Outpatient Stem Cell Transplantation Using a Reduced-Intensity Conditioning in Type 1 Diabetes Mellitus
Biology of Blood and Marrow Transplantation
HIE), cerebral palsy (CP) or congenital hydrocephalus. Most of the CB units (CBU) were privately banked through parental initiatives at the time of the baby's birth. Variable testing was performed by the banks at the time of CB cryopreservation and storage. From 2004 to 2010, the program was approached by thousands of families seeking treatment for their children. Their calls, emails and letters were catalogued in a database and characterized. CBUs were shipped to Duke from private cord blood
... rivate cord blood banks (CBB) for administration after prequalification of CBB records. To qualify, the number of total nucleated cells had to deliver 1X10e7 cells/kg of recipient body weight, have negative donor screening tests and negative sterility cultures. Patient and CB samples were tested with HLA low-resolution typing to confirm patient/unit match. A CB reference sample was also shipped to Duke for potency testing including enumeration of colony forming units, viability and CD34 counts. Upon confirmation of eligibility, shipment was arranged by Duke's Stem Cell Laboratory and the private CBB. From March 2004 to December 2010, the PBMT program received approximately 2,667 inquiries about CB stem cell treatment for children with brain injuries. Of those, 745 parents had privately banked their child's CB. When asked to provide the CB report from the CBB, 139 did not send a report and had no further contact with the program. Some children (351) were ineligible: 264 due to patient reasons including diagnosis (165) , incomplete records (81) , and parents declining (10); and 91 due to CB reasons including low cell count (57), positive sterility cultures (24), and bank issues (10). A total of 255 children were successfully infused with their own CB without any serious adverse events. Units were shipped from 57 different CBBs. In summary, 27.9% of contacts had privately stored their baby's CB. Of these, 34.2% were eligible for infusion. Of ineligible patients, 25.9% were due to problems with CBU quality. The infusions were well tolerated (Sun et al, Transfusion 2010). Studies to determine efficacy in babies and children with HIE, CP and congenital hydrocephalus are ongoing.