Knowra Hydroxycarbamide Hydroxycarbamide Hydroxycarbamide, also called hydroxyurea, is a medication that inhibits ribonucleotide reductase and slows DNA synthesis. It is used to treat sickle cell disease and several cancers.
Ribonucleotide reductase : An enzyme that converts ribonucleotides into deoxyribonucleotides, supplying DNA synthesis. Hydroxycarbamide inhibits this enzyme, depleting the building blocks needed to copy DNA.
Sickle cell disease : A group of inherited disorders in which abnormal hemoglobin causes red blood cells to sickle and damage tissues. Hydroxycarbamide reduces painful crises and other complications for many people with this disorder.
L-glutamine : A prescription amino-acid therapy used to reduce acute complications of sickle cell disease. It offers a different sickle cell treatment strategy without hydroxycarbamide's DNA-synthesis inhibition.
Complete blood count : A blood test measuring red cells, white cells, platelets, hemoglobin, and related indices. Regular counts detect the bone-marrow suppression that can require hydroxycarbamide dose adjustment or interruption.
Fetal hemoglobin : The hemoglobin produced mainly before birth, composed of two alpha and two gamma globin chains. Hydroxycarbamide raises fetal hemoglobin, which reduces sickling in many people with sickle cell disease.
Polycythemia vera : A chronic blood cancer in which the bone marrow produces too many red blood cells. Hydroxycarbamide can lower blood-cell counts and reduce clotting risk in selected patients.
Voxelotor : A hemoglobin-binding drug developed to reduce sickling by stabilizing oxygenated hemoglobin. It targeted hemoglobin directly rather than increasing fetal hemoglobin, though it was withdrawn from markets in 2024.
Myelosuppression : Reduced bone-marrow production of blood cells, causing low counts in one or more cell lineages. It is a dose-limiting effect of hydroxycarbamide and requires ongoing blood-count monitoring.
Nitric oxide : A small signaling molecule that relaxes blood vessels and participates in diverse cellular processes. Hydroxycarbamide may promote nitric oxide signaling, another proposed contributor to its effects in sickle cell disease.
Essential thrombocythemia : A chronic myeloproliferative neoplasm marked by excessive platelet production. Hydroxycarbamide is used to reduce platelet counts and prevent vascular complications in some patients.
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