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erythropoietin + hydroxyurea
- Development stage
- Phase 1
- Lead developer
- Amgen
- Modality
- Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules, Recombinant Proteins and Enzymes
- Administration
- Oral, Subcutaneous, Intravenous
01
Overview
Erythropoietin + hydroxyurea is a combination therapy used primarily in the management of sickle cell disease (SCD), particularly for patients with chronic anemia or those who are non-responders to single-agent therapy. Erythropoietin (often as recombinant human erythropoietin, rHuEPO or epoetin alfa) is a glycoprotein hormone that stimulates erythropoiesis by promoting the proliferation and differentiation of red blood cell precursors. Hydroxyurea is an antineoplastic agent that increases fetal hemoglobin (HbF) production and reduces sickling in SCD by inhibiting ribonucleotide reductase. The combination aims to improve hematologic parameters such as hemoglobin concentration and HbF levels, reduce hemolysis, and allow more aggressive dosing of hydroxyurea in high-risk SCD patients or those with mild renal insufficiency. Clinical studies have shown this combination can be effective and generally well-tolerated in SCD patients stabilized on hydroxyurea[1][2][3][4][5].
- Other names
- EPO + HUrHuEPO + hydroxyureaepoetin alfa + hydroxyurea
02
Targets
Erythropoietin ReceptorRNR (Ribonucleotide reductase)
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