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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.

SUBMITTER: Levine JE 

PROVIDER: S-EPMC8062984 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature

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

Levine John E JE   Antin Joseph H JH   Allen Carl E CE   Burroughs Lauri M LM   Cooke Kenneth R KR   Devine Steven S   Heslop Helen H   Nakamura Ryotaro R   Talano Julie An JA   Yanik Gregory G   DiFronzo Nancy N  

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 20200205 5

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 urgen  ...[more]

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