Аннотация:Summary 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 ( FLT 3 ‐ ITD ), NPM 1 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, FLT 3 ‐ ITD was solely associated with worse overall survival. These results support the distinctive features of the category ‘ AML ‐ MRC ’ even in children.
