Sickle-cell disease

group of genetic blood disorders

Hydroxycarbamide, also known as hydroxyurea, is an antimetabolite medication used in sickle-cell disease, essential thrombocythemia, chronic myelogenous leukemia, polycythemia vera, and cervical cancer. In sickle-cells disease it increases fetal hemoglobin and decreases the number of attacks. It is taken by mouth.

Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. Voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients. It initially appeared to have an acceptable safety profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity noted in clinical trials. It was developed by Global Blood Therapeutics, a subsidiary of Pfizer.

Crizanlizumab, sold under the brand name Adakveo among others, is a monoclonal antibody medication that binds to P-selectin. It is a medication used to reduce the frequency of vaso-occlusive crisis in people aged 16 years and older who have sickle cell anemia. It is given by injection into a vein.

experimental gene therapy for sickle cell disease and transfusion-dependent beta thalassemia