Conventional Medical Treatment for Sickle Cell Disease
What is sickle cell disease?
Sickle cell disease is an inherited red blood cell disorder that causes hemoglobin to become irregularly shaped. Hemoglobin is a protein in red blood cells that delivers oxygen throughout the body. Normal red blood cells with hemoglobin are smooth, flexible and round. Sickle cell disease results in hemoglobin evolving into a sickle shape. They also become stiff and stick together, causing difficulty moving through blood vessels. Sickle cell disease can cause blockages in blood flow, resulting in anemia, pain, infections, organ failure, etc.
Conventional medical treatment for sickle cell disease
Treatment for sickle cell disease will depend on many factors, including age and overall health. It is important to begin treatment as soon as possible; early treatment can help prevent organ damage, strokes, and infection. Conventional medical treatment for sickle cell disease may include medications, treating infections, transfusions, bone marrow transplant, and gene therapy.
A physician may prescribe pain medications for acute or chronic pain. This could include prescription-strength NSAIDs, corticosteroids or opioids, depending on the level of pain experienced. Other medications that help in the prevention of a pain crisis may include the following:
- Hydroxyurea is a medication used to decrease the occurrences of pain and acute chest syndrome. It can also lower the need for blood transfusions and reduce organ damage.
- Crizanlizumab can prevent sickle cells from clumping together. This helps blood flow and decreases the likelihood of a pain crisis.
- L-Glutamine is an amino acid that helps reduce pain and infections. Although the body produces this amino acid, additional amounts are needed for those with sickle cell disease due to increased rates of pain.
- Voxelotor prevents red blood cells from easily breaking down. It also strengthens their ability to carry oxygen.
Infections can occur frequently when sickle cell disease is present. Treatment of infections include the following:
- Vaccines, as a preventative against infections
Blood can be used to treat symptoms and prevent complications of sickle cell disease. Blood transfusions that may be beneficial include the following:
- Acute transfusions treat anemia or an acute stroke. They are also used to prevent surgical complications.
- Red blood cell transfusions increase the amount of normal red blood cells.
- Ongoing transfusions can reduce the risk of a stroke.
Bone marrow transplant
Stem cell therapy via bone marrow transplant offers a potential cure for individuals with sickle cell disease. This treatment is typically done in youth, as risk increases with age.
Research is ongoing into the use of gene therapy for the treatment of sickle cell disease. These therapies would modify hematopoietic stem cells for those who cannot secure a bone marrow match. The modified cells would then be injected into the blood in order to reach the bone marrow. The availability of this treatment is currently unknown.