Unrelated Allogeneic Cord Blood Banking and Transplantation Forum
August 14 and 15, 2000 - Slide Presentation
Ellen F. Lazarus, M.D.
Division of Hematology
OBRR, CBER, FDA
Unrelated Allogeneic Cord Blood Forum
- Co-sponsored by NHLBI and CBER
- Masur Auditorium NIH, Aug. 14-15, 2000
- Predominantly scientific meeting - cord blood bank, clinical transplant and scientific presentations followed by panel discussions
- Presentations of cord blood clinical and non-clinical data submitted in response to FDA request for data for proposed HSPC standards
- Unedited transcript will be available on CBER website
Objectives
- Discuss the current status of unrelated allogeneic placental/umbilical cord blood banking and transplantation
- Discuss scientific issues regarding the characterization of placental/umbilical cord blood grafts and data supporting development of cord blood product standards
- Identify future research directions
Experiences of Unrelated Cord Blood Banks
- US cord blood banks: New York Blood Center, COBLT, St. Louis, American Red Cross, National Marrow Donor Program
- International organization: NETCORD
- Canada: Alberta Cord Blood Bank
- Cord blood storage: Transient warming events
Panel discussion: What differences are important?
- Various cord blood centers perform and report cord blood product cell counts differently
- Uniform criteria for product cell counting, possibly supported by a voluntary laboratory certification program, would be desirable
- TNC count, ABO/Rh, hemoglobinopathy, HLA
- HLA typing - optimal level of resolution?
- Donor exclusion criteria are not uniform
Performing the search
- Both cord blood bank and transplant center perspectives were presented
- Procedures used to find and select the optimal cord blood unit for the patient
- HLA matching, cell dose, IRB protocol
- Most systems automated, on-line; web-based search systems being developed
- Multi-bank cooperation models described
Transplant outcome analysis
- Transplant outcome data describing the effects of HLA disparity, cell dose, and recipient age and underlying disease on engraftment and survival
- Comparison of pediatric BMT and UCBT from HLA-identical sibling donors
- NYBC, St. Louis, Duke and U. Minn. (consolidated data), transplant outcomes in adult recipients, Japan
- UCB has been successfully transplanted in hundreds of pediatric patients and some adult patients
- Cell dose is the major determinant of transplant outcome; extent of HLA disparity also important
Other scientific presentations
- Ex Vivo expansion
- Approaches to identifying and characterizing hematopoietic stem cells and their correlation with engraftment potential
- Potential new markers of HSCs
- Importance of CFU-Meg
- Correlation of CD34+ cells with CFU
Summary: Possible product standards
- Product processed and stored in an accredited lab/cord blood bank, collected iaw standards
- Donor ID screening/testing, maternal and family history, informed consent obtained
- Minimum product volume 30 ml; sterile
- ABO/Rh, HLA typed at A,B, and DRß1
- Post-processing TNC, viability
- Minimum 3/6 locus HLA match
- Cell dose: 2 x 107/kg (<50kg), 1 x 107/kg (>50kg)
Summary: Future directions
- Voluntary certification programs for CBBs
- Techniques to increase cell dosing - ex vivo expansion, combining units, other strategies
- DNA-based technology for ID and sterility testing
- Development of markers to detect "true" HSPCs
- Prospective, comparative studies of CBU vs. BM transplantation in pediatric patients
- Studies of efficacy of CBU transplant for adults
- Investigate possible multi-potentiality of cord blood cells
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