Treatment Options For Thalassemia

October 26, 2023

Thalassemia is an inherited blood disorder common in Asia, Africa, and the Mediterranean. This condition occurs when there are fewer red blood cells and hemoglobin (what allows the red blood cells to carry oxygen) in an individual's body than what is considered normal. The genetic abnormality that causes thalassemia is due to two genes and results in a deficiency of beta globulins, an essential part of the hemoglobin molecule, which transports oxygen. This culminates in markedly reduced or no hemoglobin in the red blood cells.

Thalassemia minor is usually mild anemia with up to a fifty percent reduction in hemoglobin. Individuals in this state are generally able to lead fairly normal lives. In fact, this carrier state protects the patient from malaria, which is endemic in those areas of the world. Thalassemia major results in severe anemia, which must be treated. A child with symptomatic beta-thalassemia is pale, sometimes jaundiced, with deformed, often decayed teeth, and a bumpy skull. The spleen and liver will often be enlarged, and the patient may also be in heart failure. Without treatment, most children with this condition will not live past their teenage years, which is why treatment is crucial.

With all of this in mind, get to know the major approaches to treating thalassemia now.

Observational Approach

Neonatal screening, especially in high-risk populations, targets the children who need to be watched. Some states do not require screening for hemoglobinopathies, and immigrant children may be missed as well. An observational approach will be taken with identified children, sometimes for years. Observation is usually the first line of treatment for an individual with a minor case of thalassemia.

During this time, a patient's growth and development, glucose and thyroid metabolism, iron levels, and potential dental problems will be monitored. The chronic anemia causes short stature and delay of puberty. Iron overload is a major problem and can affect the development of the thyroid and pancreas. Other major concerns during this time are the onset of pulmonary hypertension, heart failure, and osteoporosis. Cardiac evaluation with an MRI is recommended every year, starting eight or nine years old. Supplementing with folic acid is recommended.

Learn how to effectively treat thalassemia with food now.

Avoiding Too Much Iron

Excessive iron absorption is a major problem in thalassemia patients, so avoiding too much iron is essential. Iron absorption will increase in anyone with anemia. The body recognizes the anemia, and the gut increases its transport of iron into the bloodstream, where it is stored in the liver. Too much iron, however, damages the liver and causes cirrhosis. Avoiding too much iron is an important part of the treatment for thalassemia since transfusions also import iron into the patient.

A low iron diet is essential for affected individuals. Parents and children are told to stay away from red meats, pork, most cereals, peanut butter, leafy green vegetables, and many other foods normally considered healthy. Oranges and orange juice facilitate the absorption of iron in the gut. Most commercial bread has extra iron added as well. Because of the prevalence of iron in food, individuals are advised to read the labels on products to determine how much iron they contain. Children need to be taught appropriate eating habits very early as well.

Speaking of eating habits, learn more about how they can help treat thalassemia.

Maintaining A Healthy Diet

Despite these mentioned limitations, it is essential for thalassemia patients to get adequate nutrition. Maintaining a healthy diet is somewhat difficult, but there are still a few options. Milk and dairy products are encouraged since calcium helps lessen iron absorption. Chicken and turkey, as well as most vegetables and fruits, are all important in maintaining a healthy diet as well. Parents will need to become quite knowledgeable about their child's dietary needs. Consultation with a dietitian is critical and should be part of the parents' and the child's early education.

Reveal the next treatment option for thalassemia when it goes beyond food now.

Blood Transfusions

One of the hallmark ways to treat serious cases of thalassemia is through blood transfusions. The hemoglobin level helps determine when to start blood transfusions, but when the level falls and stays below six to seven grams per deciliter (g/dL), the patient has usually already become symptomatic. The best time to start chronic transfusion therapy is before the affected individual reaches this stage. Maintaining the hemoglobin level above nine to ten g/dL can usually be achieved with transfusions every three to four weeks. Complications from blood transfusion therapy include iron overload, infection, and graft vs. host disease.

Discover another considerable thalassemia treatment, and potential cure, now.

Stem Cell Transplants

Thalassemia can be treated with blood transfusions, but it can only be cured by stem cell transplants. All human cells arise from stem cells. Stem cells are primordial, able to become many different kinds of cells as the fetus grows. There are two types of stem cells: adult and embryonic. Adult stem cells can be obtained from bone marrow and from the blood in umbilical cords, which is routinely collected and banked in many places now. Embryonic stem cells are obtained from embryos, usually those leftover from procedures done at fertility clinics.

Obtaining adult stem cells generally harms no one, though obtaining embryonic stem cells destroys the embryo, which leads to ethical questions. Stem cell transplants are complex and fraught with dangers to the patient. The patient's bone marrow, which produces abnormal cells, must be destroyed and then replaced with the stem cells. Infection and host versus graft disease are the major concerns, both of which can be fatal. However the chances for a cure for thalassemia, even in adults, is usually far greater than the risks of the procedure. Cure rates are much higher in children with thalassemia than in adults (sixty-five percent) who have more the advanced disease.

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