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hydroxyurea + thalidomide
- Development stage
- Phase 4
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Hydroxyurea + thalidomide is a combination therapy consisting of two small molecule drugs, hydroxyurea and thalidomide. This combination has been investigated primarily for the treatment of transfusion-dependent beta-thalassemia and sickle cell disease. Hydroxyurea acts as an antimetabolite that increases fetal hemoglobin (HbF) production, reduces inflammation, and decreases hypercoagulability. Thalidomide is an immunomodulatory and anti-angiogenic agent that also induces γ-globin gene expression, leading to increased erythroid cell proliferation and elevated hemoglobin levels. The combination aims to reduce transfusion requirements by synergistically increasing hemoglobin levels in patients who are partial or non-responders to monotherapy with either drug[1][2][3][4].
- Other names
- thalidomide and hydroxyurea combination therapy
02
Targets
TNFA (Tumor necrosis factor alpha (soluble))CRBN (Cereblon)RK (Ribokinase)RNR (Ribonucleotide reductase)HBG1 (Hemoglobin subunit gamma-1)
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