Sibling Donor Cord Blood Transplantation for Thalassemia Major: Experience of the Sibling Donor Cord Blood Program

The Sibling Donor Cord Blood (SDCB) Program was initiated in 1998 as a resource to collect, characterize, and release cord blood units (CBUs) from families affected by malignant and nonmalignant disorders for trans- plantation. Families in the United States were recruited by telephone after re- ferrals by community and academic physicians. Collection kits were mailed to prospective participants and family members were instructed about CBU pro- curement from community hospitals and shipping to a central laboratory. Data about the infant's delivery and CBU harvest, CBU processing, prethaw char- acteristics, sterility, and human leukocyte antigen (HLA) typing were collected. Standard outcome data were collected after CBU release for transplantation. Descriptive analyses of CBU collections, processing, release, and transplanta- tion outcomes were performed. Currently, 1617 CBU collections have been pro- cessed from families with thalassemia (6%), sickle cell disease (28%), malignant disorders (49%), and other rare hematological disorders (17%). Thirty-two of 96 donor-recipient pairs with thalassemia major were HLA identical and 14 have received cord blood transplantation, either alone or in combination with bone marrow or peripheral blood progenitor cells (N = 4) from the same donor. Eleven of the 14 survive free of thalassemia after trans- plantation. These preliminary results confirm the feasibility and utility of remote-site sibling donor cord blood collection and subsequent transplantation for hematological disorders, with a very high rate of usage from a cord blood bank dedicated to performing these unique collections. It was concluded that cord blood transplantation from sibling donors represents a suitable alterna- tive to bone marrow transplantation.