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Abstract：
BACKGROUND: Type of conditioning regimen impacts the outcome of patients who undergo allogeneic HSCT since graft versus host disease (GVHD), infections, regimen related toxicities (RRT) are important causes of post-transplant mortality. Despite the RRT profile of busulfan, it is frequently used worldwide. Treosulfan has advantages in terms of dose of administration, lower incidence of sinusoidal obstruction syndrome and lower neurotoxicity. We retrospectively investigated outcomes of patients who underwent allogeneic HSCT with treosulfan or busulfan based conditioning regimens in our institution.
METHODS: Treosulfan was administered to 94 patients while 85 patients received busulfan. Our outcomes were RRT, chronic and acute GVHD, relapse related mortality (RRM), non-relapse mortality, and fungal infection. The clinical follow up data, regarding the primary and secondary endpoints of our study, of the patients who received treosulfan or busulfan based conditioning regimens were statistically analyzed.
RESULTS: The median follow-up was 14 months for the treosulfan group while it was 11 months for the busulfan group (p = 0.16). RRT was 11.7% and 7.1% for treosulfan and busulfan respectively. The incidence of extensive chronic GVHD was less frequent in the treosulfan group compared to the busulfan group (15.7% vs. 32.1%) (p < 0.001). The incidence of acute GVHD (Grade 3 or higher) was 32.2% in the treosulfan group while it was 31.6% in the busulfan group. The RRM was 17% in the treosulfan group while it was 34% in the busulfan group. The non-relapse mortality was 35.5% and 29.4% in the treosulfan group and in the busulfan group respectively (p = 0.962).
CONCLUSIONS: Treosulfan, with a lower RRM, lower chronic GVHD incidence and with a similar RRT profile appears to be a safe alternative to busulfan.
METHODS: Treosulfan was administered to 94 patients while 85 patients received busulfan. Our outcomes were RRT, chronic and acute GVHD, relapse related mortality (RRM), non-relapse mortality, and fungal infection. The clinical follow up data, regarding the primary and secondary endpoints of our study, of the patients who received treosulfan or busulfan based conditioning regimens were statistically analyzed.
RESULTS: The median follow-up was 14 months for the treosulfan group while it was 11 months for the busulfan group (p = 0.16). RRT was 11.7% and 7.1% for treosulfan and busulfan respectively. The incidence of extensive chronic GVHD was less frequent in the treosulfan group compared to the busulfan group (15.7% vs. 32.1%) (p < 0.001). The incidence of acute GVHD (Grade 3 or higher) was 32.2% in the treosulfan group while it was 31.6% in the busulfan group. The RRM was 17% in the treosulfan group while it was 34% in the busulfan group. The non-relapse mortality was 35.5% and 29.4% in the treosulfan group and in the busulfan group respectively (p = 0.962).
CONCLUSIONS: Treosulfan, with a lower RRM, lower chronic GVHD incidence and with a similar RRT profile appears to be a safe alternative to busulfan.
摘要：
背景：类型的预处理方案会影响移植物抗宿主病（GVHD）后接受同种异体HSCT的患者的预后，感染,治疗方案相关毒性(RRT)是移植后死亡的重要原因.尽管白消安的RRT特征，它在世界范围内经常使用。曲硫丹在给药剂量方面具有优势，正弦阻塞综合征的发生率较低，神经毒性较低。我们回顾性调查了在我们机构中接受基于曲硫丹或白消安的预处理方案的同种异体HSCT患者的结局。
方法：对94例患者服用了曲硫丹，85例患者服用了白消安。我们的结果是RRT,慢性和急性GVHD，复发相关死亡率（RRM），非复发死亡率，和真菌感染。临床随访数据,关于我们研究的主要和次要终点，对接受以曲硫丹或白消安为基础的调理方案的患者进行统计学分析。
结果：曲硫安组的中位随访时间为14个月，白消安组的中位随访时间为11个月（p=0.16）。曲硫丹和白消安的RRT分别为11.7%和7.1%。与白消安组相比，曲硫安组广泛慢性GVHD的发生率较低(15.7%vs.32.1%)(p<0.001)。急性GVHD（3级或更高）的发生率在曲硫丹组为32.2％，而在白消安组为31.6％。曲硫丹组的RRM为17％，而白消安组的RRM为34％。曲硫安组和白消安组的非复发死亡率分别为35.5%和29.4%(p=0.962)。
结论：曲硫丹，较低的RRM，较低的慢性GVHD发病率和相似的RRT表现似乎是白消安的安全替代品.
方法：对94例患者服用了曲硫丹，85例患者服用了白消安。我们的结果是RRT,慢性和急性GVHD，复发相关死亡率（RRM），非复发死亡率，和真菌感染。临床随访数据,关于我们研究的主要和次要终点，对接受以曲硫丹或白消安为基础的调理方案的患者进行统计学分析。
结果：曲硫安组的中位随访时间为14个月，白消安组的中位随访时间为11个月（p=0.16）。曲硫丹和白消安的RRT分别为11.7%和7.1%。与白消安组相比，曲硫安组广泛慢性GVHD的发生率较低(15.7%vs.32.1%)(p<0.001)。急性GVHD（3级或更高）的发生率在曲硫丹组为32.2％，而在白消安组为31.6％。曲硫丹组的RRM为17％，而白消安组的RRM为34％。曲硫安组和白消安组的非复发死亡率分别为35.5%和29.4%(p=0.962)。
结论：曲硫丹，较低的RRM，较低的慢性GVHD发病率和相似的RRT表现似乎是白消安的安全替代品.
