Brachet C, Azzi N, Demulder A, Devalck C, Gourdin A, Gulbis B, Klein A, Le P Q, Loop M, Sariban E, Ferster A
1Haemato-Oncology Unit, Hôpital Universitaire des Enfants-ULB, Brussels, Belgium.
Bone Marrow Transplant. 2004 Apr;33(8):799-803. doi: 10.1038/sj.bmt.1704443.
Since 1988, 24 children have undergone haematopoietic stem cell transplantation (HSCT) for severe sickle cell disease (SCD) in our unit, 13 being grafted after having been exposed to hydroxyurea (HU) to control SCD-related complications. Different pre-transplant conditioning regimens were given over time: Bu14/Cy200 in six patients (group 1), Bu16/Cy200/antithymocyte globulin (ATG) in five (group 2) and Bu16/Cy200/ATG with HU prior to HSCT in 13 (group 3). The aim of this study is to compare the outcome after HSCT of these groups of patients, which differ according to pre-transplant drug exposure. Overall, 20 of the 24 transplanted children had stable engraftment and have remained free of SCD-related symptoms after HSCT; 19 of them are currently alive and cured of SCD. In group 1 (HU-, ATG-), we observed one unexplainable late death, one absent engraftment, one late rejection and one mixed stable chimerism. In group 2 (HU-, ATG+), we observed the absence of engraftment in two patients and one early rejection. In group 3 (HU+, ATG+), we observed no cases of either absent engraftment, mixed stable chimerism or late rejection. In our experience, pre-transplant treatment with HU seems to be associated with a lower incidence of rejection/absent engraftment in severe SCD patients. These results need to be confirmed with a larger number of patients.
自1988年以来，我们科室有24名儿童因重症镰状细胞病（SCD）接受了造血干细胞移植（HSCT），其中13名在接受羟基脲（HU）治疗以控制SCD相关并发症后进行了移植。随着时间的推移，采用了不同的移植前预处理方案：6例患者采用白消安14mg/kg/环磷酰胺200mg/kg（第1组），5例采用白消安16mg/kg/环磷酰胺200mg/kg/抗胸腺细胞球蛋白（ATG）（第2组），13例在HSCT前采用白消安16mg/kg/环磷酰胺200mg/kg/ATG并联合HU（第3组）。本研究的目的是比较这些患者组HSCT后的结局，这些组因移植前药物暴露情况不同而有所差异。总体而言，24例接受移植的儿童中有20例移植稳定，HSCT后未出现SCD相关症状；其中19例目前存活且已治愈SCD。在第1组（未使用HU、未使用ATG）中，我们观察到1例无法解释的晚期死亡、1例未植入、1例晚期排斥和1例混合稳定嵌合体。在第2组（未使用HU、使用ATG）中，我们观察到2例患者未植入和1例早期排斥。在第3组（使用HU、使用ATG）中，我们未观察到未植入、混合稳定嵌合体或晚期排斥的病例。根据我们的经验，对于重症SCD患者，移植前使用HU似乎与较低的排斥/未植入发生率相关。这些结果需要更多患者来证实。
