Guidelines on Sickle Cell Disease Treatments

Sickle cell disease is a type of red blood cell disorder. Healthy red blood cells are round and flexible, so they travel quickly through the blood cells. However, in people with sickle cell disease, a genetic mutation inherited from their parents causes the red blood cells to take on a sickle or crescent shape. They also have a more rigid structure and are prone to sticking to blood vessel walls, so blood flow becomes disrupted or blocked. Sickle cells even die earlier than healthy red blood cells do, resulting in a low red blood cell count or anemia.

Symptoms of sickle cell disease start to show up as early as six months of age, and the condition will require management throughout one’s lifetime. Bone marrow transplant can be an effective cure for sickle cell disease, though it is not realistic for many people. Because it is a very risky procedure, and the body may ultimately reject the transplant, few people with sickle cell pursue this option.

Because symptoms of sickle cell can vary from mild to severe, treatment is often focused on managing the presented symptoms and preventing the development of complications.

Preventive medication

Patients with sickle cell are often prescribed drugs for different concerns. 

• Voxelotor is an oral medication that helps prevent red blood cells from becoming sickle-shaped and sticking to each other.
• L-glutamine oral powder (Endari) reduces the incidence of sickle cell crises.
• Crizanlizumab (Adakveo) is administered intravenously to prevent blood cells from binding together, sticking to vessel walls, and causing blockage.
• Hydroxyurea (Droxia, Hydrea, Siklos) increases hemoglobin and reduces the need for transfusions in treating anemia.

These medications often have side effects, so it’s important to have a specialist assess if they are the best treatment option.

Anemia

Healthy red blood cells usually last 120 days, but sickle cells only last for up to 20 days. The shorter turnover creates a red blood cell shortage. With fewer red blood cells, the body receives less oxygen. Patients with sickle cell may experience fatigue, jaundice, difficulty in breathing, elevated heart rate, and developmental delays in children.

Blood transfusions are used to treat anemia in severe cases. However, multiple transfusions pose a risk of iron overload, so additional therapies may be needed to manage iron levels.

Swelling and pain

When sickle cells cause blockages in the veins, these can cause episodes of pain that can last for weeks. According to the Centers for Disease Control and Prevention, this can be prevented by staying hydrated and avoiding temperature extremes and high altitudes. Treatment consists of pain medications like ibuprofen and aspirin, but in severe cases, opioids and hospitalization may be required.

These blockages may also cause the hands and feet to swell, usually accompanied by a fever. The condition is called dactylitis and is generally treated with pain medication and increased fluid intake.

Infection

Patients with sickle cell disease are more vulnerable to infections because sickle cells can damage the spleen. Vaccines play an important role in disease management, including regular childhood vaccines and yearly flu shots. Children and some adult patients may be prescribed to take penicillin daily to reduce the risk of contracting severe infections, particularly if the spleen is removed as a preventive measure.

Though not all sickle cell disease patients may qualify for the curative bone marrow transplant, available treatments can effectively manage symptoms and prevent further complications. 

PicnicHealth is supporting new research on sickle cell disease. Learn more by visiting PicnicHealth.com.