About Sickle Cell Disease
What is Sickle Cell Disease?
Sickle Cell Disease is a lifelong inherited blood condition that affects the ability of red blood cells to deliver oxygen to the body’s tissues and organs.
In people with Sickle Cell Disease, red blood cells lose their normal shape and become sickle-shaped and rigid. Sickle-shaped red blood cells get stuck in small blood vessels and block the flow of blood and oxygen to the body, which can cause severe pain and inflammation.
People born with Sickle Cell Disease inherit two abnormal sickle cell genes, one from each parent who was carrying a sickle cell trait.
What are current treatment options for Sickle Cell Disease?
Most current treatment options, including antibiotics, pain medications, and blood transfusions, focus on preventing infections, relieving pain, and controlling complications as they occur; however, they do not affect how red blood cells work. Hydroxyurea and Endari (L-glutamine) are currently the only approved medications for the treatment of Sickle Cell Disease.
A bone marrow transplant is the only cure for Sickle Cell Disease; however, this risky procedure is available for a limited number of patients and may have serious side effects.
Because of the lack of treatments that target the underlying cause of sickle cell disease, people with Sickle Cell Disease often struggle with a range of severe symptoms, including severe pain crises, anemia, fatigue, frequent infections, and inflammation that start when they are infants and last a lifetime.
Who is most affected by Sickle Cell Disease?
Sickle Cell Disease is most common in people with ancestors from certain parts of the world including parts of sub-Saharan Africa, India, and Spanish-speaking regions in the Western Hemisphere including South America, Central America, and the Caribbean. In the United States, Sickle Cell Disease occurs in approximately 1 out of every 365 African-American births and 1 out of every 16,300 Hispanic-American births. Among African-Americans, Sickle Cell Disease is the most common genetic disorder passed on from parent to child.
Although medications may affect people with varying genetic traits differently, African Americans, Hispanics, and other minorities have not historically participated in clinical research studies in numbers large enough to generate significant data on the interactions between certain medications and their genetic characteristics. It is important for members of minority groups most frequently affected by Sickle Cell Disease to participate in clinical research studies to help scientists develop better and safer treatments.