Hematopoietic cell transplantation (HCT) clinical trials face unique challenges, including:
- The relatively small number of procedures performed at any single center
- Diverse indications for HCT, and now other cell therapies, requiring dissimilar approaches
- The complex nature of the interventions
- The risk of multiple competing complications in the immediate post-transplantation period
- The risk of important, though infrequent, late effects
The ability to perform multicenter HCT trials was long hampered by:
- Limited funding
- Low priority afforded to HCT trials by networks focused on non-HCT cancer therapy and by pharmaceutical companies
- Lack of an effective multicenter HCT trials infrastructure
To address this need, in 2001, the National Heart, Lung, and Blood Institute (NHLBI), and National Cancer Institute (NCI) issued a Request for Applications (HLA-01-004), inviting participation in the BMT CTN. This resulted in cooperative agreements for a Data and Coordinating Center and multiple Clinical Centers that has evolved into an effective infrastructure for planning, implementing, and completing HCT and, more recently, non-HCT cellular therapy trials, providing definitive answers to questions that have led to changes in practice. In 2021, the BMT CTN celebrated its 20th anniversary. Over those 20 years, it enrolled >13,000 patients on >55 trials and published >135 manuscripts. For a full description of the Network and its accomplishments see here.