Almost 18,000 HCTs are performed in the United States (US) annually, and the number increases by approximately 5% per year. This increase reflects the utility of HCT in treating both malignant and non-malignant diseases, higher donor availability, and treatment advances that allow HCT to be performed in older and sicker patients. However, this number reflects less than half of number of the patients who could benefit from transplantation if the safety and efficacy of the procedure and the ability to successfully perform transplants across histocompatibility barriers were improved.  Such improvements require development and testing of new transplant strategies.

Yet, HCT clinical trials face unique challenges, including the relatively small number of transplantations performed at any single center, the diverse indications for HCT, the complexities of the procedure, and multiple post-transplant complications. 

The BMT CTN was established to address these challenges and execute multicenter HCT trials with broad national participation. The Network:

• Effectively fosters development of innovative and important concepts into well-designed trials that answer questions in the most efficient manner;
• Supports timely implementation and completion of those trials;
• Ensures protection of subjects (both donors and recipients);
• Provides high-quality data and adherence to regulatory requirements in an increasingly complex environment.

The BMT CTN has answered relevant research questions in both common and rare diseases and critical issues such as optimal graft sources, conditioning intensity, regimen-related toxicity, engraftment, GVHD, infection, disease control, and quality of life.

The BMT CTN has held two State of the Science Symposia, the most recent in 2007.  That meeting resulted in very specific recommendations for high priority clinical trials to be implemented by the US transplant community using the resources of the BMT CTN, NCI Cooperative Groups, and other programs.  Most of these trials have been launched and some have already been successfully completed.

In 2014, BMT CTN will host a third State of the Science Symposium at which 13 scientific committees will survey the HCT landscape and identify areas with the greatest need for high-impact Phase II and III clinical trials. The results of this Symposium are expected to guide national multicenter trial efforts in BMT over the next 5-6 years.

Physicians who refer for transplant need to be aware of the current state of the art in transplant, key transplant research, best practices and future research direction that aid in their clinical decision-making.