Unrelated Cord Blood Helps Infants With Metabolic Disorders
MONDAY, Dec. 10 -- Umbilical cord transplants from unrelated donors can benefit infants born with life-threatening metabolic disorders such as Hurler disease and Krabbe leukodystrophy, according to a study led by Duke University Medical Center researchers.
These kinds of metabolic disorders -- which cause organ failure and early death -- occur in about one in 10,000 births. Without intervention, most children with these disorders die before they reach their first birthday.
Bone marrow transplantation has been the traditional way of treating these disorders but can only be done using a matched donor. Both bone marrow and umbilical cord blood transplant replace missing enzymes and allow affected organs to develop more normally.
This study included 159 children with inherited metabolic disorders who received unrelated donor cord blood transplants at Duke between 1995 and 2007.
"We saw that there were advantages to the unrelated cord blood transplant," lead investigator Dr. Vinod Prasad, a pediatric hematologist/oncologist at Duke, said in a prepared statement. "For instance, cord blood is more readily available than bone marrow, and there was a decreased risk of complications, including a lower incidence of serious and potentially fatal graft-versus-host disease, which occurs when donor cells perceive a recipient's tissues and organs as foreign."
The study results also suggest that patients who receive cord blood transplants while they're still relatively healthy have better outcomes than those who receive bone marrow transplants.
"Over 88 percent of this subset of patients were alive one year after their cord blood transplants, and close to 80 percent were alive five years afterwards. One reason for this could be the cord blood cells are immunologically more naive than the blood-forming stem cells derived form bone marrow and therefore they may be more adaptable and less reactive once they get into the patient's body," Prasad said.
The study was expected to be presented Monday at the American Society of Hematology annual meeting in Atlanta.
The U.S. National Institute of Neurological Disorders and Stroke has more about Krabbe disease.
Posted: December 2007