Sunday, March 11, 2001
Match need not always be perfect
UC-led study finds hope for some transplants
By Tim Bonfield
The Cincinnati Enquirer
Young children with leukemia and other rare immune-system diseases may no longer have to wait for a perfect match to get a lifesaving bone-marrow transplant.
A study led by a doctor at Children's Hospital Medical Center reports that children under 5 fare at least as well after transplants with imperfectly matched bone marrow as with perfectly matched donations.
The study details are scheduled to be published Thursday in Blood, the journal of the American Society of Hematology.
The study focused on 170 children worldwide who suf fer from Wiskott-Aldrich Syndrome, a rare and deadly blood disorder that affects three in a million male births.
Children's Hospital treats about six cases a year.
The disease often is detected once infant boys suffer unexplained bleeding in the mouth or skin, severe skin rashes or recurrent serious infections. It also can be diagnosed during pregnancy.
With lesser treatments, more than half of boys with the syndrome die before age 5. Few live past age 15.
Doctors have known since 1968 that a successful bone-marrow transplant, a complex and risky procedure, can cure the disease. But until now, most boys with Wiskott-Aldrich Syndrome never got transplants because doctors waited for perfect matches, said Dr. Lisa Filipovich, a professor of pediatrics at Children's Hospital.
The study found that transplants that matched nine or 10 of the 12 possible antigens between donor and patient were just as successful as matching all 12 antigens. Antigens are proteins or other substances to which the body reacts by producing antibodies.
Dr. Filipovich said the findings are important because any child with Wiskott-Aldrich should be able to find a donor with nine or 10 matching antigens.
The benefit of mismatched bone marrow isn't limited to one rare disease.
Nationwide, several hundred young children a year need bone-marrow transplants for leukemia, severe combined immune deficiency disease (the bubble boy disease), and other rare disorders.
We think this can in fact apply to other diseases in young children because their immune systems are more plastic, Dr. Filipovich said. It hasn't been studied formally, but that's the way it's looking.
However, it appears that older children and adults are less likely to benefit from mismatched bone-marrow transplants because their immune systems are more developed, she said.
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