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CIBMTR: Advancing Transplant Research

The CIBMTR is a global collaboration of researchers, clinicians, and centers working together to improve patient outcomes in transplant medicine. With over 500,000 registered patients and 1,300 publications, they conduct practice-changing research, understand successful outcomes, and address access to care. Their work includes cellular therapy outcomes, clinical trials, bioinformatics, and more.

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CIBMTR: Advancing Transplant Research

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  1. CIBMTR 2019 What your work makes possible Bronwen Shaw Tuesday February 19, 2019 >500,000 PATIENTS >2,700 RESEARCHERS >400 CENTERS >1,300 PUBLICATIONS >40 COUNTRIES

  2. A WORLD OF RESEARCHERS >5,000 researchers, clinicians, and others >400 transplant centers >40 countries WORKING TOGETHER >195 studies in progress >1,300 publications >2.6 million biorepository samples for >150,000 donors and recipients FOR LIFE >500,000 patients registered ~25,000 new patients registered annually

  3. GLOBAL COLLABORATION

  4. CUMULATIVE TRANSPLANT PATIENTS REGISTERED

  5. Geographic Distribution of All Transplants Registered,1968 through 2018* United States Canada Europe Asia Australia / New Zealand Mideast / Africa Central / South America Allogeneic transplants N=290,164 Autologous transplants N=276,384 *Transplants continue to be registered for 2018; Data are incomplete

  6. CELLULAR THERAPY OUTCOMES REGISTRY • State of the Art Cellular Therapy Outcomes Registry established, designed to address to the needs for long-term follow-up of patients receiving genetically manipulated cells • Integrated with HCT outcomes registry • Pilot study of cellular therapy data collection completed • New versions of cellular therapy data collection forms using input from the pilot study, industry partners and regulatory agencies • Time studies to estimate appropriate reimbursement schedules

  7. Type of Cells Used for Cellular Therapies: 2016-2018

  8. Prior History of Transplant among CAR T cell Recipients: 2016 to 2018 (N= 646)

  9. CAR T Cell Indications: 2016-2018 (N=646) *Centers: 84 US Median age: 55 y (1-81)y Prior allo HCT: 12%

  10. Active Projects Utilizing the CT Registry

  11. WHO WE ARE – MORE THAN A DATABASE Large Network of Clinical Centers Scientific and Statistical Expertise Unique and Extensive Clinical Database CIBMTR RESEARCH

  12. WHY WE ARE - IMPROVING PATIENTS’ LIVES Dedicated to improving survival, treatment, and quality of life Conduct practice-changing research helping patients and physicians UNDERSTAND THE BIOLOGY OF SUCCESSFUL OUTCOMES SELECT DONORS, GRAFTS, TREATMENT REGIMENS ADDRESS ACCESS TO CARE AND FUTURE WORKFORCE NEEDS EVALUATE PATIENT RISK IDENTIFY LONG-TERM EFFECTS OF TRANSPLANTATION AND GUIDE MEDICAL CARE FOR SURVIVORS

  13. HOW WE DO IT Collecting clinical outcomes data worldwide for 45 years 6 major areas of research activity

  14. SCIENTIFIC WORKING COMMITTEES 15 Scientific Working Committees 47HCT global experts chair committees in their field – thousands participate 109 publications In 2018 65 presentations this year >195 ongoing studies >2,700 worldwide researchers

  15. PUBLICATIONS BY YEAR – 109 in 2018

  16. MEDICARE CLINICAL TRIALS AND STUDIES Coverage with Evidence Development (CED) studies allow CMS to provide coverage for patients whose data are reported to CIBMTR; CIBMTR generates the data that will inform policy decisions Currently engaged in 5 CMS CED studies

  17. MEDICARE CLINICAL TRIALS AND STUDIES

  18. MEDICARE CLINICAL TRIALS AND STUDIES

  19. STEM CELL THERAPEUTIC OUTCOMES DATABASE (SCTOD) Transplant Center Volumes 2013-2017 Center-Specific Survival Analysis Contract RENEWED with HRSA to track and analyze all allogeneic transplants performed in the US and transplants performed globally with products from the US Research Summaries for Patients

  20. RESEARCH REPOSITORY 70,992 from unrelated donors and 9,211from related donors 63,678 from unrelated recipients and 9,579 from related recipients >2.6 million samples 12,153 from unrelated cord blood units >408,000 samples from >10,600 patients on BMT CTN trials

  21. BIOINFORMATICS • Develop pipelines to analyze NGS data and immune genes (HLA, KIR) • and whole genomes • Investigate the role of genetic ancestry in transplantation • Develop data standards and tools for making data portable • Investigate global HLA populations • Develop methods for HLA association studies

  22. HSR ACTIVITIES • Access to and • Health Economics • of HCT • Unmet need for HCT • Reimbursement analysis of HCT costs in older patients Costs of HCT by transplant setting • Survivorship • Quality of Life and • Patient Reported Outcomes • Individualized survivorship care plans • Patient-centered outcomes research agenda in HCT • Stepped care self-management program for survivors • Treatment Decision • Making • Translation of clinical research into practice

  23. Blood and Marrow Transplant Clinical Trials Network (BMT CTN) >10,600patients enrolled on BMT CTN trials since 2003 Renewed for 4th, 7-year cycle >408,000 research sample repository 46 clinical trials >95 publications

  24. Resource for Clinical Investigations in BMT (RCI BMT) • >36,000patients accrued to • 18 studies, including >21,000 on a long-term (20 years) donor follow-up study • 6 studies enrolling patients (Includes 2 INDs and 1 IDE) • 7 studies in development • 4 RCI BMT studies (2 INDs) • 3 BMT CTN studies

  25. Resource for Clinical Investigations in BMT (RCI BMT) • Implemented a new electronic patient‐reported outcomes (ePRO) system to be used by Survey Research Group to support: • Clinical trials • Long term follow‐up/direct patient contact • Quality of life assessment for patients and donors • 3 upcoming BMT-CTN studies will use the ePRO system to collect QOL data • 1702/1703/1704 • 1702 will all have pre-HCT data collected in FormsNet

  26. Developing statistical models to use in HCT and cellular therapy research and comparing new models to existing solutions using the CIBMTR Research Database • Statistical integrity of CIBMTR scientific activities ensured • Articles on HCT‐related statistical issues for clinical audiences supported • Working Committee investigators supported in developing scientific studies

  27. ADDRESSING IMPORTANT ISSUES • Neurocognitive dysfunction in HCT recipients • Autologous HCT versus allogeneic HCT in patients with follicular lymphoma • Impact of Upper GI GVHD • Comparing BM and PBSC in children HCT • Practice pattern changes and improvements in HCT for primary immuno-deficiencies • A younger unrelated donor results in the best outcomes • Engaging patients in setting a patient-centered outcomes research agenda • What do transplant physicians think about palliative care?

  28. 76 PRESENTATIONS

  29. SHARING KNOWLEDGE – in ways that are useful to you INFORMATION REQUEST SERVICE > 433 custom analyses PUBLIC WEBSITE >393,418 views in 2018 DATA SHARING and ANALYTIC TOOLS eDBtC – 261 unique users, 189 different centers accessed 3,455eDBtC sessions, 159 users from 137 centers downloaded data 906 times Disease Risk Index (DRI) calculator – 4,310 views VOD Risk Calculator –1,226 views Survival Calculator – 3,779 views Center Performance Analytics - 97 users, 377 sessions Data for RFI – 142 users, 687 sessions

  30. SHARING KNOWLEDGE – in ways that are useful to you DATA MANAGEMENT 2018 Data management manual – 219,983 unique page views Data collection forms – 42,964 unique page views Training and reference –68,327 unique page views

  31. Everyone has a donor CIBMTR The Future CAR-T cells Gene Therapy New indications New Regimens

  32. EVERYONE HAS A DONOR HLA-identical sibling BM/PB The Challenge: Which donor is best? What are the barriers to getting to HCT? HLA-mismatched relative BM/PB HLA-matched and mismatched unrelated BM/PB Unrelated umbilical cord blood

  33. NEW INDICATIONS: CHALLENGES New endpoints Tissue repair – regenerative medicine Cardiac Neurological Orthopedic Fibrosis New centers/community New experts New diseases

  34. LONG TERM FOLLOW UP: CHALLENGES Patient-reported outcomes Direct Patient contact Consent to provide contact details to CIBMTR Long term follow up Subsequent neoplasms Pregnancy

  35. AGNIS • The goal of AGNIS is to make data exchange easier by creating a: • Standardized vocabulary for BMT data collection utilizing industry tools like the caDSR • Data sharing system that facilitates electronic form submission from local databases to the CIBMTR database with access to the data via the FormsNet3 application

  36. AGNIS • 2018 Accomplishments • 25 centers submitting data (6 direct /19 with BMT vendors) and additional 9 centers retrieving data • Completion of internal development and testing of the CIBMTR Cellular Therapy forms • Implemented AGNIS tool that improves external testing practices by allowing users to reset test form submission back to due status • 2019 Plans • Work with external partner centers to test the CIBMTR Cellular Therapy forms and then release them to active production use • Support the CIBMTR form revisions planned for 2019 including 6 TED and at least 2 CRF forms

  37. AGNIS on FHIR • A new messaging interface to AGNIS using healthcare informatics standards (HL7 FHIR) that embrace modern approaches to data exchange • Working to reduce data submission burden for centers by introducing greater interoperability between CIBMTR and transplant center EHR solutions & HLA Laboratory systems • 2019 - Expanding Proof of Concept

  38. DATA TRANSFER Automatic CRID assignment The Challenge: How do we reduce the burden of data entry to the sites? GVHD smart form HLA Cytogentics

  39. TOGETHER, WE CAN MEET THE CHALLENGE

  40. VALUE TO THE COMMUNITY

  41. ONLINE RESOURCES CIBMTR.ORG @CIBMTR theCIBMTR

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