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August 2014 Newsletter  Volume 20, Issue 2 Table of Contents: Perspectives - 10th Anniversary of the CIBMTR Congratulations to 3 Working Committees Pediatric Cancer Working Committee Immunobiology Working Committee 2015 BMT Tandem Meetings Center-Specific Outcomes Report and Analysis Forum ASBMT Clinical Case Forum Surpasses 60 Cases Blood and Marrow Transplant Clinical Trials Network 2014 CIBMTR Advisory Committee Members Our Supporters Abbreviations  Perspectives - 10th Anniversary of the CIBMTR By Paul Martin, MD Humans often experience a “mid-life crisis” as we enter the fourth decade of life.  We rightly ask, “Where have I been, where am I going, and what will it mean in the end?” The IBMTR / CIBMTR has entered its fourth decade of existence, and although organizations have longer lifespans than humans, it is nonetheless timely to ask similar questions by examining the past decade of accomplishments. This examination is especially appropriate as we celebrate the 10th anniversary of the affiliation agreement between the NMDP and the Medical College of Wisconsin that transformed the IBMTR into the CIBMTR.   The past decade has seen remarkable growth in the numbers of CIBMTR publications and involved authors representing different institutions. In the two successive five-year periods from 2003 to 2007 and 2008 to 2012, the number of peer-reviewed publications nearly doubled from 119 to 235, the number of authors more than doubled from 357 to 775, and the number of represented institutions increased from 211 to 339.   Major funding for the CIBMTR comes from three main sources: a U24 grant from the NCI, NHLBI, and NIAID to support CIBMTR operations; a subcontract from DHHS to support the SCTOD for HRSA; and a U10 grant from the NCI and NHLBI to support the BMT CTN. Each of these grants was renewed twice during the past decade. In addition, the CIBMTR has attracted increasing support from industry contracts and foundation grants.   Formation of the BMT CTN in 2001 represented a critically important milestone in the life-history of the IBMTR / CIBMTR by transforming a purely retrospective vision to a mission that encompasses both critical retrospective studies and innovative prospective clinical trials answering important questions related to hematopoietic cell transplantation. Since its inception, the BMT CTN has opened 33 protocols, 7 of which are currently open to enrollment. To date, more than 6,500 patients have enrolled in clinical trials through the BMT CTN.    The CIBMTR has also facilitated laboratory research by establishing a repository of specimens from more than 20,000 unrelated and 1,800 related donor and recipient pairs. More than 15,000 aliquots were distributed to investigators last year.   The past decade has seen dramatic advances in transplantation and other cellular strategies along with greatly improved methods for procuring, sharing, and analyzing data. Keys to our future success include a focus on quality, inclusiveness, and collaboration with improved flexibility, accessibility, speed, and efficiency.  What is the most valuable asset of the CIBMTR?  Without question, it is the people who bring medical, scientific, statistical, and IT and data management expertise from within and outside the CIBMTR, working together and sharing a mission and vision to advance human health by improving hematopoietic cell transplantation.   By joining NMDP Research Operations with the IBMTR as the CIBMTR, the affiliation agreement between the NMDP and the Medical College of Wisconsin created a new entity, in much the same way that two people form a new entity in marriage. Given the successes of the CIBMTR during the past decade, let’s raise a congratulatory toast:Happy 10th Anniversary, and here is to the next 10 years!   Return to Top   Congratulations to 3 Working Committees   After the 2013-2014 review of the CIBMTR’s Scientific Working Committees, three have been recognized as outstanding by the Advisory Committee: Graft Sources and Manipulation Immunobiology Pediatric Cancer These committees were identified because of their strong promotion of studies that will have a notable impact on the field, willingness to forgo studies that will not have a significant impact on the field, and focus on the effective use of CIBMTR resources. In addition, these committees have cultivated external collaborations and are notably well-organized and highly productive.   Congratulations to the leadership of these committees: Mary Eapen, MD, MS – Scientific Director, Graft Sources and Manipulation and Pediatric Cancer Marcelo Fernandez-Vina, PhD – Chair, Immunobiology Daniel Fowler, MD – Chair, Graft Sources and Manipulation Gregory Hale, MD – Chair, Pediatric Cancer Carrie Kitko, MD – Chair, Pediatric Cancer Stephanie Lee, MD – Scientific Director, Immunobiology Carlheinz Mueller, MD, PhD – Chair, Immunobiology Miguel-Angel Perales, MD – Chair, Graft Sources and Manipulation Vanderson Rocha, MD, PhD – Chair, Graft Sources and Manipulation Adriana Seber, MD – Chair, Pediatric Cancer Stephen Spellman, MBS – Scientific Director, Immunobiology Elizabeth Thiel, MD, MS – Scientific Director, Pediatric Cancer Michael Verneris, MD – Chair, Immunobiology   Return to Top   Pediatric Cancer Working Committee   Autologous and allogeneic HCT are important treatment options in the multidisciplinary care for children and adolescents with cancer.  As definitions of cancer risk groups change and as new therapeutic agents are incorporated into treatment plans, the need to understand the role and timing of HCT in pediatric cancers is essential.  Rare cancers, the role of transplantation in patients with Down syndrome, the role of the conditioning regimen intensity in treatment outcomes, demonstration of graft-versus-leukemia effects, and the timing of autologous stem cell transplantation in pediatric lymphomas and neuroblastoma are just some of the topics which have been or are currently being evaluated.   The Pediatric Cancer Working Committee (PCWC) focuses on cellular therapy for children and adolescents with cancer, has conducted numerous studies addressing a wide range of issues in both allogeneic and autologous transplantation, and has worked with other Working Committees and Pediatric Cancer Consortiums collaboratively.   The PCWC is led by 3 co-chairs: Carrie Kitko, MD (University of Michigan); Gregory Hale, MD (All Children’s Hospital Johns Hopkins Medicine); and Parinda Mehta, MD (Cincinnati Children’s Hospital). The Committee's CIBMTR Scientific Director is Elizabeth Thiel, MD, and the CIBMTR statistician is Jeanette Carreras, MPH.   The Pediatric Cancer Working Committee has identified the following as its foci and priorities: Role and timing of HCT as primary pediatric cancer therapy outcomes improve, especially in identifying risk-appropriate populations; Alternative donor transplantation and graft source selection in pediatric cancers; Identification of variables in predicting outcomes of HCT in pediatric cancers; Broadening the participation of pediatric transplant physicians at all levels in the scientific endeavors of the WC. Since 2012, the Pediatric Cancer Committee has published four manuscripts (including two in Blood, one in Pediatric Blood and Cancer, and one in Biology of Blood and Marrow Transplantation) and conducted four abstract presentations at the American Society of Pediatric Hematology/Oncology annual conference, American Society of Hematology annual conference, and BMT Tandem Meetings. The PCWC has four ongoing studies addressing important topics, such as the role of HCT for hypodiploid ALL and T-cell ALL as well as identifying risk factors for relapse and leukemia-free survival in the pediatric population. The opportunity for future research is promising as the number of registration level allogeneic transplants from 2000-2013 was 15,228 and autologous transplants was 1,655. These numbers are indicative of the importance of conducting future studies to more fully understand the timing and process of HCT in children and adolescents with cancer. The PCWC is interested in studies designed to answer questions that are highly relevant for advancing pediatric patient care, particularly in areas where data from prospective trials are not available, such as rare diseases where registry data is essential for providing hypothesis-generating data that inform future prospective HCT trials.   Return to Top   Immunobiology Working Committee   The Immunobiology Working Committee (IBWC) is the largest committee of the CIBMTR. Its three co-chairs - Carlheinz Mueller, MD, PhD (German National Bone Marrow Donor Registry, Ulm, Germany); Michael Verneris, MD (University of Minnesota); and Katharina Fleischhauer, MD (Essen, Germany) - are assisted by scientific directors Stephanie Lee, MD, MPH, and Stephen Spellman, MBS, as well as statisticians Tao Wang, PhD; Hai-Lin Wang, MS; and Michael Haagenson, MS.   The IBWC addresses scientific questions about the association between genetic factors and successful transplantation outcomes. The committee endorses studies that assess genes and gene products of the major histocompatibility complex, natural killer cell repertoire, cytokine / proinflammatory cytokine and immune-response determinants, minor histocompatibility loci, and other genetic factors. The committee’s studies also include comparisons of clinical outcomes from different donor types (eg, mismatched related versus unrelated donors) and exploration of novel biostatistical and analytic approaches to investigate the impact of specific HLA mismatches. In addition, the NMDP Research Sample Repository provides a unique resource for investigators conducting retrospective analyses of immune-response determinants and transplant outcome. Currently, samples are available from more than 20,000 unrelated donor / cord blood-recipient pairs and 1,800 related donor pairs for whom complete clinical data have been collected and validated. Last year, the Repository distributed more than 15,000 aliquots to investigators. Current inventory may be viewed and requests for samples may be submitted using the instructions on the CIBMTR website.   For studies that examine the clinical role of the immune system in transplantation and do not require complete high-resolution HLA typing data and / or samples, the CIBMTR can provide clinical data on more than 42,000 HLA-identical sibling, 6,600 other-related, and 36,000 unrelated donor transplants. The IBWC currently lists 45 studies in progress, some in collaboration with other research organizations, such as the International Histocompatibility Working Group, European Group for Blood and Marrow Transplantation, and Eurocord. In the past year, nine manuscripts have been published or submitted, and seven new proposals were accepted. Publications from the IBWC may be accessed on the CIBMTR website.   Because the committee is so busy, it may seem that additional proposals would be unwelcome. However, the exact opposite is true; the success of the committee depends on vibrant scientific interactions, new ideas and testable hypotheses, and participation by individuals with different perspectives and scientific backgrounds. The IBWC encourages all investigators with an interest in immunology, immunobiology, and human genetics to become actively involved with this committee. Study proposals may be submitted year-round on the CIBMTR website. Working committee meetings convene annually at the BMT Tandem Meetings although other venues for interaction are also available. Please contact one of the chairs or a member of the scientific staff to learn more or to discuss your research ideas and proposals. We look forward to chatting with you and seeing you at our meetings!   Return to Top   2015 BMT Tandem Meetingsby D'Etta Waldoch, CMP   The BMT Tandem Meetings - the combined annual meetings of the CIBMTR and ASBMT - are North America’s largest international gathering of blood and marrow transplant clinicians and investigators, laboratory technicians, advanced practice professionals, transplant nurses, pharmacists, administrators and clinical research associates, since 1999.    Meeting Topics & Special Sessions: Adult AML Autoimmune Disease B-Cells in Chronic GVHD Checkpoint Inhibitors Fungal Infection Health Care Economics HLA for Rare Disease HLA in Transplantation Pharmacologic and Cellular Therapy for CMV Infections Transplant for T-Cell Malignancies NMDP Session Statistical Session Stem Cell Biology WBMT International Session   Plus: E. Donnall Thomas Lecture Mortimer M. Bortin Lecture 108 Oral Abstracts and 2 Poster Sessions 15 CIBMTR Working Committee Meetings 8 Meet-the-Professor Luncheon Sessions February 11-15, 2015, will find leading authorities from around the world in San Diego to present the latest developments in blood and marrow transplantation during the BMT Tandem Meetings at the Manchester Grand Hyatt. Along with state-of-the-art educational offerings, industry-supported satellite sessions and product theaters will broaden the spectrum of presentations. In addition to an outstanding scientific program, the 2015 meetings offer peripheral sessions for BMT pharmacists, BMT center administrators, coordinators, investigators, medical directors, clinical research professionals / data managers, transplant nurses, and advanced practitioners.   Keep an eye on the CIBMTR and ASBMT websites to create your own personal meeting agenda. The online registration, abstract, and housing site opened on August 1st and will firm up over the next few months. The early registration and abstract deadline is October 9th. After registering, take advantage of special conference guest room rates at the Manchester Grand Hyatt and neighboring hotels.  Don’t forget to reserve your ticket to the Saturday evening Tandem Reception aboard the USS Midway to end a memorable week with colleagues and old friends!   Questions regarding support opportunities at the 2015 BMT Tandem Meetings may be directed to Sherry Fisher at For general information, please e-mail the conference office at   We look forward to seeing you in San Diego!   Return to top   Center-Specific Outcomes Report and Analysis Forumby Carol Doleysh, BS, CPA   The SCTOD is part of the HRSA-funded C. W. Bill Young Cell Transplantation Program that collects data on all allogeneic transplants performed in the US and on transplants performed elsewhere using cellular products that originated in the US. Recent activity within the SCTOD has focused on the center-specific outcomes report and forum.   HCT outcomes reports for US HCT centers are needed to provide information requested by patients, insurers, and government agencies and to comply with current laws. The SCTOD contract requires that the CIBMTR conduct an analysis of one-year survival rates at each transplant center in the US and make these data available to the public. The 2014 Center-Specific Outcomes Report, which is currently being prepared, will include both related and unrelated HCTs performed at US transplant centers from 2010-2012. The report generated by the CIBMTR is sent to transplant center medical directors and US payors, and it is meant to be useful as a quality improvement tool for transplant centers. The data are also made available to the public on the Be The Match website.   In order to fairly address the complex issues and maintain a transparent scientific approach to center outcomes reporting, a fourth Center-Specific Outcomes Analysis Forum was held in June 2014 in collaboration with the NMDP’s Defining Quality and Value in Stem Cell Transplant meeting. Participants included representatives of the HCT community, including transplant physicians and center directors, the ASBMT, governmental funding agencies, patients, private payors, and statisticians. The major topics addressed at this year’s forum included the following. Socio-demographic factors to collect and analyze Collection and reporting of transplant center characteristics associated with outcomes Cellular infusion types to be included in the center-specific outcome analysis Reports the CIBMTR can use to assist centers in understanding performance and directing quality improvement New / future quality measures for consideration of public reporting by the CIBMTR A summary of the meeting will be distributed to US Medical Directors and posted on the CIBMTR website.   Return to Top   ASBMT Clinical Case Forum Surpasses 60 Cases ASBMT Clinical Case Forum users have already posted more than 60 cases in the short period of time since the ASBMT launched this new member benefit in February. For a limited-time only, the Forum is also open to non-members! Check out a few of the active case discussions by logging in now Visit the site and review the cases by disease state category or post your own cases for peer feedback. Questions? Email   Return to Top   Blood and Marrow Transplant Clinical Trials Network By Amy Foley The BMT CTN, with its 20 core and approximately 100 affiliate centers, has enrolled over 6,500 patients since 2003. The CIBMTR shares administration of the BMT CTN Data and Coordinating Center with NMDP and The EMMES Corporation. These three organizations together support all BMT CTN activities.   The BMT CTN Steering Committee is currently under the leadership of Chair Fred Appelbaum (Fred Hutchinson Cancer Research Center). Steve Devine (Ohio State University Medical University) continues to serve as Vice Chair, and Ginna Laport (Stanford University) is serving as Immediate Past Chair.   Clinical Trials: Open Enrollment The BMT CTN encourages widespread transplant community participation in clinical trials. If your center is interested in participating, please visit the BMT CTN website.   There are seven trials open, one released to sites, and five in development. The following BMT CTN trials are open or will soon be opened for enrollment: BMT CTN 0903 – Phase II study for allogeneic transplantation for hematologic malignancy in HIV+ patients BMT CTN 1101 – Phase III study comparing HLA-haploidentical related donor bone marrow vs. double umbilical cord blood (haplo vs. double cord) with RIC for patients with hematologic malignancy BMT CTN 1102 – Biologic assignment trial comparing RIC HCT to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate-2 and high risk myelodysplastic syndrome BMT CTN 1202 – Prospective cohort of biologic samples for the evaluation of biomarkers predicting risk of complications and mortality following allogeneic HCT BMT CTN 1203 – Randomized Phase II study of novel approaches for GVHD prophylaxis compared to CIBMTR controls BMT CTN 1204 - RIC for children and adults with hemophagocytic syndromes or selected primary immune deficiencies BMT CTN 1205 – Easy-to-read informed consent for HCT clinical trials BMT CTN 1304 / DFCI 10-106 – Phase III study comparing conventional dose treatment using a combination of lenalidomide, bortezomib, and dexamethasone (RVD) to high-dose treatment with peripheral stem cell transplant in the initial management of myeloma in patients up to 65 years (Note: this study is managed by Dana Farber Cancer Institute, but BMT CTN has endorsed the study and is providing accrual credit to BMT CTN centers).   Return to Top   2014 CIBMTR Advisory Committee Members   The Advisory Committee, made up of members from across the globe, maintains careful oversight of the CIBMTR research agenda. The 2014 committee members are listed on the CIBMTR website, and we are pleased to welcome the newest members of the committee. Lee Ann Weitekamp, MD – Collection Center Representative Jason Gangewere – Donor Center Representative Maureen Beaman – Patient / Family Representative   Return to Top   Our Supporters   The CIBMTR is supported by Public Health Service Grant / Cooperative Agreement U24-CA76518 from the NCI, NHLBI, and NIAID; a Grant / Cooperative Agreement 5U10HL069294 from NHLBI and NCI;  a contract HHSH250201200016C with HRSA / DHHS; two Grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research; and grants from our corporate and private contributors, which are listed on the CIBMTR website.    Return to Top   Abbreviations   ​Need an acronym defined? Review our list of common abbreviations.   Return to Top

Last Updated: 11/18/2014 1:41 PM