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Unrelated cord blood transplants cure X-linked immunodeficiencies
Last Updated: 2001-04-06 13:50:33 EDT (Reuters Health)
By Will Boggs, MD
WESTPORT, CT (Reuters Health) - Transplantation of banked, unrelated umbilical cord blood generated normal immune systems in three boys with X-linked immunodeficiencies, according to a report in the April issue of The Journal of Pediatrics.
Dr. E. Richard Stiehm, from UCLA, and colleagues transplanted two brothers with X-linked lymphoproliferative syndrome (XLP) and one boy with X-linked hyper-IgM immunodeficiency (XHIM) using unrelated, banked umbilical cord blood cells that were in all cases partially (5/6 loci) HLA matched. The clinicians say "pretransplantation cytoreduction and posttransplant graft-verus-host prophylaxis were given."
The two boys with XLP developed mild graft-versus-host reactions, but by 6 months posttransplant they were off all medications and had normal T cells, immunoglobulins, and antibody function, the authors report.
The boy with XHIM also developed mild graft-versus-host disease, requiring temporary treatment, the results indicate. All medications were discontinued 12 months after transplantation and his immune function had become normal.
Point mutations responsible for the immunodeficiencies that were present in all patients before transplantation were no longer detectable in any of the boys at 1 year posttransplantation, the results indicate. Moreover, posttransplant B-cell lines had converted to the genotype of the donor.
Now 3 years' posttransplantation, "the patients are fine off all medications," Dr. Stiehm told Reuters Health. He said that umbilical cord blood transplants are as good as bone marrow transplants from a parent or a matched unrelated donor, "but not as good as a matched sibling [transplant]."
Dr. Alexandra Filipovich, from Children's Hospital Medical Center, in Cincinnati, Ohio, discusses the future prospects of cord blood transplants in a related editorial, and concludes, "If the ongoing study of feasibility of cord blood transplantation supported by the National Heart, Lung, and Blood Institute shows benefit to recipients of 4/6 or even 3/6 matched cord blood grafts, this could establish use of cord blood as a valuable addition to the armamentarium of hematopoietic stem cell sources for patients lacking other options and provide the rationale for funding future cord blood banking."
And if that day arrives, it will increase the demand for umbilical cord blood. Anticipating that eventuality, Dr. Stiehm counsels, "If asked, donate your baby's cord blood; it may help him/her or save another life."
J Pediatrics 2001;138:570-573,459-461.
-Westport Newsroom 203 319 2700
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