Unmatched Bone Marrow Transplant Can Be As Effective As A Perfect Match in Young Children, Says New Study
CINCINNATI -- A new study raises the hope that no child who needs a bone marrow transplant to treat a disease will die because matched marrow is not available.
The Children's Hospital Medical Center of Cincinnati study involves a rare and lethal immune deficiency. But the findings suggest that young children can accept a transplant from someone who is not a perfect match, not only for this particular syndrome, Wiskott-Aldrich, but for other more common disorders as well.
The international study, led by Alexandra H. Filipovich, M.D, professor of Pediatrics at Cincinnati Children's, shows that marrow from unrelated donors can be as effective as perfectly matched marrow from related donors for the treatment of Wiskott-Aldrich syndrome in young children.
"The number of potential volunteer donors in registries around the world has reached a number where a suitable donor can be found for virtually any child who needs a transplant," says Dr. Filipovich, the study's main author. "With advances in treatment, the use of unrelated donors, which is a more complex treatment, has improved to the point where we can get excellent results."
The international, multi-center study involved 170 children who received transplants for Wiskott-Aldrich between 1968 and 1996. Wiskott-Aldrich is an immune deficiency that affects three of every million boys, and only boys, in the population. Complications can include life-threatening infection, a bleeding disorder, cancers and autoimmune disease. On average, children with Wiskott-Aldrich live to be 15. More than half die before their fifth birthday.
Bone marrow transplants are the only effective treatment for Wiskott-Aldrich. Since the first transplant was done in 1968, it has become acceptable to do transplants if there is a perfect match in the family. These transplants are about 90 percent effective.
Dr. Filipovich's study also looked at transplants for Wiskott-Aldrich involving marrow from unrelated donors. In cases in which 9 or 10 of a possible 12 tissue antigens matched, the success rate of the transplant in boys treated when less than 5-years-old was equivalent to that of perfect matches, nearly 90 percent.
"Techniques have been found to successfully overcome the barriers of matches that aren't perfect," says Dr. Filipovich. "Wiskott-Aldrich can be seen as the 'star' disease for unrelated donor transplants.
It appears generalizable that young children, let's say under 5, can accept a transplant from someone who is not a perfect match not only for this disease but for others as well. So this is a treatment technique that is particularly successful in young children."
Dr. Filipovich also predicts these transplants, which are done without radiation, to be at least as effective as transplants using cord blood for the treatment of similar diseases.
Dr. Filipovich has treated more cases of Wiskott-Aldrich with bone marrow transplantation, about 60 or 70, than anyone else in the world. She also was the first physician to begin using unrelated transplants for Wiskott-Aldrich and other immune deficiencies.
Because Wiskott-Aldrich is so rare, the study was conducted throughout the world using three databases, including the International Bone Marrow Transplant Registry, which Dr. Filipovich chairs. The study is published in the March 15 issue of Blood, the journal of the American Society of Hematology. It is the first published example of such an international collaborative study and was funded in part by the National Institutes of Health (NIH).
Contact Information
Jim Feuer (
jfeuer@cchmc.org), 513-636-4420
