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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. © 2012 Blackwell Publishing Ltd.


Akira Kikuchi, Daisuke Hasegawa, Yoshitoshi Ohtsuka, Kazuko Hamamoto, Seiji Kojima, Jun Okamura, Tatsutoshi Nakahata, Atsushi Manabe, Japanese Paediatric Myelodysplastic Syndrome (MDS) Study Group. Outcome of children with refractory anaemia with excess of blast (RAEB) and RAEB in transformation (RAEB-T) in the Japanese MDS99 study. British journal of haematology. 2012 Sep;158(5):657-61

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PMID: 22734597

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