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Editorial
. 2020 May;26(5):e94-e100.
doi: 10.1016/j.bbmt.2020.01.024. Epub 2020 Feb 5.

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

John E Levine  1 , Joseph H Antin  2 , Carl E Allen  3 , Lauri M Burroughs  4 , Kenneth R Cooke  5 , Steven Devine  6 , Helen Heslop  7 , Ryotaro Nakamura  8 , Julie An Talano  9 , Gregory Yanik  10 , Nancy DiFronzo  11
Affiliations
Editorial

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

John E Levine et al. Biol Blood Marrow Transplant. 2020 May.

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.

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Figures

Figure 1:
Figure 1:
Overall survival, event free survival and GVHD-free event free survival in 23 patients with bone marrow failure disorders who underwent HCT using treosulfan-based conditioning
See this image and copyright information in PMC

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