一项评估西罗莫司在儿童和青年硬纤维瘤型纤维瘤病患者中应用的试点研究。

A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis.

作者信息

Weiss Aaron R, Dry Sarah, Maygar Clara, Cutler Anya, Lary Christine W, Khoo Carmen, Fergione Jillian E, Hounchell Melanie M, Glick Kathleen, Browning Meghen, Choo Sun Ha, Hawkins Douglas S, Lagmay Joanne, Manalang Michelle, Skapek Stephen X, Weigel Brenda, Verwys Stephanie, Federman Noah

机构信息

Division of Hematology/Oncology, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.

Department of Pathology Bone, Soft Tissue and GI Pathology, University of California Los Angeles, Los Angeles, California, USA.

出版信息

Pediatr Blood Cancer. 2023 Jun 7:e30466. doi: 10.1002/pbc.30466.

DOI:10.1002/pbc.30466

PMID:37283290

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

当韧带样型纤维瘤病（DT）中的APC/β-连环蛋白通路被破坏时，mTOR通路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能阻断mTOR通路（主要目的），以及确定在术前环境中给予西罗莫司是否安全，能否减小儿童和年轻成人DT患者的肿瘤大小/复发率，并减轻肿瘤相关疼痛（次要目的）。2014年至2017年期间，在四个中心招募了9名年龄在5至28岁的受试者。西罗莫司是可行的，并且与pS706K激活的非统计学显著降低相关。