DURHAM, N.C.- After 16 years of perfecting an experimental treatment, doctors at Duke University Medical Center report they can save most babies born with a rare and fatal immune disorder by giving them a family member's bone marrow within the first 3 1/2 months of life.
All but one of 22 babies who received a transplant in this time frame survived, according to results of a study published in the Feb. 17 issue of the New England Journal of Medicine.
Without a timely transplant, infants with severe combined immune deficiency (SCID) are destined to die within a year, overcome by infections that attack their bodies because they lack an immune system, said Dr. Rebecca Buckley, chief of Duke's division of pediatric allergy and immunology.
Giving them a bone marrow transplant soon after birth allows them to build a healthy immune system before opportunistic infections can take hold and jeopardize the success of the transplant. Early treatment also reduces the cost of care by hundreds of thousands of dollars. A transplant within the first few months can cost less than $30,000, whereas waiting until the child is seriously ill can boost the price tag into the millions and may fail, Buckley said.
Taken together, Buckley's approach has eradicated the need for toxic chemotherapy, sterile environments and lengthy hospital stays that once typified the ordeal of children with "bubble boy" disease, the term assigned to David Vetter, a young Texas patient who lived for 12 years in a plastic, germ-free bubble and died in 1984. Children can now be treated as outpatients without a hospital stay.
"This once-fatal disease should be seen as a pediatric emergency that requires immediate diagnosis and treatment because now there is a proven therapy that can save a child's life," Buckley said in an interview.
Buckley's program has yielded the highest reported success rate in the
world and appears to provide a full cure f
Contact: Rebecca A. Levine
Duke University Medical Center