what is the most common treatment for beta-thalassemia

• Treatment by Type. Beta thalassemia affects your body's ability to make hemoglobin, a protein that helps your red blood cells carry oxygen to all your organs.
• Blood Transfusions. If you have beta thalassemia major, you'll need blood transfusions every 2 to 4 weeks to raise the number of your red blood cells.
• Chelation Therapy. The blood cells you get during a transfusion have a lot of iron. After you have many transfusions, iron can build up in your body.
• Spleen Removal Surgery. Your spleen filters blood cells. In beta thalassemia, too much iron and too many abnormal red blood cells can collect in that organ and make it swell.
Stem Cell Transplant. If you or your child doesn't get better with blood transfusions and other treatments, your doctor may suggest a stem cell transplant.
Treatments for Complications. Beta thalassemia can cause problems like liver damage, weak bones, and infections. Your doctor will give you medicines and other treatments for these problems if they happen.

What is the best treatment for thalassemia?

Your healthcare provider will figure out the best treatment based on:

• Your age, overall health, and medical history
• How sick you are
• How well you can handle certain medicines, procedures, or therapies
• How long the condition is expected to last
• Your opinion or preference

How do you treat beta thalassemia?

The patients of beta thalassemia, according to doctors, need lifelong care that includes regular blood transfusions and medicines. The disease is quite common, like sickle cell anemia, among the Tharu community.

What is beta thallasemia and is it dangerous?

The homozygous form of beta thalassemia (thalassemia major) is not immediately lethal because there is some production of Hb F, which does not contain beta chains. However, there is severe anemia with complications including progressive enlargement of the liver , spleen and heart and malformation of bones.

How long can you live with thalassemia?

Without treatment, the median survival time for myelodysplastic syndromes range from less than a year to approximately 12 years, depending on factors such as number of chromosome abnormalities and level of red blood cells. However, treatment is often successful, especially for certain types of this condition.

What are some treatments for beta thalassemia?

Treatment for beta thalassemia may include:Regular blood transfusions.Medications (to decrease amount of iron in the body, called chelation therapy)Surgical removal of the spleen (if necessary)Daily doses of folic acid.Possible surgical removal of the gallbladder.No iron supplements.Bone marrow transplantation.

How is β thalassemia currently treated?

Stem cell or bone marrow transplants are the only cure for thalassaemia, but they're not done very often because of the significant risks involved. Stem cells are produced in bone marrow, the spongy tissue found in the centre of some bones, and have the ability to develop into different types of blood cells.

What is the best medicine for thalassemia?

To help rid your body of the extra iron, you might need to take an oral medication, such as deferasirox (Exjade, Jadenu) or deferiprone (Ferriprox). Another drug, deferoxamine (Desferal), is given by needle. Stem cell transplant.

What is the definitive cure for beta thalassemia major?

HSCT is currently the only treatment shown to provide an effective, definitive cure for β-thalassemia.

Are there any treatment or cure for thalassemia?

Bone marrow and stem cell transplant from a compatible related donor is the only treatment to cure thalassemia.

How is thalassemia treated in adults?

To prevent iron overload, people with thalassemia may need chelation therapy, which is when doctors give a medicine – either a pill or a shot under the skin – to remove excess iron before it builds up in the organs.

What is the fastest way to increase red blood cells?

5 nutrients that increase red blood cell countsred meat, such as beef.organ meat, such as kidney and liver.dark, leafy, green vegetables, such as spinach and kale.dried fruits, such as prunes and raisins.beans.legumes.egg yolks.

Which medicine should be avoided in thalassemia?

Some drugs, such as aspirin and throat lozenges, as well as certain 'health foods', may contain vitamin C and should be avoided.

Can thalassemia take iron tablets?

If you have thalassemia trait, your physician may prescribe iron supplements because your red blood cells are smaller than is typical. Unless you also have iron deficiency in addition to thalassemia, this is not needed and should be avoided.

Can beta thalassemia minor turn to major?

One mutated gene, you'll have mild signs and symptoms. This condition is called thalassemia minor or beta-thalassemia. Two mutated genes, your signs and symptoms will be moderate to severe. This condition is called thalassemia major, or Cooley anemia.

Is beta thalassemia rare?

Beta thalassemia is relatively rare in the United States, but is one of the most common autosomal recessive disorders in the world. The incidence of symptomatic cases is estimated to be approximately 1 in 100,000 individuals in the general population.

Which thalassemia is most common?

Frequency. Beta thalassemia is a fairly common blood disorder worldwide. Thousands of infants with beta thalassemia are born each year. Beta thalassemia occurs most frequently in people from Mediterranean countries, North Africa, the Middle East, India, Central Asia, and Southeast Asia.

How do you treat sickle beta thalassemia?

Education is essential to provide supportive and preventive care. May require Hydroxyurea if major complications occur. The only cure for Sickle Cell disease is bone marrow transplantation; gene therapy will be a curative therapy and will be coming available soon.

How is transfusion dependent thalassemia treated?

Hematopoietic stem cell allogenic transplant is the only approved cure for β-thalassemia; however, it is still limited by clinical conditions and the availability of matched donors as well as by potential graft-versus-host disease (GVHD).

Can someone with beta thalassemia could be treated with EPO?

Because of its stimulating effect on RBC production, erythropoietin (Epo) is used to treat anemia, for example, in patients on dialysis or on chemotherapy. In β-thalassemia, where Epo levels are low relative to the degree of anemia, Epo treatment improves the anemia state.

Can you treat thalassemia with iron?

People with thalassaemia may be advised to avoid iron supplements because they are at risk of iron overload. Carriers of the thalassaemia gene may be asymptomatic but pregnancy can precipitate anaemia and there is a chance that this may be inappropriately treated with iron replacement.

If I Have Thalassemia, How Does It Affect My body?

Since your body has fewer red blood cells when you have thalassemia, you may have symptoms of a low blood count, or anemia. When you have anemia, y...

How Is Thalassemia Treated?

The type of treatment a person receives depends on how severe the thalassemia is. The more severe the thalassemia, the less hemoglobin the body has...

How Do Blood Transfusions Affect My body?

People who receive a lot of blood transfusions are at risk for iron overload. Red blood cells contain a lot of iron, and over time, the iron from a...

How often do you need blood transfusions for beta thalassemia?

If you have beta thalassemia major, you'll need blood transfusions every 2 to 4 weeks to raise the number of your red blood cells. Some people with this type of beta thalassemia need transfusions only at certain times, such as when they have an illness or infection.

How many types of beta thalassemia are there?

There are three types of beta thalassemia, and each has a different treatment.

What age does beta thalassemia start?

Beta thalassemia major is the most severe type. It often starts by age 2 and causes severe anemia and other serious symptoms. Treatment involves regular blood transfusions. If your child has beta thalassemia major or intermedia, make sure to reach out to family and friends for the emotional backing you need.

What is a chelation pill?

Chelation therapy is a treatment that removes the additional iron from your body. You'll get a pill or shot of a medicine like deferasirox (Exjade, Jadenu), deferiprone (Ferriprox), or deferoxamine (Desferal). These drugs bind to iron in your body, which then leaves your body when you pee or have a bowel movement.

Why do people need blood transfusions?

Beta thalassemia intermedia causes more serious anemia. Some people will need blood transfusions to boost their red blood cell count. Beta thalassemia major is the most severe type.

What organs are affected by thalassemia?

Your spleen filters blood cells. In beta thalassemia, too much iron and too many abnormal red blood cells can collect in that organ and make it swell. An enlarged spleen can cause your hemoglobin levels to drop even lower, plus lower the number of blood cells called platelets that help your blood clot.

What is it called when you have too few red blood cells?

Too few red blood cells is called anemia. Without enough oxygen, you'll feel tired and weak.

Why is iron chelation therapy necessary?

Therefore, iron chelation therapy is also necessary to remove the excess iron before irreversible damage to internal organs occurs. A splenectomy is often recommended for patients with β-thalassemia major, because it decreases the number of blood transfusions that may be required.

Is thalassemia major a blood transfusion?

Ongoing treatment is required in individuals suffering from β-thalassemia major. Routine blood transfusions are necessary to maintain hemoglobin levels at 9-10g/dL. However, this treatment can also lead to further complications due to an excess of iron.

Can a person with -thalassemia intermedia have a blood transfusion?

β-Thalassemia Intermedia. People that have β-thalassemia intermedia may require blood transfusions and iron chelation therapy, depending on the severity of their symptoms. Individuals with β-thalassemia intermedia are also at higher risk of developing blood clots, especially during surgery.

How is thalassemia treated?

Hear Robert’s tips on how to successfully transition to adult care for thalassemia.

What is the best vitamin for thalassemia?

Many times people with thalassemia are prescribed a supplemental B vitamin, known as folic acid, to help treat anemia. Folic acid can help red blood cells develop. Treatment with folic acid is usually done in addition to other therapies.

Why is thalassemia considered immunocompromised?

Because of this, people with thalassemia are said to be “immunocompromised,” which means that some of the body’s defenses against infection aren’t working. When you are immunocompromised, it is easier for you to get infections and you sometimes need extra protection, like flu shots and other vaccines.

How to tell if you have thalassemia?

Since your body has fewer red blood cells when you have thalassemia, you may have symptoms of a low blood count, or anemia. When you have anemia, you might feel tired or weak. You might also experience: 1 Dizziness 2 Shortness of breath 3 A fast heart beat 4 Headache 5 Leg cramps 6 Difficulty concentrating 7 Pale skin

Can thalassemia cause low blood count?

Please try again later. Since your body has fewer red blood cells when you have thalassemia, you may have symptoms of a low blood count, or anemia. When you have anemia, you might feel tired or weak. You might also experience: Your body will try very hard to make more red blood cells.

Do people with thalassemia need blood transfusions?

People with thalassemia minor or trait usually do not need blood transfusions because they either do not have anemia or have only a mild anemia.

How to manage thalassemia?

You can help manage your thalassemia by following your treatment plan and adopting healthy-living habits. Avoid excess iron. Unless your doctor recommends it, don't take vitamins or other supplements that contain iron. Eat a healthy diet. Healthy eating can help you feel better and boost your energy.

What is the best treatment for thalassemia in children?

Stem cell transplant. Also called a bone marrow transplant, a stem cell transplant might be an option in some cases. For children with severe thalassemia, it can eliminate the need for lifelong blood transfusions and drugs to control iron overload.

How to tell if a child has thalassemia?

If your doctor suspects your child has thalassemia, he or she can confirm a diagnosis with blood tests. Blood tests can reveal the number of red blood cells and abnormalities in size, shape or color.

When should a baby be tested for thalassemia?

Testing can be done before a baby is born to find out if he or she has thalassemia and determine how severe it might be. Tests used to diagnose thalassemia in fetuses include:

Can thalassemia cause excess iron?

Some people with thalassemia who don't have regular transfusions can also develop excess iron. Removing the excess iron is vital for your health. To help rid your body of the extra iron, you might need to take an oral medication, such as deferasirox (Exjade, Jadenu) or deferiprone (Ferriprox).

How to cure thalassemia major?

The long-term hope is that thalassemia major will be cured by insertion of the normal beta-chain gene through gene therapy or by another modality of molecular medicine.

What is beta thalassemia?

People with thalassemia make defective hemoglobin, the compound in blood that carries oxygen.

What is the most common type of thalassemia?

The most familiar type of thalassemia is beta thalassemia. It involves decreased production of normal adult hemoglobin (Hb A), the predominant type of hemoglobin from soon after birth until death. (All hemoglobin consists of two parts: heme and globin). The globin part of Hb A has 4 protein sections called polypeptide chains. Two of these chains are identical and are designated the alpha chains. The other two chains are also identical to one another but differ from the alpha chains and are termed the beta chains. In persons with beta thalassemia, there is reduced or absent production of beta globin chains.

What is the difference between thalassemia minor and major?

There are two forms of beta thalassemia. They are thalassemia minor and thalassemia major (which is also called Cooley's anemia ).

What is Mediterranean anemia?

Because of this, thalassemia major has been called Mediterranean anemia.

How does thalassemia affect a newborn?

Infants with thalassemia major are well at birth because of a special form of hemoglobin present in the fetus and newborn. Eventually, however, this hemoglobin is replaced by defective hemoglobin. Symptoms emerge late in the first year of life. The child develops pale skin, irritability, growth retardation, swelling of the abdomen due to enlargement of the liver and spleen (hepatosplenomegaly) with jaundice. This is associated with severe anemia with rupture of the red blood cells (hemolytic anemia). The child with thalassemia major becomes dependent on blood transfusions and, although they do help, they create further problems including iron overload. Folic acid supplementation is often given. At this time, the primary treatments are directed at relieving symptoms of the illness. Selected patients may qualify for bone marrow or stem cell transplants. Gene therapy remains a potential treatment for the future.

Why is Cooley's anemia named Cooley's anemia?

At birth the baby with thalassemia major seems entirely normal. This is because the predominant hemoglobin at birth is still fetal hemoglobin (HbF). HbF has two alpha chains (like Hb A) and two gamma chains (unlike Hb A). It has no beta chains so the baby is protected at birth from the effects ...

What is the phase 1 trial for beta thalassemia?

A phase I clinical trial using autologous CD34 + hematopoietic progenitor cells transduced with a lentiviral vector encoding the normal human beta-globin gene for treatment of beta thalassemia major is currently under way ( NCT01639690 ).

When was gene therapy first used for thalassemia?

Since the first successful gene therapy for thalassemia major, in 2007, researchers have worked to improve the efficacy and safety of the procedure. [ 14, 18, 19] In this process, autologous hematopoietic stem cells (HSCs) are harvested from the patient and then genetically modified with a lentiviral vector expressing a normal globin gene. After the patient has undergone appropriate conditioning therapy to destroy existing HSCs, the modified HSCTs are reinfused into the patient. Clinical trials of gene therapy for thalassemia are currently recruiting participants. [ 20, 21]

What drugs are used to increase fetal hemoglobin?

The two most widely studied drugs in this area are butyrates and hydroxyurea. [ 24] More recently, new therapeutic targets have been reported, such as BCL11A, which regulates fetal hemoglobin expression. [ 25, 26]

What is the goal of hypertransfusional support?

The goal of long-term hypertransfusional support is to maintain the patient's hemoglobin level at 9-10 g/dL, thus improving his or her sense of well being while simultaneously suppressing enhanced erythropoiesis. This strategy treats the anemia and suppresses endogenous erythropoiesis so that extramedullary hematopoiesis and skeletal changes are suppressed. Patients receiving long-term transfusion therapy also require iron chelation. (See Medication .)

Does thalassemia minor require treatment?

Thalassemia minor. Patients with thalassemia minor usually do not require any specific treatment. Inform patients that their condition is hereditary and that physicians sometimes mistake the disorder for iron deficiency.

Can thalassemia cause iron deficiency?

Patients with thalassemia minor usually do not require any specific treatment. Inform patients that their condition is hereditary and that physicians sometimes mistake the disorder for iron deficiency. Some pregnant patients with the beta thalassemia trait may develop concurrent iron deficiency and severe anemia; they may require transfusional support if they are not responsive to iron repletion modalities.

Is patient activity limited secondary to severe anemia?

Patient activity may be limited secondary to severe anemia.

Diagnosis

Treatment

Lifestyle and Home Remedies

• You can help manage your thalassemia by following your treatment plan and adopting healthy-living habits. 1. Avoid excess iron.Unless your doctor recommends it, don't take vitamins or other supplements that contain iron. 2. Eat a healthy diet. Healthy eating can help you feel better and boost your energy. Your doctor might also recommend a folic ac...

See more on mayoclinic.org

Coping and Support

Preparing For Your Appointment