September 19, 2014

Thalassemia Major is a genetic blood disorder that causes a severe type of anemia in the babies it afflicts. The disorder causes the blood to produce an irregular amount of the oxygen-carrying protein hemoglobin, which leads to poor oxygen transportation through the body, resulting in red blood cell death. It can cause bone deformities, fatigue, growth failure (failure to thrive), shortness of breath, jaundice, and enlargement of the liver and spleen. If left untreated, it will lead to death of the patient, usually before the age of 10.

Until recently, the only way Thalassemia Major could be treated was through blood transfusions for the patient’s entire life. However, new discoveries made with the use of cord blood stem cells are now making a difference for those with Thalassemia Major, by curing the disease and potentially freeing them from a lifetime of regular treatments.

When using cord blood to treat this genetic disorder, the patient’s blood cannot be used due to the genetic defects it contains. The most likely candidate for a genetic stem cell match would be a sibling, which results in nearly twice the success rates when compared to those from a non-relative donor.

When using any allogeneic stem cell transplant, there is a possibility of the body rejecting the transplant and the patient developing Graft vs. Host Disease. Researchers and medical professionals sought a way to reduce the chances of that side effect, thereby increasing the likelihood of a successful cure in those treated. In a study that concluded in 2003, 44 children with Thalassemia Major were given an allogeneic cord blood transfusion1. They were then separated into smaller groups who each received a different combination of treatments and medications in an effort to prevent Graft vs. Host Disease. After an average of 24 months, 36 of the 44 children showed no sign of having the disease, and of those, only 6 developed any form of rejection or Graft vs. Host Disease, with varying degrees of severity.

This study showed a 100-percent survivability rate in the participants and predicted a 90-percent “event-free” (lack of complications) survival rate for the two years following the transplant. Cord blood banking helps parents ensure that their child and all siblings in the family have a possible treatment option when it comes to Thalassemia Major or any of the other 80 diseases currently treated with cord blood stem cells. More is being discovered all the time in the field of stem cell research, which means that banking your child’s cord blood could have even more potential for a bright medical future for your family now and for a lifetime.

1. Related umbilical cord blood transplantation in patients with thalassemia and sickle disease. Franco Locatelli, F. et al. Blood 101(6) 2137-2143 doi: 10.1182/blood-2002-07-2090. Published online March 15, 2003. Found online at http://goo.gl/m8rDs3

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