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.

中文翻译：

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改善非恶性血液病结果的优先次序：血液和骨髓移植临床试验网络的报告。

非恶性血液病，例如骨髓衰竭疾病，免疫失调疾病和血红蛋白病，通常会导致寿命缩短和生活质量下降。这些疾病中的许多可以通过同种异体造血细胞移植治愈，但是由于认为疗效不足和/或毒性过大，通常不为患者提供该程序。在2018年，血液和骨髓移植临床试验网络召集了一个工作队，以鉴定最迫切需要但可行的临床试验，以改善非恶性疾病患者的预后。本报告总结了工作组的讨论，并指定了用于非恶性血液病临床试验开发的网络计划。