Duke Study Shows Umbilical Cord Blood Transplant Is Viable Treatment For Genetic Disorders

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MIAMI -- Children whose genetic disorders have previously been a death sentence have a reasonable chance for life with umbilical cord blood transplants, according to Duke University Medical Center researchers.

The physicians found that of the patients treated, those younger than 2 years old did the best, with 80 percent surviving.

Results of a study prepared for presentation Sunday at the American Society of Hematology annual conference show an overall long-term survival rate of 68 percent for children with a variety of genetic diseases.

"What we've seen is that diseases which in the past have been thought incurable can now be cured if recognized early and referred to appropriate resources - centers that can treat pediatric patients with umbilical cord blood transplant," said Dr. Rick Howrey, who led the study.

Doctors think the success rate of transplants at early ages may be related to the period of the infants' developing immune systems, which make it less likely that they will suffer serious graft vs. host disease. That occurs when the transplanted cells (the graft) begin growing and later attacking the rest of the body, as if the host's other cells are foreign to the newly developing immune system. Replacing the patient's defective marrow before the genetic disease has caused serious damage may also contribute to the higher success in younger patients, Howrey said. In addition, the patients' smaller size increases the ratio of stem cells per kilo of body weight, which is probably the most important factor, he said.

"A lot of parents of children with these genetic disorders have been told by their doctors, 'There's nothing we can do,' which in the past has been true. But with this new form of treatment, there is now hope for children with many genetic diseases which in t

Contact: Karen Hines
Duke University Medical Center

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