Permanent cure of beta thalassemia major

We often receive these questions:

  • How to cure beta thalassemia?
  • Is there a cure for beta thalassemia?
  • Is beta thalassemia curable?

The answer is that the only cure available today for beta thalassemia major is Bone Marrow Transplant(BMT),developed by Prof. Guido Lucarelli about 30 years ago in Italy. Since then,more than 3,000 children worldwide have done a BMT for thalassemia.

After a successful BMT,the child stops doing blood transfusions. Anyway,BMT has always risks associated.
The best age to do it is in young children.

It is necessary to have a donor,and the main options are:

To know if a donor is HLA compatible,a blood test called HLA typing has to be done.

The results of the BMT depend on the age of the child,his health conditions and who is the donor. Young low risk children with an HLA matched compatible sibling have a 89% success rate. You can read more results in the FAQ.

Dr. Pietro Sodani doesn’t recommend the BMT for patients older than 17 years of age,because the risk is too high. For adult patients,there are some trials going on using Gene Therapy,you can read more in this section of the Thalassemia Patients and Friends forum.

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