Since the first report of a young girl affected by sickle cell anemia, treated successfully by bone marrow transplantation (BMT) for acute myeloid leukemia, more than 200 patients have been transplanted worldwide for sickle cell anemia. The disease-free survival (DFS) is good (80-85% in several series), even though many children who received allografts had already significant sickle-related complications. The best results are obtained in young children who have HLA-identical sibling donors and are transplanted early in the course of the disease (DFS: 93%). Future directions in the field of stem cell transplantation of sickle cell anemia include (1) the establishment of new protocols with less toxicity, but still effective, (2) adapted conditioning regimen for adult patients, and (3) new sources of stem cells for broader application: umbilical cord blood and volunteer unrelated donors.

中文翻译：

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镰状细胞病中的造血干细胞移植。

自首次报道镰状细胞性贫血的年轻女孩通过骨髓移植（BMT）成功治疗急性髓细胞性白血病以来，全世界已有200多例患者因镰状细胞性贫血而被移植。即使许多接受同种异体移植的儿童已经患有严重的与镰刀有关的并发症，其无病生存期（DFS）仍然很好（在多个系列中为80-85％）。在具有与HLA相同的同胞供体并在病程早期进行移植的幼儿中可获得最佳结果（DFS：93％）。镰状细胞性贫血的干细胞移植领域的未来方向包括（1）建立毒性较小但仍有效的新方案，（2）针对成年患者的适应性调理方案，以及（3）用于干细胞贫血的新干细胞来源广泛的应用：