使用噻替派、氟达拉滨、抗胸腺细胞球蛋白、低剂量环磷酰胺、200厘戈瑞全身照射及移植后环磷酰胺对镰状细胞病患者进行单倍体相合造血干细胞移植。 - Suppr

Haploidentical hematopoietic stem cell transplant for patients with sickle cell disease using thiotepa, fludarabine, thymoglobulin, low dose cyclophosphamide, 200 cGy tbi and post transplant cyclophosphamide.

作者信息

Frangoul Haydar, Evans Misty, Isbell Julie, Bruce Katie, Domm Jennifer

机构信息

Pediatric Stem Cell Transplant Program Children's Hospital at TriStar Centennial and Sarah Cannon Research Institute, Nashville, TN, 37203, USA.

出版信息

Bone Marrow Transplant. 2018 May;53(5):647-650. doi: 10.1038/s41409-017-0077-1. Epub 2018 Jan 16.

DOI:10.1038/s41409-017-0077-1

PMID:29339800

Abstract

摘要

相似文献

Haploidentical hematopoietic stem cell transplant for patients with sickle cell disease using thiotepa, fludarabine, thymoglobulin, low dose cyclophosphamide, 200 cGy tbi and post transplant cyclophosphamide.使用噻替派、氟达拉滨、抗胸腺细胞球蛋白、低剂量环磷酰胺、200厘戈瑞全身照射及移植后环磷酰胺对镰状细胞病患者进行单倍体相合造血干细胞移植。

Bone Marrow Transplant. 2018 May;53(5):647-650. doi: 10.1038/s41409-017-0077-1. Epub 2018 Jan 16.

Haploidentical Stem Cell Transplant With Post Transplant Cyclophosphamide for Chronic Granulomatous Disease With Thiotepa, Busulfan, and Fludarabine as Conditioning.采用硫替派、白消安和氟达拉滨进行预处理，联合移植后环磷酰胺的单倍体相合干细胞移植治疗慢性肉芽肿病

J Pediatr Hematol Oncol. 2021 May 1;43(4):155-156. doi: 10.1097/MPH.0000000000002015.

A reduced intensity conditioning regimen of fludarabine, cyclophosphamide, antithymocyte globulin, plus 2 Gy TBI facilitates successful hematopoietic cell engraftment in an adult with dyskeratosis congenita.

Bone Marrow Transplant. 2012 Sep;47(9):1254-5. doi: 10.1038/bmt.2011.257. Epub 2012 Jan 16.

Optimal conditioning regimen for haplo-identical stem cell transplantation in adult patients with acquired severe aplastic anemia: Prospective de-escalation study of TBI and ATG dose.成人获得性重型再生障碍性贫血患者行单倍体相合造血干细胞移植的最佳预处理方案：TBI 和 ATG 剂量的前瞻性降阶梯研究。

Am J Hematol. 2018 Nov;93(11):1368-1375. doi: 10.1002/ajh.25257. Epub 2018 Sep 30.

Total body irradiation and cyclophosphamide plus antithymocyte globulin regimen is well tolerated and promotes stable engraftment as a preparative regimen before T cell-replete haploidentical transplantation for acute leukemia.全身照射及环磷酰胺加抗胸腺细胞球蛋白方案耐受性良好，并且作为急性白血病T细胞充足的单倍体相合移植前的预处理方案，可促进稳定植入。

Biol Blood Marrow Transplant. 2014 Aug;20(8):1176-82. doi: 10.1016/j.bbmt.2014.04.012. Epub 2014 Apr 18.

Fludarabine, cyclophosphamide, anti-thymocyteglobulin, and low-dose total body irradiation conditioning enables 1-HLA-locus-mismatched hematopoietic stem cell transplantation for very severe aplastic anemia without affecting ovarian function.

Am J Hematol. 2009 Mar;84(3):167-9. doi: 10.1002/ajh.21355.

Reduced toxicity, myeloablative HLA-haploidentical hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for sickle cell disease.降低毒性的清髓性人类白细胞抗原半相合造血干细胞移植联合移植后环磷酰胺治疗镰状细胞病

Ann Hematol. 2017 Aug;96(8):1373-1377. doi: 10.1007/s00277-017-3030-x. Epub 2017 Jun 1.

Successful engraftment with fludarabine, cyclophosphamide, and thymoglobulin conditioning regimen in unrelated transplantation for severe aplastic anemia: A phase II prospective multicenter study.氟达拉滨、环磷酰胺和抗胸腺球蛋白预处理方案在非亲缘造血干细胞移植治疗重型再生障碍性贫血中的Ⅱ期前瞻性多中心研究。

Biol Blood Marrow Transplant. 2010 Nov;16(11):1582-8. doi: 10.1016/j.bbmt.2010.05.010. Epub 2010 May 26.

A well-tolerated regimen of 800 cGy TBI-fludarabine-busulfan-ATG for reliable engraftment after unmanipulated haploidentical peripheral blood stem cell transplantation in adult patients with acute myeloid leukemia.在成年急性髓系白血病患者中，一种耐受性良好的800厘戈瑞全身照射-氟达拉滨-白消安-抗胸腺细胞球蛋白方案用于非处理单倍体相合外周血干细胞移植后可靠植入。

Biol Blood Marrow Transplant. 2015 Jan;21(1):119-29. doi: 10.1016/j.bbmt.2014.09.029. Epub 2014 Oct 6.

Using fludarabine to reduce exposure to alkylating agents in children with sickle cell disease receiving busulfan, cyclophosphamide, and antithymocyte globulin transplant conditioning: results of a dose de-escalation trial.在接受白消安、环磷酰胺和抗胸腺细胞球蛋白移植预处理的镰状细胞病患儿中，使用氟达拉滨减少烷化剂暴露：剂量递减试验结果

Biol Blood Marrow Transplant. 2015 May;21(5):900-5. doi: 10.1016/j.bbmt.2015.01.015. Epub 2015 Jan 21.

引用本文的文献

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review.镰状细胞病的造血干细胞移植：多维综述。

Cell Transplant. 2024 Jan-Dec;33:9636897241246351. doi: 10.1177/09636897241246351.

Knowledge to date on secondary malignancy following hematopoietic cell transplantation for sickle cell disease.目前关于镰状细胞病造血细胞移植后继发恶性肿瘤的知识。

Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):266-271. doi: 10.1182/hematology.2022000371.

Debating the Future of Sickle Cell Disease Curative Therapy: Haploidentical Hematopoietic Stem Cell Transplantation vs. Gene Therapy.镰状细胞病治愈性疗法的未来辩论：单倍体相合造血干细胞移植与基因疗法

J Clin Med. 2022 Aug 16;11(16):4775. doi: 10.3390/jcm11164775.

Clonal Hematopoiesis and the Risk of Hematologic Malignancies after Curative Therapies for Sickle Cell Disease.镰状细胞病根治性治疗后的克隆性造血与血液系统恶性肿瘤风险

J Clin Med. 2022 Jun 2;11(11):3160. doi: 10.3390/jcm11113160.

Choice of Donor Source and Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Sickle Cell Disease.镰状细胞病造血干细胞移植的供体来源选择和预处理方案

J Clin Med. 2019 Nov 15;8(11):1997. doi: 10.3390/jcm8111997.

Effect of increased dose of total body irradiation on graft failure associated with HLA-haploidentical transplantation in patients with severe haemoglobinopathies: a prospective clinical trial.增加全身照射剂量对重度血红蛋白病患者HLA单倍型移植相关移植物失败的影响：一项前瞻性临床试验。

Lancet Haematol. 2019 Apr;6(4):e183-e193. doi: 10.1016/S2352-3026(19)30031-6. Epub 2019 Mar 14.

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Haploidentical hematopoietic stem cell transplant for patients with sickle cell disease using thiotepa, fludarabine, thymoglobulin, low dose cyclophosphamide, 200 cGy tbi and post transplant cyclophosphamide.

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