Sickle Cell Disease Treatment
In September 2024, Pfizer withdrew voxelotor (also called Oxbryta) from the market. The medicine was used to prevent sickling in red blood cells. Pfizer said the benefits of using voxelotor do not outweigh the risks in the approved sickle cell population.
There are many ways to manage sickle cell disease. Your medical team will probably include a doctor who specializes in blood diseases, called a hematologist. If your child is diagnosed with sickle cell disease after their newborn screening result is confirmed, set up a visit with a hematologist as soon as possible.
Your healthcare provider may recommend one or more medicines to help manage symptoms and lower the risk of complications. Blood transfusions or pain medicine may also be needed. For certain patients, transformative therapies with potential for cure like a blood and bone marrow transplant or gene therapy may be options.
It is important to work as a team with your provider to set up a treatment plan. Sticking with your treatment plan can help reduce symptoms and lower the risk of complications. Family members may be able to provide additional caregiving to help you manage your child’s condition or your own.
Medicines
The U.S. Food and Drug Administration (FDA) has approved four medicines to treat sickle cell disease. In 1998, hydroxyurea was approved. Later, L-glutamine and crizanlizumab‑tmca were approved. They treat different aspects of the disease.
First line medicine to prevent sickling and reduce complications
Hydroxyurea is an oral medicine that can reduce sickling of red blood cells and help prevent serious symptoms of sickle cell disease, including pain crises. Healthcare providers usually prescribe daily hydroxyurea for infants as young as 9 months old who have sickle cell disease. If it does not manage symptoms enough, your provider will prescribe other medicines as well.
- Use in adults: Hydroxyurea helps reduce serious symptoms such as pain crises and acute chest syndrome. It can also improve anemia and reduce the need for blood transfusions and hospitalizations. It is not yet known whether hydroxyurea can prevent stroke in adults.
- Use in children: Hydroxyurea can help prevent pain crises and hospitalizations for children with sickle cell disease. A study of infants between ages 9 and 18 months shows that the medicine lowers the number of pain events and . Providers may recommend regular use of hydroxyurea as a substitute for transfusions for children at higher risk of stroke to prevent a first stroke after a period of blood transfusions. Studies suggest that hydroxyurea does not affect growth in children, but more studies are needed.
- Pregnancy: Pregnant women should not take hydroxyurea.
Possible side effects include a low 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. Hydroxyurea can also potentially help people with sickle cell disease live longer.
Treating Sickle Cell Disease With Hydroxyurea
Learn facts, risks, and benefits of hydroxyurea, an oral medicine that can help you reduce sickle cell disease complications.
Medicines to prevent or treat pain
- L-glutamine is approved for people ages 5 years and older. Taking L-glutamine may lead to fewer hospital admissions, fewer pain crises, less need for blood transfusions, and a lower risk of acute chest syndrome. The medicine is a powder that is mixed with a drink or foods like applesauce or cereal. Side effects may include nausea, , chest pain, and pain in bones or muscles.
- Over-the-counter pain medicine, such as acetaminophen or ibuprofen, can treat mild to moderate pain caused by sickle cell disease.
- Prescription pain medicines may be needed for serious pain. Sometimes treatment is given in a clinic or hospital. A healthcare provider may prescribe medicines called opioids for serious pain or pain that lasts a long time. Learn about safe use of opioid medicines.
Tips to Manage Your Pain From Sickle Cell Disease
Learn about pain management and how you can prevent serious problems associated with sickle cell disease.
- Crizanlizumab-tmca is approved for adults and children ages 16 years and older who have sickle cell disease. The medicine is given through an intravenous (IV) line in the vein once a month. The medicine helps prevent blood cells from sticking to blood vessel walls and blocking blood flow. It also helps prevent pain crises, lowers the need for transfusions, and calms .
- A healthcare provider will watch the patient closely while they get the IV medicine and also afterward to be sure they don’t have a serious reaction. The patient should notify their provider right away if they have any of the following symptoms within 24 hours after treatment: fever, chills, nausea, vomiting, tiredness, dizziness, sweating, rash, hives, itching, wheezing, or difficulty breathing. Other side effects may include joint and back pain.
Medicine to lower risk of infection
Daily treatment with an antibiotic called penicillin helps lower children’s chance of having a serious infection in the bloodstream. It is available as a liquid for babies and young children and as tablets for those who are older.
Many healthcare providers stop prescribing penicillin after a child turns 5 years old. Some providers prefer to continue to prescribe this antibiotic throughout a person’s life. Those who have had surgical removal of the spleen (called a splenectomy) or pneumonia caused by bacteria called pneumococcus should keep taking penicillin.
Blood transfusions
Your healthcare provider may recommend a blood transfusion to treat and prevent certain sickle cell disease complications. Blood transfusion is a safe and common procedure during which a patient receives donated blood through an IV placed in one of their veins. It takes 1 to 4 hours for the transfusion, depending on how much the person needs. Healthcare providers will be nearby in case of problems. Learn more about transfusions.
Red blood cell transfusions raise the number of red blood cells and provide normal red blood cells that are more flexible than red blood cells that have sickled hemoglobin.
- Acute transfusions treat serious anemia complications. Providers may also give transfusions when a person with sickle cell disease has had a stroke or an acute chest syndrome or is experiencing multi-organ failure.
- Intermittent transfusions, given as needed, may help relieve symptoms. People who have sickle cell disease usually receive blood transfusions before surgery to prevent complications.
Regular or ongoing blood transfusions may help lower the chances of another stroke in people who have already had one stroke. Healthcare providers also recommend blood transfusions for children who have abnormal results of a transcranial scan (ultrasound of the head). Transfusions can lower the chance of having a first stroke.
Pregnant people may need regular transfusions to reduce sickle cell symptoms and reduce the risk of complications.
Transfusions may help lower complications that do not improve with hydroxyurea alone. Providers may give transfusions to people who do not respond well to hydroxyurea.
Possible complications of transfusion include , which occurs when the patient’s immune system recognizes the donated blood as a foreign invader and attacks it. Infections and iron overload (buildup in body organs) can also occur. Patients with sickle cell disease require special blood matching for blood transfusions to decrease the chance of alloimmunization.
Blood and bone marrow transplant
A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease. To be successful, transplants require a well-matched donor, usually a close blood relative.
Another name for a blood and bone marrow transplant is a hematopoietic stem cell transplant. People who get a transplant are often called “recipients.” People who provide the cells for the transplant are “donors.”
Human leukocyte antigen (HLA) testing helps assess the match between donor and recipient. HLAs play an important part in the body’s immune response to foreign substances. They make up a person’s tissue type, which varies from person to person.
Currently, most blood and bone marrow transplants are done in children who have had complications, such as strokes, acute chest crises, and repeated pain crises. Transplants are riskier in adults. Some people may have a transplant if medicines are not working well to prevent serious pain episodes.
Scientists are studying 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 90% of children when the donor is a blood relative and an HLA match. Even with this high success rate, receiving a transplant still has risks. Healthcare providers take many steps to prevent transplant complications, but they can still happen.
Possible complications include the following:
- Graft-versus-host disease, which is caused by transplanted cells attacking the recipient’s organs
- Higher risk of some cancers later in life
- Infertility
- Seizures
- Serious infections
- Transplant failure, which causes sickle cell disease to return
About 5% of recipients under 16 years old have died from transplant procedures. About 9% of recipients 16 years or older have died after receiving a transplant. For some recipients, a transplant does not cure long-term pain linked with sickle cell disease. A transplant cannot always correct all the damage that sickle cell disease has caused.
To prepare the donor for a blood and bone marrow transplant, their provider gives them a “mobilizing” medicine to raise the number of in their bloodstream. The provider then collects the stem cells from the donor’s blood.
To make space in the recipient’s bone marrow for the donor’s cells, the health provider will give them “conditioning” chemotherapy medicines or use radiation treatments. The provider will then infuse the donor’s cells into the recipient’s blood. 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 have normal hemoglobin that doesn’t sickle.
Because transplantation can affect future fertility, a prospective recipient should talk with their healthcare provider about fertility preservation options before the transplant.
Gene therapies
Gene therapies aim to treat or cure conditions by adding new DNA or changing existing DNA. In December 2023, the FDA approved two gene therapies to treat sickle cell disease:
- Exagamglogene autotemcel adds a modified gene to the body.
- Lovotibeglogene autotemcel makes changes to a gene that is already in the body.
Researchers at the NHLBI are exploring other types of gene therapies that may help provide new treatments or a cure for sickle cell disease. Gene therapies that change a person’s own blood-forming stem cells may provide a cure for people who have sickle cell disease but don’t have a well-matched donor.
Gene therapy involves either restoring activity of a faulty or missing gene or adding a new gene to improve the way the cell works. A healthcare provider will give the patient a medicine to “mobilize” the adult stem cells in their bone marrow. The provider then collects the stem cells from the person’s blood and modifies them in a lab using genetic methods.
Similar to a blood and bone marrow transplant, the provider will administer “conditioning” chemotherapy or radiation to make space in the bone marrow. Then, the provider infuses the genetically modified cells into the person’s blood. 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 don’t sickle.
It is important to remember that even if a person’s disease is cured with a blood and bone marrow transplant or gene therapy, they can still pass the hemoglobin S gene to their children.
Complementary and alternative medicine (CAM)
CAM seems to help some people deal with pain caused by sickle cell disease. The following approaches may lower pain, especially if it is not well managed with medicines:
- Cognitive behavioral therapy (a type of counseling)
- Acupuncture
- Exercise or movement programs, such as yoga
- Massage
- Meditation and mindfulness practices
- Virtual reality, a computer-generated 3D environment you can see with special goggles
Read more about CAM approaches for managing pain.