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Recombinant erythropoietin (rEPO) is a biosynthetic version of the human hormone erythropoietin, produced via recombinant DNA technology. It acts as an erythropoiesis-stimulating agent (ESA) by binding to the erythropoietin receptor (EPOR) on erythroid progenitor cells in the bone marrow. This binding stimulates the proliferation, maturation, and survival of these cells, leading to increased red blood cell production. In the context of myelodysplastic syndromes (MDS), rEPO is used to treat symptomatic anemia, particularly in patients with a low blast count and low endogenous erythropoietin levels. The Fondazione Italiana Sindromi Mielodisplastiche-ETS (FISM) has conducted Phase III clinical research (such as trial NCT00252707) to evaluate the efficacy of rEPO as a standalone therapy compared to its use in combination with differentiating agents like isotretinoin and calcitriol in patients with MDS without an excess of blasts.
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