Types of Medications for Sickle Cell Disease

For about 20 years, the only medicine available to treat sickle cell disease (SCD) was hydroxyurea. Since 2017, 3 new medicines have been approved by the U.S. Food and Drug Administration (FDA):

• Endari™ (L-glutamine)
• Adakveo® (crizanlizumab-tmca)
• Oxbryta® (voxelotor)

These newer drugs can all be used alone or in combination with hydroxyurea. Access, adherence (taking medicine as prescribed), and cost remain issues with these drugs.

People with SCD use medicines that prevent infections and relieve pain. Other medicines are also commonly used to treat specific complications.

What medicines treat sickle cell disease?

Hydroxyurea

Hydroxyurea was the first medicine approved by the FDA to treat sickle cell disease in 1998. It was first developed as a chemotherapy, or anti-cancer, drug. There is now more than 20 years of evidence for its benefits in SCD.1-3

Hydroxyurea treats SCD in multiple ways. Most importantly, it increases your amount of fetal hemoglobin. This type of hemoglobin is normally only present in newborns. It transports oxygen well and can counteract the negative effects of sickle hemoglobin.^2,4

Studies have shown that hydroxyurea can:^2,5

• Reduce episodes of acute pain crises and acute chest syndrome
• Reduce hospitalizations and the need for transfusions
• Improve anemia
• Lower the risk for chronic organ damage
• Lower the risk of stroke
• Reduce delays in growth and puberty
• Improve quality of life

Unfortunately, not enough people use hydroxyurea, either because of side effects, access, or cost.

Like other drugs, hydroxyurea has risks. There have been reports of skin complications during long-term therapy. Some of these complications are serious. Though rare, hydroxyurea has been linked to cases of skin cancer with long-term use.^6-9

Other serious complications of hydroxyurea include bone marrow suppression. This is when fewer blood cells are made in the bone marrow. Bone marrow is the spongy, middle part of the long bones in the body and is where most of the body’s blood cells are made. Bone marrow suppression is common in drugs that treat cancer, like hydroxyurea. This may include:^6-9

• Anemia, decreased oxygen-carrying red blood cells
• Neutropenia, decreased white blood cells that help fight infection
• Thrombocytopenia, decreased platelets that help stop bleeding
• Pancytopenia, a general decrease in all of these types of blood cells

Endari

The FDA approved Endari in 2017 to treat SCD in people 5 years old and older. We do not yet know exactly how it works, but it is thought to neutralize oxidative stress which helps sickle cells become more flexible. This reduces the risk of blood cells getting trapped in blood vessels and blocking blood flow.^3,10

Endari reduces the number of acute pain crises and acute chest syndrome episodes. It also reduces hospitalizations and the amount of time spent in the hospital. This gives people a non-chemotherapeutic option. However, its powder form makes it more challenging to adhere to treatment than hydroxyurea tablets.^11,12

Oxbryta

The FDA approved Oxbryta in 2021 to treat SCD in people 4 years old and older. It works by preventing sickle hemoglobin from sticking together inside red blood cells. This helps stop red blood cells from sickling.^1,13

Oxbryta improves anemia by increasing hemoglobin levels. It also lowers bilirubin levels and reticulocyte counts. These are additional signs that Oxybryta can treat complications of anemia.¹³

Adakveo

The FDA approved Adakveo in 2019 to treat SCD in people 16 years old and older. It contains an antibody that blocks a protein in blood vessels and platelets that make them more sticky. The protein, P-selectin, can cause clots to form between blood vessel walls, platelets, red blood cells, and white blood cells, blocking blood flow.^1,14

Adakveo reduces the number of acute episodes that lead to a hospital visit. It also slightly reduces the length of hospital stays. People usually receive treatment by IV infusion once a month.¹⁴

What is the cost of SCD drugs?

SCD drug costs can be very expensive. Because insurance coverage varies from person to person, it is hard to guess costs. However, some drugs cost up to $100,000 a year before insurance. Depending on your state, Medicaid may not yet cover these expensive treatments. Talk to your doctor about your drug choices, including what these might cost for you.¹²

The costs of SCD drugs may be outweighed by reducing expensive hospitalizations. People with SCD who take hydroxyurea pay lower total healthcare costs. We do not yet know the long-term financial impact of the newer drugs.¹¹

What supportive medicines do people use?

Antibiotics and immunizations

Infections are life-threatening for people with SCD. Treatments to prevent and manage infections have greatly improved childhood survival rates. This usually includes daily penicillin from 2 months old to 5 years old. It also includes all routine vaccinations, as well as the pneumococcal and meningococcal vaccines.^12,13

Pain medicines

Pain is the most common symptom of SCD. It is most often treated at home with non-steroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen or diclofenac. When home management with pain medicines and hydration does not relieve pain, treatment in an ER is necessary. Doctors may give stronger pain medicines, such as opioids.^1,4

Other medicines

Sickle cell disease is much more than a pain condition. It can cause complications in many parts of the body, which vary in severity for each person. Depending on your specific situation, your doctor may recommend other medicines, such as:^4,6

• Asthma drugs
• Iron chelation therapy
• Skin medicines and ointments
• ACE inhibitors
• Antidepressants

Other things to know

All drugs can cause side effects, and these side effects can vary depending on the specific drug you are taking. Talk to your doctor about what to expect when taking these drugs. You also should call your doctor if you have any changes that concern you when taking them.

Before beginning treatment for SCD, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you take. This includes over-the-counter drugs.