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The Autoimmune Diseases and Cellular Therapy Working Committee (ACWC) was formed in 2014 via the merging of two existing committees, the Autoimmune Diseases WC and the Cellular Therapy WC. This created a unique committee with a broad scope and diverse applications. The unifying aspect of these two areas is the requirement of outreach to other specialties for collaboration in projects. Also, there is overlap with the use of cellular therapy for treatment of autoimmune diseases.
The ACWC’s mission is to promote studies of HCT and other cellular therapies for autoimmune disease and to investigate novel uses of cellular therapies for both malignant and non-malignant indications. The committee’s leadership is comprised of individuals with expertise in transplantation, cellular therapy, cell processing, and autoimmune diseases.
Autoimmune disease remains an uncommon transplant indication. To date, physicians have primarily transplanted patients with multiple sclerosis (MS) and scleroderma but also transplanted patients with systemic lupus erythematous, inflammatory bowel diseases, autoimmune cytopenias, diabetes, and other diseases. Most transplants are autologous, which rely on immune-ablative regimens to control autoimmunity. Applications of allogeneic transplant remain mostly investigational. From 2008 to 2014, 148 autologous and 40 allogeneic HCTs were reported to the CIBMTR by 42 centers.
The main challenge for this committee remains the availability of disease specific data for more comprehensive studies. Disease assessment in autoimmune diseases is often complex and requires organ specific disability assessments, and input from disease specialties is ideal. Data collection for these diseases is different from other more common hematological or malignant disease transplant indications, in which disease specific information is readily available to data managers. The CIBMTR has undertaken several efforts to improve data collection, including simplifying report forms. For example, the committee reached out to the EBMT Autoimmune Working Party and harmonized the MS forms. Similar efforts on scleroderma forms are ongoing. Still, studies in this committee require additional active data collection to supplement information about disease status at transplant, disease response, and progression events after transplant.
One of the studies recently completed, AI 0901 (PIs: Paolo Muraro, MD, and Ricardo Saccardi, MD), was made possible by the collaboration of the EBMT Autoimmune Working Party and centers from Brazil with high activity in transplants for autoimmune disease. In this study, 283 autologous transplant recipients with MS were analyzed, making this the largest study with the longest follow-up to date. The analysis demonstrated that responses to transplant are long lasting and patients who receive transplants while in earlier and more inflammatory forms of the disease (relapsing remitting) have better disease control. This study emphasized the importance of transplant as a therapeutic option for MS patients and will lead to further studies, which are anticipated to result in a change in practice. Given the success of the MS study, the committee is focusing on similar collaborations to describe long-term outcomes of patients who receive transplants for scleroderma (AC 14-01 – PI: Dominique Farge, MD).
Cellular therapy application is a rapidly emerging area with a very large scope. The CIBMTR initiated efforts to study the use of cellular therapy in 2008 through the SCTOD, which assessed alternate uses of hematopoietic cells but did not include promotion of hematopoietic cell engraftment. At that time, applications in cardiology were rapidly expanding the use of autologous stem cells with the intent to restore or heal cardiomyocyte, known as regenerative medicine. The CIBMTR Cellular Therapy Working Committee reached out to the cardiology community to establish this collaboration.
The CIBMTR also modified the data collection structure to accommodate collection of cellular therapy. This required the development of a structure that did not use transplantation as the main milestone for data collection, as these patients are not transplant recipients. Thus, patients who receive cellular product without the intent to establish hematopoiesis are directed to the cellular therapy track when completing the CIBMTR Identification Form (CRID form). The cellular therapy form, called Form 4000 or CTRM, is a single stand-alone form without follow-up. Its intent is to capture activity, and it incorporates limited outcome data related to infusion adverse events. Since its implementation, data for 534 patients who received cellular therapy for several indications were reported by 10 centers. The most common indication is the use of autologous cord blood unit for treatment of congenital neurologic disorders, mainly cerebral palsy and congenital hydrocephalus. However, this is not representative of all activity, as only a small number of centers report this data to the CIBMTR.
The cellular therapy field has evolved over the past five years, with the use of genetically modified cells for treatment of malignancy. These are mainly chimeric antigen receptors (CARs) in lymphocytes for cancer immunotherapy. The CIBMTR started to receive reports of CAR T cells for treatment of acute leukemia in the CTRM. The committee recognized a need to adapt to the evolving nature and practices on cellular therapy and developed a task force to revise the data collection practices and expand the focus of cellular therapy to include the use of treatment of malignancies. During the last in-person committee meeting, many committee members recommended expanding the activities of the committee to capture more details on these indications. After a large number of members volunteered to participate on the task force, the committee leadership decided to hold a forum on cellular therapy and data collection. The objectives of this forum are to revise the structure of data collection for cellular therapies by the CIBMTR, develop potential projects that could jump start the data collection, and network with the centers that are most actively involved in these therapies.
In parallel to studies of cellular therapy in the non-transplant setting, the CTWC investigates more frequent uses of cell therapy in allogeneic transplant. After allogeneic transplant, so called donor cellular infusion (DCI) is performed for treatment of disease relapse, falling chimerism, GVHD, infections, and other post-transplant issues. The transplant community is very interested in understanding indications and efficacies of DCI. The CTWC has two studies focusing on these indications:
In summary, the ACWC has a number of exciting projects. For both areas of focus, there is increasing activity, which the CIBMTR can assist in further advancements.
The Donor Health and Safety Working Committee (DHSWC), now in its 10th year, is led by Chairs Paul O’Donnell, MD, PhD, FACP; Michael Pulsipher, MD; and Galen Switzer, PhD. The DHSWC’s research priority is to understand the impact of donation on both related and unrelated hematopoietic stem cell donors. A number of important retrospective and prospective studies over the past five years are increasing our understanding of the medical and psychosocial risks involved in marrow or PBSC donation and have or are likely to change clinical practice in the management of adult and pediatric donors. Two recently reported studies found significant differences in the incidence and duration of adverse events following marrow vs. PBSC donation by volunteer unrelated donors, and a third study showed the impact of race and ethnicity on collection of PBSC from unrelated donors. With respect to related donors, the important RDSafe study, supported by the DHSWC and an RO1 NIH grant held by committee Chairs, Drs. Pulsipher and Switzer, is examining a number of aspects of the donation process on donor outcomes, including severity of adverse events and donor quality of life (QOL). Prior to the RDSafe study, no prospective study of a large cohort of related donors had been performed. This study completed accrual in 2014. Analysis is progressing, and results are being submitted for publication. In research published in 2010, a CIBMTR survey examining practice patterns of related donor care in US transplant centers raised issues of conflict of interest in the management of related donors and helped lead to a change in the 5th Edition of the FACT Standards. This encouraged the separation of care of related donors and recipients, a practice which has always been routine in the management of unrelated donors. A follow-up CIBMTR survey of donor practice patterns recently completed, and soon to be submitted for publication, documents a favorable change in the separation of donor-recipient clinical care at US transplant centers in response to FACT Standards, a change which is now mandatory in the current 6th Edition Standards.
The DHSWC is pleased to see a steady increase in attendance at the committee meetings over the past three BMT Tandem Meetings. We encourage participation from the transplant community, especially new members, in ongoing studies or through the submission of new proposals.
The DHSWC would like to acknowledge the important contributions to the research focus and operations of the committee by Dennis Confer, MD, who guided the committee as Scientific Director from its inception in 2005 until recently taking the position of Ex Officio Senior Advisor of DHSWC. We would also like to thank Bronwen Shaw, MD, PhD, who succeeded him as Scientific Director of the committee and who previously served as a Chair of the committee until her recent appointment as Associate Scientific Director of the CIBMTR. We also want to acknowledge the essential contributions of the dedicated Biostatisticians who support this committee including Brent Logan, PhD; Pintip Chitphakdithai, PhD; and Deidre Kiefer, MPH, in the design, analysis, and publication of the committee’s research.
To view more photos from the 2015 BMT Tandem Meetings, visit the CIBMTR Facebook page.
If you answered “yes” to any of these questions, then we invite you to join the newly established Health Economics Interest Group.
The CIBMTR Health Services Research Program, in conjunction with NMDP/Be The Match Payer Policy, held the inaugural meeting of the Interest Group at the 2015 BMT Tandem Meetings in San Diego. Our goal was to bring together investigators interested in these topics and determine if there was enough enthusiasm to work towards developing a health economics research agenda in transplant.
Approximately 40 transplanters, researchers, and payer representatives attended the inaugural meeting. Michael Boo, JD, Chief Strategy Officer for NMDP/Be The Match, opened the session by stressing the relevance of health economics in transplant and was followed by Navneet Majhail, MD, Director of the BMT Program at Cleveland Clinic and former Medical Director of the Health Services Research Program. Dr. Majhail provided an overview of the Program’s accomplishments in economics research to date. Linda Burns, MD, the current Medical Director of the Health Services Research Program, then led the group in a lively discussion of the economic landscape and challenges in transplant.
Attendees voiced concerns regarding the numerous challenges facing our field, including increasing pressure to reduce the cost of transplants, improve quality, respond to the changing landscape of health care reform, optimize resource utilization, and justify transplant versus non-transplant therapies. Attendees from centers outside the US provided an international perspective on resource utilization. Payers explained they need evidence from transplant centers that they are providing the best outcomes for their patients while being transparent in all components of cost and cost allocation.
All present agreed there was considerable enthusiasm to continue to work together as an Interest Group. Next steps include a webinar later this year to enhance peer learning and promote standard investigational methodology in health economics. A follow-up meeting is planned in conjunction with a relevant professional society meeting (location and date to be determined). Stay tuned for more information, and if you’d like to join the Health Economics Interest Group in the meantime, send your contact information to Ellen Denzen, Health Services Research Program Senior Manager, at firstname.lastname@example.org. Looking forward to partnering with you!
This change applies to the forms required for autologous transplant recipients who do not give consent to participate in the Research Database protocol to ensure the continued epidemiological integrity of the CIBMTR outcomes registry and to meet our obligations to provide the US government with an accurate assessment of transplant activity.
The Pre-TED form may be submitted to the CIBMTR without the consent of the recipient because the CIBMTR meets the definition of a Public Health Authority (PHA) under the Health Insurance Portability and Accountability Act (HIPAA). In this capacity, the CIBMTR is authorized to collect individually identifiable health information without consent or authorization of the individual. The PHA designation also allows transplant centers, which fit the definition of covered entities, to disclose these data to the CIBMTR under 45 CFR 164.512 (Privacy Rule) without the direct consent or authorization of the recipient.
For autologous transplant recipients who do not provide consent for the CIBMTR Research Database, completion of forms other than the Pre-TED will not be required. Data collected on the Pre-TED forms will not be used in research. Important factors supporting this change include:
We thank you for approaching all HCT recipients at your center, regardless of their graft source, to ask them to consider participating in the CIBMTR Research Database protocol. The success of the CIBMTR research program is built on the dedicated contributions of our transplant centers.
The BMT CTN Steering Committee is currently under the leadership of Chair Fred Appelbaum, MD (Fred Hutchinson Cancer Research Center). Steve Devine, MD (Ohio State University Medical University) is now serving as Chair-Elect, and Rick Jones (Johns Hopkins University) started his term as Vice-Chair on January 1.
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 nine trials open to accrual, three released to sites, and seven in development. The following BMT CTN trials are open or will soon be opened for enrollment.
Institute for Healthcare Advancement’s Health Literacy Conference (May)
APOS/IPOS World Congress on Psycho-Oncology 2015 (July)
There are 50 BMT CTN published articles, including 14 primary analyses. The following manuscripts were recently published.
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The Advisory Committee, made up of members from across the globe, maintains careful oversight of the CIBMTR research agenda. The 2015 committee members are listed on the CIBMTR website, and we are pleased to welcome the newest members of the committee:
The CIBMTR is supported by Public Health Service Grant / Cooperative Agreement 5U24CA076518 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.
Need an acronym defined? Review our list of common abbreviations.