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, eye damage, and strokes; and control complications (if they occur).

Bone marrow transplants may offer a cure in a small number of sickle cell anemia cases. Researchers continue to look for new treatments for the disease. These include gene therapy and improved bone marrow transplants.

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 and pediatric hematologists specialize in treating adults and children who have blood diseases and disorders.

Treating Pain

Medicines and Fluids

Mild pain often is treated with over-the-counter medicine and heating pads. Severe pain may need to be treated in a hospital.

The usual treatments for acute (short-term) pain crises are fluids and pain-killing medicines. 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.

Common medicines used to treat pain crises include acetaminophen, nonsteroidal anti-inflammatory drugs (NSAIDs), and narcotics such as meperidine, morphine, oxycodone, and others. (Narcotic abuse and addiction are issues that must be considered in any pain control plan.)

Treatment for mild-to-moderate pain usually begins with NSAIDs or acetaminophen. If pain continues, a narcotic may be added. Moderate-to-severe pain is often treated with narcotics. The narcotic may be used alone or with NSAIDs or acetaminophen.

Hydroxyurea

If you have severe sickle cell anemia, you may need a medicine called hydroxyurea. This medicine helps reduce the number of painful crises you have. Hydroxyurea is used to prevent painful crises, not to treat them when they occur.

Given daily, this medicine reduces how often painful crises and acute chest syndrome occur. People taking the medicine also need fewer blood transfusions and have fewer hospital visits.

Early studies of hydroxyurea in children show that they have improved growth. These studies also suggest that this medicine may help preserve organ function.

Hydroxyurea can cause serious side effects, including an increased risk for dangerous infections. People who take hydroxyurea must be carefully watched. Your doctor may need to adjust the dose of this medicine to reduce the risk for side effects.

Doctors are studying the long-term effects of hydroxyurea on people who have sickle cell anemia. Studies are under way to find out whether hydroxurea prevents organ damage or other sickle cell complications.

Talk to your doctor about the risks and benefits of taking hydroxyurea.

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 enlargement of the spleen is a common reason for a blood transfusion.

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

Having routine blood transfusions can cause side effects. Patients must be carefully watched. Side effects can include a dangerous buildup of iron in the blood (which must be treated) and an increased risk of infection from the transfused blood.

For more information, see the Diseases and Conditions Index Blood Transfusion article.

Infections

Infections are a major complication of sickle cell anemia. In fact, pneumonia is the leading cause of death in children who have sickle cell anemia. Other common infections linked to sickle cell anemia include meningitis, influenza, and hepatitis.

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

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

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

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

Eye Damage

Sickle cell anemia can damage the blood vessels in the eyes. Parents should ask their child's doctor about regular checkups with an eye doctor who specializes in diseases of the retina. The retina is a thin layer of tissue inside the back of the eye.

Adults who have sickle cell anemia also should have regular checkups with an eye doctor.

Strokes

Stroke prevention and treatment is now possible for children and adults who have sickle cell anemia. Starting at age 2, children who have sickle cell anemia often get routine ultrasound scans of their heads. This is called transcranial Doppler ultrasound. These scans are used to check blood flow in the brain.

The scans allow doctors to find out which children are at high risk for a stroke. These children are then treated with routine blood transfusions. This treatment has been found to greatly reduce the number of strokes in children.

Treating Other Complications

Acute chest syndrome is a severe and life-threatening complication of sickle cell anemia. Treatment usually requires hospitalization and may include oxygen, blood transfusions, antibiotics, pain medicine, and checking the body's fluids.

If you have leg ulcers due to sickle cell anemia, you may be given strong pain medicines. Ulcers can be treated with cleansing solutions and medicated creams or ointments. Skin grafts may be needed if the condition continues. Bed rest and keeping the legs raised to reduce swelling are helpful, although not always possible.

Gallbladder surgery may be needed if the presence of gallstones leads to gallbladder disease.

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

Regular Health Care for Children

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

All children younger than 2 need to see the doctor often. Children who have sickle cell anemia may need additional checkups. After age 2, children who have sickle cell anemia may need to see the doctor less often, but usually 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 prevent some sickle cell complications.

New Treatments

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

Bone Marrow Transplant

A bone marrow transplant can work well for treating sickle cell anemia. This treatment may even offer a cure in a small number of cases.

However, the procedure is risky and can lead to serious side effects or even death. Because of this, only some people can or should have this procedure.

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

Bone marrow used for a transplant must come from a closely matched donor. This is usually a close family member who doesn't have sickle cell anemia.

Researchers continue to look for ways to reduce the risks of this procedure.

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 in the bone marrow 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 cell gene or "turn on" a gene that makes red blood cells behave normally.

New Medicines

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

• Butyric acid. This is a food additive that may increase normal hemoglobin in the blood.
• Nitric oxide. This medicine may make sickle cells less sticky and keep blood vessels open. People who have sickle cell anemia have low levels of nitric oxide in their blood.
• Decitadine. This medicine increases hemoglobin F levels (this type of hemoglobin carries more oxygen). It may be a good choice instead of hydroxyurea.