February, Keystone, CO
Dr. Mary Horowitz
Dr. Paul Martin
While laboratory advances have increased fundamental knowledge of transplantation and tumor biology and suggested strategies for improving transplant results, implementing and completing clinical trials that definitively test the efficacy of new transplant approaches has proven difficult. Regulatory and funding agencies, pharmaceutical companies and transplant physicians often have divergent perspectives and objectives that hinder achieving consensus on optimal approaches.
- Review the current state of clinical trials in hematopoietic stem cell transplantation including major obstacles to implementing and completing trials in a timely manner.
- Discuss clinical trial designs most likely to be successful in this area.
- Discuss streamlined systems for reporting adverse events that ensure patient safety but do not impose excessive burden on clinical investigators.
- Discuss strategies for achieving consensus on diagnosis and grading transplant-specific outcomes particularly engraftment and graft-versus-host disease.
- Summarize in a written document recommendation for clinical trial design, adverse event reporting and analysis of transplant-specific outcomes. This document will be submitted for publication.
Invited participants (limit of 20) and observers will include representatives from the academic transplant community, the Food and Drug Administration, the National Institutes of Health, the American Society for Blood and Marrow Transplantation, the Foundation for the Accreditation of Hematopoietic Stem Cell Transplantation, the International Society of Hematotherapy and Graft Engineering, the National Marrow Donor Program, the International Bone Marrow Transplant Registry and the Autologous Blood and Marrow Transplant Registry. Participants will be assigned to one of three subcommittees charged with developing recommendations and leading a discussion I one of the three main Forum areas: Clinical Trial Design, Adverse Event Reporting and Transplant-Specific Outcomes. Observers will be invited to comment on the recommendations.