STEM CELL TRANSPLANTATION FROM UNRELATED DONORS FOR CORRECTION OF PRIMARY IMMUNODEFICIENCIES

Abstract

The use of unrelated donor stem cell transplantation has increased exponentially over the past 5 years. The greatest benefits of URD BMT have accrued to younger patients, those with favorable pretransplant disease characteristics, and patients treated at transplant centers that have considerable experience with URD BMT specifically. Children with nonmalignant congenital disorders and immunodeficiencies in particular enjoy the highest long-term survival rates among recipients of unrelated donor BMT. Success rates range between 40% and 90% for the majority of immunodeficiency diagnoses curable by allogeneic transplantation. Early results of quality of life assessments support the notion that the functional status of long-term survivors of URD BMT is comparable to that of patients treated with matched sibling BMT. In the current era, children with a variety of lethal congenital immunodeficiencies lacking sibling donors deserve consideration for unrelated grafting at institutions with specific expertise in this therapy.