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 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. |