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ADDITION OF THALIDOMIDE AND HYDROXYUREA
Thalidomide and hydroxyurea work synergistically to inhibit angiogenesis, promote apoptosis of abnormal cells, and enhance fetal hemoglobin production while reducing sickling and hemolysis.
Thalidomide and hydroxyurea work synergistically to inhibit angiogenesis, promote apoptosis of abnormal cells, and enhance fetal hemoglobin production while reducing sickling and hemolysis. Used for Sickle cell disease (Phase 2), Multiple myeloma or other hematologic malignancies (investigational).
At a glance
| Generic name | ADDITION OF THALIDOMIDE AND HYDROXYUREA |
|---|---|
| Also known as | Hydrea and Thalidomide |
| Sponsor | Pakistan Blood and Marrow Transplant (PBMT) Group |
| Drug class | Immunomodulatory agent + ribonucleotide reductase inhibitor combination |
| Target | Cereblon (thalidomide); ribonucleotide reductase (hydroxyurea) |
| Modality | Small molecule |
| Therapeutic area | Oncology / Hematology |
| Phase | Phase 2 |
Mechanism of action
Thalidomide is an immunomodulatory agent that inhibits tumor necrosis factor-alpha production and angiogenesis while promoting T-cell proliferation and cereblon binding. Hydroxyurea increases fetal hemoglobin (HbF) levels and reduces sickling through ribonucleotide reductase inhibition. In combination, these agents target multiple pathways relevant to hematologic malignancies and severe hemolytic anemias.
Approved indications
- Sickle cell disease (Phase 2)
- Multiple myeloma or other hematologic malignancies (investigational)
Common side effects
- Peripheral neuropathy
- Thromboembolism
- Myelosuppression
- Teratogenicity (thalidomide)
- Hepatotoxicity
Key clinical trials
- Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia (PHASE2)
- A Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor Treatment (PHASE3)
- Long Term Beta Thalassemia Treatment: Findings From The Extension Period (PHASE2)
- Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients (PHASE3)
- Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |