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 – Endari, Adakveo, and Oxbryta – have been approved by the U.S. Food and Drug Administration (FDA). These all reduce the number of pain crises and hospitalizations. 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 medications.
People with SCD use medicines that prevent infections and relieve pain. Other medicines are also commonly used to treat specific complications.
What medications treat sickle cell disease?
Hydroxyurea was the first medicine approved by the FDA to treat sickle cell disease in 1997. It was originally 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, but 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
Unfortunately, not enough people use hydroxyurea, either because of side effects, access, or cost. There are also misconceptions about it causing cancer that lower adherence to treatment. Mild side effects (headache and nausea) are possible, but there is no evidence of hydroxyurea increasing the risk of cancer.2,4
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 prevents red blood cells from sickling. This reduces the risk of blood cells getting trapped in blood vessels and blocking blood flow.3,6
Endari reduces the number of acute pain crises and acute chest syndrome episodes. It also reduces hospitalizations and 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.7,8
The FDA approved Oxbryta in 2019 to treat SCD in people 12 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.1,8
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. Like hydroxyurea, it is taken once daily as a tablet.9
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 that sticks to sickle cells. This helps prevent pain and inflammation when sickle cells block blood flow.1,8
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.10
What is the cost of SCD medications?
Annual costs of generic hydroxyurea are about $1,200. The newer drugs are much more expensive. Annual costs may be up to $20,000 a year for Endari and $100,000 a year for Adakveo and Oxbryta. Depending on your state, Medicaid may not yet cover these expensive treatments.8
However, the costs 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 medications.11
What supportive medications 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 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 medications and hydration does not relieve pain, treatment in an ER is necessary. Doctors may administer stronger pain medications, such as opioids or ketamine.1,4
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 medications, such as:4,6