对患有非恶性疾病的儿童进行同种异体造血干细胞移植前的最佳调理是正在进行的研究的主题。这项前瞻性、随机、2 期试验比较了白消安与基于三硫丹的准备方案的安全性和有效性。患有非恶性疾病的儿童接受氟达拉滨和静脉注射 (IV) 白消安（4.8 至 3.2mg/kg/天）或静脉注射三硫丹（10、12 或 14g/m2/天）。噻替哌给药（2×5mg/kg）由研究者决定。主要终点是免于移植（治疗）相关死亡率（免于 TRM），定义为第 -7 天至第 100 天之间的死亡。总体而言，分析了至少 12 个月随访的 101 名患者（白消安 50 例、三硫丹 51 例）。使用白消安后，TRM 的清除率为 90.0%（95% CI：78.2%、96.7%），而使用三硫丹后，TRM 清除率为 100.0%（95% CI：93.0%、100.0%）。次要结局（移植相关死亡率[12.0% vs 3.9%]）和总生存率（88.0% vs 96.1%）有利于三硫丹。与白消安治疗后(n = 2)相比，三硫丹治疗后移植失败更常见(n = 11)，而除一名白消安患者外，所有患者均通过第二次手术获救。两组的 CTCAE III 级不良事件相似。这项研究证实三硫丹是白消安的绝佳替代品，可以安全地用于患有非恶性疾病的儿童的调理治疗。© 2023。作者。

Optimal conditioning prior to allogeneic hematopoietic stem cell transplantation for children with non-malignant diseases is subject of ongoing research. This prospective, randomized, phase 2 trial compared safety and efficacy of busulfan with treosulfan based preparative regimens. Children with non-malignant diseases received fludarabine and either intravenous (IV) busulfan (4.8 to 3.2 mg/kg/day) or IV treosulfan (10, 12, or 14 g/m2/day). Thiotepa administration (2 × 5 mg/kg) was at the investigator's discretion. Primary endpoint was freedom from transplantation (treatment)-related mortality (freedom from TRM), defined as death between Days -7 and +100. Overall, 101 patients (busulfan 50, treosulfan 51) with at least 12 months follow-up were analyzed. Freedom from TRM was 90.0% (95% CI: 78.2%, 96.7%) after busulfan and 100.0% (95% CI: 93.0%, 100.0%) after treosulfan. Secondary outcomes (transplantation-related mortality [12.0% versus 3.9%]) and overall survival (88.0% versus 96.1%) favored treosulfan. Graft failure was more common after treosulfan (n = 11), than after busulfan (n = 2) while all patients were rescued by second procedures except one busulfan patient. CTCAE Grade III adverse events were similar in both groups. This study confirmed treosulfan to be an excellent alternative to busulfan and can be safely used for conditioning treatment in children with non-malignant disease.© 2023. The Author(s).