If you or a loved one has sickle cell disease, finding the right treatment is important for overall health and quality of life. Treatments are even used to prevent complications like stroke and pain crises and to extend the lifespan. Treatment options can vary widely based on your unique situation and type of disease. 

To find the right treatment, it’s best to talk to your healthcare provider. Some treatments are better suited for certain disease types, and your doctor will be able to talk to you about your options. However, being informed about the different treatments available can help give you agency in your care. Here are some of the common treatment options for sickle cell disease. 

Blood transfusions 

This procedure can also help prevent additional complications such as strokes. Some people with sickle cell disease are more prone to strokes because their sickle cells can clump together, and they don’t move as well through the body. If this applies to you, a blood transfusion might be a very valuable treatment. 

Blood transfusions can also help prevent anemia, which can sometimes get worse in people with sickle cell disease after an infection. 

Gene therapy 

Gene therapy works by introducing copies of a gene that doesn’t work properly in people with sickle cell disease. Once the process is completed, you’re able to make healthy red blood cells again. During the process, you’ll need to undergo chemotherapy because this removes cells from the bone marrow to make room for the new stem cells. 

Currently, there are two different gene therapies available, Casgevy (Exagamglogene autotemcel) and Lyfgenia (Lovotibeglogene autotemcel). Casgevy adds a modified gene to your body. Lyfgenia uses your own blood stem cells, modifies them and then adds them back via an infusion. 

Stem cell transplants 

Also called a bone marrow transplant, a stem cell transplant can not only treat the symptoms of but also cure sickle cell disease. This works by putting healthy red blood stem cells from a donor in the bone marrow, which can trigger the growth of additional healthy red blood cells. Often, this treatment is used for children with severe sickle cell disease. Finding an appropriate donor can be difficult, and the best match is often a close family member, such as a sibling. 

Medication 

Various medications can be used to reduce the number of sickle cells in the blood, improve anemia, and reduce pain. Antibiotics are also often used for people with sickle cell disease to prevent infection because they are more susceptible to infections. When people with sickle cell disease experience pain crises, they may be treated with IV fluids and pain medication. 

Hydroxyurea is one option for medication, and it reduces the sickling of red blood cells. It can also help prevent pain crises and improve anemia. It’s an oral medication that can be used by adults and children as young as 9 months old. 

L-glutamine is another medication option. It’s used to reduce hospital admissions, reduce the number of pain crises, and lower the number of blood transfusions needed. It’s a powder that can be mixed with food or drinks and is available for adults and children aged 5 and older. 

Crizanlizumab-tmca is a medication that helps prevent blood cells from sticking to blood vessel walls. It can also help reduce the number of transfusions needed, prevent pain crises, and reduce inflammation. It’s administered via an IV, and it’s available for adults and children aged 16 and older. 

Oxbryta was another medication used to treat sickle cell disease, but it was recalled in September 2024. It was used to prevent the red blood cells from sickling. However, it was found that the benefits of the drug did not outweigh the risks, as taking the drug increased the chance of developing VOCs (vaso-occlusive crises). This is when sickle cells block blood flow, which can be painful and even deadly. 

What are the complications of sickle cell disease treatment? 

Treating sickle cell disease is important for management and quality of life, but sometimes, you will experience side effects during treatment. Side effects of some medications can include low white blood cell count, low platelet count, worsening anemia, nausea, fatigue, and pain. Side effects or complications of blood transfusions can include alloimmunization, infections, and iron overload. Complications of bone marrow transplants can include graft-versus-host disease, increased cancer risk, infertility, seizures, serious infections, and transplant failure. 

There is always the risk that future complications or side effects that scientists aren’t currently aware of or that pharmaceutical companies aren’t warning about may arise. For example, those who took Oxbryta were not properly warned about the risk of VOCs. Those who have been negatively affected by the drug have begun to file lawsuits against Pfizer and Global Blood Therapeutics for failure to warn. If you believe you have been negatively affected by Oxbryta, you may be eligible to file a lawsuit.