Hydroxycarbamide

At Hydroxycarbamide it is a cytostatic agent. It is used in the treatment of malignant blood diseases such as leukemia. It is also used in anti-retroviral treatment for HIV infections.

What is hydroxycarbamide?

Hydroxycarbamide is one of the cytostatic drugs. It is mainly used in chronic myeloid leukemia (CML). It is also occasionally used in the treatment of sickle cell anemia (the formation of abnormal hemoglobin) and in anti-retroviral treatment in HIV infection.

Hydroxycarbamide is commercially available in the form of capsules. It is a hydroxylated urea, which is present as a white and crystalline, hydroscopic powder and is soluble in water. Hydroxycarbamide is also known as Hydroxyurea or Hydroxyurea.

Pharmacological effect on the body and organs

The exact mode of action of hydroxycarbamide has not yet been fully clarified. As a cytostatic, the active ingredient itself inhibits the growth, division and reproduction of cells. He intervenes in the DNA synthesis. Hydroxycarbamide is supposed to disrupt the structure of individual nucleotides. The active ingredient seems to block the enzyme that is responsible for converting ribose into deoxyribose. In addition, hydroxycarbamide could help prevent the incorporation of thymine nucleotides into the DNA chain.

The effect in the treatment of sickle cell anemia is also not yet understood. There is likely to be an increase in the concentration of hemoglobin here, as in an unborn child. This hampers the formation of fibers in the abnormal hemoglobin and thus the curvature of the red blood cells. There are no clumps because the blood remains more fluid overall.

Medical application & use for treatment & prevention

Hydroxycarbamide is used in the treatment of chronic myeloid leukemia (CML for short, characterized by the strong increase in white blood cells and granulocytes), in essential thrombocythemia (strong increase in platelets in the blood), in polycythemia vera (increase in all three blood cell rows in the blood), sickle cell anemia and thalassemia major (insufficient production of normal HbA1). It is also rarely used in antiretroviral therapy for HIV infections.

Hydroxycarbamide must always be taken according to the doctor's instructions. When treating CML, the starting dose for adults is 40 mg / kg body weight per day. Depending on the number of white blood cells, the dose is then individually adjusted.

For the treatment of polycythemia vera, the starting daily dose is 15 to 20 mg / kg body weight. Here, too, there is an individual adjustment, always depending on the number of blood cells. The dose for essential thrombocythemia is 15 mg / kg body weight daily and is individually adapted depending on the number of blood cells. The effect may be stronger in elderly patients, so the dosage is usually lower.

Use of hydroxycarbamide is not indicated in the event of hypersensitivity to the active ingredient, severe blood formation disorders in the bone marrow, a lack of blood platelets and white blood cells, or anemia.

The physician must carefully weigh the benefits and risks of using hydroxycarbamide against each other after previous similar therapy, with impaired liver or kidney function and with simultaneous treatment with cytostatics from the subgroup of antimetabolites.

Hydroxycarbamide must also not be used during pregnancy and breastfeeding. The active ingredient can be used to treat children, but it is rare, as most of these diseases do not occur in children.

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Risks & side effects

Hydroxycarbamide can cause different side effects. They can occur very rarely to frequently, but do not have to occur. Common side effects include disturbances in the formation of the bone marrow, a lack of white blood cells, the formation of megaloblasts, and constipation or diarrhea.

Occasionally, when taking hydroxycarbamide, nausea and vomiting, malaise, chills, anemia, a lack of blood platelets, reddening of the skin on the legs and arms, reddening of the face or a blotchy rash may occur.

Elevations in blood urea levels, liver enzyme levels, blood bilirubin levels, blood uric acid levels and blood creatinine levels are also not unusual.

In rare cases, headaches, hair loss, dizziness, fever, shortness of breath, confusion, delusions, urinary retention, water retention in the lungs and allergic air sacs. Impaired kidney function is one of the very rare side effects.

If hydroxycarbamide is taken in combination with antiviral agents, liver damage or inflammation of the pancreas can occur.

With accompanying or preceding concomitant cytostatic therapy or radiation therapy, certain side effects (e.g. functional disorders of the bone marrow, gastrointestinal complaints, skin reddening) may be aggravated.