September is National Sickle Cell Awareness Month, a time to recognize the perseverance of patients living with sickle cell disease (SCD) and to recommit ourselves to improving the quality of life and health outcomes for all individuals living with SCD. Read President Joe Biden’s Proclamation on National Sickle Cell Awareness Month 2022 to learn how federal agencies are working to put an end to SCD.
Sickle cell disease is a genetic condition that affects the body’s red blood cells. It occurs when a child receives two sickle cell genes—one from each parent. In someone living with this disease, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”.
When sickled red blood cells travel through small blood vessels, they get stuck and clog the blood flow. This can cause pain and other serious problems such as infection, acute chest syndrome and stroke. The pain experienced by people living with sickle cell disease can vary in intensity and last for a few hours to a few weeks.
While the disease is most common among African Americans, other racial and ethnic groups are affected, including Latinos and people of Middle Eastern, Indian, Asian and Mediterranean backgrounds. Sub-Saharan Africa has the greatest burden of disease, with more than 300,000 babies born with the disease each year. However, the disease is common enough in the United States that there are about 100,000 people currently living with sickle cell disease but uncommon enough that medical professionals rarely see sickle cell disease patients.
Sickle cell disease is most often discovered at birth during routine newborn screening tests at the hospital.
There is no single best treatment for all people with sickle cell disease. Treatment options are different for each person depending on their symptoms and many studies have shown that patients do not receive treatment for their pain as soon as, or in appropriate doses as, other patients.
Currently, the only cure for sickle cell disease is a bone marrow or stem cell transplant. However, these treatment options come with serious risks, require a close donor match (like a sibling) and are only used in severe cases.
Clinical trials related to a sickle cell disease cure that will work for all patients are being conducted as part of the National Institutes of Health’s Cure Sickle Cell Initiative. The goal of this initiative is to advance the development of new gene- and cell-based therapies for sickle cell disease within the next five to 10 years. See the NIH map of privately and publicly funded clinical studies around sickle cell disease in the U.S.
Annals of Emergency Medicine: The State of Sickle Cell Disease Care in the United States: How Can Emergency Medicine Contribute?
The Agency for Healthcare Research and Quality’s (AHRQ) new Healthcare Cost and Utilization Project (HCUP) Statistical Brief presents recent data, including historical trends and cost, on inpatient stays among patients with sickle cell disease.
There have been important strides made in caring for people living with sickle cell disease. Take a look at the Latest Advances such as the guidelines for sickle cell disease, health journals, medical reports and initiatives geared toward improving the quality of life for people living with this disease.
NHLBI Webinar
Bone Marrow Transplants, Other Therapies, and Sickle Cell Disease
September 18, 2019
The Cure Sickle Cell Initiative was created by the National Heart, Lung, and Blood Institute (NHLBI). The Initiative aims to transform the lives of individuals living with sickle cell disease by moving the most promising therapies safely into clinical trials within five to ten years.
Other goals of the NHLBI’s Cure Sickle Cell Initiative include:
The NHLBI leads or sponsors many studies on sickle cell disease, advancing our knowledge and treatment of the condition—and you can help. Learn more about enrolling in NHLBI clinical trials that are related to sickle cell disease and see if you or someone you know is eligible to participate in one of them.
The Cure Sickle Cell Initiative is a collaborative, patient-focused research effort designed to identify safe and effective ways to treat sickle cell disease at the genetic level. People living with sickle cell disease help guide the direction of research to cure the disease, as well as contribute to the education and participation of clinical trials in the patient and advocacy community. Click on the Community Stories below to learn more about the developments made in sickle cell research and the importance of community engagement to advance the cure of sickle cell disease.
