title: Acute myeloid leukaemia with myelodysplastic features in children: a report of Japanese Paediatric Leukaemia/Lymphoma Study Group.
abstract: The clinical characteristics and prognostic relevance of acute myeloid leukaemia (AML) with myelodysplastic features remains to be clarified in children. We prospectively examined 443 newly diagnosed patients in a multicentre clinical trial for paediatric de novo AML, and found 'AML with myelodysplasia-related changes' (AML-MRC) according to the 2008 World Health Organization classification in 93 (21·0%), in whom 59 were diagnosed from myelodysplasia-related cytogenetics alone, 28 from multilineage dysplasia alone and six from a combination of both. Compared with 111 patients with 'AML, not otherwise specified' (AML-NOS), patients with 'AML-MRC' presented at a younger age, with a lower white blood cell count, higher incidence of 20-30% bone marrow blasts, unfavourable cytogenetics and a lower frequency of Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD), NPM1 and CEBPA mutations. Complete remission rate and 3-year probability of event-free survival were significantly worse in 'AML-MRC' patients (67·7 vs. 85·6%, P < 0·01, 37·1% vs. 53·8%, P = 0·02, respectively), but 3-year overall survival and relapse-free survival were comparable with 'AML-NOS' patients. By multivariate analysis, FLT3-ITD was solely associated with worse overall survival. These results support the distinctive features of the category 'AML-MRC' even in children.
authors.affiliation: Department of Paediatrics, St. Marianna University School of Medicine, Kawasaki, Japan.
authors.forename: Akitoshi
authors.initials: A
authors.lastname: Kinoshita
authors.forename: Hayato
authors.initials: H
authors.lastname: Miyachi
authors.forename: Hiromichi
authors.initials: H
authors.lastname: Matsushita
authors.forename: Miharu
authors.initials: M
authors.lastname: Yabe
authors.forename: Tomohiko
authors.initials: T
authors.lastname: Taki
authors.forename: Tomoyuki
authors.initials: T
authors.lastname: Watanabe
authors.forename: Akiko M
authors.initials: AM
authors.lastname: Saito
authors.forename: Daisuke
authors.initials: D
authors.lastname: Tomizawa
authors.forename: Takashi
authors.initials: T
authors.lastname: Taga
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Takahashi
authors.forename: Hidemasa
authors.initials: H
authors.lastname: Matsuo
authors.forename: Kumi
authors.initials: K
authors.lastname: Kodama
authors.forename: Kentaro
authors.initials: K
authors.lastname: Ohki
authors.forename: Yasuhide
authors.initials: Y
authors.lastname: Hayashi
authors.forename: Akio
authors.initials: A
authors.lastname: Tawa
authors.forename: Keizo
authors.initials: K
authors.lastname: Horibe
authors.forename: Souichi
authors.initials: S
authors.lastname: Adachi
mesh_terms: D000293:Adolescent; D001853:Bone Marrow / Q000473:pathology*; D002648:Child; D002675:Child, Preschool; D020732:Cytogenetic Analysis; D005260:Female; D006801:Humans; D060828:Induction Chemotherapy; D007223:Infant; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000188:drug therapy / Q000401:mortality / Q000473:pathology*; D008297:Male; D000090243:Nucleophosmin; D011379:Prognosis; D012307:Risk Factors; D016896:Treatment Outcome
keywords: WHO classification; multilineage dysplasia; myelodysplasia; paediatric acute myeloid leukaemia