Sickle Cell Disease Treatment

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Medicine to prevent the sickling of red blood cells

Voxelotor treats sickle cell disease in adults and children 4 years old and older. The oral medicine prevents red blood cells from forming the sickle shape and binding together. This may reduce the destruction of some red blood cells, which in turn lowers the risk for anemia and improves blood flow to your organs.

Possible side effects include headache, diarrhea, abdominal pain, nausea, fatigue, and fever. Rarely, allergic reactions may occur, causing rashes, hives, or mild shortness of breath. Talk to your healthcare provider about other medicines you take.

Medicine to reduce vaso-occlusive and pain crises

Crizanlizumab-tmca is approved for adults and children 16 years old and older who have sickle cell disease. The medicine, which is given through an intravenous (IV) line in the vein, helps prevent blood cells from sticking to blood vessel walls and causing blood flow blockage, inflammation, and pain crises.

Possible side effects include nausea, joint pain, back pain, and fever.

Medicine to reduce or prevent multiple complications

Hydroxyurea is an oral medicine that has been shown to reduce or prevent several complications of sickle cell disease.

• Use in adults: Many studies of adults with hemoglobin SS or hemoglobin Sβ (sickle cell beta) thalassemia showed that hydroxyurea lowered the number of episodes of pain crises and acute chest syndrome. It also improved anemia and reduced the need for transfusions and hospital admissions.
• Use in children: Studies in children with severe hemoglobin SS or Sβ thalassemia showed that hydroxyurea lowered the number of vaso-occlusive crises and hospitalizations. A study of children between the ages of 9 and 18 months who had hemoglobin SS or Sβ thalassemia also showed that hydroxyurea lowered the number of pain episodes and the incidence of dactylitis (painful swelling of the hands and feet). There is no information about how safe or effective hydroxyurea is in children under 9 months old.
• Pregnancy: Pregnant people should not use hydroxyurea.

Since hydroxyurea can decrease several complications of sickle cell disease, most experts recommend daily use for children and adults with hemoglobin SS or Sβ0 thalassemia who have frequent painful episodes, recurrent chest crises, or severe anemia.

Possible side effects include a lowered white blood cell count or platelet count. Rarely, hydroxyurea can worsen anemia. These side effects usually go away quickly if a patient stops taking the medicine. When the patient restarts it, the healthcare provider usually prescribes a lower dose.

It is still unclear whether hydroxyurea can cause problems later in life in people who have sickle cell disease and take the medicine for many years. Studies suggest that hydroxyurea does not put people at a higher risk of cancer and does not affect growth in children, but further studies are needed.

Handout

Sickle Cell Disease: Hydroxyurea: What You Need to Know

Learn more about hydroxyurea. 

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Medicine to treat pain

The FDA approved L-glutamine for people age 5 and older to lower the number of pain crises. Research showed that patients taking L-glutamine had fewer hospital admissions than patients taking a placebo. The medicine is prescribed as a powder that is mixed into liquids or foods like applesauce or cereal.

Possible side effects include nausea, fatigue, chest pain, and musculoskeletal pain. L-glutamine has not yet been tested in older adults.

Over-the-counter pain medicine, such as acetaminophen or ibuprofen, can be used to treat mild to moderate pain. Stronger or more serious pain may need to be treated in a clinic or hospital. Your provider may prescribe stronger medicines called opioids for severe pain.

Fact sheet

Managing Pain With Sickle Cell Disease Fact Sheet

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Medicine to reduce risk of infection

Penicillin taken twice a day helps lower children's chance of having a serious infection in the bloodstream. Newborns need to take liquid penicillin. Older children can take tablets. 

Many healthcare providers will stop prescribing penicillin after a child has reached the age of 5. Some providers prefer to continue to prescribe this antibiotic throughout a person's life, particularly if the person has hemoglobin SS or hemoglobin Sβ0 thalassemia, since people who have sickle cell disease are still at risk. All people who have had surgical removal of the spleen (called a splenectomy) or a past infection with pneumococcus should keep taking penicillin throughout their lives.

Your healthcare provider may recommend transfusions to treat and prevent certain sickle cell disease complications. You may have one or more of the following kinds of transfusions.

• Acute transfusions treat complications that cause severe anemia. Providers may also use transfusions for patients in cases of an acute stroke, in many cases of acute chest crises, and in multi-organ failure. A patient who has sickle cell disease usually receives blood transfusions before surgery, to prevent complications.
• Red blood cell transfusions increase the number of red blood cells and also provide normal red blood cells that are more flexible than red blood cells with sickle hemoglobin.
• Regular or ongoing blood transfusions may help lower the chances of another stroke in people who have had an acute stroke.

Healthcare providers also recommend blood transfusions for children who have abnormal transcranial Doppler (TCD) ultrasound results, because transfusions can lower the chance of having a first stroke. Some healthcare providers use this approach to treat complications that do not improve with hydroxyurea. Providers may also use transfusions in people who have too many side effects from hydroxyurea. Possible complications include alloimmunization , which occurs when the transfusion recipient develops antibodies to the blood being transfused, making it hard to find a matching unit of blood for a future transfusion. Infection and iron overload can also occur.