低リスク骨髄異形成症候群の治療目標

Myelodysplastic syndrome (MDS) is an abnormality of hematopoietic stem cells with limited effective drug therapy. Owing to the diversity of disease subtypes, treatment options must be decided on a case-by-case basis based on predictive prognostic scoring systems. The recently published revised WHO and ICC classifications incorporate genetic abnormalities into the diagnosis, and IPSS-M, which incorporates genetic mutations into the IPSS-R, allows for more individualized prognostic prediction. The treatment of low-risk MDS focuses on managing infection and bone marrow failure while preserving the quality of life: pharmacotherapy such as erythropoiesis-stimulating agents, lenalidomide, azacitidine, luspatercept, and allogeneic hematopoietic stem cell transplantation. The treatment choice depends on the profile at diagnosis and the results of predictive prognostic studies. Although there is little clear evidence regarding the timing of treatment initiation and goals of treatment, the NCCN guidelines suggest a hemoglobin level of 10-12 g/dl. Otsuka's HemeSight^® hematological malignancy gene panel test, which will be introduced in Japan this year, is expected to contribute to the development of personalized treatments based on genetic information.