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Priorities for Improving Outcomes for Nonmalignant Blood Diseases: A Report from the Blood and Marrow Transplant Clinical Trials Network. Biol Blood Marrow Transplant 2020 05;26(5):e94-e100



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Pubmed Central ID




Scopus ID

2-s2.0-85081220914   2 Citations


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.

Author List

Levine JE, Antin JH, Allen CE, Burroughs LM, Cooke KR, Devine S, Heslop H, Nakamura R, Talano JA, Yanik G, DiFronzo N


Julie-An M. Talano MD Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Bone Marrow
Hematologic Diseases
Hematopoietic Stem Cell Transplantation
Quality of Life
Transplantation Conditioning