Regular blood transfusions help prevent complications, including stroke. Medications can also ease pain episodes and reduce infections. They include hydroxyurea (hy-drahk-see-uh), voxelotor, and L-glutamine therapy.
People with sickle cell disease have a faulty gene that makes red blood cells stuck in small blood vessels, blocking their flow and depriving the body of oxygen. This can cause anemia and severe, painful episodes called pain crises.
Symptoms
The abnormal sickle-shaped red blood cells block the flow of oxygen and nutrients to tissues. This causes extreme pain episodes, known as crises. These can happen anywhere in the body but are most common in the arms, abdomen, and chest. The pain can be described as sharp, intense, or stabbing and is often more painful than surgery or childbirth. It can also be accompanied by fever, fatigue, and shortness of breath.
Children with sickle cell disease tend to become iron-overloaded (too much iron). This can damage the organs, particularly the liver, kidneys, and brain. Your doctor will recommend medication to reduce your child’s chance of experiencing this. They may prescribe hydroxycarbamide or crizanlizumab, both given by injection. These medicines prevent the sickle-shaped red blood cells from sticking to blood vessels, which reduces pain crises.
Some people with severe sickle cell disease need a blood transfusion regularly. This helps to prevent complications such as infections and a condition called hematologic crisis, which occurs when large numbers of red blood cells are destroyed quickly, leaving your body with low levels of hemoglobin or the protein that carries oxygen throughout the body. Treatment options for hematologic crises include pain medicines, IV fluids, and treatments that help prevent or treat the infection.
Treatment
Medications, blood transfusions, and other treatments can help reduce pain episodes, treat complications, and prevent problems that arise over time. People with sickle cell disease may also need regular visits with a hematologist or doctor specializing in blood diseases.
Sickle-shaped red cells can stick together and block blood flow. This limits oxygen and can cause severe, painful attacks called crises. These crises usually start in the chest and can last hours or days. They can also occur in the bones, joints, and spleen. People with sickle cell disease are at high risk for a stroke that can cause brain damage or death. The condition can also lead to eye damage, delayed growth, and puberty in children.
Treatment options include blood transfusions, antibiotics to fight infections, and medicines to reduce symptoms. Hydroxyurea (Droxia, Hydrea, Siklos) cuts the frequency of pain crises and can help reduce hospitalizations. A newer drug, crizanlizumab, is an injection into a vein that relieves pain crises. It can be given on its own or in combination with hydroxyurea.
It is essential for people who have sickle cell disease to follow their doctor’s advice about healthy living and take all prescribed medications. They should avoid too much heat or cold, get enough rest, and drink plenty of fluids. Support from a family member or sickle cell support groups can be helpful. People who want to have children can talk to a genetic counselor to learn about the risk and options for prenatal testing.
Prevention
Symptoms of sickle cell disease occur because of a lack of blood that prevents vital organs from getting the oxygen they need. Over time, this can cause severe problems like cognitive impairment, stroke, kidney failure, and early-onset osteoporosis. The average life expectancy for people with sickle cell is in their mid-40s.
Sickle-shaped red cells can get stuck in blood vessels, blocking their exit and causing pain crises that last hours or days. They can be triggered by cold weather, dehydration, illness, or stress and may be aggravated by changes in altitude or environment. A person might have one or many crisis episodes each year.
Other complications include leg ulcers (painful open sores), gallstones, and decreased energy. Kids with sickle cell anemia are also more likely to have a life-threatening episode called splenic sequestration crisis, in which the spleen traps many abnormal red blood cells and enlarges. Symptoms include paleness, extreme tiredness, and belly pain.
Medications used to treat sickle cell anemia can reduce the frequency of pain crises and other symptoms. These medications include antibiotics to fight infections and medicines to reduce pain during situations. A drug called hydroxyurea (Droxia, Hydrea, Siklos) cuts the number of painful crises and can help reduce the need for blood transfusions. Other drugs that ease pain during situations include voxlelotor and L-glutamine oral powder (Endari). Bone marrow transplants can cure sickle cell anemia in some children and teens.
Life expectancy
Historically, sickle cell disease has been an extremely deadly disease. Few patients survive beyond childhood. Today, with improved standards of medical care, life expectancy for people with sickle cell disease is increasing significantly.
However, this improvement masks significant differences in survival rates between countries and among different ethnic groups and the impact of symptom severity at the time of death. This is why it’s essential to have accurate knowledge of contemporary mortality in sickle cell disease, including what factors are associated with early death and the clinical conditions that increase an individual’s risk.
Many patients with sickle cell disease die due to a painful episode or complications such as acute chest syndrome, stroke, or infection (Escherichia coli, Staphylococcus aureus, hepatitis C virus, tuberculosis, and human immunodeficiency virus). A significant number of these deaths occur in relatively healthy patients who do not have overt chronic organ failure.
Various things trigger these episodes, including colds, stress, illness, or dehydration. They can last a few hours or days and cause back legs, arms, or chest pain. If the symptoms are severe, they can lead to hospitalization. People with sickle cell disease are also at increased risk of developing jaundice caused by a buildup of yellow pigment hemoglobin in the blood.
