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范可尼贫血的造血干细胞移植:来自印度的单中心经验

Hematopoietic Stem Cell Transplantation for Fanconi Anemia: A Single Center Experience from India.

作者信息

Doval Divya, Choudhary Dharma, Sharma Sanjeev Kumar, Khandelwal Vipin, Kumar Meet, Handoo Anil, Setia Rasika

机构信息

Department of Hemato-Oncology and Bone Marrow Transplantation, BLK Superspeciality Hospital, New Delhi, 110005 India.

Laboratory Services, Department of Haematology, BLK Superspeciality Hospital, New Delhi, 110005 India.

出版信息

Indian J Hematol Blood Transfus. 2020 Jul;36(3):565-568. doi: 10.1007/s12288-020-01254-3. Epub 2020 Feb 24.

Abstract

Hematopoietic stem cell transplantation (HSCT) is the only treatment option for the hematological manifestations of Fanconi anemia (FA). Fludarabine based reduced intensity conditioning regimens have helped in improving outcomes significantly in FA patients. We retrospectively analyzed the outcomes of FA patients who underwent allogeneic HSCT at BLK Superspeciality Hospital, New Delhi from June 2011 to September 2019. Twenty FA patients underwent 23 transplants at our center. Overall survival and disease free survival were 65% and 50%, respectively at a median of 23 months. Overall mortality was 30%. HSCT for FA is a feasible option even in developing countries although children present late to transplant centers after multiple transfusions and infections.

摘要

造血干细胞移植(HSCT)是范可尼贫血(FA)血液学表现的唯一治疗选择。基于氟达拉滨的减低强度预处理方案有助于显著改善FA患者的治疗效果。我们回顾性分析了2011年6月至2019年9月在新德里BLK超级专科医院接受异基因HSCT的FA患者的治疗效果。20例FA患者在我们中心接受了23次移植。中位随访23个月时,总生存率和无病生存率分别为65%和50%。总死亡率为30%。尽管儿童在多次输血和感染后很晚才到移植中心,但FA的HSCT即使在发展中国家也是一种可行的选择。

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Haematopoeitic cell transplantation for Fanconi anaemia - when and how?范可尼贫血的造血细胞移植:何时及如何进行?
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Fanconi anemia and its diagnosis.范可尼贫血及其诊断。
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