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

Authors

Aaron R. Weiss, Division of Hematology/Oncology, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.
Sarah Dry, Department of Pathology Bone, Soft Tissue and GI Pathology, University of California Los Angeles, Los Angeles, California, USA.
Clara Maygar, Department of Pathology Bone, Soft Tissue and GI Pathology, University of California Los Angeles, Los Angeles, California, USA.
Anya Cutler, MaineHealth Institute for Research, Portland, Maine, USA.
Christine W. Lary, Roux Institute and the Bouve College of Health Sciences at Northeastern University, Portland, Maine, USA.
Carmen Khoo, MaineHealth Institute for Research, Portland, Maine, USA.
Jillian E. Fergione, Division of Hematology/Oncology, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.
Melanie M. Hounchell, Division of Hematology/Oncology, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.

Document Type

Article

Publication Date

6-7-2023

Institution/Department

Pediatrics

Journal Title

Pediatric blood & cancer

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.

First Page

e30466

Recommended Citation

Weiss AR, Dry S, Maygar C, et al. A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis [published online ahead of print, 2023 Jun 7]. Pediatr Blood Cancer. 2023;e30466. doi:10.1002/pbc.30466