Abstract：
Deregulation of the mTOR pathway may play an important role in tumor biology when the APC/β-catenin pathway is disrupted in desmoid-type fibromatosis (DT). A pilot study was conducted to determine whether sirolimus can block the mTOR pathway (primary aim) as well as determine whether it can safely be given in the preoperative setting, decrease tumor size/recurrence, and decrease tumor-associated pain in children and young adults (secondary aims) with DT. Nine subjects ages 5-28 years were enrolled from 2014 to 2017 across four centers. Sirolimus was feasible and was associated with a nonstatistically significant decrease in pS706K activation.
摘要：
当在纤维瘤样型纤维瘤病（DT）中APC/β-连环蛋白途径被破坏时，mTOR途径的失调可能在肿瘤生物学中起重要作用。进行了一项初步研究，以确定西罗莫司是否可以阻断mTOR通路（主要目标），以及确定是否可以在术前设置安全给予。减小肿瘤大小/复发，并减少儿童和年轻人的肿瘤相关疼痛（次要目标）与DT。从2014年到2017年，在四个中心招募了9名年龄在5-28岁之间的受试者。西罗莫司是可行的，并且与pS706K活化的无统计学意义的降低相关。
