Blood and Marrow Transplant Clinical Research Network

BACKGROUND:

Several new developments in the field of allogeneic hematopoietic stem cell transplantation have substantially decreased transplant-related morbidity and promise to make major improvements in both the safety and efficacy of transplantation, allowing transplantation to be extended to more patients. Use of matched unrelated marrow donors, unrelated cord blood units, and partially matched family donors have greatly expanded the options for patients who lack an HLA identical sibling donor. Use of stem cells mobilized and collected from peripheral blood rather than harvested from bone marrow speeds engraftment and may improve treatment outcome. Manipulation of the transplant to adjust the dose of stem cells and lymphocytes to optimum numbers (graft engineering) may also be beneficial. The use of non-myeloablative transplants (mini-transplants) promises to extend the safe application of stem cell allografts to individuals over 60 years and to patients with non-lethal hematologic diseases. Recently identified human minor histocompatibility antigens may provide another variable for predicting the severity of graft-versus-host disease.

There is an urgent need to evaluate these promising new therapeutic approaches to hematopoietic stem cell transplantation and to disseminate the findings to health care professionals, patients and the public. Each year, thousands of patients undergo hematopoietic stem cell transplants in the United States, yet few of these patients are offered the option to enroll in a research protocol to study and improve the outcome of this life-saving but toxic and expensive procedure. There are several reasons why a blood and marrow transplant clinical research network would accelerate clinical research and evaluate new approaches to transplantation. The heterogeneity of hematopoietic stem cell transplant patients makes it difficult to accumulate a large number of comparable patients in one center. Multi-center trials will reduce the number of patients needed at each clinical center and allow accrual to be completed more rapidly. Further, a common treatment protocol will reduce variables that contribute to patient outcome and allow valid comparisons between treatments. Finally, the Network approach will increase the number of comparative trials that are conducted by providing a framework for rapid initiation of important studies, a focus on randomized studies, and efficient use of pooled clinical expertise and data management resources.

The Request for Applications was released in January, 2001. Awards were made in September, 2001.

DESIGN NARRATIVE: