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

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

doi:10.1002/pbc.30466

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

Pediatr Blood Cancer. 2023 Jun 7:e30466. doi: 10.1002/pbc.30466. Online ahead of print.

Authors

Aaron R Weiss¹, Sarah Dry², Clara Maygar², Anya Cutler³, Christine W Lary⁴, Carmen Khoo³, Jillian E Fergione¹, Melanie M Hounchell¹, Kathleen Glick¹, Meghen Browning⁵, Sun Ha Choo⁶, Douglas S Hawkins⁷, Joanne Lagmay⁸, Michelle Manalang⁹, Stephen X Skapek¹⁰, Brenda Weigel¹¹, Stephanie Verwys¹, Noah Federman¹²

Affiliations

¹ Division of Hematology/Oncology, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.
² Department of Pathology Bone, Soft Tissue and GI Pathology, University of California Los Angeles, Los Angeles, California, USA.
³ MaineHealth Institute for Research, Portland, Maine, USA.
⁴ Roux Institute and the Bouve College of Health Sciences at Northeastern University, Portland, Maine, USA.
⁵ Division of Hematology/Oncology, Department of Pediatrics, Children's Hospital of Wisconsin, Milwaukee, Wisconsin, USA.
⁶ Division of Hematology/Oncology, Department of Pediatrics, Rady Children's Hospital, San Diego, California, USA.
⁷ Division of Hematology/Oncology, Department of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, Washington, USA.
⁸ Division of Hematology/Oncology, Department of Pediatrics, University of Florida Health Science Center, Gainesville, Florida, USA.
⁹ Department of Pediatrics, Marshfield Marshfield Medical Center, Marshfield, Wisconsin, USA.
¹⁰ Division of Hematology/Oncology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
¹¹ Division of Hematology/Oncology, Department of Pediatrics, University of Minnesota/Masonic Cancer Center, Minneapolis, Minnesota, USA.
¹² Departments of Pediatrics and Orthopaedic Surgery, David Geffen School of Medicine, University of California, Los Angeles, California, USA.

PMID: 37283290
DOI: 10.1002/pbc.30466

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.

Keywords: children; desmoid tumor; sirolimus; young adults.

Abstract

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