Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France
Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France
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DOI：
10.3324/haematol.2018.213207
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发表时间：
2020-01-01
期刊：
影响因子：
10.1
通讯作者：
Socie, Gerard
中科院分区：
文献类型：
--
作者：
Bernaudin, Francoise;Dalle, Jean-Hugues;Socie, Gerard
异基因干细胞移植仍然是治疗镰状细胞性贫血(SCA)的唯一方法，但清髓性调理在这一过程中的地位仍有待确定。本研究的目的是分析法国一系列30岁以下的SCA患者的长期结果，包括嵌合体、SCA相关事件和生物学数据(血红蛋白、网织红细胞、HBS%)和生育力，这些患者在1988年至2012年接受了标准的骨髓清除性调节[白果、环磷酰胺和兔抗胸腺细胞球蛋白(ATG)]的匹配同胞供者干细胞移植。自从该系列的第一份报告(1988-2004年)以来，已有151名连续的新的SCA患者进行了类似的移植。考虑死亡、未植入或排斥(供体细胞
Allogeneic stem cell transplantation remains the only curative treatment for sickle cell anemia (SCA), but the place of myeloablative conditioning in the procedure remains to be defined. The aim of the present study was to analyze long-term outcomes, including chimerism, SCA-related events and biological data (hemoglobin, reticulocytes, HbS%), and fertility in a French series of 234 SCA patients under 30 years of age who, from 1988 to 2012, received a matched-sibling-donor stem cell transplantation following standardized myeloablative conditioning [busulfan, cyclophosphamide and rabbit anti-thymocyte globulin (ATG)]. Since the first report of the series (1988-2004), 151 new consecutive patients with SCA have been similarly transplanted. Considering death, non-engraftment or rejection (donor cells