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The Health Services and International Issues Working Committee (HSIS WC) has a complementary but distinct study portfolio from other disease or outcome based WCs of the CIBMTR. We focus on population-based studies, health services research, health care and resource utilization, disparities research, secondary data analysis, clinical practice variation (on a national and global scale), international studies, survey research, and more! We have experience merging CIBMTR data sets with those from the Japan Society for Hematopoietic Cell Transplantation, US CMS / Medicare, US Veteran’s Administration Database, BMT CTN, Pediatric Health Information Service, and others. We are also familiar with the use of publicly available data such as SEER, US zip codes, US census, national GDP, etc. The HSIS WC is the result of the 2013 merger of the Health Policy / Psychosocial Issues WC and the International Studies WC. We bring together an enthusiastic and diverse group of BMT investigators worldwide, representing varied clinical and research backgrounds. Our WC gives the opportunity for international centers to ask research questions regarding their populations. If you want to ask and answer a really big question or have an idea for an innovative outcomes-related or international study, join us at the next BMT Tandem Meetings to see our committee in action.
In the last five years, our combined WC has published 15 papers, including these examples:
Our current portfolio contains 11 active studies:
The HSIS WC also collaborates with the CIBMTR Health Services Research Program operated by NMDP/Be The Match’s Patient and Health Professional Services department. The Health Services Research Program typically conducts investigator-initiated studies that require expertise and resources beyond those usually needed for CIBMTR studies. Currently, the HSIS WC and Health Services Research Program are partnering on three studies, one of which is conducted through the WC:
The Regimen-Related Toxicities and Supportive Care Working Committee (RRTWC) was created to study factors associated with morbidity and mortality after HCT. RRTWC studies focus on patient- and transplant-related factors that may contribute to toxicity and mortality. The main goal of this committee is to advance approaches that make transplants safer.
Over the past year, the RRTWC has completed important studies focusing on conditioning regimens, early transplant complications, and comorbidities. Patient factors, unrelated to disease, are closely linked with outcomes. Age and performance score are frequently applied to assess eligibility and to estimate transplant or non-relapse related mortality. The development of the hematopoietic cell transplantation-comorbidity index (HCT-CI) by Sorror et al (Blood 2005) showed that a systemic evaluation of key comorbidities present at the time of transplant can assist in predicting post-transplant outcomes, such as non-relapse mortality and overall survival. The CIBMTR and the RRTWC collaborated with Sorror by incorporating the elements from HCT-CI in the pre-TED form with the intent of validating this tool (RT07-01 – Prospective validation of the impacts of the HCT–CI, alone and combined with aging on HCT outcomes for malignant diseases). The data was collected prospectively over two years, and the results were recently published (1). We validated and quantified the effect of higher comorbidity with adverse outcomes in allogeneic transplants in myeloablative and reduced intensity settings, as well as in autologous transplants. Interestingly, the study evaluated the degree of agreement between the HCT-CI coding from the TED form and a clinician at the same center. Among four centers, there was a significant variability in HCT-CI scores, which reinforces the need for education to standardize HCT-CI scores. Clearer guidance is now available, including an online calculator. An additional question currently being addressed is the ability of HCT-CI to predict early mortality after transplants for non-malignant diseases. The registry now contains HCT-CI on all patients since 2007, which improves adjustment and comparisons for all CIBMTR studies.
Our committee helps investigators identify additional patient characteristics associated with outcomes. Artz conducted a study [RT10-01 – C-reactive protein (CRP), albumin and ferritin to predict non-relapse mortality after allogeneic HCT] to validate the impact of ferritin, albumin, and CRP on overall survival and transplant-related mortality. Assessment of ferritin and CRP was done by ELISA at the University of Chicago while albumin levels were reported by centers. The results confirmed that these three biomarkers were associated with mortality post-transplant, independent of comorbidity and other factors, and they could be added to a pre-transplant assessment to better refine predictions of morbidity and mortality.
The RRTWC conducted several studies to assess the impact of obesity on transplant outcomes. The most recent study on obesity by Aplenc and Bunin et al (RT 0902 - Effect of body mass in children with hematologic malignancies undergoing allogeneic bone marrow transplantation) focused on children with malignant diseases. The results demonstrated no significant impact of obesity, measured by age-adjusted body mass index, on transplant outcomes (2).
Conditioning regimen strongly influences transplant toxicity and, as a modifiable factor, is particularly relevant. The RRTWC assisted in the creation of the currently used working definition of conditioning regimen intensity for CIBMTR analyses (3). During the past year, the RRTWC conducted several studies addressing different aspects of conditioning regimens. Hong et al (RT12-04 - Comparison of non-myeloablative conditioning regimens for lymphoproliferative disorders) compared non-myeloablative regimens used for transplants in lymphoma based on the use of total body irradiation (TBI) (4). The study demonstrated that TBI-based non-myeloablative conditioning regimens for lymphoproliferative diseases are currently used less frequently than chemotherapy-based regimens. The latter are similar to regimens commonly used in the non-transplant setting. The outcomes after transplant between regimens with or without TBI were similar. Chen and Lane et al (RT11-01 Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous HCT) analyzed different conditioning regimens for autologous transplant in lymphoma (5). The study compared commonly used conditioning regimens and demonstrated disparate outcomes according to the regimen and the type of lymphoma histology. The results suggest a more tailored approach in selecting a high dose regimen for lymphoma. For example, the use of busulfan-based regimens in Hodgkin disease was associated with worse outcomes than a BEAM (carmustine, etoposide, cytarabine, melphalan) regimen, and CBV (cyclophosphamide, carmustine and etoposide) with standard carmustine dose (300mg/m2) in follicular lymphoma was associated with better outcomes than other regimens.
Another conditioning question related to the sequence of agents was answered by the publication by Holter-Chakrabarty et al (RT 1201 - The sequence of cyclophosphamide and myeloablative TBI in HCT for patients with acute leukemia) where the sequence of TBI and cyclophosphamide (Cy) for acute leukemia was explored (6). The findings demonstrated that CyTBI and TBICy were equally common and not defined by center. Outcomes were similar between the two different sequences, arguing against previous concerns with altered TBI sequence and worse outcomes. The RRTWC also developed a prospective cohort study sponsored by Otsuka Pharmaceutical Development and Commercialization to compare intravenous busulfan (BU) based myeloablative conditioning regimen to TBI based conditioning. The results of this large cohort study demonstrated that the outcomes with Bu were superior to TBI for myeloid malignancies undergoing myeloablative regimens (7).
Primary graft failure was also addressed by the RRTWC. The first study by Schriber et al reported on dismal outcomes for second allogeneic transplants for patients who experienced primary graft failure (8). In a follow-up study by Olsson et al (RT 0901 Primary graft failure following allogeneic HCT for the treatment of hematological malignancies – Leukemia 2015), factors associated with the development of graft failure were developed and a score was proposed to identify patients with high risk of graft failure (9).
The RRTWC collaborates with Hahn and Sucheston-Campbell on a large genome-wide association study to identify genetic determinants for early mortality after unrelated donor HCT [RT09-04/IB09-06a - Adjudication of cause-specific mortality after unrelated donor allogeneic HCT for acute leukemia and myelodysplastic syndrome: The primary endpoint of a future genome-wide association study (GWAS)]. The adjudication of causes of death reported to the CIBMTR and a proposal for ranking and categorization for this and other studies was recently published (10).
Moving forward, the RRTWC has a comprehensive portfolio of new studies. The major themes of approved studies include exploring toxicity in pediatric transplants, assessing determinants of critical care in transplant patients, reporting on incidence and prognostic factors for endothelial injury syndromes, and updating conditioning regimen intensity definitions.
Dick van Bekkum, one of the pioneers of BMT, passed away on July 17 after a short illness. He would have turned 90 on July 30. Professor van Bekkum dedicated his life to science and medicine, contributing to the HCT field in numerous ways, including seminal contributions to the IBMTR. Read the obituary written by Bob Lowenberg to remember Dick van Bekkum’s life and many achievements.
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Meeting Topics and Special Sessions
The SCTOD is part of the HRSA-funded C. W. Bill Young Cell Transplantation Program (Program), which collects data on all allogeneic transplants performed in the US and on transplants performed elsewhere using cellular products that originated in the US.
Managed by the Program, the HRSA Blood Cell Transplant website features basic transplant, cord blood, and donor information; a description of the Program and its contractors; and a search feature for patient survival and center volumes data. An increasing amount of information useful to patients is being made available on this website, including the Transplant Activity Report for HCTs performed from 2008 to 2012 and center volumes data for HCTs performed from 2009 to 2013.
The Transplant Activity Report was posted for the first time in 2015 and provides static reports of the number of transplants performed at US transplant centers. These data include all types of transplants, including autologous, related allogeneic, and unrelated allogeneic as reported by transplant centers. The tables provide annual transplant numbers by age of patient; cell source (bone marrow, peripheral blood stem cells, and umbilical cord blood); disease; donor type (autologous, unrelated allogeneic, and related allogeneic); gender; race; state of transplant center; and year.
New or updated data has recently been made available on these websites:
Center Volumes Data – data has been updated so queries can be run by center or disease on HCTs performed from 2009 to 2013
The BMT CTN Steering Committee is currently under the leadership of Chair Fred Appelbaum, MD (Fred Hutchinson Cancer Research Center), Chair-Elect Steve Devine, MD (Ohio State University Medical University), and Vice-Chair Rick Jones (Johns Hopkins University).
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 ten trials open to accrual, two released to sites, and eight in development. The following BMT CTN trials are open or will soon be opened for enrollment.
There are 51 BMT CTN published articles, including 14 primary analyses. The following manuscript was recently published.
Three of our trials recently closed to accrual when they met their protocol accrual goals. RCI BMT also opened three new trials recently of which all three have had their first accruals. We are actively collecting data on 14 different studies and projects managed by RCI BMT staff. Staff members are working with the statistical and protocol team members to prepare data sets on four studies for either upcoming abstracts or manuscripts.
The Survey Research Group call tracking enhancement project went live on March 23. This new call tracking system increases the stability and efficiency of study, time point, subject, and contact attempt management and processes. It allows the Survey Research Group to view and manage all activities for multiple studies in one place and automates several workflow, analysis, and study management activities that were previously very manual, difficult, and inconsistent across studies.
One of the most valuable features of the system is it auto-assigns contact attempt tasks to staff members daily. When a new subject is added to the system, the first contact attempts for each current and future time point are automatically created. As staff members contact a subject within their time point window, they can select when the next contact attempt should be by day and timeframe (morning, afternoon, evening, or anytime) as well as the task type (call, email, letter, etc).
Another valuable feature of this new system is the reporting functionality. With data about all studies, subjects, and time points in one place, staff members can create and save complex reports. These reports can incorporate multiple data points, and staff members can easily refresh the reports with the latest data or review any specified period of time. Currently staff members are using the system for four studies, and the plan is to add all future studies the Survey Research Group supports.
Final approval was made to proceed with Medidata Solutions, Inc. RAVE® and CTMS™ for the RCI BMT clinical trial data collection and trial management system. We are excited to begin implementing this interconnected system, which will allow RCI BMT to effectively and efficiently provide support for a wide array of clinical trials and research studies.
The Health Services Research Program partners with the Health Services and International Issues Working Committee (HSIS WC) and the NMDP/Be The Match Payer Policy Department to maximize clinical, research, and policy expertise in the following focus areas:
We recently initiated a number of exciting research and patient-centered projects:
Our research portfolio also includes on-going research (select studies):
Recent Health Services Research Program peer-review publications:
Finally, we are planning next steps for the Health Economics Interest Group, which held its first meeting at the BMT Tandem Meetings in February. Interest Group members will receive an update in August.
For any questions about the Health Services Research Program or the Health Economics Interest Group, please contact Ellen Denzen at firstname.lastname@example.org.
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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 sincerely thank each one for their time and efforts.
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.