Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report

The occurrence of myelodysplastic syndrome (MDS) in children is infrequently documented and most commonly manifests in the form of refractory cytopenia of childhood (RCC). The management of pediatric MDS remains controversial and involves primarily chemotherapy, immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT). However, therapeutic data for patients with RCC who are ineligible for either HSCT or IST are scarce. In the present case, the 8-year-old patient achieved disease stabilization following decitabine monotherapy, thereby suggesting a potential novel therapeutic avenue for children with low-risk chronic MDS or RCC.