Sickle cells are destroyed rapidly in the body of people with the disease causing anemia, jaundice and the formation of gallstones.
The sickle cells also block the flow of blood through vessels resulting in lung tissue damage (acute chest syndrome), pain episodes (commonly in the arms, legs, chest and abdomen), stroke and priapism (painful prolonged and unwanted erection). It also causes damage to most organs including the spleen, kidneys, eyes and liver. Damage to the spleen makes sickle cell disease patients, especially young children, easily infected by certain bacterial. Therefore, any fever in someone with sickle cell disease must be taken very seriously.
Treatment
Health maintenance for patients with sickle cell disease starts with early diagnosis, preferably in the newborn period and includes penicillin prophylaxis, vaccination against pneumococcus bacteria and folic acid supplementation.
Treatment of complications often includes antibiotics, pain management, intravenous fluids, blood transfusion and surgery all backed by psychosocial support. Like all patients with chronic disease patients are best managed in a comprehensive multi-disciplinary program of care.
Blood Transfusions
Blood transfusions help benefit sickle cell disease patients by reducing recurrent pain crises, risk of stroke and other complications. Because red blood cells contain iron, and there is no natural way for the body to eliminate it, patients who receive repeated blood transfusions can accumulate iron in the body until it reaches toxic levels. It is important to remove excess iron from the body, because it can gather in the heart, liver, and other organs and may lead to serious organ damage and even death. Treatments are available to eliminate iron overload (see below).
Hydroxyurea for treating Sickle Cell Disease
A single randomized clinical trial of 299 patients with SCD demonstrated that compared to placebo, hydroxyurea was associated with lower annual rates of pain crises, pain episodes of acute chest syndrome and need for transfusions. Hydroxyurea was first approved by the FDA for SCD in 1998 and is now available for adults and children with sickle cell anemia.
Transfusional Iron Overload
Patients with sickle-cell disease (SCD) receiving chronic transfusions of red blood cells are at risk of developing transfusional iron overload over time. Transfusional iron overload is characterized by an increase of labile plasma iron (i.e., non-transferrin bound iron) in the body, which can lead to functional impairment in vital organs. The organs that are at risk of damage due to iron overload include the liver, heart, pancreas, thyroid, pituitary gland, and other endocrine organs. Buildup of labile plasma iron in these organs can lead to hepatic cirrhosis, cardiomyopathy, diabetes mellitus, hypoparathyroidism, impaired growth, infertility, hypogonadism and even death. The body does not have a way to get rid of iron received from blood transfusions. An agent that chelates iron is therefore needed to get rid of excess iron in the body. There are two agents. One agent is given intravenously and the other is by mouth.