Stem Cells For Thalassaemia
What is Thalassaemia?
Thalassaemia is an inherited blood-related disorder, commonly found in regions bordering the Mediterranean and in Southeast Asia. This disorder reduces the body’s production of haemoglobin, the oxygen-carrying component of the red blood cells which circulates oxygen throughout the body and transports them to organs.
Haemoglobin is made up of alpha- and beta-proteins. An insufficient production of the alpha-protein results in alpha-thalassaemia, while a beta protein insufficiency results in beta-thalassaemia. There are also three types of Thalassaemia – Thal. Major, Thal. Minor and Thal. Intermediate.
Thalassaemia Major
Thalassaemia Major is the most severe form of Thalassaemia. Children with Thalassaemia major are born normal but tend to become anaemic when they reach the age of three months or older.
These children constantly require medical treatment and transfusion of a blood component called “packed red cells” every three to four weeks. Regular blood transfusions, however, cause the accumulation of excessive iron in the body which gets accumulated in the vital organs leading to extensive organ damage. Thalassaemia Major patients must be treated with a drug called Desferioxamine (Desferal) to combat this. The drug is injected into the body over an eight hour period and repeated at least five times a week, using a syringe pump. This kind of burdensome subcutaneous therapy is disliked by most people and definitely more by children.
Thalassaemia Minor
Individuals with Thalassaemia Minor are ‘carriers’ of the disorder. They are healthy and normal but some may have slight anaemia.
Most people who are Thalassaemia Minor are unaware of their condition, until they undergo a special blood test or one of their children is found to be Thalassaemia Major. If both parents are carriers there is a 25% chance that their newborn will suffer from Thalassaemia Major.
Thalassaemia Intermediate
This type of Thalassaemia has clinical characteristics between those of Thalassaemia Major and Minor. The majority of these patients are reasonably well and do not require blood transfusions until their 2nd birthday. Some may not even have the diagnosis until they are 7 years old. Children with this type of Thalassaemia suffer from the symptoms of the disorder a little later than those with Thalassaemia Major.
What are the current treatments?
- Regular blood transfusions, coupled with Iron-chelating drugs e.g. Desferioxamine
- Stem cell transplant – to replace the entire blood system.
How can stem cells help my child?
A stem cell transplant can help one suffering from thalassaemia major. This can be done if a “match” is found for the patient.
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