Sickle cell anemia is the most severe form of sickle cell disease (SCD). Treatments for sickle cell anemia include medications, blood transfusions, and bone marrow transplants.

SCD is a group of genetic disorders that causes misshapen red blood cells (RBCs), which are curved and resemble sickles. These RBCs do not travel easily through blood vessels and can cause blockages in blood flow, potentially leading to severe complications.

People with sickle cell anemia should consult a doctor to find out which treatment options may be best for them.

This article discusses a variety of treatments for sickle cell anemia.

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Voxelotor (Oxbryta) is a medication that prevents RBCs from developing a sickle shape and binding to each other. This may stop the body from destroying some RBCs and, as a result, may reduce the risk of anemia and improve blood flow to the organs.

This medication is for adults and children ages 4 years and older who are living with SCD.

Side effects and risks

Possible side effects include:

In rare cases, people may experience allergic reactions, which can cause hives, rashes, and mild breathlessness.

This medication helps reduce the risk of blood cells clinging to blood vessel walls. As a result, crizanlizumab-tmca (Adakveo) may reduce a person’s risk of pain crises, blood vessel blockages, and inflammation.

The Food and Drug Administration (FDA) has approved this medication as an intravenous (IV) injection for people ages 16 years and older.

Side effects and risks

Possible side effects include:

Doctors may recommend hydroxyurea for adults and children with either of two specific types of SCD: hemoglobin SS and sickle cell beta-thalassemia. It may help prevent several severe complications.

In adults, this medication may help reduce pain crises and lower the risk of acute chest syndrome, which is a medical emergency. It may also improve the number of RBCs and reduce the number of times a person needs blood transmissions and hospital admissions.

In children, hydroxyurea may reduce the number of blood vessel blockages and hospital admissions. It may also help prevent pain episodes and swelling in the hands and feet.

Side effects and risks

Hydroxyurea may reduce the number of white blood cells or platelets in the blood. In rare cases, it can make anemia worse. However, stopping the use of hydroxyurea can reverse these side effects. Doctors may then prescribe it again at a lower dose.

Pregnant people should not take hydroxyurea, and its effects on children under 9 months of age are not known.

People may take over-the-counter (OTC) or prescription medications to manage pain. These might include:

  • L-glutamine: This may help reduce pain crises and hospital admissions in people ages 5 years and older. It comes as a powder that people can mix into liquids and foods.
  • OTC pain relief: Acetaminophen and ibuprofen may help soothe mild to moderate pain.
  • Opioids: A doctor may prescribe these for severe pain.

Side effects and risks

L-glutamine may cause:

According to the United Kingdom’s National Health Service (NHS), ibuprofen may cause the following negative effects:

Opioids have a high potential for addiction and may cause:

People should always speak with a healthcare professional for guidance before taking any pain relief medications.

Children with sickle cell anemia may need two daily doses of penicillin to reduce their risk of a severe infection traveling through the blood. Newborns take penicillin as a liquid, and older children can take penicillin in pill form.

Some doctors stop prescribing this medication after 5 years of age. Others may recommend penicillin as a lifelong medication, especially for people who have had surgery to remove their spleen or have had an infection called pneumococcus.

Doctors may also recommend that people with hemoglobin SS and sickle cell beta-thalassemia take penicillin for the rest of their lives.

Side effects and risks

Different types of penicillin have varying side effects. These might include:

Blood transfusions can help replace sickle-shaped RBCs with typical ones that travel more efficiently around the body. They can also help treat complications that may lead to severe anemia, stroke, acute chest crises, and multiple organ failure.

Before a person with sickle cell anemia undergoes any type of surgery, a doctor may request that they receive a transfusion to reduce the risk of complications.

Transfusions may decrease the likelihood of another stroke in people who have already had a stroke due to sickle cell-related blockages. They may also decrease the chances of a first stroke in children who have abnormal transcranial Doppler ultrasound results that suggest an increased risk of stroke.

Additionally, transfusions can be an alternative to hydroxyurea for those who do not tolerate its side effects well.

Side effects and risks

A process called alloimmunization can cause the body to “reject” the blood transfusion, making it difficult to find a match for the next blood donation.

Transfusions also pose a risk of iron overload and infections.

Currently, the only way to cure sickle cell anemia is by replacing a person’s bone marrow with bone marrow from a donor who is a close genetic match. However, not everyone has a well-matched donor.

Most transplants for sickle cell anemia occur in children who have experienced pain crises, recurring chest crises, and strokes. The procedure is riskier for adults.

Bone marrow transplants are successful in around 85% of children who receive bone marrow from a well-matched, related donor.

Side effects and risks

Bone marrow transplants can lead to serious complications, including:

The mortality rate due to complications after bone marrow transplant procedures is around 5%. Some medications can reduce the risk of complications.

In 2023, the FDA approved two cell-based gene therapies, Casgevy and Lyfgenia, to treat SCD in people ages 12 years and older.

Both therapies involve modifying a person’s own blood stem cells and then returning them to the person’s body through an infusion as part of a stem cell transplant. These cells then help the person produce functioning RBCs.

Side effects and risks

The most common side effects of Casgevy include:

  • nausea
  • vomiting
  • itching
  • mouth sores
  • headache
  • abdominal pain
  • musculoskeletal pain
  • low levels of platelets and white blood cells
  • febrile neutropenia (fever and low white blood cell count)

Possible side effects of Lyfgenia include:

People should also note that Lyfgenia comes with a boxed warning regarding the risk of blood cancer, so doctors will monitor anyone who receives this therapy for blood cancer.

Below are some common questions and answers about sickle cell anemia.

What is the most common treatment for sickle cell anemia?

According to the American Society of Hematology, hydroxyurea and pain relief medications are common treatments for SCD.

A person may also receive blood transfusions and other treatments as needed.

What worsens sickle cell anemia?

According to the NHS, potential triggers for sickle cell crises include:

  • dehydration
  • extreme temperatures
  • high altitudes, although air travel is fine since planes have a managed oxygen level
  • very intense exercise
  • alcohol consumption
  • smoking
  • stress

Is sickle cell disease a lifelong condition?

SCD is a lifelong condition, although it is curable through bone marrow transplants. However, these procedures are risky, especially in adults.

People may manage SCD with medications and blood transfusions.

Sickle cell anemia treatments include medications to manage pain and prevent complications such as infection and stroke. Blood transfusions can provide more functional RBCs that can help treat and manage crises and prevent further complications.

A bone marrow transplant can cure SCD. However, this procedure has a high risk of complications, and donors often need to have a close genetic link to the recipient.

People with SCD should talk with their doctor to find out which treatments may work best for them.