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Treatment for Sickle Cell Disease.*

Bone marrow transplant offers the only potential cure for sickle cell anemia. But very few people have a suitable donor for transplant. As a result, treatment for sickle cell anemia is usually aimed at avoiding crises, relieving symptoms and preventing complications. If you have sickle cell anemia, you'll need to make regular visits to your doctor to check your red blood cell count and monitor your health. You may also require treatment from specialists at a hospital or sickle cell anemia clinic. Treatments may include medications to reduce pain and prevent complications, blood transfusions and supplemental oxygen, as well as bone marrow transplant.

Medications
Medications used to treat sickle cell anemia include:

Antibiotics. Children with sickle cell anemia usually begin taking the antibiotic penicillin when they're about 2 months of age and continue until they're 5 years old. Doing so helps prevent infections, such as pneumonia, which can be life-threatening to an infant or child with sickle cell anemia. Antibiotics may also help adults with sickle cell anemia fight certain infections.
Pain-relieving medications. To relieve pain during a sickle crisis, your doctor may advise over-the-counter pain relievers and application of heat to the affected area. You may also need stronger prescription painkillers.
Hydroxyurea (Droxia, Hydrea). This prescription drug, normally used to treat cancer, may be helpful for adults with severe disease. When taken daily, it reduces the frequency of painful crises and may reduce the need for blood transfusions. It seems to work by stimulating production of fetal hemoglobin — a type of hemoglobin found in newborns that helps prevent the formation of sickle cells. There is some concern about the possibility that long-term use of this drug may cause tumors or leukemia in certain people. Your doctor can help you determine if this drug may be beneficial for you.

Blood transfusions
In a red blood cell transfusion, red blood cells are removed from a supply of donated blood. These donated cells are then given intravenously to a person with sickle cell anemia.
Blood transfusions increase the number of normal red blood cells in circulation, helping to relieve anemia. In children with sickle cell anemia at high risk of stroke, regular blood transfusions can decrease their risk of stroke.
Blood transfusions carry some risk. Blood contains iron. Regular blood transfusions cause an excess amount of iron to build up in your body. Because excess iron can damage your heart, liver and other organs, people who undergo regular transfusions must often receive treatment to reduce iron levels. In 2005, the Food and Drug Administration approved deferasirox (Exjade), the first oral medication that can reduce excess iron levels, for use in people older than the age of 2.

Supplemental oxygen
Breathing supplemental oxygen through a breathing mask adds oxygen to your blood and helps you breathe easier. It may be helpful if you have acute chest syndrome or a sickle cell crisis.

Bone marrow transplant
This procedure allows people with sickle cell anemia to replace their bone marrow — and its sickle-shaped red blood cells — with healthy bone marrow from a donor who doesn't have the disease. It can be a cure, but the procedure is risky, and it's difficult to find suitable donors. Researchers are still studying bone marrow transplants for people with sickle cell anemia. Currently, the procedure is recommended only for people who have significant symptoms and problems from sickle cell anemia.
In a bone marrow transplant, your bone marrow is first destroyed using chemotherapy or radiation. A matched donor's healthy bone marrow is removed using a minor surgical technique. You then receive a transfusion of that healthy marrow, and the healthy marrow may start producing normal blood cells. Sometimes, however, the transplant doesn't work or the recipient's body rejects the new marrow.
The procedure requires a lengthy hospital stay. After the transplant, you'll need drugs to help prevent rejection of the donated marrow.

Treating complications
Doctors treat most complications of sickle cell anemia as they occur. Treatment may include antibiotics, blood transfusions, pain-relieving medicines, other medications and possibly surgery, such as to correct vision problems or to remove a damaged spleen.

Experimental treatments
Scientists continue to gain new insights into the symptoms and causes of sickle cell anemia. Some possible new treatments researchers are studying include:

Gene therapy
Because sickle cell anemia is caused by a defective gene, researchers are exploring whether correcting this gene and inserting it into the bone marrow of people with sickle cell anemia will result in the production of normal hemoglobin. Scientists are also exploring the possibility of turning off the defective gene while reactivating another gene responsible for the production of fetal hemoglobin — a type of hemoglobin found in newborns that prevents sickle cells from forming.

Butyric acid
Some studies show that this commonly used food additive may increase the amount of fetal hemoglobin in the blood.
Clotrimazole. Normally used to treat fungal infections, this over-the-counter medication helps prevent a loss of water from red blood cells, which may reduce the number of sickle cells that form.

Nitric oxide People with sickle cell anemia have low levels of nitric oxide, a gas that helps keep blood vessels open and reduces the stickiness of red blood cells. Treatment with nitric oxide may prevent sickle cells from forming.

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