Myelodysplastic syndromes (MDS)

Abstract

MDS in children is a rare group of hematopoietic stem cell clonal disorder. Allogeneic HSCT is the only curative treatment. HLA typing and the search for a compatible donor must be carried out upon diagnosis, for all patients.  However, patients with refractory cytopenia of childhood without an unfavorable karyotype can keep the disease stable for a long time. Thus, in the absence of transfusion dependence or severe neutropenia, a careful observation strategy without treatment is recommended. The treatment of children diagnosed with MDS with excess blasts remains a major challenge. Allogeneic HSCT is the only curative treatment, although the data published in the literature generally include a small number of patients, heterogeneously transplanted. For children with MDS secondary to therapy, despite HSCT, the evolution is generally unfavorable.