Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adults. Therefore, we prospectively evaluated the safety and efficacy of treosulfan-based conditioning in children with hematological malignancies in this phase II trial. Overall, 65 children with acute lymphoblastic leukemia (35.4%), acute myeloid leukemia (44.6%), myelodysplastic syndrome (15.4%), or juvenile myelomonocytic leukemia (4.6%) received treosulfan intravenously at a dose of 10 mg/m²/day (7.7%), 12 g/m²/day (35.4%), or 14 g/m²/day (56.9%) according to their individual body surface area in combination with fludarabine and thiotepa. The incidence of complete donor chimerism at day +28 was 98.4% with no primary and only one secondary graft failure. At 36 months, NRM was only 3.1%, while relapse incidence was 21.7%, and overall survival was 83.0%. The cumulative incidence of acute graft-vs.-host disease was 45.3% for grades I–IV and 26.6% for grades II–IV. At 36 months, 25.8% overall and 19.4% moderate/severe chronic graft-vs.-host disease were reported. These data confirm the safe and effective use of treosulfan-based conditioning in pediatric patients with hematological malignancies. Therefore, treosulfan/fludarabine/thiotepa can be recommended for myeloablative conditioning in children with hematological malignancies.

异体移植前基于四硫烷的调理已显示出与成年后非复发死亡率（NRM）降低相关的清髓，免疫抑制和抗肿瘤作用。因此，我们在此II期临床试验中前瞻性地评估了以硫丹为基础的调理对血液系统恶性肿瘤儿童的安全性和有效性。总体上，有65例急性淋巴细胞白血病（35.4％），急性髓细胞性白血病（44.6％），骨髓增生异常综合征（15.4％）或青少年骨髓单核细胞白血病（4.6％）患儿接受了10 mg / m ² /天的静脉注射硫代硫丹（7.7％），12 g / m ² /天（35.4％）或14 g / m ²/天（56.9％），具体取决于他们与氟达拉滨和噻替帕合用的个体表面积。在第+28天完全供体嵌合的发生率为98.4％，无一次移植失败，仅有一次移植失败。在36个月时，NRM仅为3.1％，复发率则为21.7％，总生存率为83.0％。I–IV级急性移植物抗宿主病的累积发生率分别为45.3％和II–IV级26.6％。在36个月时，报告总体为25.8％，中度/重度慢性移植物抗宿主病。这些数据证实在患有血液恶性肿瘤的小儿患者中安全有效地使用基于硫磺的调理剂。因此，对于血液系统恶性肿瘤的儿童，推荐使用硫代硫丹/氟达拉滨/硫替太帕进行清髓治疗。