The Blood and Marrow Transplant Clinical Trials Network: An Effective Infrastructure for Addressing Important Issues in Hematopoietic Cell Transplantation

Abstract

Hematopoietic cell transplantation (HCT) is a rapidly evolving field with active preclinical and clinical development of new strategies for patient assessment, graft selection and manipulation, and pre- and post-transplantation drug and cell therapy. New strategies require evaluation in definitive clinical trials; however, HCT trials face unique challenges, including the relatively small number of transplantations performed at any single center, the diverse indications for HCT requiring dissimilar approaches, the complex nature of the intervention itself, the risk of multiple complications in the immediate post-transplantation period, and the risk of important, though infrequent, late effects. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) was established by the US National Heart Lung and Blood Institute and the National Cancer Institute to meet these challenges. In its 15 years as a network, the BMT CTN has proven to be a successful infrastructure for planning, implementing, and completing such trials and for providing definitive answers to questions leading to improvements in the understanding and practice of HCT. It has opened 37 trials, about one-half phase 2 and one-half phase 3, enrolled more than 8000 patients, and published 57 papers addressing important issues in the treatment of patients with life-threatening malignant and nonmalignant blood disorders. This review describes the network's accomplishments, key components of its success, lessons learned over the past 15 years, and challenges for the future.