Alpha Thalassemia (cont.)
How Are Thalassemias Treated?
Treatments for thalassemias depend on the type and severity of the disorder.
People who are carriers or who have alpha or beta thalassemia trait have mild or
no symptoms. They need little or no treatment.
Doctors use three standard treatments for moderate and
severe forms of thalassemia. These include blood transfusions, iron chelation
(ke-LAY-shun) therapy, and folic acid supplements. Other treatments have been developed or are
being tested, but they're used much less often.
Standard Treatments
Blood Transfusions
Transfusions of red blood cells are the main treatment
for people who have moderate or severe thalassemias. A blood transfusion, given
through a needle in a vein, gives you healthy red blood cells with normal hemoglobin. Red blood
cells live for only about 120 days. So, you may need repeated transfusions to
maintain a supply of healthy red blood cells.
If you have hemoglobin H disease or beta thalassemia intermedia, you may need
blood transfusions on occasion. For example, you may need this treatment when
you have an infection or other illness, or when your anemia is severe enough to
cause tiredness.
If you have beta thalassemia major, or Cooley's anemia, you need regular
blood transfusions (often every 2 to 4 weeks). These will help you maintain
normal hemoglobin levels and red blood cell numbers. Blood transfusions allow
you to feel better, enjoy normal activities, and live into adulthood.
Blood transfusions are lifesaving, but they're expensive and carry a risk of
transmitting infections and viruses (for example, hepatitis). However, this risk
is very low in the United States because of careful blood screening.
Iron Chelation Therapy
Because the hemoglobin in red blood cells is an iron-rich protein, regular
blood transfusions can lead to a buildup of iron in the blood. This condition is
called iron overload. It damages the liver, heart, and other parts of the body.
To prevent this damage, iron chelation therapy is needed to remove excess
iron from the body. Two medicines are used for iron chelation therapy.
- Deferoxamine
(Desferal) is a liquid medicine that's given slowly under the skin, usually
with a small portable pump used overnight. This therapy takes time and can be
mildly painful. Side effects include loss of vision and hearing.
- Deferasirox is a pill taken once a day. Side effects
include headache,
nausea (feeling sick to the stomach),
vomiting,
diarrhea,
joint pain, and fatigue
(tiredness).
Folic Acid Supplements
Folic acid is a B vitamin that helps build healthy red blood cells. You may
need to take folic acid supplements in addition to blood transfusions and/or
iron chelation therapy.
Other Treatments
Other treatments have been developed or are being tested, but they're used
much less often.
Blood and Marrow Stem Cell
Transplant
A blood and marrow stem cell transplant replaces your
abnormal or faulty stem cells with healthy ones from another person (a donor). Stem cells are the cells
inside bone marrow that make red blood cells and other types of blood cells.
A stem cell transplant is the only treatment that can
cure thalassemia. But
only a small number of people who have the severe form of the disorder are able
to find a good match among donors and have the risky procedure.
Possible Future Treatments
Researchers are working to find new treatments for thalassemias. For example,
it may be possible someday to insert a normal hemoglobin gene into stem cells in
bone marrow. This will allow people to make their own healthy red blood cells
and hemoglobin.
Researchers also are studying ways to trigger 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 make up for the lack of healthy adult hemoglobin.
Treating Complications
Better treatments now allow people who have moderate and severe thalassemias
to live much longer. As a result, these people must cope with complications that
occur over time.
An important part of managing thalassemias is treating complications.
Treatment may be needed for heart or liver diseases, infections, osteoporosis,
and other problems.
Next: Can thalassemias be prevented? »
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