镰状细胞病管理的现状:干细胞移植治疗的简明综述

George Nkansah Rost Fordjour
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引用次数: 0

摘要

镰状细胞病的治疗在很大程度上仍然是姑息性的。虽然它可以改善生活质量,但患有镰状细胞病的人仍然遭受严重的镰状危象、终末器官损伤和预期寿命缩短。越来越多的研究导致了干细胞移植和基因治疗作为镰状细胞病潜在治疗策略的确定和进展。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,但受限于有限的移植供体池、移植并发症和选择标准。本文回顾了镰状细胞病的文献,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。关键词:脐带血,单倍体,镰状细胞病,干细胞移植
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The Current State of Sickle Cell Disease Management: A Concise Review on Stem Cell Transplant as a Cure
Treatment of sickle cell disease remains largely palliative. While it may improve the quality of life, persons with sickle cell disease still suffer from extreme sickling crises, end-organ damage, and reduced life expectancy. Increasing studies have led to the identification and advancement of stem cell transplant and gene therapy as potential curative strategies for sickle cell disease. However, there have been various factors that have hindered their clinical application. Stem cell transplantation, the more propitious of the two, is limited by restricted transplant donor pool, transplant complications, and selection criteria. The current paper reviewed the literature on sickle cell disease, current treatment options, and more particularly on 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. Keywords: Cord blood, Haploidentical, Sickle cell disease, Stem cell transplantation
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