斯里兰卡单倍体同种异体造血干细胞移植治疗遗传性骨髓衰竭的报道。

IF 0.3 Q3 MEDICINE, GENERAL & INTERNAL

Ceylon Medical Journal Pub Date : 2022-03-31 DOI:10.4038/cmj.v67i1.9554

Wasanthi Wickramasinghe, Revathi Raj, Shama Goonatillake, Anuja Abayadeera, Malik Samarasinghe, Jeewaka Galhenage, Pavithra Aarewatte, Devan Mendis, Chandima Thevarapperuma, Ruwangi Dissanayake, Lallindra Gooneratne

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引用次数: 0

摘要

摘要:儿童骨髓衰竭(BMF)可为特发性(70-80%)或遗传性。造血干细胞移植(HSCT)是治疗这两种疾病的唯一方法。同种异体造血干细胞移植需要合适的供体。许多孩子没有匹配的HLA的兄弟姐妹或无血缘关系的供体。所有的孩子都可以获得单倍体相同的供体，因为父母双方至少有50%的HLA匹配。这是斯里兰卡首例7岁BMF女孩接受单倍体造血干细胞移植治疗的报告，强调了开发单倍体造血干细胞移植计划作为治疗这种预后不佳的疾病的潜在方法的重要性。

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A report of haploidentical allogeneic haematopoietic stem cell transplantation for inherited bone marrow failure in Sri Lanka.

Abstract: Bone marrow failure (BMF) in children can be idiopathic (70-80%) or inherited. Haematopoietic stem cell transplantation (HSCT) is the only cure for both causes. Allogeneic HSCT requires a suitable donor. Many children will not have a HLA matched sibling or unrelated donor. A haploidentical donor is available for all children as eaazch parent will have at minimum a 50% HLA match. This report of a 7-year old girl with BMF treated with a haplo-HSCT, the first in Sri Lanka, highlights the importance of developing a haploidentical HSCT programme as a potential cure for a disease with a dismal outcome.

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来源期刊

Ceylon Medical Journal MEDICINE, GENERAL & INTERNAL-

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期刊介绍： The Ceylon Medical Journal, is the oldest surviving medical journal in Australasia. It is the only medical journal in Sri Lanka that is listed in the Index Medicus. The CMJ started life way back in 1887 as the organ of the Ceylon Branch of the British Medical Association. Except for a brief period between 1893 and 1904 when it ceased publication, the CMJ or its forbear, the Journal of the Ceylon Branch of the British Medical Association, has been published without interruption up to now. The journal"s name changed to the CMJ in 1954.