Hydroxyurea and blood transfusions have been the mainstays in the prevention of anemia and vaso-occlusive crises in sickle cell disease. Vaso-occlusive pain crises and anemia are hallmark complications associated with sickle cell disease. Polymerization also reduces the life span of HgS and therefore leads to hemolysis and anemia in sickle cell patients. The occlusion of small blood vessels can lead to tissue ischemia which may present as vaso-occlusive pain crisis, acute chest syndrome, stroke, or organ failure. 4 As a result, a more rigid, adhesive HgS is produced that sticks to other blood cells and endothelium and in turn occludes small blood vessels. Upon deoxygenation of hemoglobin, HgS undergoes polymerization, which damages the blood cell. Polymerization is a key pathway in the pathogenesis of sickle cell disease ( FIGURE 1). 3 Other common disorders include sickle–hemoglobin C disease and sickle beta thalassemia, both of which are caused when a patient inherits one HgS allele and another abnormal beta-globin chain mutation.

Sickle cell anemia, HgSS, is the most common disorder it occurs when a patient inherits both HgS alleles and accounts for 60% to 70% of patients with sickle cell disease. Sickle cell disease encompasses a group of disorders in which at least one HgS allele is present. Sickle cell disease is caused by a mutation in which glutamine is replaced with valine at the sixth position of the beta globin gene (HBB) and is characterized by the production of sickled hemoglobin (HgS). 2 Sickle cell disease also has a global impact, with a high prevalence among people of sub-Saharan Africa, South America, the Caribbean, Central America, Saudi Arabia, India, and the Mediterranean. 1 Sickle cell disease occurs predominantly in African American and Hispanic American patients one in every 365 African American and one in every 16,300 Hispanic American children are born with sickle cell disease. Sickle cell disease is the most common genetic disease in the United States and affects approximately 100,000 Americans. Sickle cell disease is a group of inherited red blood cell disorders in which patients inherit two defective hemoglobin genes. Current treatment options and these novel agents can be used to successfully manage these two major complications of sickle cell disease. Recently, three novel agents were approved to reduce the incidence of vaso-occlusive pain crises and/or anemia in sickle cell patients. Despite current therapies, sickle cell patients still experience frequent anemia and vaso-occlusive crises yearly. Hydroxyurea and blood transfusions have been the gold standard of therapy for the management of sickle cell disease. As a result, patients are at an increased risk for complications associated with anemia and vaso-occlusion. 2020:45 (7)(Specialty& Oncology suppl):8-12.ĪBSTRACT: Sickle cell disease is a group of inherited blood disorders in which patients are born with sickled hemoglobin.

Ohapi principles with sickle cell