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Sickle Cell Disease 101

In Houston alone, 50 babies are diagnosed with sickle cell disease every year and 800 to 1,000 are followed by Texas Children’s Hospital and Baylor College of Medicine physicians. More public awareness and education about sickle cell disease is critically important. Although many are aware of the disease, the causes, effects and treatment aren’t widely understood.

Sickle cell disease is an inherited, genetic blood disease that occurs when the shape of the blood cells change as a result of mutation in the hemoglobin gene and disrupts normal blood flow. When the cells are misshaped, they cannot flow as easily as the round, normal cells and get clogged in the blood vessels. The abnormal blood flow can result in a number of serious issues, including pain, pneumonia, organ damage, strokes and life-threatening infections when the spleen gets clogged. The disease primarily affects people of African-American, Middle Eastern or Indian descent where malaria is, or was, common.

Because sickle cell disease is an inherited, genetic disease, it cannot be prevented. Our main goal is to prevent complications from the disease. All babies in the United States are screened for sickle cell at birth. If they are identified as having the disease, they are referred to pediatric sickle cell centers, where they are given yearly care and treatment and parents are educated about complications and management. In the last 100 years we have learned a lot about the genetics, biochemistry and molecular biology of sickle cell disease but have only recently learned how to treat it properly.

Historically, treatment has focused on pain relief and mitigating complications and included blood transfusions and, in some severe cases, bone marrow transplants. However, in the last 20 years, a new approach to treatment has given promise to researchers like me.

Hydroxyurea is a new treatment, approved by the U.S. Food and Drug Administration for adults with sickle cell and is increasingly prescribed to children. It reduces the severity of sickle cell disease by stimulating production of a form of hemoglobin present in the fetus and small infants that has the ability to block the sickling action of red blood cells. Approximately 98% of sickle cell disease patients in the United States will live into adulthood, but many do not lead healthy lives. Recurrent bouts of pain, frequent hospitalizations, and poor quality of life are common in sickle cell disease, which is why prevention of complications using hydroxyurea is now an attractive option. It is critical that we continue to research this disease and learn how to treat and manage it.