Various treatment options for thalassemia

Thalassemia is a hereditary blood disorder that makes the body unable to produce hemoglobin (Hb). As a result, they will experience anemia. The thing that often becomes the question is, can this disease be cured? If possible, what are the treatment or drug options for thalassemia sufferers?

Can thalassemia be cured?

The cause of thalassemia is a genetic mutation in the body. This means that someone born in a family carrying this mutated gene is at greater risk of developing thalassemia. So, because this is a hereditary disease, can thalassemia be cured?

The answer, of course you can. It's just that, the choices are very limited and have a high risk. Until now, the only treatment that has been able to completely treat thalassemia is a bone marrow transplant.

Unfortunately, it is not easy to find a suitable bone marrow donor. Additionally, bone marrow transplant procedures (BMT / Bone Marrow Transplant) is still considered very risky.

According to the National Heart, Lung, and Blood Disease website, experts are still looking for other remedies that might treat and cure thalassemia. For example, it is possible that in the future there will be technology capable of inserting the normal hemoglobin gene into stem cells in bone marrow.

In this way, the body of a thalassemia sufferer is expected to be able to produce healthy red blood cells and hemoglobin.

Researchers are also studying ways to stimulate a person's ability to make fetal hemoglobin after birth. This type of hemoglobin is found in fetuses and newborns.

After birth, the body switches to making adult hemoglobin. Making more fetal hemoglobin may compensate for the lack of healthy adult hemoglobin.

What are the available treatments for thalassemia?

Treatment depends on the type of thalassemia and the severity suffered by each patient.

People with thalassemia minor, either alpha or beta, usually only show mild symptoms of thalassemia, or even no symptoms at all. People with this condition may need little or no treatment.

For cases of severe and mild thalassemia, doctors recommend three types of standard treatment, namely blood transfusions, iron chelation therapy (iron chelation therapy), and folic acid supplements. Other types of treatment that have been developed or are in the process of being tested are used less frequently.

Treatment options for thalassemia

The following are some treatment options that can be undertaken by thalassemia sufferers:

1. Blood transfusions

Red blood cell transfusions are the mainstay of treatment for people who have moderate or severe thalassemia. This treatment can increase the number of healthy red blood cells with normal hemoglobin.

During a blood transfusion, a needle is used to insert an intravenous (IV) into one of the blood vessels, until healthy blood enters the body. This procedure usually takes 1-4 hours.

Red blood cells only last about 120 days. Therefore, you may need to have repeated transfusions to maintain a healthy supply of red blood cells.

For people with hemoglobin H or beta thalassemia intermedia, you may need blood transfusions under certain conditions, for example when you have an infection or other illness, or when you have severe anemia that causes fatigue.

For those with beta thalassemia major (Cooley's anemia), you may need regular blood transfusions (every 2-4 weeks). This transfusion will help you maintain normal levels of hemoglobin and red blood cells.

Blood transfusions can help you feel better, enjoy daily activities, and live a normal life. Although very useful for mental safety, this treatment is expensive and carries the risk of transmitting infections and viruses (such as hepatitis). Even so, the risk of transmitting infections and viruses is very low in the United States because it has gone through strict blood screening.

2. Iron chelation therapy

Hemoglobin in red blood cells is a protein that is rich in iron. Through regular transfusions, iron in the blood will accumulate in certain organs, such as the liver, heart and other organs. This condition is called iron overload or iron overload .

To prevent this damage, doctors use iron chelation therapy to remove excess iron from the body. The two main drugs used in iron chelation therapy for thalassemia are:

• Deferoxamine is a liquid thalassemia medication that is given slowly under the skin, usually via a small portable pump that is used overnight. Thalassemia treatment takes time and is a little painful. The side effects of this drug in people with thalassemia are vision and hearing problems.
• Deferasirox is a pill that is taken once a day. The side effects of this thalassemia drug are headache, nausea (stomach discomfort), vomiting, diarrhea, joint pain, and fatigue.

3. Folic acid supplements

Folic acid is a type of B vitamin that plays a role in the formation of healthy red blood cells. Doctors recommend folic acid supplements in addition to blood transfusion medication and / or iron chelation therapy.

4. Blood and bone marrow transplant

This treatment for thalassemia has actually been developed and is going through the testing phase. However, for Indonesia itself, bone marrow transplant, aka BMT (bone marrow transplant) is done less frequently.

Blood and marrow stem cell transplants are performed to replace damaged stem cells with healthy ones from a donor. Stem cells (stem cells) are cells in the bone marrow that play a role in the production of red blood cells and other types of blood cells.

Stem cell transplant is the only treatment that can cure thalassemia. However, only a few patients with severe thalassemia were able to find a suitable donor.

Current treatment allows moderate and severe thalassemia patients to live longer. However, they have to deal with complications of thalassemia that may occur from time to time.

An important part of treating thalassemia is treating its complications. Complication treatment may be needed to treat heart problems or liver disease, infections, osteoporosis, and other health problems.