Sickle cell anemia is a severe form of an inherited blood disorder known as sickle cell disease. Someone with this condition produces abnormal red blood cells, often sickle- or C- shaped, that can block small blood vessels, preventing parts of the body from receiving healthy blood flow, which can cause severe pain and damage. Although sickle cell disease is not a cancer, its treatment can be similar to therapies used for leukemia and lymphoma.

Some people with sickle cell disease have only mild symptoms. Others have severe symptoms and are often hospitalized for such complications as stroke, infections, and severe pain. It is important for patients with sickle cell to seek treatment from a care team with experience treating the disease.

Physicians at Johns Hopkins are leading experts in the research and treatment of sickle cell disease and offer the most advanced treatments and care.

Treatment Options

Two treatments can potentially cure sickle cell disease: bone marrow transplantation and gene therapy. Not all patients are eligible for those therapies.

Treatments that can relieve the symptoms and complications of sickle cell include blood transfusions and a drug called hydroxyurea, as well as antibiotics to fight infection and medications to relieve pain during flare-ups of the disease.

Any treatment can have side effects. It’s important to talk to your care team about how you’re feeling so they can help manage these effects.

Bone Marrow Transplant

Physician scientists at Johns Hopkins have developed a bone marrow transplant procedure that has been very successful in treating sickle cell disease. It involves giving a patient healthy stem cells -- which are immature cells found in the fatty tissue inside of bones that grow into blood cells -- provided by a donor. Normally, that donor would have to be a match with the patient, meaning they have the same kind of proteins known as human leukocyte antigen. The procedure pioneered by Johns Hopkins, however, requires just a half-match, meaning that there are far more people are eligible to be the donors.

The bone marrow transplantation procedure for sickle cell disease is also different in that patients are given just enough chemotherapy to prevent the immune system from rejecting the donated bone marrow, rather than destroying the immune system. Three days after the transplant, the patient is given a high dose of a drug that “reboots” the immune system.

Gene Therapy

Gene therapy was approved for the treatment of sickle cell disease in 2023. Although it is new, it has shown much promise. In testing of the treatment, sickle cell patients who had been hospitalized with severe pain due to blood vessel blockage two or more times in the previous two years were nearly all free of such symptoms for the nine months after they received gene therapy. Reports from the testing said the patients’ quality of life improved in every way: physically, emotionally, socially and functionally.

The gene therapy process takes about a year and is similar to a bone marrow transplantation in which no donor is needed. Stem cells are collected from the patient and treated in a lab while the patient undergoes chemotherapy to remove abnormal cells from their bone marrow. Then the treated stem cells are injected back into the patient’s body.

Blood Transfusion

Transfusions are often used to help control the complications of sickle cell disease, such as anemia (lack of healthy red blood cells, marked by fatigue) and stroke. They also help prevent pain crises. Because blood transfusions can cause iron in the blood to hit levels that are toxic to the body’s organs, patients who undergo transfusions are also often given drugs that link to the iron so it can be excreted from the body.

Hydroxyurea

Hydroxyurea is used to prevent pain crisis episodes and reduce the need for blood transfusions in patients with sickle cell disease. It works by making red blood cells rounder and more flexible, and less likely to form a sickle shape and block blood vessels.