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Thalassemia



Thalassemia is an inherited blood disorder in which the body makes an abnormal form of hemoglobin. Hemoglobin is the protein molecule in red blood cells that carries oxygen.

The disorder results in excessive destruction of red blood cells, which leads to anemia. Anemia is a condition in which your body doesn’t have enough normal, healthy red blood cells.

Thalassemia is inherited, meaning that at least one of your parents must be a carrier of the disease. It’s caused by either a genetic mutation or a deletion of certain key gene fragments.

Thalassemia minor is a less serious form of the disorder. There are two main forms of thalassemia that are more serious. In alpha thalassemia, at least one of the alpha globin genes has a mutation or abnormality. In beta thalassemia, the beta globin genes are affected.
Each of these forms of thalassemia has different subtypes. The exact form you have will affect the severity of your symptoms and your outlook.
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The impact can range from mild to severe and life-threatening. Around 100,000 newborns are delivered each year with severe forms of thalassemia. It is most common with Mediterranean, South Asian, and African ancestry.



Symptoms

The symptoms of thalassemia vary depending on the type of thalassemia.
Symptoms will not show until the age of 6 months in most infants with beta thalassemia and some types of alpha thalassemia. This is because neonates have a different type of hemoglobin, called fetal hemoglobin.
After 6 months "normal" hemoglobin starts replacing the fetal type, and symptoms may begin to appear.
These include:
·       Jaundice and pale skin
·       Drowsiness and fatigue
·       Chest pain
·       Cold hands and feet
·       Shortness of breath
·       Leg cramps
·       Rapid heart beat
·       Poor feeding
·       Delayed growth
·       Headaches
·       Dizziness and faintness
·       Greater susceptibility to infections
·       Skeletal deformities may result as the body tries to produce more bone marrow.
·       If there is too much iron, the body will try to absorb more iron to compensate. Iron may also accumulate from blood transfusions. Excessive iron can harm the spleen, heart, and liver.

Patients with hemoglobin H are more likely to develop gallstones and an enlarged spleen. Untreated, the complications of thalassemia can lead to organ failure.



Thalassemia occurs when there’s an abnormality or mutation in one of the genes involved in hemoglobin production. You inherit this genetic defect from your parents.
If only one of your parents is a carrier for thalassemia, you may develop a form of the disease known as thalassemia minor. If this occurs, you probably won’t have symptoms, but you’ll be a carrier of the disease. Some people with thalassemia minor do develop minor symptoms.
If both of your parents are carriers of thalassemia, you have a greater chance of inheriting a more serious form of the disease.
According to the Centers for Disease Control and Prevention (CDC), thalassemia is most common in people from Asia, the Middle East, Africa, and Mediterranean countries such as Greece and Turkey.


Thalassemia is really a group of blood problems, not just one.
To make hemoglobin you need two proteins, alpha and beta. Without enough of one or the other, your red blood cells can’t carry oxygen as they should.
Alpha thalassemia means you lack alpha hemoglobin. With beta thalassemia, you lack beta hemoglobin.
Your doctor may also talk about thalassemia minor and thalassemia major, or Cooley’s anemia. The minor type is less serious than the major one, and your type won’t change.

There are three main types of thalassemia (and four subtypes):

Beta thalassemia, which includes the subtypes major and intermedia

Alpha thalassemia, which include the subtypes hemoglobin H and hydrops fetalis
thalassemia minor

All of these types and subtypes vary in symptoms and severity. The onset may also vary slightly.

Diagnosis

Most children with moderate to severe thalassemia receive a diagnosis by the time they are 2 years old.
People with no symptoms may not realize that they are carriers until they have a child with thalassemia.
Blood tests can detect if a person is a carrier or if they have thalassemia.

A complete blood count (CBC): This can check levels of hemoglobin and the level and size of red blood cells.
A reticulocyte count: This measures how fast red blood cells, or reticulocytes, are produced and released by the bone marrow. Reticulocytes usually spend around 2 days in the bloodstream before developing into mature red blood cells. Between 1 and 2 percent of a healthy person's red blood cells are reticulocytes.

Iron: This will help the doctor determine the cause of anemia, whether thalassemia or iron deficiency. In thalassemia, iron deficiency is not the cause.
Genetic testing: DNA analysis will show whether a person has thalassemia or faulty genes.

Prenatal testing: This can show whether a fetus has thalassemia, and how severe it might be.

Chorionic villus sampling (CVS): a piece of placenta is removed for testing, usually around the 11th week of pregnancy.

Amniocentesis: a small sample of amniotic fluid is taken for testing, usually during the 16th week of pregnancy. Amniotic fluid is the fluid that surrounds the fetus.




Thalassemia also brings up different concerns related to pregnancy. The disorder affects reproductive organ development. Because of this, women with thalassemia may encounter fertility difficulties.

To ensure the health of both you and your baby, it’s important to plan ahead of time as much as possible. If you want to have a baby, discuss this with your doctor to make sure that you’re in the best health possible. Your iron levels will need to be carefully monitored. Preexisting issues with major organs are also considered.
Pregnancy carries the following risk factors in women with thalassemia:


a higher risk for infections
gestational diabetes

heart problems
hypothyroidism, or low thyroid
increased number of blood transfusions
low bone density

Living with Thalassemia

You’ll want to work closely with your doctor and keep up with your treatments.
If you have thalassemia, follow these habits to stay well:

Don’t take iron pills.

Eat a healthy diet to keep your bones strong and give you energy.

Ask your doctor about supplements like calcium and vitamin D.

Stay away from sick people and wash your hands often.
If you get a fever or feel ill, see your doctor.

You may also want to look into joining a support group, so you can connect with other people who have the condition.



WebMD Medical Reference Reviewed by Jennifer Robinson, MD on January 09, 2015

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