Pediatric Myeloproliferative Disorders/Myelodysplastic Syndromes/Overlap Syndromes
摘要
Myelodysplastic syndromes (MDS) are a diverse collection of hematopoietic clonal disease defined by poor hematopoiesis. In absence of prompt treatment, this disease progresses to leukemia. These entities are rare as well as challenging to treat in children. A variety of genetic disorders and inherited bone marrow failure syndromes are associated with this disease. This has led to its incorporation in disease classification and has implications in therapy. While some disorders like transient abnormal myelopoiesis are self-limiting, the majority require allogeneic stem cell transplant and are preferred treatment and standard of care. Adults have shown potential with newer therapies, which are currently in advanced stages of clinical trials.