In sickle cell anemia, a mutation causes the normally “donut-shaped” red blood cells that transport oxygen throughout the body to be deformed into a rigid sickle-like shape. As a result, the cells can become trapped in narrow blood vessels, restricting blood flow and preventing oxygen from efficiently reaching all parts of the body.

Among other symptoms, this causes inflammation, damage, and pain. Because the defective red blood cells are recycled quickly by the body, the condition can also be associated with abnormally low red blood cell counts.

Sickle cell anemia may be cured in some individuals through stem cell transplants, and there are also several therapies approved to help manage the condition and its related complications.

Stem cell transplant

A stem cell transplant is currently the only therapy approved by the U.S Food and Drug Administration that may be able to reverse sickle cell anemia in some individuals.

The stem cells in the bone marrow are the source of all new blood cells in the body. A stem cell transplant is intended to replace the stem cells in a patient’s bone marrow with those obtained from a healthy donor. This should result in the production of normal red blood cells.

A stem cell transplant requires finding a suitable donor for the patient, which can be difficult and may not be possible for some patients. The procedure itself can also be very risky, involving chemotherapy and potential fatality if the cells are rejected by the patient’s body.

Generally, the procedure is best carried out at a younger age if possible as this may reduce the risk of complications.


Endari (L-glutamine), developed by Emmaus Life Sciences, is a therapy that acts to make red blood cells more flexible, reducing their risk of becoming trapped inside blood vessels. This improves blood flow and ultimately increases the amount of oxygen reaching tissues.

Endari was approved by the FDA in July 2017 for the treatment of sickle cell anemia.


Hydroxyurea was the first treatment approved by the FDA in 1997 for the treatment of sickle cell anemia.

The mutation that causes sickle cell anemia is in the HBB gene, which provides the instructions to make adult hemoglobin, the protein inside the red blood cells to which oxygen binds.

Hydroxyurea acts to promote the production of another type of hemoglobin called fetal hemoglobin, which is normally only produced during development. This type of hemoglobin is very effective in oxygen transport. Hydroxyurea may, therefore, help manage pain and other complications associated with the condition.

Treatments to manage pain

Pain is a common symptom of sickle cell anemia. It may be managed through various treatments, depending on severity.

Common pain medications include nonsteroidal anti-inflammatory drugs (NSAIDs) such as ibuprofen, diclofenac, or acetaminophen. If the pain continues, it can be treated with opioid narcotics, such as codeine, or stronger opioids such as morphine.

Treatments to manage infections

Preventing infections, or treating them quickly if they occur, is essential in sickle cell anemia patients. Children with the disease can benefit from receiving daily doses of penicillin from as early as 2 months old and should be kept up to date with any routine vaccinations.

If infections occur, they should be quickly treated with the appropriate antibiotic medications.

Other treatments

A transfusion of red blood cells from a healthy blood donor can temporarily increase levels of healthy red blood cells in the patient’s body. This can help relieve the symptoms of sickle cell anemia, but may also be used as a preventive measure for complications such as stroke in high-risk patients.


Sickle Cell Anemia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.