Umbilical cord blood transplants, even from unrelated donors,
can help save the lives of babies born with certain inherited
metabolic disorders, US researchers reported.
Usually, bone marrow transplants are the only option for such
infants, who can die from organ failure and early death.
Bone marrow transplants can be difficult to get and donors are
rare.
Umbilical cord blood, however, can be donated with every birth and
also contains immature cells known as stem cells that can restore
missing or damaged cells in a patient.
Stem cells are the body's master cells and there are several
kinds.
Stem cells from the bone marrow or cord blood are partly
differentiated, or transformed, and can be used to restore the
immune systems of patients undergoing cancer treatment, for
example.
Dr Vinod Prasad and colleagues at Duke University in North Carolina
studied 159 children with inherited metabolic disorders who
received transplants of cord blood from unrelated new-borns at Duke
between 1995 and 2007.
"We saw that there were advantages to the unrelated cord blood
transplant," Prasad said in a 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."
Speaking to an American Society of Haematology meeting in Atlanta,
Prasad said more than 88% of patients who got cord blood
transplants before they began to show too many symptoms of illness
lived for at least a year.
"One reason for this could be the cord blood cells are
immunologically more naive than the blood-forming stem cells
derived from bone marrow and therefore they may be more adaptable
and less reactive once they get into the patient's body," he
said.
One metabolic disease Prasad's team treated is Krabbe disease, also
known as Krabbe leukodystrophy, which affects the nervous system.
Another is Hurler disease, which affects the heart, liver and
brain.
"These disorders are rare when taken individually - some of them
occur in only one in a million births - but if you put them
together they have a sizeable incidence, maybe 1 in 10,000 births,"
Prasad said.
