Levine John E, Antin Joseph H, Allen Carl E, Burroughs Lauri M, Cooke Kenneth R, Devine Steven, Heslop Helen, Nakamura Ryotaro, Talano Julie An, Yanik Gregory, DiFronzo Nancy
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Division of Hematologic Malignancies, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts.
Biol Blood Marrow Transplant. 2020 May;26(5):e94-e100. doi: 10.1016/j.bbmt.2020.01.024. Epub 2020 Feb 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 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.
非恶性血液疾病，如骨髓衰竭症、免疫调节紊乱症和血红蛋白病，常常导致寿命缩短和生活质量低下。这些疾病中的许多都可以通过异基因造血细胞移植治愈，但由于认为疗效不足和/或毒性过大，患者往往无法接受该治疗程序。2018年，血液和骨髓移植临床试验网络召集了一个特别工作组，以确定最迫切需要且可行的临床试验，这些试验有可能改善非恶性疾病患者的治疗结果。本报告总结了特别工作组的讨论情况，并明确了非恶性血液疾病临床试验开发的网络计划。
