Sickle Cell Disease Treatment
A bone marrow transplant is currently the only cure for some patients who have sickle cell disease. After early diagnosis, your healthcare provider may recommend medicines or transfusions to manage complications, including chronic pain.
Babies who have sickle cell disease may see a hematologist, a doctor who specializes in blood diseases such as sickle cell disease. For newborns, the first sickle cell disease visit should take place before 8 weeks of age.
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.
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.
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 , 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.
Blood and bone marrow transplant
A blood and bone marrow transplant is currently the only cure for sickle cell disease, but it is not for everyone. Many patients who have sickle cell disease do not have a relative who is a close enough genetic match to be a donor. A well-matched donor is needed for a patient to have the best chance for a successful transplant.
Currently, most transplants to treat sickle cell disease are performed in children who have had complications, such as strokes, acute chest crises, and recurring pain crises. These transplants usually use a matched donor. Blood and bone marrow transplants are riskier in adults.
Several medical centers are looking into new ways to help more people who have sickle cell disease get a transplant. These ways include blood and bone marrow transplant techniques in children and adults who do not have a matched donor in the family or who are older than most recipients.
Blood and bone marrow transplants are successful in about 85% of children when the donor is related and human leukocyte antigen (HLA)–matched. Even with this high success rate, transplants still have risks. Complications can include serious infections, seizures, and other clinical problems. About 5% of people who have received such transplants have died. Sometimes transplanted cells attack the recipient’s organs. This is called graft-versus-host disease. You will get medicine to prevent many of the complications, but they can still happen.
Potential genetic therapy treatments
Researchers at the NHLBI are exploring ways genetic therapies may help provide new treatments or cure for sickle cell disease. Genetic therapies aim to treat or cure conditions by adding new DNA or changing existing DNA.
Genetic therapy involves either restoring a faulty or missing gene or adding a new gene that improves the way the cell works. Researchers take blood or bone marrow from a patient and modify their stem cells in a laboratory using genetic therapies.
Genetic therapies that modify a person’s own hematopoietic stem cells may provide a cure for people who have sickle cell disease and do not have a well-matched donor. Modified stem cells can be injected into the blood; then the cells travel in the bloodstream to the marrow spaces inside the bones. Once inside the bone marrow, the cells can produce healthy red blood cells that do not sickle.