Researchers set new standard of care for adult cord blood transplant patients

Trial results presented at the Annual American Society of Hematology Conference

University of Minnesota researchers will present the promising results from adult umbilical cord blood studies for patients with cancers of the blood and bone marrow. These studies’ findings provide solutions to the problems outlined in recently published studies in the New England Journal of Medicine (NEJM). “The results of our studies are a triumph in a treatment that has been largely viewed as only possible in children and adolescents,” said John Wagner, M.D., professor of pediatrics and scientific director of clinical research, Blood and Marrow Transplantation Program and Stem Cell Institute.

A major limitation of adult cord blood transplantation highlighted in the NEJM articles is the high likelihood of patients dying early after transplant. This likelihood is due to: poor recovery of the patient’s blood cell counts as a result of the relatively low number of cells in cord blood compared to bone marrow, or toxicity from the high doses of chemotherapy and radiation used in conventional transplant regimens.

The University of Minnesota Blood and Marrow Transplant Program have addressed these issues in two separate studies. In the first study, the combined use of two cord blood units from different donors was investigated as a way to increase the number of cord blood cells given to the patient on transplant day. Thirty-one patients were given high-dose chemotherapy and radiation, in which all the patient’s bone marrow is destroyed, and then transplanted with two partially matched umbilical cords. The matching criteria is less stringent in cord blood than bone marrow. All of the patients engrafted successfully with cord blood cells with a low incidence of serious transplant complications and very promising survival compared to the previous studies.

In the second study, a group of 59 patients who were not eligible for high doses of chemotherapy and radiation (due to their older age, extensive prior chemotherapy treatment, or other serious co-existing diseases) were treated with a “reduced intensity” regimen involving lower doses of chemotherapy and radiation. These patients were then transplanted with cord blood from either one or two donors. In this study, 89 percent of the patients engrafted successfully overall with 98 percent of patients engrafting if they had recent chemotherapy before the transplant or a prior transplant. Despite the high-risk nature of this patient group, the risk of life-threatening transplant complications was relatively low even in older patients or those with a prior transplant.

“These remarkable results represent a significant advance in the practice of adult cord blood transplantation. These approaches allow us to offer potentially life-saving transplant therapy to many patients who have previously been denied such treatment,” said Juliet Barker, MBBS(Hons), assistant professor of medicine, Adult Blood and Marrow Transplant Program.