While sickle cell anemia is a lifelong illness and there is no cure for most people with the disease, treatment can help to relieve pain, prevent complications, and prolong life.
Treatment for sickle cell anemia is focused on avoiding and relieving pain and symptoms as well as preventing complications and includes medications, blood transfusions, and, for a small number of select patients, blood and bone marrow transplant (stem cell transplant), which is currently the only cure for sickle cell anemia.
For patients with sickle cell anemia who have had a severe episode of splenic sequestration (trapped sickle cells in the spleen), removal of the spleen (splenectomy) may be necessary.
Medications – Medications used to treat sickle cell anemia include hydroxyurea (Droxia, Hydrea, and Siklos) and l-glutamine oral powder (Endari), cirzanlizumab (Adakyeo), all of which help to decrease the number of pain crises; Voxelotor (Oxybryta), which helps improve anemia; and pain-relieving medications, such as ibuprofen and aspirin or opioids for severe pain.
Children with sickle cell anemia between the ages of 2 months-to-5-years-old may be given penicillin to prevent infections, such as pneumonia, while some adults with sickle cell anemia may need lifelong penicillin, especially if they have had pneumonia or their spleen removed (splenectomy). Children with sickle cell anemia are especially at risk for childhood illness; therefore, vaccinations for pneumonia, meningitis, and an annual flu shot are necessary.
Blood transfusions – Red blood cell transfusions are administered to patients with sickle cell anemia in order to increase the number of normal red blood cells and reduce symptoms and complications.
Patients with sickle cell anemia who have frequent blood transfusions may be given iron chelation therapy to reduce excess iron levels (hemosiderosis) that can damage the liver, heart, pancreas, and other organs.
Bone marrow transplantation (Stem cell transplantation) – In selected patients, usually children with sickle cell anemia who have significant symptoms and complications, bone marrow transplantation, also know as stem cell transplantation, may be performed using healthy bone marrow from a matched donor, such as a sibling, after which immunosuppressant drugs must be taken for life.