-014-0214-6.
7
Mixed chimerism in haemoglobinopathies: from risk of graft rejection to immune tolerance.血红蛋白病中的混合嵌合体：从移植物排斥风险到免疫耐受
Tissue Antigens. 2014 Mar;83(3):137-46. doi: 10.1111/tan.12313.
8
Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling.血红蛋白病患者接受 HLA 相同同胞来源的脐带血或骨髓移植的结果。
Blood. 2013 Aug 8;122(6):1072-8. doi: 10.1182/blood-2013-03-489112. Epub 2013 May 21.
9
French multicenter 22-year experience in stem cell transplantation for beta-thalassemia major: lessons and future directions.法国多中心 22 年β-地中海贫血重型患者干细胞移植经验：教训与未来方向。
Biol Blood Marrow Transplant. 2013 Jan;19(1):62-8. doi: 10.1016/j.bbmt.2012.08.005. Epub 2012 Aug 11.
10
Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease.在因重型地中海贫血或镰状细胞病而接受骨髓移植后长期、持续存在造血混合嵌合体的患者中，存在定量不同的红细胞/有核细胞嵌合体。
Haematologica. 2011 Jan;96(1):128-33. doi: 10.3324/haematol.2010.031013. Epub 2010 Oct 7.