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

doi:10.1016/j.bbmt.2020.01.024

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 journal › Editorial

5 Scopus citations

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 language	English
Pages (from-to)	e94-e100
Journal	Biology of Blood and Marrow Transplantation
Volume	26
Issue number	5
DOIs	https://doi.org/10.1016/j.bbmt.2020.01.024
State	Published - May 2020

Keywords

Bone marrow failure
Nonmalignant
Thalassemia
Transplantation

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10.1016/j.bbmt.2020.01.024

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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

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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 = "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",

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Levine, JE, Antin, JH, Allen, CE, Burroughs, LM, Cooke, KR, Devine, S, Heslop, H, Nakamura, R, Talano, JA, 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, vol. 26, no. 5, pp. e94-e100. https://doi.org/10.1016/j.bbmt.2020.01.024

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T1 - Priorities for Improving Outcomes for Nonmalignant Blood Diseases

T2 - A Report from the Blood and Marrow Transplant Clinical Trials Network

AU - Levine, John E.

AU - Antin, Joseph H.

AU - Allen, Carl E.

AU - Burroughs, Lauri M.

AU - Cooke, Kenneth R.

AU - Devine, Steven

AU - Heslop, Helen

AU - Nakamura, Ryotaro

AU - Talano, Julie An

AU - Yanik, Gregory

AU - DiFronzo, Nancy

PY - 2020/5

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N2 - 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.

AB - 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.

KW - Bone marrow failure

KW - Nonmalignant

KW - Thalassemia

KW - Transplantation

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AN - SCOPUS:85081220914

SN - 1083-8791

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ER -

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

Abstract

Keywords

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