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

Abstract Non-malignant blood diseases such as bone marrow failure disorders, immune dysregulation disorders, and hemoglobinopathies often lead to shortened lifespans 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 non-malignant diseases. This report summarizes the task force discussions and specifies the network plans for clinical trial development for non-malignant blood diseases.