hydroxycarbamide

Action and Spectrum

Actions and spectrum: 

hydroxycarbamide, also known as hydroxyurea, is a medication with a variety of actions and a broad spectrum of uses. It primarily acts as an antimetabolite and an antineoplastic agent, meaning it interferes with the growth and proliferation of cells. It is used in the treatment of various conditions, including: 

• Cancer: hydroxycarbamide is used to treat certain types of cancer, particularly leukemia, melanoma, and some types of solid tumors. It works by inhibiting growth of cancer cells. 
• Sickle Cell Disease: It is also used in the management of sickle cell disease to reduce the frequency of painful vaso-occlusive crises and other complications associated with the disease. 
• Myeloproliferative Disorders: hydroxycarbamide is used in myeloproliferative disorders like polycythemia vera and essential thrombocythemia to reduce the excessive production of blood cells. 
• Psoriasis: In some cases, hydroxycarbamide can be used to treat psoriasis that does not respond to other treatments. 

Drug Interaction

Adverse Reaction

Frequency not defined 

Bone marrow suppression 

erythrocyte abnormalities 

secondary neoplasms 

Peripheral neuropathy 

headache 

disorientation 

Asthenia 

seizure 

acute delirium 

dizziness 

malaise 

hallucination 

Peripheral oedema 

Diarrhoea 

Nausea 

anorexia 

GI irritation 

Stomatitis 

gastric distress 

oral mucosa ulcer 

constipation 

Mucositis 

Vomiting 

Dyspnoea 

asthma 

pulmonary fibrosis 

Elevated liver enzyme 

Dysuria 

renal tubular disease 

Hyperuricaemia 

Maculopapular rash 

Panniculitis 

Pruritus 

Eczema 

Severe myelosuppression 

Black Box Warning

Black Box Warning: 

hydroxycarbamide may cause severe myelosuppression, including anemia, neutropenia, and thrombocytopenia. This medication should be used with caution, and the potential benefits should outweigh the risks, especially in patients with preexisting hematologic conditions. 

Contraindication / Caution

Contraindication/Caution: 

Contraindication: 

• Hypersensitivity: Patients who have a known hypersensitivity or allergy to hydroxycarbamide or any of its components should not use this medication. 
• Severe Bone Marrow Suppression: hydroxycarbamide can cause myelosuppression, including anemia, neutropenia, and thrombocytopenia. It should not be used in individuals who already have severe bone marrow suppression. 
• Pregnancy and Breastfeeding: hydroxycarbamide may cause harm to a developing fetus. It is contraindicated during pregnancy unless the potential benefits outweigh the risks.  
• Children and Adolescents: hydroxycarbamide’s safety and efficacy in children and adolescents under certain ages may not be established, and its use in these populations may be contraindicated or require careful monitoring. 
• Live Vaccines: Patients on hydroxycarbamide treatment should avoid live vaccines due to the potential for decreased immune response. 

Caution: 

• Renal Impairment: hydroxycarbamide is primarily excreted by the kidneys. In individuals with renal impairment, dose adjustments may be necessary to prevent excessive drug accumulation and potential toxicity. Close monitoring of kidney function is advisable. 
• Hepatic Impairment: Patients may require dose adjustments, as the liver plays a role in metabolizing hydroxycarbamide. Liver function should be monitored regularly. 
• Bone Marrow Suppression: hydroxycarbamide can cause bone marrow suppression, leading to anemia, neutropenia, and thrombocytopenia. Patients should have regular blood tests to monitor their blood cell counts. Dose adjustments or discontinuation may be necessary if significant bone marrow suppression occurs. 
• Infections: hydroxycarbamide can suppress the immune system. Patients should be cautious about exposure to infections and should promptly report any signs of infection to their healthcare provider. 
• Pediatric Use: hydroxycarbamide’s safety and efficacy in pediatric patients may vary depending on the specific condition being treated. Caution should be used when prescribing this medication to children and adolescents, and careful monitoring is essential. 
• Elderly Patients: they are more susceptible to the side effects of hydroxycarbamide, including bone marrow suppression. Lower starting doses and more frequent monitoring may be needed.

Comorbidities: 

• Sickle Cell Disease: hydroxycarbamide is commonly used to treat sickle cell disease (SCD) and reduce the frequency and severity of painful crises. It has been shown to be effective in adults and children with SCD. 
• Polycythemia Vera: hydroxycarbamide is used to manage polycythemia vera, a condition characterized by the overproduction of red blood cells. It helps reduce the number of red blood cells and lower the risk of complications like blood clots. 
• Myeloproliferative Disorders: hydroxycarbamide may be used in various myeloproliferative disorders, such as essential thrombocythemia and chronic myeloid leukemia, to reduce high platelet counts and control the progression of the disease. 
• Thalassemia: In some cases of thalassemia, hydroxycarbamide may be considered to increase fetal hemoglobin levels, which can improve the symptoms of the condition. 
• HIV Infection: hydroxycarbamide has been investigated as an adjuvant therapy in HIV-infected individuals to increase fetal hemoglobin levels, which may inhibit HIV replication. However, its use in this context is still being studied. 
• Psoriasis: hydroxycarbamide may be used topically to treat psoriasis, a chronic skin condition characterized by scaly, itchy skin patches. 

Pregnancy / Lactation

Pregnancy consideration: Pregnancy Category: D 

Lactation: excreted into human milk: unknown  

Pregnancy category: 

• Category A: well-controlled and Satisfactory studies show no risk to the fetus in the first or later trimester. 
• Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women. 
• Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.   
• Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits. 
• Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women. 
• Category N: There is no data available for the drug under this category. 

Pharmacology

Pharmacology: 

hydroxycarbamide, also known as hydroxyurea, is a medication with diverse pharmacological actions. Its primary mechanism of action involves inhibiting the enzyme ribonucleotide reductase, which leads to a reduction in the production of deoxyribonucleotides, essential building blocks for DNA.

This inhibition interferes with DNA synthesis and repair. In clinical practice, hydroxycarbamide is used to treat various conditions, including sickle cell disease, polycythemia vera, and certain types of cancer. It helps by reducing the production of abnormal blood cells and increasing the production of fetal hemoglobin, which can alleviate symptoms and improve outcomes in these disorders. hydroxycarbamide’s pharmacology makes it a valuable tool in managing these conditions.  

Pharmacodynamics: 

• Inhibition of DNA Synthesis: hydroxycarbamide inhibits the enzyme ribonucleotide reductase, which reduces the conversion of ribonucleotides to deoxyribonucleotides. This leads to a decreased availability of deoxyribonucleotides, essential for DNA synthesis, and repair. As a result, the drug interferes with the replication and repair of DNA. 
• Stimulation of Fetal Hemoglobin: hydroxycarbamide can stimulate the production of fetal hemoglobin (HbF) in individuals with sickle cell disease and thalassemia. Increased levels of HbF can help mitigate the symptoms and complications associated with these conditions. 
• Reduction in Abnormal Blood Cells: In conditions like polycythemia vera and essential thrombocythemia, hydroxycarbamide reduces the production of abnormal blood cells, such as excessive red blood cells and platelets. This helps lower blood viscosity and the risk of clot formation. 
• Antineoplastic Effects: hydroxycarbamide is also used as a chemotherapy agent in the treatment of various cancers, including some leukemias. It exerts its antineoplastic effects by disrupting DNA synthesis and interfering with the growth and replication of cancer cells. 
• Anti-Inflammatory Properties: hydroxycarbamide may have anti-inflammatory properties, which can be beneficial in certain inflammatory conditions, including myeloproliferative neoplasms.

Pharmacokinetics: 

Absorption 

• hydroxycarbamide is administered orally in the form of capsules or tablets. 
• It is absorbed from gastrointestinal tract after oral ingestion. 

Distribution 

• hydroxycarbamide is distributed throughout the body, and it readily crosses the blood-brain barrier. 
• It can distribute into the intracellular compartment, where it exerts its effects on DNA synthesis. 

Metabolism 

• hydroxycarbamide does not undergo significant hepatic metabolism. 
• The drug is primarily converted to its active form, which inhibits ribonucleotide reductase and affects DNA synthesis. 

Elimination and excretion 

• hydroxycarbamide and its metabolites are primarily excreted through the kidneys. 
• It can be excreted in the urine within a few hours of administration. 

Adminstartion

Administration: 

• Dosage: The dosage of hydroxycarbamide is determined by the prescribing healthcare provider based on the patient’s medical condition, body weight, and other factors. It is important to follow the prescribed dose exactly as directed. 
• Frequency: hydroxycarbamide is usually taken once daily. Again, the frequency may vary based on individual patient needs, so follow your healthcare provider’s instructions. 
• Consistency: Try to take hydroxycarbamide at the same time each day to maintain a consistent level of the medication in your bloodstream. 
• Duration of Treatment: The duration of hydroxycarbamide treatment will be determined based on condition. It may be a short-term or long-term treatment, so continue taking it as prescribed unless instructed otherwise. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: hydroxycarbamide 

Pronounced: (hye-drok-see-KAR-buh-mide)  

Why do we use hydroxycarbamide? 

• Sickle Cell Disease (SCD): hydroxycarbamide is commonly used to treat sickle cell disease. It helps to reduce frequency and severity of painful crises and other complications associated with SCD. 
• Myeloproliferative Disorders: hydroxycarbamide may be used in the management of myeloproliferative disorders, such as essential thrombocythemia and polycythemia vera. It helps reduce the overproduction of blood cells in these conditions. 
• Psoriasis: In some cases, hydroxycarbamide may be prescribed to treat severe psoriasis, a chronic skin condition. 
• AIDS-Related Kaposi’s Sarcoma: hydroxycarbamide may be used in the treatment of AIDS-related Kaposi’s sarcoma, a type of cancer that can develop in individuals with HIV/AIDS.