Myelodysplastic syndromes in childhood--classification, epidemiology, and treatment.

Much of the applied terminology of myelodysplastic syndromes (MDS) in childhood is confusing and not mutually exclusive. It is therefore proposed that the FAB classification of MDS is used in children in order to improve diagnostic precision and to facilitate epidemiologic, clinical, and therapeutic comparisons. The true incidence of childhood MDS is unknown but the rate may approximate the incidence of acute myelogenous leukemia. A pooled analysis of eight larger series representing 110 children less than 15 years old at diagnosis with de novo MDS classified according to the FAB recommendations showed that the more aggressive subtypes dominated, which partly may reflect that the less advanced cases are underdiagnosed. The median age at presentation was 6.0 years. The male/female ratio was 1.6. Monosomy 7 was the most frequent cytogenetic abnormality. The median survival was 13 months and the probability of survival three years from diagnosis was 16%. Spontaneous remission may be observed very infrequently. Allogeneic bone marrow transplantation (BMT) represents the only potentially curative treatment. The survival rate three years after BMT is about 50%. Major differences between childhood and adult MDS exist with respect to the distribution of FAB subgroups, the rate of progression, and the cytogenetic findings. The literature on MDS in children is still sparse and there is an obvious need for more studies designed to determine the incidence, clinical and laboratory characteristics, the natural course, and the efficacy of contemporary treatment options.