What Is ThalassemiaWhat Is Thalassemia?? Thalassemia is an inherited blood disorder Thalassemia is an inherited blood disorder

that causes mild or severe that causes mild or severe anemiaanemia (uh-NEE- (uh-NEE-me-uh). The anemia is due to reduced me-uh). The anemia is due to reduced hemoglobin (HEE-muh-glow-bin) and fewer hemoglobin (HEE-muh-glow-bin) and fewer red blood cells than normal. Hemoglobin is red blood cells than normal. Hemoglobin is the protein in red blood cells that carries the protein in red blood cells that carries oxygen to all parts of the body.oxygen to all parts of the body.

In people with thalassemia, the genes that In people with thalassemia, the genes that code for hemoglobin are missing or code for hemoglobin are missing or variant (different than the normal genes). variant (different than the normal genes). Severe forms of thalassemia are usually Severe forms of thalassemia are usually diagnosed in early childhood and are diagnosed in early childhood and are lifelong conditions. lifelong conditions.

The two main types of thalassemiaThe two main types of thalassemia

alpha and beta, are named for the two protein alpha and beta, are named for the two protein chains that make up normal hemoglobin. The chains that make up normal hemoglobin. The genes for each type of thalassemia are passed genes for each type of thalassemia are passed from parents to their children. Alpha and beta from parents to their children. Alpha and beta thalassemias have both mild and severe forms. thalassemias have both mild and severe forms.

Alpha thalassemiaAlpha thalassemia occurs when one or more of the four genes occurs when one or more of the four genes

needed for making the alpha globin chain of needed for making the alpha globin chain of hemoglobin are variant or missing. Moderate hemoglobin are variant or missing. Moderate to severe anemia results when more than two to severe anemia results when more than two genes are affected. The most severe form of genes are affected. The most severe form of alpha thalassemia is known as alpha alpha thalassemia is known as alpha thalassemia major. It can result in miscarriage.thalassemia major. It can result in miscarriage.

Beta thalassemia Beta thalassemia occurs when one or both of the two genes occurs when one or both of the two genes

needed for making the beta globin chain of needed for making the beta globin chain of hemoglobin are variant. The severity of illness hemoglobin are variant. The severity of illness depends on whether one or both genes are depends on whether one or both genes are affected and the nature of the abnormality. If affected and the nature of the abnormality. If both genes are affected, anemia can range from both genes are affected, anemia can range from moderate to severe. The severe form of beta moderate to severe. The severe form of beta thalassemia is also known as Cooley’s anemia. thalassemia is also known as Cooley’s anemia. Cooley’s anemiaCooley’s anemia is the most common severe is the most common severe form of thalassemia in the United States.form of thalassemia in the United States.

Alpha ThalassemiasAlpha Thalassemias Alpha thalassemia “silent carrier” Alpha thalassemia “silent carrier” Mild alpha thalassemia, also called alpha Mild alpha thalassemia, also called alpha

thalassemia minor or alpha thalassemia trait thalassemia minor or alpha thalassemia trait Hemoglobin H disease Hemoglobin H disease Hydrops fetalis, or alpha thalassemia major Hydrops fetalis, or alpha thalassemia major

Beta ThalassemiasBeta Thalassemias Beta thalassemia minor, also called Beta thalassemia minor, also called

thalassemia minor or thalassemia trait thalassemia minor or thalassemia trait Beta thalassemia intermedia, also called Beta thalassemia intermedia, also called

thalassemia intermedia or mild Cooley’s thalassemia intermedia or mild Cooley’s anemia anemia

Beta thalassemia major, also called Beta thalassemia major, also called thalassemia major or Cooley’s anemia thalassemia major or Cooley’s anemia

Mediterranean anemia Mediterranean anemia

Cooley ’s anemiaCooley ’s anemia Cooley’s anemiaCooley’s anemia is another name for the is another name for the

severe form of beta thalassemia. The name is severe form of beta thalassemia. The name is sometimes used to refer to any type of sometimes used to refer to any type of thalassemia that requires treatment with thalassemia that requires treatment with regular blood transfusions.regular blood transfusions.

Thalassemia is caused by variant or missing Thalassemia is caused by variant or missing genes that affect how the body makes genes that affect how the body makes hemoglobin. Hemoglobin is the protein in hemoglobin. Hemoglobin is the protein in red blood cells that carries oxygen. People red blood cells that carries oxygen. People with thalassemia make less hemoglobin and with thalassemia make less hemoglobin and fewer circulating red blood cells than fewer circulating red blood cells than normal. The result is mild or severe normal. The result is mild or severe anemiaanemia

Many possible combinations of variant genes Many possible combinations of variant genes cause the various types of thalassemia. cause the various types of thalassemia. Thalassemia is always inherited (passed from Thalassemia is always inherited (passed from parents to children). People with moderate to parents to children). People with moderate to severe forms of thalassemia received variant severe forms of thalassemia received variant genes from both parents. A person who inherits a genes from both parents. A person who inherits a thalassemia gene or genes from one parent and thalassemia gene or genes from one parent and normal genes from the other parent is a carrier normal genes from the other parent is a carrier (thalassemia trait). Carriers often have no signs of (thalassemia trait). Carriers often have no signs of illness other than mild anemia, but they can pass illness other than mild anemia, but they can pass the variant genes on to their children. the variant genes on to their children.

Hemoglobin includes two kinds of protein Hemoglobin includes two kinds of protein chains called alpha globin chains and beta chains called alpha globin chains and beta globin chains. If the problem is with the alpha globin chains. If the problem is with the alpha globin part of hemoglobin, the disorder is alpha globin part of hemoglobin, the disorder is alpha thalassemia. If the problem is with the beta thalassemia. If the problem is with the beta globin part, it is called beta thalassemia. There globin part, it is called beta thalassemia. There are both mild and severe forms of alpha and are both mild and severe forms of alpha and beta thalassemia. Severe beta thalassemia is beta thalassemia. Severe beta thalassemia is often called often called Cooley’s anemiaCooley’s anemia..

Alpha ThalassemiaAlpha Thalassemia

Four genes are involved in making the alpha Four genes are involved in making the alpha globin part of hemoglobin—two from each globin part of hemoglobin—two from each parent. Alpha thalassemia occurs when one or parent. Alpha thalassemia occurs when one or more of these genes is variant or missing. more of these genes is variant or missing.

People with only one gene affected are called People with only one gene affected are called silent carriers and have no sign of illness. silent carriers and have no sign of illness.

People with two genes affected (called alpha People with two genes affected (called alpha thalassemia trait, or alpha thalassemia minor) have thalassemia trait, or alpha thalassemia minor) have mild anemia and are considered carriers. mild anemia and are considered carriers.

People with three genes affected have moderate to People with three genes affected have moderate to severe anemia, or hemoglobin H disease. severe anemia, or hemoglobin H disease.

Babies with all four genes affected (a condition Babies with all four genes affected (a condition called alpha thalassemia major, or hydrops fetalis) called alpha thalassemia major, or hydrops fetalis) usually die before or shortly after birth. usually die before or shortly after birth.

If two people with alpha thalassemia trait If two people with alpha thalassemia trait (carriers) have a child, the baby could have (carriers) have a child, the baby could have a mild or severe form of alpha thalassemia a mild or severe form of alpha thalassemia or could be healthy.or could be healthy.

Beta ThalassemiaBeta Thalassemia

Two genes are involved in making the beta Two genes are involved in making the beta globin part of hemoglobin—one from each globin part of hemoglobin—one from each parent. Beta thalassemia occurs when one or parent. Beta thalassemia occurs when one or both of the two genes are variant. both of the two genes are variant.

If one gene is affected, a person is a carrier and If one gene is affected, a person is a carrier and has mild anemia. This condition is called beta has mild anemia. This condition is called beta thalassemia trait, or beta thalassemia minor. thalassemia trait, or beta thalassemia minor.

If both genes are variant, a person may have If both genes are variant, a person may have moderate anemia (beta thalassemia intermedia, or moderate anemia (beta thalassemia intermedia, or mild Cooley’s anemia) or severe anemia (beta mild Cooley’s anemia) or severe anemia (beta thalassemia major, or Cooley’s anemia). thalassemia major, or Cooley’s anemia).

Cooley’s anemia, or beta thalassemia major, is a Cooley’s anemia, or beta thalassemia major, is a rare condition. A survey in 1993 found 518 rare condition. A survey in 1993 found 518 Cooley’s anemia patients in the United States. Cooley’s anemia patients in the United States. Most of these persons had the severe form of the Most of these persons had the severe form of the illness, but there may be more who are not illness, but there may be more who are not diagnosed. diagnosed.

If two people with beta thalassemia trait (carriers) have a If two people with beta thalassemia trait (carriers) have a

baby, one of three things can happenbaby, one of three things can happen::

The baby could receive two normal genes (one from The baby could receive two normal genes (one from each parent) and have normal blood (1 in 4 chance, or each parent) and have normal blood (1 in 4 chance, or 25 percent). 25 percent).

The baby could receive one normal gene from one The baby could receive one normal gene from one parent and one variant gene from the other parent and parent and one variant gene from the other parent and have thalassemia trait (2 in 4 chance, or 50 percent). have thalassemia trait (2 in 4 chance, or 50 percent).

The baby could receive two thalassemia genes (one The baby could receive two thalassemia genes (one from each parent) and have a moderate to severe form from each parent) and have a moderate to severe form of the disease (1 in 4 chance, or 25 percent). of the disease (1 in 4 chance, or 25 percent).

Who Is At Risk for ThalassemiaWho Is At Risk for Thalassemia?? 1.1. Thalassemia is passed from parents to children Thalassemia is passed from parents to children

through their genes. through their genes. 2.2. Thalassemia affects both males and females. Thalassemia affects both males and females. 3.3. Beta thalassemias affect people of Mediterranean Beta thalassemias affect people of Mediterranean

origin or ancestry (Greek, Italian, Middle Eastern) origin or ancestry (Greek, Italian, Middle Eastern) and people of Asian and African descent. and people of Asian and African descent.

4.4. Alpha thalassemias mostly affect people of Alpha thalassemias mostly affect people of Southeast Asian, Indian, Chinese, or Filipino origin Southeast Asian, Indian, Chinese, or Filipino origin or ancestry. or ancestry.

What Are the Signs and Symptoms of What Are the Signs and Symptoms of ThalassemiaThalassemia??

The symptoms of thalassemia depend on the The symptoms of thalassemia depend on the type and severity of the disease. Symptoms type and severity of the disease. Symptoms occur when not enough oxygen gets to various occur when not enough oxygen gets to various parts of the body due to low hemoglobin and a parts of the body due to low hemoglobin and a shortage of red blood cells in the blood shortage of red blood cells in the blood (anemia). (anemia).

““Silent carriers” and persons with alpha Silent carriers” and persons with alpha thalassemia trait or beta thalassemia trait thalassemia trait or beta thalassemia trait (also called carriers) usually have no (also called carriers) usually have no symptoms. Those with alpha or beta symptoms. Those with alpha or beta thalassemia trait often have mild anemia thalassemia trait often have mild anemia that may be found by a blood test. that may be found by a blood test.

In In more severe types of thalassemia, such as Cooley’s anemia, more severe types of thalassemia, such as Cooley’s anemia, signs of the severe anemia are seen in early childhood and signs of the severe anemia are seen in early childhood and

may includemay include:: 1.1. Fatigue (feeling tired) and weakness Fatigue (feeling tired) and weakness 2.2. Pale skin or jaundice (yellowing of the skin) Pale skin or jaundice (yellowing of the skin) 3.3. Protruding abdomen, with enlarged spleen Protruding abdomen, with enlarged spleen

and liver and liver 4.4. Dark urine Dark urine 5.5. Abnormal facial bones and poor growth Abnormal facial bones and poor growth

Babies with all four genes affected (a condition Babies with all four genes affected (a condition called alpha thalassemia major, or hydrops fetalis) called alpha thalassemia major, or hydrops fetalis) usually die before or shortly after birthusually die before or shortly after birth

How Is Thalassemia DiagnosedHow Is Thalassemia Diagnosed??

1.1. Thalassemia is diagnosed using blood tests, including a Thalassemia is diagnosed using blood tests, including a complete blood count (CBC) and special hemoglobin complete blood count (CBC) and special hemoglobin studies.studies.

2.2. A CBC provides information about the amount of A CBC provides information about the amount of hemoglobin and the different kinds of blood cells, such as hemoglobin and the different kinds of blood cells, such as red blood cells, in a sample of blood. People with red blood cells, in a sample of blood. People with thalassemia have fewer red blood cells than normal and thalassemia have fewer red blood cells than normal and less hemoglobin than normal in their blood. Carriers of less hemoglobin than normal in their blood. Carriers of the trait may have slightly small red blood cells as their the trait may have slightly small red blood cells as their only sign. only sign.

3.3. Hemoglobin studies measure the types of hemoglobin in a Hemoglobin studies measure the types of hemoglobin in a blood sample. blood sample.

Cooley’s anemiaCooley’s anemia

is usually diagnosed in early childhood because of is usually diagnosed in early childhood because of signs and symptoms, including severe anemia. signs and symptoms, including severe anemia. Some people with milder forms of thalassemia may Some people with milder forms of thalassemia may be diagnosed after a routine blood test shows that be diagnosed after a routine blood test shows that they have anemia. Doctors suspect thalassemia if a they have anemia. Doctors suspect thalassemia if a child has anemia and is a member of an ethnic child has anemia and is a member of an ethnic group that is at risk for thalassemia. group that is at risk for thalassemia.

To distinguish anemia caused by iron deficiency To distinguish anemia caused by iron deficiency from anemia caused by thalassemia, tests of the from anemia caused by thalassemia, tests of the amount of iron in the blood may be done. Iron-amount of iron in the blood may be done. Iron-deficiency anemia occurs because the body doesn’t deficiency anemia occurs because the body doesn’t have enough iron for making hemoglobin. The have enough iron for making hemoglobin. The anemia in thalassemia occurs not because of a lack anemia in thalassemia occurs not because of a lack of iron, but because of a problem with either the of iron, but because of a problem with either the alpha globin chain or the beta globin chain of alpha globin chain or the beta globin chain of hemoglobin. Iron supplements do nothing to hemoglobin. Iron supplements do nothing to improve the anemia of thalassemia, because improve the anemia of thalassemia, because missing iron is not the problem. missing iron is not the problem.

Family genetic studies are also helpful in Family genetic studies are also helpful in diagnosing thalassemia. This involves diagnosing thalassemia. This involves taking a family history and doing blood taking a family history and doing blood tests on family members. tests on family members.

Prenatal testing can determine if an unborn Prenatal testing can determine if an unborn baby has thalassemia and how severe it is baby has thalassemia and how severe it is likely to be.likely to be.

How Is Thalassemia TreatedHow Is Thalassemia Treated??

Treatment for thalassemia depends on the type and severity Treatment for thalassemia depends on the type and severity of the disease. of the disease.

People who are carriers (they have thalassemia trait) People who are carriers (they have thalassemia trait) usually have no symptoms and need no treatment. usually have no symptoms and need no treatment.

Those with moderate forms of thalassemia (for example, Those with moderate forms of thalassemia (for example, thalassemia intermedia) may need blood transfusions thalassemia intermedia) may need blood transfusions occasionally, such as when they are experiencing stress occasionally, such as when they are experiencing stress due to an infection. If a person with thalassemia due to an infection. If a person with thalassemia intermedia worsens and needs regular transfusions, he or intermedia worsens and needs regular transfusions, he or she is no longer considered to have thalassemia she is no longer considered to have thalassemia intermedia; instead, the person is said to have thalassemia intermedia; instead, the person is said to have thalassemia major, or Cooley’s anemia. major, or Cooley’s anemia.

1.1. Those with severe thalassemia have a Those with severe thalassemia have a serious and life-threatening illness. They serious and life-threatening illness. They are treated with regular blood transfusions, are treated with regular blood transfusions, iron chelation (ke-LAY-shun) therapy, and iron chelation (ke-LAY-shun) therapy, and bone marrow transplants. Without bone marrow transplants. Without treatment, children with severe thalassemia treatment, children with severe thalassemia do not live beyond early childhood. People do not live beyond early childhood. People with severe thalassemia who are able to with severe thalassemia who are able to continue therapy successfully may live continue therapy successfully may live into their thirties, forties, and beyond. into their thirties, forties, and beyond.

1.1. Blood TransfusionsBlood Transfusions Severe forms of thalassemia are treated by Severe forms of thalassemia are treated by

regular blood transfusions. A blood regular blood transfusions. A blood transfusion, given through a needle in a vein, transfusion, given through a needle in a vein, provides blood containing normal red blood provides blood containing normal red blood cells from healthy donors. In thalassemia cells from healthy donors. In thalassemia treatment, blood transfusions are done on a treatment, blood transfusions are done on a schedule (often every 2–4 weeks) to keep schedule (often every 2–4 weeks) to keep hemoglobin levels and red blood cell numbers hemoglobin levels and red blood cell numbers at normal levels. Transfusion therapy can at normal levels. Transfusion therapy can allow a person with severe thalassemia to feel allow a person with severe thalassemia to feel better, enjoy normal activities, and live longer. better, enjoy normal activities, and live longer.

Transfusion therapy, while lifesaving, is Transfusion therapy, while lifesaving, is expensive and carries a risk of transmitting viral expensive and carries a risk of transmitting viral and bacterial diseases (for example, hepatitis). and bacterial diseases (for example, hepatitis). Transfusion also leads to excess iron in the Transfusion also leads to excess iron in the blood (iron overload), which can damage the blood (iron overload), which can damage the liver, heart, and other parts of the body. To liver, heart, and other parts of the body. To prevent damage, iron chelation therapy is prevent damage, iron chelation therapy is needed to remove excess iron from the body. needed to remove excess iron from the body.

2-2-Iron Chelation TherapyIron Chelation Therapy Iron chelation therapy uses medicine to Iron chelation therapy uses medicine to

remove the excess iron that builds up in the remove the excess iron that builds up in the body when a person has frequent blood body when a person has frequent blood transfusions. If the iron is not removed, it transfusions. If the iron is not removed, it damages body organs, such as the heart and damages body organs, such as the heart and liver. liver.

The medicine, deferoxamine (deh-fer-ROX-uh-The medicine, deferoxamine (deh-fer-ROX-uh-meen), works best when given slowly under the meen), works best when given slowly under the skin, usually with a small portable pump skin, usually with a small portable pump overnight. This therapy is demanding and overnight. This therapy is demanding and sometimes is mildly painful, so some people sometimes is mildly painful, so some people stop chelation therapy. A pill form of iron stop chelation therapy. A pill form of iron chelation therapy, deferasirox, was approved in chelation therapy, deferasirox, was approved in November 2005 for use in the United States. November 2005 for use in the United States.

People who have iron overload should not take People who have iron overload should not take vitamins or other supplements that contain iron. vitamins or other supplements that contain iron.

33--SurgerySurgery Surgery may be needed if body organs, such as Surgery may be needed if body organs, such as

the spleen or gall bladder, are affected. For the spleen or gall bladder, are affected. For example, if the spleen becomes inflamed and example, if the spleen becomes inflamed and enlarged, it may be removed. If gallstones enlarged, it may be removed. If gallstones develop, the gall bladder may be removed. develop, the gall bladder may be removed.

A-A-Bone Marrow or Stem Cell Bone Marrow or Stem Cell TransplantsTransplants

Bone marrow or stem cell transplants have Bone marrow or stem cell transplants have been used successfully in some children with been used successfully in some children with severe thalassemia. This is a risky procedure, severe thalassemia. This is a risky procedure, but it offers a cure for those children who but it offers a cure for those children who qualify. qualify.

44--Other TreatmentsOther Treatments People with severe thalassemia are more likely People with severe thalassemia are more likely

to get infections that can worsen their anemia. to get infections that can worsen their anemia. They should get an annual flu shot and the They should get an annual flu shot and the pneumonia vaccine to help prevent infections. pneumonia vaccine to help prevent infections.

Folic acid is a B vitamin that helps build red Folic acid is a B vitamin that helps build red blood cells. People with thalassemia should blood cells. People with thalassemia should take folic acid supplements. take folic acid supplements.

Researchers are also studying other treatments, Researchers are also studying other treatments, such as gene therapy and fetal hemoglobin. such as gene therapy and fetal hemoglobin.

55--Gene therapyGene therapy

Someday, it may be possible to cure Someday, it may be possible to cure thalassemia in an unborn child by inserting a thalassemia in an unborn child by inserting a normal gene into the child’s stem cells.normal gene into the child’s stem cells.

66--Fetal hemoglobinFetal hemoglobin Researchers are studying ways to enhance Researchers are studying ways to enhance

production of fetal hemoglobin in people with production of fetal hemoglobin in people with thalassemia. Fetal hemoglobin is the type of thalassemia. Fetal hemoglobin is the type of hemoglobin made by the body before birth. After hemoglobin made by the body before birth. After birth, the body usually switches from making fetal birth, the body usually switches from making fetal hemoglobin to the adult form of hemoglobin. Some hemoglobin to the adult form of hemoglobin. Some children have a gene variant that prevents the children have a gene variant that prevents the switch, and their continuing production of fetal switch, and their continuing production of fetal hemoglobin lessens the severity of their illness. hemoglobin lessens the severity of their illness. Researchers are testing ways to enhance fetal Researchers are testing ways to enhance fetal hemoglobin production after birth.hemoglobin production after birth.

How Can Thalassemia Be Prevented?How Can Thalassemia Be Prevented?

Although thalassemia cannot be prevented, Although thalassemia cannot be prevented, it can be identified before birth by prenatal it can be identified before birth by prenatal diagnosis. diagnosis.

People who have or believe that they may People who have or believe that they may carry the thalassemia genes can receive carry the thalassemia genes can receive genetic counseling to avoid passing the genetic counseling to avoid passing the disorder to their children.disorder to their children.

Living With ThalassemiaLiving With Thalassemia

1.1. The Cooley’s Anemia Foundation offers The Cooley’s Anemia Foundation offers support to people with various types of support to people with various types of thalassemia through its Thalassemia Action thalassemia through its Thalassemia Action Group. Group.

2.2. If you have moderate or severe thalassemia, If you have moderate or severe thalassemia, you need to take care of your overall health. you need to take care of your overall health.

Follow your treatment plan. See your doctor regularly Follow your treatment plan. See your doctor regularly for checkups and treatment. for checkups and treatment.

If you must have regular blood transfusions and iron If you must have regular blood transfusions and iron chelation therapy, it is important to continue with chelation therapy, it is important to continue with treatment as recommended. treatment as recommended.

If you have regular blood transfusions, you should If you have regular blood transfusions, you should avoid taking vitamins or other supplements containing avoid taking vitamins or other supplements containing iron. iron.

Maintain a healthy diet. Your doctor may also give you Maintain a healthy diet. Your doctor may also give you a supplement of folic acid (a B vitamin) every day to a supplement of folic acid (a B vitamin) every day to help your body make new red blood cells. help your body make new red blood cells.

Get a flu shot every year and the pneumococcal vaccine Get a flu shot every year and the pneumococcal vaccine to prevent infectto prevent infect

Key PointsKey Points Thalassemia is an inherited blood disorder that Thalassemia is an inherited blood disorder that

can cause mild to severe anemia. can cause mild to severe anemia. Thalassemia involves problems with the Thalassemia involves problems with the

production of hemoglobin in red blood cells. production of hemoglobin in red blood cells. As a result, a person with thalassemia doesn’t As a result, a person with thalassemia doesn’t have enough hemoglobin or red blood cells to have enough hemoglobin or red blood cells to carry oxygen throughout the body (anemia). carry oxygen throughout the body (anemia).

Two main types of thalassemia are alpha and Two main types of thalassemia are alpha and beta thalassemia. Alpha thalassemia occurs beta thalassemia. Alpha thalassemia occurs when there is a problem with the alpha globin when there is a problem with the alpha globin chain that is part of hemoglobin. Beta chain that is part of hemoglobin. Beta thalassemia occurs when there is a problem with thalassemia occurs when there is a problem with the beta globin chain. the beta globin chain.

Mild, moderate, and severe forms of Mild, moderate, and severe forms of thalassemia occur. Severe beta thalassemia is thalassemia occur. Severe beta thalassemia is often called Cooley’s anemia. often called Cooley’s anemia.

The most common severe form of thalassemia The most common severe form of thalassemia seen in the United States is beta thalassemia seen in the United States is beta thalassemia major, or Cooley’s anemia. It mainly affects major, or Cooley’s anemia. It mainly affects people from Mediterranean countries and Asia. people from Mediterranean countries and Asia.

Some people are “silent carriers” with no Some people are “silent carriers” with no symptoms. Other carriers have mild anemia but symptoms. Other carriers have mild anemia but usually need no treatment. Carriers can pass usually need no treatment. Carriers can pass thalassemia genes on to their children. thalassemia genes on to their children.

Severe thalassemia is treated with frequent Severe thalassemia is treated with frequent blood transfusions and iron chelation blood transfusions and iron chelation therapy to remove excess iron that builds up therapy to remove excess iron that builds up in the body from the transfusions. in the body from the transfusions.

Bone marrow or stem cell transplants have Bone marrow or stem cell transplants have cured thalassemia in some children, but this cured thalassemia in some children, but this treatment is not available for most people treatment is not available for most people with thalassemia. with thalassemia.

Researchers are studying new treatments, Researchers are studying new treatments, including ways to cure thalassemia through including ways to cure thalassemia through stem cell and gene therapies. stem cell and gene therapies.

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