Hydroxyurea is a medication primarily used in the treatment of various blood disorders, including sickle cell disease, myeloproliferative neoplasms, and certain types of cancer. It belongs to the class of chemotherapy drugs known as antimetabolites, which work by interfering with the synthesis of DNA and RNA in rapidly dividing cells, ultimately leading to their destruction. In sickle cell disease, hydroxyurea helps increase the production of fetal hemoglobin, which can improve symptoms and reduce the frequency of painful vaso-occlusive crises.

One of the key benefits of hydroxyurea is its ability to reduce the frequency and severity of symptoms in individuals with sickle cell disease, including pain crises, acute chest syndrome, and the need for blood transfusions. By increasing the levels of fetal hemoglobin, hydroxyurea helps prevent the polymerization of sickle hemoglobin and the subsequent formation of sickle-shaped red blood cells, leading to improved oxygen delivery and reduced organ damage. Additionally, hydroxyurea has been shown to slow down the progression of certain types of cancer, such as chronic myeloid leukemia and essential thrombocythemia, by inhibiting the growth of malignant cells and promoting apoptosis.