Department of Paediatrics, Teikyo University School of Medicine, Tokyo, Japan.
Br J Haematol. 2012 Sep;158(5):657-61. doi: 10.1111/j.1365-2141.2012.09210.x. Epub 2012 Jun 26.
We report the outcome of 16 children with refractory anaemia with excess of blasts (RAEB; n = 4) and RAEB in transformation (RAEB-T; n = 12) following induction therapy with etoposide, cytarabine and mitoxantrone (ECM) prior to haematopoietic stem cell transplantation (HSCT). The median observation period was 77 months (range 5-123). Complete remission rate was 81% following induction; no toxic deaths occurred. Eight-year event-free survival and overall survival was 50% and 56%, respectively. None of the three patients with a complex karyotype survived, suggesting karyotype is a crucial prognostic factor for survival. This study indicates the safety and high remission rate of ECM and high survival rates after HSCT for paediatric RAEB and RAEB-T.
我们报告了 16 例接受依托泊苷、阿糖胞苷和米托蒽醌（ECM）诱导治疗后进行造血干细胞移植（HSCT）的难治性贫血伴原始细胞过多（RAEB；n=4）和转化后 RAEB（RAEB-T；n=12）的儿童的结果。中位观察期为 77 个月（范围 5-123）。诱导后完全缓解率为 81%；无治疗相关死亡。8 年无事件生存率和总生存率分别为 50%和 56%。3 例复杂核型患者均未存活，提示核型是生存的关键预后因素。这项研究表明 ECM 诱导治疗具有安全性和高缓解率，并且儿童 RAEB 和 RAEB-T 进行 HSCT 后具有较高的生存率。
