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The focus of the Chronic Leukemia Working Committee is to define the optimal timing and improve the outcome of patients undergoing transplantation for MDS, CML, CLL, and myeloproliferative disorders. Over the last five years, the members of the committee published 11 manuscripts and presented several oral and poster presentations. Topics included comparing conditioning regimens and the intensity of the conditioning regimen in an attempt to improve outcomes and reduce regimen-related toxicity. One study used a large data set to validate and refine an index to predict a patient’s risk of relapse after transplantation. Studies under development focus on the optimal timing of transplantation in diseases where new non-transplant therapies have emerged. The committee is also responsible for reviewing and revising data collection forms, which will serve as the basis for future studies. Below we highlight a few published studies and proposals currently underway.
Immediate Past Chairs:
Dr. Edward Copelan has led a number of studies comparing ablative conditioning regimens and assessing outcomes and toxicity. In a recent study, the writing committee compared cyclophosphamide in combination with intravenous or oral busulfan and total body irradiation in patients with CML undergoing myeloablative transplantation. This study adds to a growing literature that busulfan-based conditioning regimens are more effective in disease control with an acceptable toxicity profile. A current study looks to define the appropriate timing of transplantation for patients with CML who have progressed on imatinib. The study requires comparing outcomes in a non-transplant patient database, which resides at an outside institution to the CIBMTR Research Database. This type of collaboration is strongly encouraged.
Dr. Philippe Armand led an important study refining the Disease Risk Index, which estimates a patient’s risk of relapse after transplantation based on pre-transplant characteristics. This information is valuable to clinicians when discussing expectations about transplantation with patients and family. Equally important is the establishment of uniform criteria, which can be applied to patients in other clinical trials so that a more direct comparison of patient populations can be made and outcomes compared.
The committee published a number of studies focusing on myeloproliferative diseases. Myeloproliferative neoplasms are rare diseases, and registry studies have a unique role in reporting outcomes in this patient population after transplantation. One study compared the outcome of patients after ablative and non-ablative regimens. These data help guide clinicians in their choice of conditioning regimens for this difficult-to-treat group of diseases. A future study will compare transplantation with non-transplant strategies in order to define the role and timing of transplantation for these diseases.
The committee welcomes new participants as well as new proposals. We also encourage collaboration with other committees and the use of outside data sets, which can be used to better define the role and timing of transplantation as new non-transplant strategies emerge. The committee encourages individuals to help in revising and updating the data collection forms. With emergence of molecular data, database form revisions will be needed to strengthen the Research Database and provide a valuable resource for years to come. We encourage young investigators to participate in this effort as it is an excellent opportunity to become familiar with the current data and make use of this powerful resource. The next in-person meeting of the Chronic Leukemia Working Committee is at the CIBMTR/ASBMT BMT Tandem Meetings in Hawaii in February 2016. We look forward to seeing you there and welcoming new proposals. A link has been provided that outlines the work of this committee over the last several years and current proposals under development. We hope this information will stimulate your thoughts on new proposals.
Based on the CIBMTR Summary Slides from 2014, infection is reported as the primary cause of death in up to 17% of patients receiving autologous and allogeneic transplantation. However, transplant clinicians recognize that infection accounts for significantly greater morbidity in our patients. This burden of infections and its correlation with post-transplant immune reconstitution is the focus of the Infection and Immune Reconstitution Working Committee’s efforts.
Our committee faces unique challenges due to the complex interactions of multiple time-dependent co-variates as well as the complexities associated with reporting multiple (and often recurrent) infections caused by a diverse range of pathogens. The rates of post-transplant infections are also associated with factors such as graft type, donor-recipient mismatch, recipient age, and GVHD incidence. Furthermore, infections prior to the transplant also impact transplant outcomes. Given the complexity of these factors, our analyses rely heavily on novel statistical techniques provided by our excellent MS Statistician, Min Chen, and our PhD Statistician, Kwang Woo Ahn. The committee welcomes Soyoung Kim, a new PhD Statistician who joined our group in September 2015. We look forward to her ideas and contributions.
The committee recognizes that data collection for infections and immune reconstitution is complex and particularly demanding. In part, the significant effort required to document infection endpoints has contributed to under-reporting biases evident in prior analyses. Given this complexity, we greatly appreciate the time dedicated by all data managers to provide us with the high quality data needed to understand and improve infection-related transplant outcomes. To streamline and improve the quality of infection-related data, the committee leadership has formed a task force with the recently formed ASBMT Infectious Disease Special Interest Group to redesign data collection mechanisms. This will include improved information collection regarding antimicrobial prophylaxis and greater detail regarding certain infectious complications. Dr. Riches is currently collaborating with a group of investigators to pilot these proposed collection forms in an oligo-center prebiotic trial that is currently in development.
Several manuscripts from our committee are currently in press, under review, or ready for submission. These include:
While all studies to date have focused on infections, the committee would like to expand efforts to study correlates of immune recovery, including lymphocyte subsets and immunoglobulin levels, especially as the quantity and quality of these data improve over time. The committee welcomes proposals to further investigate these issues.
The CIBMTR’s Research Database has information on approximately _______ patients.
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CIBMTR investigators will present the following 25 abstracts at ASH:
Visit the 2016 BMT Tandem Meetings homepage to register and view additional details. At the early registration deadline, there were more than 1,300 participants registered. The last day for general registration rates is January 16, 2016, and the last day to book your hotel is January 20, 2016. After registering, take advantage of special conference guest room rates at a wide variety of hotels within the BMT Tandem Meetings room block. Remember to reserve your ticket to the Tandem Reception on Sunday evening at the Royal Hawaiian Hotel on the Ocean Lawn and in the Monarch Room.
The BMT CTN has completed its first SCD trial, BMT CTN 0601. This study, using unrelated donors for transplantation of children with SCD, was undertaken because of the limitations of identifying suitably matched siblings as donors. The trial employed a reduced intensity-conditioning regimen and bone marrow grafts from volunteer donors or banked umbilical cord blood. The trial’s primary objective was event-free survival at 1-year. (The events are death or graft failure.) The primary results will be presented at the ASH annual meeting in December. (See details in ASH Presentations section below). The cord blood arm of the trial was closed earlier for excess graft failure (Kamani N et al. Biology of Blood and Marrow Transplantation, 2012).
Given the successful accrual of the 0601 study, the BMT CTN has been recognized as a network that effectively brings together hematologists and transplant physicians to treat SCD patients on transplant clinical trials. This collaboration has resulted in the development of two subsequent SCD protocols:
Both trials are slated to be released to centers and posted on the BMT CTN website by early 2016.
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 12 trials open to accrual and 9 in development. The following BMT CTN trials were recently opened for accrual:
BMT CTN Investigators will present these abstracts at the ASH annual meeting in December:
There are 57 BMT CTN published articles, including 16 primary analyses. The following manuscripts were recently published.
To get up-to-date information about BMT CTN studies, meetings, and news:
The patient-based session provided novel insights into the HCT care delivery process and clearly drove discussions throughout the meeting. At the meeting’s conclusion, the most highly audience-rated areas of interest to tackle in the next one to two years were measures of quality of life and care coordination. Within the broad area of quality of life, key topics lending themselves to measuring outcomes included a return to “normal,” financial health, psychosocial well-being, and long-term follow-up care issues. Within the area of care coordination, voiced interests were timely HLA typing, referral for transplant consultation, a multidisciplinary team approach to post-transplant complications including GVHD management, and communication at times of care transitions. Furthermore, the primary theme that emerged from the meeting was: “We need to create and retain a patient-centered focus.”
This patient-based session clearly demonstrated how impactful patient engagement can be and the urgent need to include patient-centered outcomes research (PCOR) within the Health Services Research Program’s portfolio. PCOR addresses the questions and concerns most relevant to patients, with other key stakeholders including caregivers, healthcare providers, researchers, payers, and policy makers. Involvement by patients and other non-researchers guides research prioritization and innovations in care delivery and impacts healthcare policy. Importantly, PCOR helps patients and their care providers make informed healthcare decisions and, ultimately, improves healthcare delivery.
For more than a decade, Dr. Douglas Rizzo has led CIBMTR efforts to collect patient-reported outcomes (PRO). A quality of life study was performed in the late 1990’s, and PRO have been collected in a substantial number of BMT CTN studies. Led by Drs. Bronwen Shaw and Douglas Rizzo, the CIBMTR is currently completing feasibility analyses of collecting PRO directly from patients after enrollment through the transplant center.
The CIBMTR and Health Services Research Program will lead efforts in PCOR and measure development within the HCT community. Stay tuned for more details in the coming months about how we plan to get going and how you can be involved. In the meantime, contact Ellen Denzen, Senior Manager of the Health Services Research Program, at firstname.lastname@example.org with any questions or suggestions.
Summaries are created through a collaborative process involving CIBMTR Consumer Advocacy Committee members; CIBMTR and NMDP/Be The Match Medical Writers, Communications Specialists, and Patient Education Specialists; and CIBMTR Scientific Directors. Developing these summaries is one of the main initiatives of the Consumer Advocacy Committee.
The Consumer Advocacy Committee was created in 2005 as a subcommittee of the Advisory Committee to communicate CIBMTR research results and data to the non-medical community and to provide patient and donor perspectives during the development of the CIBMTR research agenda. Many members have personal experience as a donor, recipient, or family member.
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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 all of our committee members for their time and efforts.
The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U10HL069294 from NHLBI and NCI; a contract HHSH250201200016C with Health Resources and Services Administration (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.