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Umbilical-Cord Blood Transfusion Appears Effective
American Health Line
19 May 2005
Infants diagnosed with Krabbe disease, an often fatal enzyme disorder, who receive a transplant of donor umbilical-cord blood soon after birth can go on to have normal cognitive development and fewer physical problems, according to a study published Thursday in the New England Journal of Medicine, the Raleigh News & Observer reports (Avery, Raleigh News & Observer, 5/19). In the study led by Maria Escolar, director of the University of North Carolina's program for neurodevelopment function in rare disorders, researchers from Duke University and UNC performed umbilical-cord blood transfusions on 25 infants over a six-year period. Of those infants, 11 who had a family history of Krabbe were tested for the disease at birth and received transplants within days of birth. Transplants were performed on the other 14 infants between the ages of four months and 11 months after they started showing symptoms of the disease (Armstrong, Wall Street Journal, 5/19).
As part of the procedure, the infants underwent chemotherapy "to kill their bone marrow" and later received the cord-blood transfusions, the News & Observer reports. Researchers said that cord-blood transfusions are a more effective treatment for Krabbe disease than bone marrow transplants because cord blood cells appear to address the enzymatic deficiencies more quickly and such transfusions do not require an exact match between donors and recipients. The study found that all of the infants diagnosed at birth have reached three years of age (Raleigh News & Observer, 5/19). Nearly all infants in that group developed normal cognitive functions, behavior skills and language abilities, the study found (Wall Street Journal, 5/19). In the older group of infants, six of the 14 died, while symptoms of the survivors did not improve or worsen.
Editorial
An accompanying editorial stated that caution should be exercised before performing the procedure. "Hematopoietic stem cell transplantation is still considered by most practitioners to be a high-risk therapy of last resort," the editorial states, adding, "The tendency has been to test risky therapies first in adults or older children who can make informed decisions about participating and in patients who are already ill from their disease, for whom the risks of therapy seem less extreme" (Raleigh News & Observer, 5/19).
Implications
Researchers say the study's findings show it is imperative to screen and diagnose the disease at birth (Wall Street Journal, 5/19). Transplant physician Joanne Kurtzberg said, "Now that we know that we can dramatically alter the course of these babies' lives when a transplant is done in the first month, newborn screening should be mandatory. The most effective treatment can be administered if the diagnosis is early" (Raleigh News & Observer, 5/19). Currently, no states screen newborns for Krabbe or most of the other 44 enzyme conditions known as lysosomal-storage disorders "in part because the cases have long been considered hopeless," the Journal reports. However, the new study "gives a significant boost to efforts by patient-advocacy groups and many physicians to create a universal standard" for newborn screenings that would include "far more diseases" than most states currently test for, according to the Journal (Wall Street Journal, 5/19). The study is available online.
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