Outcome of children with Refractory Anaemia with Excess of Blast (RAEB) and RAEB in Transformation (RAEB-T) in the Japanese MDS99 study.
Outcome of children with Refractory Anaemia with Excess of Blast (RAEB) and RAEB in Transformation (RAEB-T) in the Japanese MDS99 study.
复制标题
日本 MDS99 研究中难治性贫血伴原始细胞过多 (RAEB) 和 RAEB 转化 (RAEB-T) 儿童的结果。
DOI：
10.1111/j.1365-2141.2012.09210.x
复制
发表时间：
2012
期刊：
影响因子：
--
通讯作者：
Kikuchi A.
中科院分区：
文献类型：
--
作者：
Matsuzaki J;Kagitani-Shimono K;Sugata H;Hirata M;Hirata I;Hanaie R;Nagatani F;Tachibana M;Tominaga T;Mohri I;Taniike M.;Kikuchi A.
我们报告了16例难治性贫血伴原始细胞增多(RAEB；n=4)和RAEB转化中(RAEB-T；n=12)的患儿在造血干细胞移植(HSCT)前用依托泊苷、阿糖胞苷和米托蒽醌(ECM)诱导治疗的结果。中位观察期77个月(5~123个月)。诱导后完全缓解率为81%，无中毒性死亡。8年无事件生存率和总生存率分别为50%和56%。三名核型复杂的患者无一存活，这表明核型是影响生存的关键预后因素。这项研究表明，对于儿童RAEB和RAEB-T，ECM安全、缓解率高，HSCT后存活率高。
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.