镰状细胞病的现状:干细胞移植治疗的综述

Md Sadique Hussain, Varunesh Chaturvedi
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引用次数: 0

摘要

镰状细胞病(SCD)的治疗仍然主要是姑息性的。虽然它可以提高生活水平,但患有SCD的人仍然遭受严重的镰状危象、终末器官破坏和预期寿命缩短。越来越多的研究导致了干细胞移植和基因治疗作为SCDs可能的解决方案的认识和进展。然而,有各种因素阻碍了它们的临床应用。干细胞移植是两者中较有利的一种,但受供体数量少、移植困难和资格要求的限制。本文回顾了SCDs的文献,目前的治疗方案,特别是干细胞移植的进展。它概述了干细胞移植的各种挑战,并提出了使用替代策略和修改方案条件以减少移植相关毒性和相关并发症来增加供体池的方法。
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The Present Condition of Sickle Cell Disease: An Overview of Stem Cell Transplantation as a Cure
Abstract Treatment of sickle cell disease (SCD) remains largely palliative. While it can enhance living standards, persons having SCD still suffer from extreme sickling crises, end-organ destruction, and reduced life expectancy. Increasing research has resulted in the recognition and advancement of stem cell transplantation and gene therapy as possible solutions for SCDs. However, there have been various factors that have hindered their clinical application. The more advantageous of the two, stem cell transplantation, is constrained by a small donor pool, transplant difficulties, and eligibility requirements. The current article reviewed the literature on SCDs, current treatment options, and more particularly the progress of stem cell transplants. It outlined various challenges of stem cell transplant and proposed ways to increase the donor pool using alternative strategies and modifications of regimen conditioning with minimal transplant-related toxicities and associated complications.
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