Sickle Cell Disease What Is Sickle Cell Disease?
In September 2024, Pfizer withdrew voxelotor (also called Oxbryta) from the market. The medicine was used to prevent sickling in red blood cells. Pfizer said the benefits of using voxelotor do not outweigh the risks in the approved sickle cell population.
Sickle cell disease — also called sickle cell anemia — is a group of disorders that affect , the major protein that carries oxygen in red blood cells. Normally, red blood cells are disc-shaped and flexible so they can move easily through the blood vessels. In sickle cell disease, red blood cells are misshaped, typically crescent- or “sickle”-shaped due to a gene that affects the hemoglobin molecule. When red blood cells sickle, they do not bend or move easily and can block blood flow to the rest of the body.
Serious problems may come from having sickle cell disease, including sudden onset of strong episodes of pain, called pain crises, that occur because of blocked blood flow and often require medical attention Other terms used to describe these pain episodes include “sickle cell crisis” and “vaso-occlusive crisis.” People who have sickle cell disease may also experience other serious health complications, such as chronic pain, stroke, lung problems, eye problems, infections, and kidney disease.
Sickle cell disease is a lifelong illness that may be managed through preventive screening and treatment strategies. Treatment reduces or helps manage symptoms and increase longevity. With the right care, many people who have sickle cell disease live fulfilling lives and safely participate in most activities.
Sickle cell disease affects more than 100,000 people in the United States and 8 million people worldwide. In the United States, 9 of 10 people who have sickle cell disease are of African ancestry or identify as Black:
- About 1 in 13 Black babies are born with sickle cell trait, meaning that they inherited a sickle cell gene from one parent.
- About 1 in every 365 Black babies are born with sickle cell disease, meaning they inherited a sickle cell gene from each parent.
The NHLBI leads and supports research and clinical trials to find more transformative therapies and potential cures for sickle cell disease.
Sickle Cell Disease: Milestones in Research and Clinical Progress Booklet
Learn about the history of sickle cell disease in the United States, from its discovery in 1910 to the NHLBI legacy of research that has advanced the understanding of sickle cell disease, improved clinical progress, and paved the way for widely available cures.