Outcome of children with refractory anaemia with excess of blast (RAEB) and RAEB in transformation (RAEB-T) in the Japanese MDS99 study.
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.
Department of Paediatrics, Teikyo University School of Medicine, Tokyo, Japan. akikuchi@med.teikyo-u.ac.jp
Akira
A
Kikuchi
Daisuke
D
Hasegawa
Yoshitoshi
Y
Ohtsuka
Kazuko
K
Hamamoto
Seiji
S
Kojima
Jun
J
Okamura
Tatsutoshi
T
Nakahata
Atsushi
A
Manabe
D000754:Anemia, Refractory, with Excess of Blasts / Q000473:pathology / Q000628:therapy*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002471:Cell Transformation, Neoplastic / Q000473:pathology; D002648:Child; D003561:Cytarabine / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D053208:Kaplan-Meier Estimate; D008942:Mitoxantrone / Q000008:administration & dosage; D012074:Remission Induction / Q000379:methods; D016896:Treatment Outcome
D003561:Cytarabine; D005047:Etoposide; D008942:Mitoxantrone