APPENDIXES



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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program APPENDIXES

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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program A METHODS SECTION: DATA COLLECTION AND ANALYSIS Introduction As part of its statement of task, the Health Resources and Services Administration charged the Institute of Medicine Committee on Establishing a National Cord Blood Stem Cell Bank Program to “make recommendations for the optimal structure for the cord blood program and address pertinent issues related to maximizing the potential of this technology (e.g., collection, storage, standard setting, information sharing, distribution, reimbursement, research, and outcome measures).” To answer the questions posed to them in the statement of task, the committee members used their own technical expertise supplemented by various methods of information gathering. These methods are described below. Literature Search The committee used an extensive literature search to compile the relevant data on research on cord blood published to date in the EMBASE, PreMedline, and Medline databases of medical literature. The search was conducted in two parts. The first focused on cord blood banking issues limited to human subjects, using the keyword terms: (“blood banks” OR “blood donors” OR “Blood blood preservation” OR “preservation, biological” OR “cryopreservation” OR “freeze drying”) AND (“fetal blood” OR “hematopoietic stem cells”). This search generated 1,274 articles. The second search looked specifically at cord blood in hematopoietic progenitor

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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program cell (HPC) transplantation and at HPC transplantation in general, using the key terms “fetal blood [transplantation]” OR “cord blood stem cell transplantation” OR (“fetal blood” AND “stem cell transplantation”) OR (“umbilical cord blood” AND (“bone marrow transplantation” OR “hematopoietic stem cell transplantation” OR “peripheral blood stem cell”) OR (“umbilical cord blood” AND “stem cell transplantation”). This search was also limited to research with human subjects and provided 870 articles. The breakdown of human subjects described in the articles by age showed that fewer articles described research with adults than research with children and adolescents. The exact number of articles found for each category was: adults, 455 articles; adolescents, 200 articles; children, 271 articles; infants, 130 articles; and infants and newborns, 311 articles. Because cord blood transplantation is a relatively new therapy, the search was not limited by year. Of the 2,144 records in the list, however, only 48 articles dated from before 1980. Site Visits At the first meeting, the members of the committee decided that to best characterize the current state of cord blood banking in the United States, they would need to see cord blood banks firsthand. The committee chose a variety of banks with different affiliations, sizes, and objectives to best represent the diversity of the cord blood banking industry. The committee saw both private and public banks, as well as banks that focused on directed donations. Committee representation varied for each visit to allow each committee member to see some banks. At each bank, members of the committee asked the staff of the bank to guide them through the path of a cord blood unit from the moment that it arrives at the bank, through all processing, to the moment that the unit is frozen in storage. Specific attention was paid to the individual banks’ approaches to consent, collection processes, decisions about minimum levels for storage and for transplant, labeling and identification, processing, informatics and databases, matching, and shipping. A list of the sites visited is in Box A-1.

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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program BOX A-1 Site Visits Conducted by the Committee (by date of visit) CyroBanks International (July 26, 2004) with Dwight Brunoehler and others New York Blood Center (August 3, 2004) with Pablo Rubinstein, Cladd Stevens, Melissa Penn, Kathleen Reichert, and others Children’s Hospital Oakland (August 20, 2004) with Bert Lubin and others Cord Blood Registries (August 23, 2004) with Tom Moore and others St. Louis Cord Blood Bank (September 13, 2004) with Michael Creer, J. Mario Alonso, Donna Regan, and others J.P. McCarthy Cord Stem Cell Bank (October 13, 2004) with Voravit Ratanatharathorn, Louisa Serafimovska, and Jan Keersmaekers Michigan Community Blood Centers Cord Blood Bank (October 14, 2004) with Mary Banfill and others Survey No comprehensive data were available about indicators such as the racial makeup of cord blood donors or the cost of storage of each unit in the cord blood inventory in the United States. For this reason, the members of the committee set about gathering these data themselves. To gather these more quantitative data, the members of the committee asked cord blood banks for information in the form of a questionnaire, which was sent to 40 cord blood banks in the United States. The questions asked for: general background information; collection and storage; inventory size and composition; cost, either for storage or as reimbursements from a transplant facility; and the procedures used to search for matches and use of cord blood units (completed only if services include public banking). In addition to mailing the survey to 40 banks, the survey was also made available online (see Appendix B). The surveys were sent with prestamped, return-addressed envelopes, and second and third follow-up letters were sent to thank the banks that had responded and to request responses from those that had not yet done so. As of November 18, 2004, the committee had received 21 partially or fully completed surveys. The information gathered helped the committee make its recommendations.

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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program Raw Data Analysis Although most public banks attempt to maintain more or less complete sets of outcomes data and follow up with recipients of cord blood months and years after the transplant, many are not able to obtain the information; and even among those that can, the data are not usually shared among the different cord blood banks. The committee was given access to the outcomes data from the National Marrow Donor Program cord blood transplants, the results of the COBLT study, and the data from the New York Blood Center and did its own analysis (see Appendix G). Commissioned Papers The committee selected several areas in which it wanted analyses to be conducted by experts: human leukocyte antigen typing, the racial make-up and diversity needs of a national inventory, economic issues associated with cord blood banking, and future prospects for cord blood use. These papers were written by Karen Ballen, Margaret Goodell, David Howard, and Carolyn Hurley, respectively. Some of those papers can be found in Appendixes D to F. Committee Meetings The committee held two information-gathering meetings before commencing with the writing of the final report. The first meeting, held on June 2 and 3, 2004, in Washington, D.C., included speakers involved in creating the legislation relevant to the committee’s charge and speakers from selected federal agencies and professional organizations, as well as a speakers presenting the clinical perspective on cord blood transplantation. At the second meeting, held August 18 and 19, 2004, in Irvine, California, the committee heard from speakers on the topics of informatics and matching algorithms, cord blood bank accreditation, cord blood collection and preservation issues, ethical and legal considerations, outcomes data, and patient support issues. Speakers were chosen for their expertise in their fields. In addition, periods for participants to make open comments were included during each of the first two meetings. These allowed members of the public representing various constituencies and interested groups to address the committee. The third and fourth meetings, held September 29 and 30, 2004, in Woods Hole, Massachusetts, and December 15 and 16, 2004, in Washington, DC, respectively, were deliberative and writing meetings, during which

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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program the committee developed and refined its recommendations and the members of the committee worked together to draft the report. The committee also kept in close contact by telephone and electronic communication. Invited Participants and Guests The following individuals were invited participants and guests at meetings of the committee. James Burdick, M.D. Health Resources and Services Administration Shelly Carter, Sc.D. The EMMES Corporation Jeffrey Chell, M.D. National Marrow Donor Program Phil Coelho Thermogenesis Corporation Dennis Confer, M.D. National Marrow Donor Program Jeff Couglin American Society for Hematology Michael Fitzpatrick America’s Blood Centers Captain Robert Hartzman Director, Department of Defense Marrow Program, Navy Liana Harvath, Ph.D. National Institutes of Health Valerie Hurt National Institutes of Health Office of the General Counsel Brent Jaquet Congressman William Young’s Office, Florida Naynesh Kamani, M.D. Children’s National Medical Center Joanne Kurtzberg, M.D. Duke University Pediatric Stem Cell Transplant Center Ellen Lazarus, M.D. Food and Drug Administration

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Cord Blood: Establishing a National Hematopoietic Stem Cell Bank Program David Leitch Illinois House of Representatives Pam Murph, LCSW Association of Oncology Social Workers Sudip Parikh Senator Arlen Specter’s Office, Pennsylvania Pablo Rubenstein, M.D. National Cord Blood Program of the New York Blood Center Karen Shoos-Lipton American Association of Blood Banks Edward Snyder, M.D. American Association of Blood Banks Cladd Stevens, M.D., MPH National Cord Blood Program of the New York Blood Center Susan Stewart BMTInfonet Elizabeth Wagner National Heart, Lung, and Blood Institute Phyllis Warkentin, M.D. Foundation for the Accreditation of Cellular Therapy Jill Warner, Esq. Food and Drug Administration Thomas Weigand Operations Manager, Caitlin Raymond International Registry