Priorities for Improving Outcomes for Nonmalignant Blood Diseases: A Report from the Blood and Marrow Transplant Clinical Trials Network

John E. Levine, Joseph H. Antin, Carl E. Allen, Lauri M. Burroughs, Kenneth R. Cooke, Steven Devine, Helen Heslop, Ryotaro Nakamura, Julie An Talano, Gregory Yanik, Nancy DiFronzo

Research output: Contribution to journalEditorial

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.

Original languageEnglish (US)
Pages (from-to)e94-e100
JournalBiology of Blood and Marrow Transplantation
Volume26
Issue number5
DOIs
StatePublished - May 2020

Keywords

  • Bone marrow failure
  • Nonmalignant
  • Thalassemia
  • Transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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  • Cite this

    Levine, J. E., Antin, J. H., Allen, C. E., Burroughs, L. M., Cooke, K. R., Devine, S., Heslop, H., Nakamura, R., Talano, J. A., Yanik, G., & DiFronzo, N. (2020). Priorities for Improving Outcomes for Nonmalignant Blood Diseases: A Report from the Blood and Marrow Transplant Clinical Trials Network. Biology of Blood and Marrow Transplantation, 26(5), e94-e100. https://doi.org/10.1016/j.bbmt.2020.01.024