@article{0bcf017691344f9d9484b5b5866e4a80,
title = "Priorities for Improving Outcomes for Nonmalignant Blood Diseases: A Report from the Blood and Marrow Transplant Clinical Trials Network",
abstract = "Nonmalignant blood diseases such as bone marrow failure disorders, immune dysregulation disorders, and hemoglobinopathies often lead to shortened life spans and poor quality of life. Many of these diseases can be cured with allogeneic hematopoietic cell transplantation, but patients are often not offered the procedure because of perceived insufficient efficacy and/or excess toxicity. In 2018, the Blood and Marrow Transplant Clinical Trials Network convened a task force to identify the most urgently needed yet feasible clinical trials with potential to improve the outcomes for patients with nonmalignant diseases. This report summarizes the task force discussions and specifies the network plans for clinical trial development for nonmalignant blood diseases.",
keywords = "Bone marrow failure, Nonmalignant, Thalassemia, Transplantation",
author = "Levine, {John E.} and Antin, {Joseph H.} and Allen, {Carl E.} and Burroughs, {Lauri M.} and Cooke, {Kenneth R.} and Steven Devine and Helen Heslop and Ryotaro Nakamura and Talano, {Julie An} and Gregory Yanik and Nancy DiFronzo",
note = "Funding Information: Financial disclosure: Supported by grant U24HL138660 to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute and the National Cancer Institute. The Be The Match Foundation also provided support. The content is solely the responsibility of the authors and does not necessarily represent the official views of the abovementioned parties. Funding Information: Financial disclosure: Supported by grant U24HL138660 to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute and the National Cancer Institute. The Be The Match Foundation also provided support. The content is solely the responsibility of the authors and does not necessarily represent the official views of the abovementioned parties. Conflict of interest statement: L.B. receives research support and travel support from Medac. K.R.C. serves on the Speaker Bureau and Advisory Board for Jazz Pharmaceuticals and receives research support from Jazz Pharmaceuticals and Bristol Meyers Squibb. H.H. is a founder and has an equity stake in Allovir Marker Therapeutics; consults for Novartis, Gilead Bioscience, Kiadis, PACT Pharma, and Tessa Therapeutics; and receives research support from Cell Medica and Tessa Therapeutics. J.E.L. receives royalties from Viracor for a GVHD biomarker patent; consults for Novartis, Bluebird Bio, Ironwood, and Incyte; and receives research support from Kamada, Incyte, and Biogen. Financial disclosure: See Acknowledgments on page e99. Publisher Copyright: {\textcopyright} 2020 American Society for Transplantation and Cellular Therapy",
year = "2020",
month = may,
doi = "10.1016/j.bbmt.2020.01.024",
language = "English",
volume = "26",
pages = "e94--e100",
journal = "Transplantation and Cellular Therapy",
issn = "2666-6375",
publisher = "Elsevier BV",
number = "5",
}