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I have a young child with thalassemia who needs monthly blood transfusions in order to survive. Is a cure available for him?

Supportive care with monthly blood transfusions and appropriate medical follow-up will not cure your child, but if followed precisely, may allow your child to live up to 40 or 50 years of age with a good quality of life. The main issue is access to appropriate supportive care and blood transfusions. It is particularly important to assure safe blood, preferably not from family members but from volunteer donors. Pre-transfusion hemoglobin should be kept above 9 g/dL. After the initial 15-20 transfusions, iron from transfused red cells starts to accumulate and may cause harm to your child’s body, especially the heart and liver. This iron build-up is evaluated by measuring ferritin blood levels. When ferritin levels rise above 1,000 ng/mL its time to start iron-removing (chelation) therapy. Your child should be followed by a thalassemia center where the doctors will be able to advise you about supportive care and the different tests needed to assure that he or she will remains as healthy as possible.
 
Bone Marrow Transplantation (BMT) is the only definitive cure for thalassemia, but it has its risks. These risks depend mostly on availability of a compatible family donor, generally a sibling, and the age and health of your child at the time of transplant. It consists of replacing your child’s faulty bone marrow stem cells, from which red cells originate, with those obtained from a healthy compatible donor. It is best done when the child is between 4 to 7 years of age. This treatment has been successful in thousands of people worldwide.
 
What is thalassemia?
Thalassemia is a genetic condition where the children need monthly blood transfusion lifelong and daily medications to reduce body iron in order to sustain life. Carriers of thalassemia lead a normal life and can be detected only by performing a special blood test called Hb electrophoresis where HbA2 is elevated. When both parents are carriers, the baby has a 25% risk of being born with thalassemia major. The only curative option is a bone marrow transplantation which is not available to all children.
 
What is a Bone Marrow Transplant?
A blood and marrow stem cell transplant is a procedure that replaces a person's faulty stem cells with healthy ones.
 
Stem cells are found in bone marrow, a spongy tissue inside the bones. Stem cells develop into the three types of blood cells that the body needs:

· Red blood cells, which carry oxygen throughout the body
· White blood cells, which fight infections
· Platelets (PLATE-lets), which help the blood clot
 
BMT plays a great role in improving the quality of life for both patients and their families through decreasing medical, psychological and financial burdens of conventional continuous treatment.
 
Have more questions about bone marrow transplantation? Check out our FAQs page online- http://www.cure2children.org/faq-page#n206

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Posted by: Cure2Children