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Outcome of children with refractory anaemia with excess of blast ( RAEB ) and RAEB in Transformation ( RAEB ‐T) in the J apanese MDS 99 study
Author(s) -
Kikuchi Akira,
Hasegawa Daisuke,
Ohtsuka Yoshitoshi,
Hamamoto Kazuko,
Kojima Seiji,
Okamura Jun,
Nakahata Tatsutoshi,
Manabe Atsushi
Publication year - 2012
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2012.09210.x
Subject(s) - etoposide , medicine , cytarabine , mitoxantrone , survival rate , hematopoietic stem cell transplantation , transplantation , immunology , gastroenterology , chemotherapy
Summary 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.

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