Last reviewed 2026-04-20 · How we verify

HYDROXYUREA

At a glance

Approved indications

• Resistant chronic myeloid leukemia — 1L
• Locally advanced squamous cell carcinomas of the head and neck (excluding lip) in combination with concurrent chemoradiation — 1L

Pipeline indications

• Polycythemia vera — Phase 3
• Essential thrombocythemia — Phase 3
• Sickle cell disease — Phase 3

Common side effects

• Anemia
• Leukopenia
• Thrombocytopenia
• Nausea
• Vomiting
• Diarrhea
• Constipation
• Stomatitis
• Anorexia
• Fever
• Chills
• Malaise

Drug interactions

• Antiretroviral drugs
• Uric acid, urea, or lactic acid assays
• Continuous glucose monitoring systems

Key clinical trials

• Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007) (PHASE3)
• A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (PHASE3)
• Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases (PHASE1, PHASE2)
• Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera (PHASE3)
• Genetically Engineered Cells (CD83 CAR T Cells) for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (PHASE1)
• Comparing the Radiopharmaceutical Drug, [177Lu]Lu-DOTATATE, to Standard of Care Treatment for Patients With Meningioma That Has Come Back After Prior Treatment (PHASE2)
• Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire (PHASE4)
• A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006) (PHASE3)

Patents

Primary sources

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