Myelodysplastic Syndromes in Children: A Critical Review of Issues in the Diagnosis and Classification of 887 Cases From 13 Published Series

Abstract

Abstract Context.—Pediatric myelodysplastic syndromes (MDSs) are uncommon disorders, which may be difficult to diagnose, particularly in the absence of increased blasts. Pediatric MDSs have several unique features including their association with inherited/constitutional disorders in approximately one third of patients. The classification of pediatric MDSs has undergone significant evolution in the past 20 years. Objective.—To critically review existing classifications of pediatric MDSs and to evaluate their applicability on previously published large series. Data Sources.—Previously published pediatric MDS series containing more than 10 patients from the English literature between 1982 and 2005. Conclusions.—Data were available on 887 patients from 13 published series. Most cases (68.7%) were idiopathic/ de novo, 23.9% were associated with constitutional/inherited disorder, and 7.4% were therapy related. Approximately 10% of cases could not be classified by the French-American-British classification. Eighty-seven percent of unclassified cases were appropriately classified using the World Health Organization classification (2001), whereas 96% of them were classified with the modified World Health Organization classification for pediatric MDSs (2003). The impact of cytogenetics and constitutional/inherited disorders on the biology and outcome of the disease needs to be studied further.