Treatments for Sickle Cell Disease

Editor’s note: As of September 2024, Oxbryta^® (voxelotor) has been voluntarily withdrawn from the market by the manufacturer due to safety concerns. Pfizer, the manufacturer of Oxbryta, has also ceased distribution and discontinued all active clinical trials and expanded access programs for Oxbryta. Patients and caregivers should contact their healthcare provider about stopping Oxbryta and starting another approved medication for the treatment of sickle cell disease.

The type and severity of complications of sickle cell disease (SCD) are different for everyone. Your doctor will consider these factors, as well as your age and overall health, when determining the best treatment for you.

The only cure for SCD is a bone marrow transplant. This procedure is not right for everyone. It is risky and requires a matched donor. However, there are many treatment options for managing SCD. The goals of treatment for SCD include avoiding pain episodes, helping symptoms, and preventing complications.^1,2

Medications

There are several medicines available that treat the causes of SCD. They all work in different ways:^1,2

• Endari™ (L-glutamine) – Lowers the number of pain crises.
• Adakveo^® (crizanlizumab-tmca) – Helps prevent blood cells from sticking to the blood vessel walls.
• Hydroxyurea – Reduces the number of pain crises and acute chest syndrome episodes in adults.

Pain medications

A large part of living with SCD is managing pain. Some pain can be managed with over-the-counter pain relievers. These include:^1,3

• Nonsteroidal anti-inflammatory drugs (NSAIDs) like ibuprofen (Motrin^®, Advil^®)
• Acetaminophen (Tylenol^®)

More serious pain may require prescription pain relievers, like opioids. Severe pain may need to be treated in a hospital.^1,3

Supportive medications

Supportive medicines prevent or manage complications from SCD. The supportive medicines needed will depend on your age and symptoms.^1-3

Preventing infections in children with SCD is very important. Children should receive vaccines. This includes standard childhood vaccines and flu shots. Some children may also need pneumococcal and meningococcal vaccines. Children under age 5 usually take penicillin every day. This helps prevent infections.^1-3

Other types of supportive medicine include:^2-4

• Asthma drugs like inhalers
• Antidepressants
• Skin drugs and ointments to treat leg ulcers
• Drugs for kidney disease like angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs)

Blood transfusions

Blood transfusions are commonly used to treat and prevent some complications of SCD. Examples include:^1,2

• Red blood cell transfusions – These transfusions increase the number of healthy red blood cells.
• Acute transfusions – These transfusions treat serious complications that can cause anemia. For example, you may need a transfusion for stroke, acute chest crisis, or multi-organ failure.

You may need regular transfusions if you have had a stroke. If you need surgery for SCD, you may also get a transfusion before the surgery.¹

Gene therapy

In 2023, the US Food and Drug Administration (FDA) approved 2 gene therapies for SCD, Casgevy™ (exagamglogene autotemcel) and Lyfgenia™ (lovotibeglogene autotemcel). These treatments use a patient's stem cells. First, the stem cells are removed from the body. They are then modified using technology that can change DNA. These modified cells are returned to the body. There, they can multiply and produce healthy hemoglobin.⁵

Other procedures

Other procedures may also be used to treat specific complications of SCD. Some examples include:^3,6-9

• Surgical removal of the spleen (splenectomy) to treat or prevent splenic sequestration
• Injections, laser treatment, or surgery to address vision loss
• Devices or surgical procedures to open the airway and treat sleep issues like obstructive sleep apnea
• Injections or surgery to remove blood from the penis to treat priapism (prolonged erection)
• Dialysis or kidney transplants
• Surgery to treat bone complications
• Surgical removal of the gallbladder
• Bronchodilators or oxygen supplementation to treat acute chest syndrome

Complementary and integrative medicine

Some people with SCD may try complementary and alternative medicines (CAMs). CAM treatments do not replace traditional medical treatments but may help with additional relief. Some CAM treatments that have been researched for SCD include:^4,10

• Diet changes
• Lifestyle changes
• Zinc supplements
• Omega-3 fatty acid supplements
• Amino acid supplements
• Antioxidant supplements
• Acupuncture

Alternative medicines are not regulated by the FDA. CAM medicines may interact with your other drugs and cause a reaction. They can also cause the other drugs you take to work differently. This could make your SCD and other health conditions worse. Insurance plans may not cover CAM therapies either. Also, CAM may not work for everyone.^4,10

Research on new SCD treatments

New treatments are being developed and tested for SCD. One area of research is combination drug therapy. This involves combining different drugs for more effective treatment. Many other drugs are currently in clinical trials.¹¹

Talk to your doctor if you are interested in participating in a clinical trial. Drugs in clinical trials are experimental and not available to most people. Large trials need to establish the safety, ethics, and long-term success of these methods.¹¹