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

TitleThe Blood and Marrow Transplant Clinical Trials Network: An Effective Infrastructure for Addressing Important Issues in Hematopoietic Cell Transplantation.
Publication TypeJournal Article
Year of Publication2016
JournalBiol Blood Marrow Transplant
Volume22
Issue10
Pagination1747-1757
Date Published2016 10
ISSN1523-6536
KeywordsBone Marrow Transplantation, Clinical Trials as Topic, Hematopoietic Stem Cell Transplantation, Humans
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.

DOI10.1016/j.bbmt.2016.07.003
Alternate JournalBiol Blood Marrow Transplant
PubMed ID27418009
PubMed Central IDPMC5027144
Grant ListU10 HL069294 / HL / NHLBI NIH HHS / United States
U10 HL109526 / HL / NHLBI NIH HHS / United States
U10 HL069254 / HL / NHLBI NIH HHS / United States
U10 HL069290 / HL / NHLBI NIH HHS / United States
U10 HL069274 / HL / NHLBI NIH HHS / United States
U10 HL069278 / HL / NHLBI NIH HHS / United States
U10 HL069233 / HL / NHLBI NIH HHS / United States
U24 CA076518 / CA / NCI NIH HHS / United States
U10 HL069286 / HL / NHLBI NIH HHS / United States
U10 HL069301 / HL / NHLBI NIH HHS / United States
U10 HL069330 / HL / NHLBI NIH HHS / United States
U10 HL069291 / HL / NHLBI NIH HHS / United States
UG1 HL069278 / HL / NHLBI NIH HHS / United States
U10 HL069249 / HL / NHLBI NIH HHS / United States
U10 HL069334 / HL / NHLBI NIH HHS / United States
U10 HL069310 / HL / NHLBI NIH HHS / United States
U10 HL109137 / HL / NHLBI NIH HHS / United States
P30 CA008748 / CA / NCI NIH HHS / United States
U01 HL069294 / HL / NHLBI NIH HHS / United States
U10 HL109322 / HL / NHLBI NIH HHS / United States
U10 HL108945 / HL / NHLBI NIH HHS / United States
R01 HL069256 / HL / NHLBI NIH HHS / United States
U10 HL108987 / HL / NHLBI NIH HHS / United States
U01 HL069273 / HL / NHLBI NIH HHS / United States
U10 HL069315 / HL / NHLBI NIH HHS / United States
U10 HL069348 / HL / NHLBI NIH HHS / United States