Sibling donor cord blood transplantation for thalassemia major: Experience of the Sibling Donor Cord Blood Program.
Level 4 - case-series / case-control
Uncontrolled registry-based case series and program evaluation.
PubMed 16339667 · doi:10.1196/annals.1345.025
What was done
The Sibling Donor Cord Blood (SDCB) Program evaluated a remote-site collection, characterization, and banking protocol for families affected by malignant and nonmalignant hematological disorders. Collection kits were mailed to referred families across the United States, collected at local delivering hospitals, and shipped to a central laboratory for processing, HLA typing, sterility testing, and banking. The study reported descriptive collection statistics across all indications and transplantation outcomes specifically for sibling donor-recipient pairs with thalassemia major.
What was found
The program processed 1,617 cord blood units (CBUs): 49% for malignant disorders, 28% for sickle cell disease, 17% for other rare hematological disorders, and 6% (96 pairs) for thalassemia. Of the 96 thalassemia pairs, 32 were HLA-identical, and 14 patients underwent transplantation (10 with cord blood alone, 4 combined with bone marrow or peripheral blood progenitor cells from the same donor). Following transplantation, 11 of the 14 patients survived free of thalassemia.
Why it matters
This study shows the feasibility of a centralized remote-collection model for directed sibling cord blood banking and provides preliminary evidence supporting sibling cord blood as a viable donor source for curative transplantation in thalassemia major.
Limits
The clinical evaluation is limited to a small, uncontrolled case series of 14 transplanted patients. Four patients received supplemental bone marrow or peripheral blood progenitor cells, confounding the independent efficacy of cord blood. The abstract does not report duration of follow-up, graft failure rates, graft-versus-host disease incidence, or causes of failure in the remaining 3 patients.
Cited by
- supports Children's Hospital Oakland developed the clinical use of sibling umbilical cord blood transplantation to treat leukemia, sickle cell disease, and thalassemia.