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Sickle Cell Disease and Stroke
How Is Sickle Cell Anemia Treated?

Effective treatments are available to help relieve the symptoms and complications of sickle cell anemia, but in most cases there’s no cure. Some researchers believe that bone marrow transplants may offer a cure in a small number of cases. Researchers are looking for new treatments for sickle cell anemia, including gene therapy and safer and more effective bone marrow transplants.

People who have sickle cell anemia need regular medical care. Some doctors and clinics specialize in treating people with the condition. Doctors specializing in sickle cell anemia are often hematologists (doctors who treat people with blood disorders)?or pediatric hematologists (if they also treat children).

Goals of Treatment
The goals of treating sickle cell anemia are to relieve pain; prevent infections, eye damage, and strokes; and control complications if they occur. The treatments include medicine, blood transfusions, and specific treatment for complications.

Treating Pain
Mild painful crises can be managed with treatments such as over-the-counter medicine and heating pads. However, severe pain may need to be treated in a hospital. Painful crises are the leading cause of emergency room visits and hospitalizations of people with sickle cell anemia.

The usual treatments for acute (short-term) pain crises are pain-killing medicines and fluids, given either by mouth or through a vein, to prevent dehydration (a condition in which your body doesn’t have enough fluids). The pain-killing medicines most often used are acetaminophen, nonsteroidal anti-inflammatory drugs (NSAIDs), and narcotics such as meperidine, morphine, oxycodone, and others.

The treatment of patients who have 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 together with NSAIDs or acetaminophen. Narcotic abuse and addiction are pain management issues that must be considered in any pain control plan.

A medicine called hydroxyurea may be given to adults and older adolescents with severe sickle cell anemia to reduce their number of painful crises. This medicine is used only to prevent these crises, not to treat them when they occur. Given daily, hydroxyurea can reduce the frequency of painful crises and of acute chest syndrome. People taking the medicine also may need fewer blood transfusions.

People taking hydroxyurea must be watched carefully because the medicine can cause serious side effects, including an increased risk of dangerous infections. Some evidence suggests that long-term use of hydroxyurea can cause tumors or leukemia. Because of these risks, the medicine is usually only used in adults and older teenagers with severe sickle cell anemia. Although hydroxyurea is being tested in infants and children at this time, it won’t be approved for use in children until its long-term effects can be more closely studied.

Preventing Infections
Infection is a major complication of sickle cell anemia. In fact, pneumonia is the leading cause of death in children with the condition. Other infections common in people with sickle cell anemia include meningitis, influenza, and hepatitis. If a child with 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.

Vaccinations for pneumonia, meningitis, influenza, and hepatitis.

   - A yearly flu shot.

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

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

Preventing Strokes
Stroke prevention and treatment is now possible for children and adults who have sickle cell anemia. Starting at age 2, children with sickle cell anemia often receive regular ultrasound scans of the head (this is called transcranial Doppler ultrasound). These scans are used to monitor blood flow in the brain.

The scans allow doctors to find out which children are at high risk for a stroke and treat them with regular blood transfusions. Routine blood transfusions have been found to greatly reduce the number of strokes in children.

Blood Transfusions
Blood transfusions are 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, patients need transfusions to prevent life-threatening events such as stroke or pneumonia.

Regular blood transfusions do have side effects, and patients must be carefully watched. Side effects can include a dangerous buildup of iron in the blood (which must be treated) as well as an increased risk of infection from the transfused blood.

Treating Other Complications
Acute chest syndrome is a severe and life-threatening complication in children and adults who have sickle cell anemia. Treatment usually requires hospitalization and may include oxygen, blood transfusions, antibiotics, pain medicine, and monitoring the body’s fluids.

Leg ulcers can be painful, and patients 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 can be treated with fluids or surgery.

Regular Health Care for Children
Children with sickle cell anemia should get regular health care, just like children without the condition. They need to have their growth checked and to get the usual shots that all children receive.

It’s recommended that before age 2, children with sickle cell anemia see the doctor often (sometimes every 2 or 3 months). After age 2, children 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. Parents should ask about eye checkups and the need for an ultrasound scan of the brain.

Until age 5, daily penicillin is given to most children who have sickle cell anemia. Many patients are prescribed a vitamin called folic acid (folate) to help prevent some of the complications of sickle cell anemia.

New Treatments
Today, research on sickle cell anemia is looking at bone marrow transplants, gene therapy, and new medicines. 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 condition.

Bone Marrow Transplant
Bone marrow transplant can be a very effective treatment for sickle cell anemia, but because of its risks, only some patients can or should have this procedure.

The bone marrow transplant procedure is risky and could result in serious side effects and even death. It’s usually used only for younger patients with severe sickle cell anemia, but the decision is made on a case-by-case basis.

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

Gene Therapy
Gene therapy is being studied as a possible treatment for sickle cell anemia. Researchers are looking to see whether a normal gene can be planted in the bone marrow of a person with sickle cell anemia, and thus cause the body to produce normal red blood cells. Researchers also are studying the possibility of treatment to “turn off” the sickle cell gene or “turn on” a gene that makes red blood cells behave normally.

New Medicines
New medicines being studied are:

Butyric acid. This is a food additive that may increase normal hemoglobin in the blood.

Clotrimazole. This is used now to treat fungus infections. This medicine helps prevent
    the loss of water from a red blood cell and can keep the cell from turning into a sickle

Nitric oxide. This may make sickle cells less sticky and keep blood vessels open. People
     with sickle cell anemia have low levels of nitric acid in their blood.

Article courtesy of
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