基于treosulfan的预处理方案的数据逐渐增加，而且该方法的优点是持续的，特别是在急性毒性方面。本研究旨在描述treosulfan预处理方案在儿童中的结果，重点关注在治疗恶性和非恶性疾病时的毒性和疗效。本研究是对在西班牙接受treosulfan预处理方案进行造血干细胞移植（HSCT）的儿科患者进行的回顾性观察性研究，数据来自电子临床记录的收集。报道了158名儿童和青少年中的160例treosulfan预处理HSCT，用于治疗非恶性疾病（n=117）或恶性疾病（n=43）。 HSCT时的中位年龄为5.1岁（IQR: 2-10）。非恶性队列中最常见的诊断是原发性免疫缺陷（n=42; 36%）和镰状细胞性贫血（n=42; 36%），恶性队列中最常见的是急性淋巴细胞性白血病（n=15; 35%）。患者中有97%实现了移植成功。在非恶性队列中，中位中性粒细胞移植时间和血小板移植时间较长（分别为17天和20天；p=0.008和p=0.002）。VOD的1年累积发生率为7.98（95％CI [4.6-13.6]），各队列之间无显著差异。恶性队列中III-IV级急性移植物抗宿主病（aGvHD）的1年累积发生率较高（18% vs. 3.2%; p=0.011）。总的来说，无论是总发生率（9 vs. 3%; p<0.001）还是总cGvHD的2年累积发生率（16 vs. 1.9%; p<0.001），恶性队列中的慢性移植物抗宿主病（cGvHD）更常见。两个队列的2年累积TRM为15%，无差异。5年总生存率为80%（95％CI [72-86]），非恶性队列较高（87% vs. 61%; p=0.01）。恶性队列中的2年累积复发率为25%。5年累积GRFS率为60%（95％CI [51-70]），非恶性队列较高（72% vs. 22%; p<0.001）。基于treosulfan的无辐射预处理方案是可行的，实现了高的移植成功率和5年总生存率。treosulfan预处理方案是治疗非恶性疾病首次HSCT的新兴选择。版权所有 © 2023. Elsevier Inc. 发表。

Data on treosulfan-based conditioning regimens are little by little increasing, and the benefits of this approach are consistent, particularly regarding acute toxicity. This study aims to describe the results of treosulfan-based conditioning regimens in children, focusing on toxicity and outcomes when used to treat both malignant and non-malignant diseases.Retrospective observational study on pediatric patients treated in Spain with treosulfan-based conditioning regimens before hematopoietic stem cell transplantation (HSCT), based on data collection from electronic clinical records.One hundred and sixty treosulfan-based conditioning HSCTs to treat non-malignant diseases (n=117) or malignant diseases (n=43) in 158 children and adolescents are reported. Median age at HSCT was 5.1 years (IQR: 2-10). The most frequent diagnoses were primary immunodeficiency (n=42; 36%) and sickle cell disease (n=42; 36%) in the non-malignant cohort and acute lymphoblastic leukemia (n=15; 35%) in the malignant cohort. Engraftment occurred in 97% of the patients. There was a longer median time to neutrophil engraftment (17 vs. 14 days; p=0.008) and platelet engraftment (20 vs. 15 days; p=0.002) in the non-malignant cohort. The 1-year cumulative incidence of VOD was 7.98 (95%CI [4.6-13.6]), with no significant differences between cohorts. The 1-year cumulative incidence of grade III-IV aGvHD was higher in the malignant cohort (18% vs. 3.2%; p=0.011). Overall, cGvHD was more frequently observed in the malignant cohort for both total incidence (9 vs. 3%; p<0.001) and 2-year cumulative incidence of total cGvHD (16 vs. 1.9%; p<0.001). The 2-year cumulative TRM was 15%, with no differences between cohorts. 5-year Overall Survival was 80% (95%CI [72-86]) and was higher in the non-malignant cohort (87 vs. 61%; p=0.01). The 2-year cumulative incidence of relapse in the malignant cohort was 25%. 5-year cumulative GRFS rate was 60% (95%CI [51-70]) and was higher in the non-malignant cohort (72 vs. 22%; p<0.001).The treosulfan-based radiation-free conditioning regimen is feasible, achieving a high engraftment rate and 5-year overall survival. A treosulfan-based conditioning regimen is an emerging option for the first HSCT in non-malignant diseases.Copyright © 2023. Published by Elsevier Inc.