COMPREHENSIVE BANKING OF SIBLING DONOR CORD BLOOD FOR CHILDREN WITH MALIGNANT AND NON-MALIGNANT DISEASE Running Head: Sibling Cord Blood Banking Scientific Heading: Transfusion Medicine

250 words) Background: Banking of cord blood (CB) for unrelated hematopoietic stem cell (HSC) transplantation is well established. However, directed-donor banking of CB for siblings in a current good tissue practices (cGTP) environment has not been investigated. Methods: Families were eligible for study if they were caring for a child with a disorder treatable by HSC transplantation and expecting the birth of a full sibling. We devised standard operating procedures and policies to address eligibility, donor recruitment, donor and recipient evaluation, CB collection, shipping, graft characterization, storage and release of CB from quarantine. Many of these policies are distinctly different from those established for unrelated donor CB banks. Results: Five hundred forty families from 42 states enrolled. Collections occurred at several hundred different hospitals. No family was deferred on the basis of health history or infectious disease testing but departures from standard donor suitability criteria were documented. Disease categories for sibling recipients included: malignancy, sickle cell anemia, thalassemia major, non-malignant hematological conditions, and metabolic errors. Mean CB volume (including anticoagulant) and nucleated cell count were 103.1 ml and 8.9x10, respectively. Cell dose exceeded 1.5 x 10 nucleated cells per kilogram for 90% of banked units. Seventeen units (3.4%) have been transplanted. Sixteen of 17 CB allograft recipients had stable engraftment of donor cells. Conclusions: Remote-site collection of sibling-donor CB can be accomplished with a high success rate and in a cGTP-guided environment. The cellular products are utilized successfully for transplantation; their number and characteristics should be adequate to support the first prospective clinical investigations of sibling CB transplantation. For personal use only. by guest on June 3, 2013. bloodjournal.hematologylibrary.org From

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