Designed for transplants that replace a patient's bone marrow with stem cells from donor marrow, peripheral blood or umbilical cord blood, the procedure allows early recovery of immune function, nearly eliminates transplant rejection, and decreases the incidence and severity of "graft vs. host disease," a common complication in transplants.
Termed a "reduced-intensity" protocol, in pediatric patients it may minimize damage to sensitive growing tissues like the brain and reproductive organs.
The pilot study of the procedure is reported in the journal Bone Marrow Transplantation. It is available through advance online publication on Dec. 13 and will appear in a future print issue.
The regimen was administered to 11 pediatric and 5 adult patients at St. Louis Children's and Barnes-Jewish hospitals and the Children's Hospital of New Orleans who had non-malignant bone marrow or metabolic disorders such as sickle cell anemia, thalassemia or Hurler's syndrome. Symptoms and disease parameters stabilized or improved in all patients that underwent successful transplants.
In a successful stem cell transplant, the donor stem cells become permanently established, or engrafted, in the patient's bone marrow and continually produce healthy blood cells. To prevent the host immune system from destroying the foreign stem cells, physicians administer a pretransplant immune suppressing treatment.
"We wanted an approach that would effectively knock out the patient's immune system to let the transplanted cells engraft, but then allow immune function to recover quickly," says study leader Shalini
Contact: Gwen Ericson
Washington University School of Medicine