范可尼贫血患者异基因造血干细胞移植的疗效 - Suppr

Outcomes in allogeneic hematopoietic stem cell transplantation for Fanconi anemia.

作者信息

Gonzalez Vicent M, Peretó A, Zubicaray J, Molina B, Vinagre S, Sebastián E, Iriondo J, Castillo A, Abad L, Sanz A, Lopez de Hontanar G, Ramirez M, Sevilla J, Diaz M A

机构信息

Stem Cell Transplant Unit, H. Niño Jesus, Madrid, Spain.

Stem Cell Transplant Unit, H. Clínico, Valencia, Spain.

出版信息

Bone Marrow Transplant. 2025 Jun;60(6):914-916. doi: 10.1038/s41409-025-02591-5. Epub 2025 Apr 12.

DOI:10.1038/s41409-025-02591-5

PMID:40221635

Abstract

摘要

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Outcomes in allogeneic hematopoietic stem cell transplantation for Fanconi anemia.范可尼贫血患者异基因造血干细胞移植的疗效

Bone Marrow Transplant. 2025 Jun;60(6):914-916. doi: 10.1038/s41409-025-02591-5. Epub 2025 Apr 12.

Fertility recovery and pregnancy after allogeneic hematopoietic stem cell transplantation in Fanconi anemia patients.范可尼贫血患者异基因造血干细胞移植后生育力的恢复和妊娠。

Haematologica. 2010 Oct;95(10):1783-7. doi: 10.3324/haematol.2010.023929. Epub 2010 May 21.

Hematopoietic Stem Cell Transplant in Adolescent and Young Adults With Fanconi Anemia Is Feasible With Acceptable Toxicity, With Those Surviving 100 Days Posttransplant Having Excellent Outcomes.对患有范可尼贫血的青少年和青年进行造血干细胞移植是可行的，毒性可接受，移植后存活100天的患者预后良好。

Exp Clin Transplant. 2016 Dec;14(6):660-664. doi: 10.6002/ect.2015.0364. Epub 2016 Jul 22.

[Treatment of four cases of Fanconi anemia by allogeneic hematopoietic stem cell transplantation with low intensity conditional regimen].[低强度预处理方案异基因造血干细胞移植治疗4例范可尼贫血]

Zhonghua Xue Ye Xue Za Zhi. 2018 Mar 14;39(3):231-235. doi: 10.3760/cma.j.issn.0253-2727.2018.03.011.

[Outcomes of alternative donor allogeneic hematopoietic stem cell transplantation for Fanconi anemia: a five cases report].范可尼贫血替代供者异基因造血干细胞移植的结果：5例报告

Zhonghua Nei Ke Za Zhi. 2018 Jan 1;57(1):54-56. doi: 10.3760/cma.j.issn.0578-1426.2018.01.010.

Successful sequential liver and hematopoietic stem cell transplantation in a patient with Fanconi anemia.成功对范可尼贫血患者进行序贯肝和造血干细胞移植。

Pediatr Transplant. 2023 Aug;27(5):e14503. doi: 10.1111/petr.14503. Epub 2023 Mar 13.

Stem Cell Therapy for Fanconi Anemia.成体干细胞治疗范可尼贫血

Adv Exp Med Biol. 2018;1083:19-28. doi: 10.1007/5584_2017_67.

Pregnancy after allogeneic hematopoietic stem cell transplantation in a Fanconi anemia patient.一名范可尼贫血患者接受异基因造血干细胞移植后的妊娠情况。

Int Med Case Rep J. 2017 Jan 10;10:11-14. doi: 10.2147/IMCRJ.S124414. eCollection 2017.

Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia.大海捞针：范可尼贫血中的造血干细胞

Mutat Res. 2009 Jul 31;668(1-2):141-9. doi: 10.1016/j.mrfmmm.2009.03.010. Epub 2009 Apr 2.

Sequential treatment for allogeneic hematopoietic stem cell transplantation in Fanconi anemia with acute myeloid leukemia.范可尼贫血合并急性髓系白血病患者异基因造血干细胞移植的序贯治疗

Haematologica. 2014 Oct;99(10):e199-200. doi: 10.3324/haematol.2013.098954. Epub 2014 Aug 1.

本文引用的文献

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia.813 例范可尼贫血患儿造血干细胞移植的结果。

Blood. 2024 Sep 19;144(12):1329-1342. doi: 10.1182/blood.2023022751.

Modern management of Fanconi anemia.范可尼贫血的现代治疗管理。

Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):649-657. doi: 10.1182/hematology.2022000393.

T-Cell Depleted Haploidentical Transplantation in Children With Hematological Malignancies: A Comparison Between CD3+/CD19+ and TCRαβ+/CD19+ Depletion Platforms.血液系统恶性肿瘤患儿的T细胞去除单倍体相合移植：CD3⁺/CD19⁺与TCRαβ⁺/CD19⁺去除平台的比较

Front Oncol. 2022 Jun 20;12:884397. doi: 10.3389/fonc.2022.884397. eCollection 2022.

Excellent overall and chronic graft-versus-host-disease-free event-free survival in Fanconi anaemia patients undergoing matched related- and unrelated-donor bone marrow transplantation using alemtuzumab-Flu-Cy: the UK experience.在接受阿仑单抗-氟达拉滨-环磷酰胺预处理的范可尼贫血患者中，采用亲缘和非亲缘供者骨髓移植后，总体和慢性移植物抗宿主病无事件生存率优异：英国经验。

Br J Haematol. 2021 May;193(4):804-813. doi: 10.1111/bjh.17418. Epub 2021 Apr 14.

Haplo-identical or mismatched unrelated donor hematopoietic cell transplantation for Fanconi anemia: Results from the Severe Aplastic Anemia Working Party of the EBMT.单倍体相合或不合亲缘供者造血细胞移植治疗范可尼贫血：来自 EBMT 重型再生障碍性贫血工作组的研究结果。

Am J Hematol. 2021 May 1;96(5):571-579. doi: 10.1002/ajh.26135. Epub 2021 Mar 4.

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.基因校正的造血干细胞在非条件预处理的范可尼贫血患者中成功植入。

Nat Med. 2019 Sep;25(9):1396-1401. doi: 10.1038/s41591-019-0550-z. Epub 2019 Sep 9.

Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations.范可尼贫血症的造血细胞移植：现有证据、挑战与建议。

Expert Rev Hematol. 2017 Jan;10(1):81-97. doi: 10.1080/17474086.2016.1268048. Epub 2016 Dec 21.

Immune reconstitution in patients with Fanconi anemia after allogeneic bone marrow transplantation.范可尼贫血患者异基因骨髓移植后的免疫重建

Cytotherapy. 2014 Jul;16(7):976-89. doi: 10.1016/j.jcyt.2014.02.015. Epub 2014 May 13.

Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience.异基因造血干细胞移植治疗范可尼贫血：欧洲血液和骨髓移植学会经验。

Blood. 2013 Dec 19;122(26):4279-86. doi: 10.1182/blood-2013-01-479733. Epub 2013 Oct 21.

Malignancies after marrow transplantation for aplastic anemia and fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients.再生障碍性贫血和范可尼贫血骨髓移植后的恶性肿瘤：西雅图和巴黎对700例患者结果的联合分析

Blood. 1996 Jan 1;87(1):386-92.

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Outcomes in allogeneic hematopoietic stem cell transplantation for Fanconi anemia.

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