How Is Sickle Cell Anemia Treated?

Sickle cell anemia has no widely available cure. However, treatments can help relieve symptoms and treat complications. The goals of treating sickle cell anemia are to relieve pain; prevent infections, organ damage, and strokes; and control complications (if they occur).

Blood and marrow stem cell transplants may offer a cure for a small number of people who have sickle cell anemia. Researchers continue to look for new treatments for the disease.

Infants who have been diagnosed with sickle cell anemia through newborn screening are treated with antibiotics to prevent infections and receive needed vaccinations. Their parents are educated about the disease and how to manage it. These initial treatment steps have greatly improved the outcome for children who have sickle cell anemia.

Specialists Involved

People who have sickle cell anemia need regular medical care. Some doctors and clinics specialize in treating people who have the disease. Hematologists specialize in treating adults and children who have blood diseases or disorders.

Treating Pain

Medicines and Fluids

Mild pain often is treated at home with over-the-counter pain medicines, heating pads, rest, and plenty of fluids. More severe pain may need to be treated in a day clinic, emergency room, or hospital.

The usual treatments for acute (rapid-onset) pain are fluids, medicines, and oxygen therapy (if the oxygen level is low). Fluids help prevent dehydration, a condition in which your body doesn't have enough fluids. Fluids are given either by mouth or through a vein. Your doctor may prescribe antibiotics if you have an infection.

Treatment for mild-to-moderate pain usually begins with acetaminophen (Tylenol^®) or nonsteroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen.

If pain continues or becomes severe, stronger medicines called opioids might be needed. Talk with your doctor about the possible benefits and risks of taking strong pain medicine, especially if the medicine will be used for a long period.

Hydroxyurea

Severe sickle cell anemia can be treated with a medicine called hydroxyurea (hi-DROK-se-yu-RE-ah). This medicine prompts your body to make fetal hemoglobin. Fetal hemoglobin, or hemoglobin F, is the type of hemoglobin that newborns have.

In people who have sickle cell anemia, fetal hemoglobin helps prevent red blood cells from sickling and improves anemia.

Taken daily by mouth, hydroxyurea reduces how often painful sickle cell crises and acute chest syndrome occur. Many people taking hydroxyurea also need fewer blood transfusions and have fewer hospital visits.

Doctors are studying the long-term effects of hydroxyurea on people who have sickle cell anemia. Studies in very young children have shown that hydroxyurea can be given safely and that it improves anemia and hemoglobin F levels while reducing complications of sickle cell anemia. Long-term followup studies of adults treated with hydroxyurea suggest that those treated with the drug survive longer than those not treated with the drug.

Hydroxyurea can reduce the number of white blood cells in your blood, which can raise your risk for infections.

People who take hydroxyurea must have careful medical followup, including blood tests. The dose of this medicine might need to be adjusted to reduce the risk of side effects.

A doctor who has knowledge about hydroxyurea can tell you about the risks and benefits of taking this medicine.

Preventing Complications

Blood transfusions are commonly used to treat worsening anemia and sickle cell complications. A sudden worsening of anemia due to an infection or enlarged spleen is a common reason for a blood transfusion.

Some, but not all, people who have sickle cell anemia need regular blood transfusions to prevent life-threatening problems, such as stroke, spleen problems, or acute chest syndrome.

Having routine blood transfusions can cause side effects. Examples include allergic reactions and a dangerous buildup of iron in the body (which must be treated). In general, the blood supply is fairly safe from infections such as hepatitis and HIV.

For more information, go to the Health Topics Blood Transfusion article.

Infections

Infections can be a major complication of sickle cell anemia throughout life, but especially during childhood. Often, infections can be prevented or treated.

To prevent infections in babies and young children, treatments include:

• Daily doses of antibiotics. Treatment may begin as early as 2 months of age and continue until the child is at least 5 years old.
• All routine vaccinations (including a yearly flu shot), plus the pneumococcal vaccine.

If your child has sickle cell anemia and shows early signs of an infection, such as a fever, you should seek treatment right away.

Adults who have sickle cell anemia also should have flu shots every year and get vaccinated against pneumonia.

Eye Damage

Sickle cell anemia can damage the blood vessels in the eyes and the retinas. The retinas are the thin layers of tissue at the back of the eyes. Regular checkups with an eye doctor who specializes in diseases of the retina can help detect eye damage.

Strokes

Stroke prevention and treatment are now possible for children who have sickle cell anemia. Starting at age 2, children who have sickle cell anemia should have routine ultrasound scans of the head. This is called transcranial Doppler (TCD) ultrasound. These scans are used to check the speed of blood flow to the brain.

TCD scans allow doctors to find out which children are at high risk of stroke. Doctors can treat these children with routine blood transfusions to reduce the risk of stroke.

A doctor who has knowledge about blood transfusions and sickle cell disease can tell you about the benefits and risks of this treatment.

Treating Other Complications

Acute chest syndrome is a severe and life-threatening complication of sickle cell anemia. If acute (sudden) failure of the liver and kidneys also occurs, it's called acute multiple organ failure.

Treatment for these complications usually occurs in a hospital and may include oxygen therapy, blood transfusions, antibiotics, pain medicine, and balancing body fluids.

Leg ulcers (sores) due to sickle cell anemia can be very painful. Ulcers can be treated with cleansing solutions and medicated creams or ointments.

Skin grafts might be needed if the leg ulcers are ongoing. Bed rest and keeping the legs raised to reduce swelling are helpful. If you have a lot of pain from leg ulcers, your doctor may recommend a strong pain medicine.

Your doctor might recommend gallbladder surgery if the presence of gallstones leads to gallbladder disease.

Priapism (a painful erection in males) can be treated with fluids, medicines, or surgery.

Regular Health Care for Children

Children who have sickle cell anemia need routine health care (just like children who don't have the disease). They need to have their growth checked regularly. They also need to get the routine shots that all children get.

All children younger than 2 years old should see their doctors often. Children who have sickle cell anemia may need even more checkups. After age 2, children who have sickle cell anemia may not need to see their doctors as often, but they usually still need checkups at least every 6 months.

These visits are a time for parents to talk with their child's doctor and ask questions about the child's care. Talk with your child's doctor about eye checkups and whether your child needs an ultrasound scan of the brain.

Until age 5, daily penicillin is given to most children who have sickle cell anemia. Doctors also give many children a vitamin called folic acid (folate) to help boost red blood cell production.

Young children who have sickle cell anemia should have regular checkups with a hematologist (a blood specialist).

New Treatments

Research on blood and marrow stem cell transplants, gene therapy, and new medicines for sickle cell anemia is ongoing. The hope is that these studies will provide better treatments for the disease. Researchers also are looking for a way to predict the severity of the disease.

Blood and Marrow Stem Cell Transplant

A blood and marrow stem cell transplant can work well for treating sickle cell anemia. This treatment may even offer a cure for a small number of people.

The stem cells used for a transplant must come from a closely matched donor. The donor usually is a close family member who doesn't have sickle cell anemia. This limits the number of people who may have a donor.

The transplant process is risky and can lead to serious side effects or even death. However, new transplant approaches may improve treatment for people who have sickle cell anemia and involve less risk.

Blood and marrow stem cell transplants usually are used for young patients who have severe sickle cell anemia. However, the decision to give this treatment is made on a case-by-case basis.

Researchers continue to look for sources of bone marrow stem cells—for example, blood from babies' umbilical cords. They also continue to look for ways to reduce the risks of this procedure.

For more information, go to the Health Topics Blood and Marrow Stem Cell Transplant article.

Gene Therapy

Gene therapy is being studied as a possible treatment for sickle cell anemia. Researchers want to know whether a normal gene can be put into the bone marrow stem cells of a person who has sickle cell anemia. This would cause the body to make normal red blood cells.

Researchers also are studying whether they can "turn off" the sickle hemoglobin gene or "turn on" a gene that makes red blood cells behave normally.

New Medicines

Researchers are studying several medicines for sickle cell anemia. They include:

• Decitabine. Like hydroxyurea, this medicine prompts the body to make fetal hemoglobin. Fetal hemoglobin helps prevent red blood cells from sickling and improves anemia. Decitabine might be used instead of hydroxyurea or added to hydroxyurea.
• Adenosine A2a receptor agonists. These medicines may reduce pain-related complications in people who have sickle cell anemia.
• 5-HMF. This natural compound binds to red blood cells and increases their oxygen. This helps prevent the red blood cells from sickling.

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