Outcome of children with refractory anaemia with excess of blast (RAEB) and RAEB in Transformation (RAEB-T) in the Japanese MDS99 study

被引：13
作者：
Kikuchi, Akira [1 ]
Hasegawa, Daisuke [2 ]
Ohtsuka, Yoshitoshi [3 ]
Hamamoto, Kazuko [4 ]
Kojima, Seiji [5 ]
Okamura, Jun [6 ]
Nakahata, Tatsutoshi [7 ]
Manabe, Atsushi [2 ]
机构：
[1] Teikyo Univ, Dept Paediat, Sch Med, Itabashi Ku, Tokyo 1738605, Japan
[2] St Lukes Int Hosp, Dept Paediat, Tokyo, Japan
[3] Hyogo Coll Med, Dept Paediat, Hyogo, Japan
[4] Hiroshima Red Cross Hosp, Dept Paediat, Hiroshima, Japan
[5] Nagoya Univ, Dept Paediat, Grad Sch Med, Nagoya, Aichi 4648601, Japan
[6] Kyushu Canc Ctr Hosp, Paediat Sect, Fukuoka, Japan
[7] Kyoto Univ, Inst iPS Cell, Kyoto, Japan
来源：
BRITISH JOURNAL OF HAEMATOLOGY | 2012年 / 158卷 / 05期
关键词：
refractory anaemia with excess of blast; refractory anaemia with excess of blast in transformation; childhood; haematopoietic stem cell transplantation; ACUTE MYELOID-LEUKEMIA; JUVENILE MYELOMONOCYTIC LEUKEMIA; PEDIATRIC MYELODYSPLASTIC SYNDROMES; CHILDHOOD; CYTARABINE; THERAPY; TRIAL;
D O I：
10.1111/j.1365-2141.2012.09210.x
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
We report the outcome of 16 children with refractory anaemia with excess of blasts (RAEB; n similar to=similar to 4) and RAEB in transformation (RAEB-T; n similar to=similar to 12) following induction therapy with etoposide, cytarabine and mitoxantrone (ECM) prior to haematopoietic stem cell transplantation (HSCT). The median observation period was 77 similar to months (range 5123). 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.
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收藏
页码：657 / 661
页数：5
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