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 transplantation. Families in the United States were recruited by telephone after referrals by community and academic physicians. Collection kits were mailed to prospective participants and family members were instructed about CBU procurement from community hospitals and shipping to a central laboratory. Data about the infant's delivery and CBU harvest, CBU processing, prethaw characteristics, 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 transplantation outcomes were performed. Currently, 1617 CBU collections have been processed 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 transplantation. 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 alternative to bone marrow transplantation.
The Sibling Donor Cord Blood Program was initiated in 1998 as a resource to collect, characterize, and to release for transplantation cord blood units (CBU) from families affected by malignant and non-malignant disorders. Currently, 1686 CBUs from 1587 families have been collected among referrals from all 50 US States. The categories of participation include malignant disorders (50%), sickle cell disease (29%), thalassemia (6%), and other hereditary or rare hematological conditions (15%). The mean cell volume collected was 102 ml (range, 31–284) with a mean total nucleated cell count (TNC) of 9.5 x 108 (range, 0.6–53.6) and mean CD 34+ cell count of 3.6 x 106 (range, 0.1–88.1). The post-thaw viability of CBU released for transplantation was 94.4 % (SD ±8.7%) and only 4.4% of CBUs were not processed due to having inadequate volume. To date, 54 children have been treated by sibling donor cord blood transplantation (CBT), 38 using the cord blood unit as the sole source of stem cells. There was a very high rate of CBU utilization, particularly among thalassemia families where 18 of 105 (17%) of CBUs collected have been released for CBT. CBT recipients had hematological malignancies (N=20), thalassemia major (N=18), sickle cell anemia (N=8) or other non-malignant disorders (N=8), and all but 6 received HLA-identical allografts. The median total nucleated (TNC) and CD34+ cell dose was 3.1 x 107 TNC/kg and 0.7 x 105/kg recipient weight, respectively. The median time to ANC >500 and platelet >20,000/mm3 was 22 and 45 days, respectively. One of 52 (2%) evaluable patients had graft failure accompanied by autologous reconstitution. With a median follow-up of 9.6 (range, 0.1 – 94) months, 45 of 54 (83%) patients survive, and 9 patients (17%) died of relapse (N=6) or transplant-related causes (N=3) after CBT. Among the hemoglobinopathy patients, 23 of 26 (88%) survive, and 22 (85%) survive disease-free. Overall, the Kaplan-Meier probabilities of survival and event-free survival after sibling CBT are 79% and 72%, respectively. These results confirm the feasibility of remote site, directed donor cord blood collection and subsequent transplantation for hematological disorders. The ability to combine cord blood collections with a marrow harvest from the sibling donor effectively reduced the incidence of graft rejection when CBU cell doses were judged to be insufficient. Transplantation of sibling CBUs in lieu of bone marrow may be particularly advantageous when there is urgency for transplantation and in non-malignant disorders where graft-versus-host disease, in particular, has a negative impact upon outcome.
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