Macrocytic anaemia spans nutritional (megaloblastic) and marrow (myeloid neoplasm) causes, and modern therapy tracks that division. For anaemia associated with myeloid neoplasms — myelodysplastic syndromes and myelofibrosis — targeting the TGF-β pathway with luspatercept and using momelotinib (an ACVR1/JAK inhibitor that lowers hepcidin and improves iron availability, approved for myelofibrosis-associated anaemia) reduce transfusion dependence, and combination approaches (luspatercept with erythropoiesis-stimulating agents or momelotinib) are being explored to address the multifactorial anaemia of these disorders. For the megaloblastic anaemias of vitamin B12 or folate deficiency, prompt supplementation of the missing vitamin produces rapid haematologic recovery.
Further reading: emerging drugs for anaemia in myelofibrosis and MDS (Gangat & Tefferi, American Journal of Hematology 2025); luspatercept and momelotinib in myeloid-neoplasm anaemia.