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

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

Pediatric Hematology/Oncology

Research output: Contribution to journal › Article › peer-review

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.

Original language	English (US)
Article number	e30466
Journal	Pediatric Blood and Cancer
Volume	70
Issue number	9
DOIs	https://doi.org/10.1002/pbc.30466
State	Published - Sep 2023

Bibliographical note

Funding Information:
Aaron R. Weiss is chair of the Desmoid Tumor Research Foundation Medical Advisory Board, is on the medical advisory board for SpringWorks Therapeutics (no honoraria), and is a paid consultant for BioAtla. Noah Federman has received speaking and advisory honoraria from Bayer AG. Noah Federman receives grant support from NCI/NCATS CTSA and CIRM.

Funding Information:
Funding for this study was provided by a grant from the Desmoid Tumor Research Foundation. Partial drug supply was provided through a grant from Pfizer. We gratefully acknowledge all the patients and their families, care providers, and research personnel who participated in this study, and a special thank you to Clara Maygar for all her tremendous efforts in assisting with the biological aims of the study. Noah Federman receives support from NCI/NCATS CTSA (Grant/Award Number: UL1TR001881) and CIRM (Grant/Award Number: INFRA4‐13685).

Publisher Copyright:
© 2023 Wiley Periodicals LLC.

Keywords

children
desmoid tumor
sirolimus
young adults

PubMed: MeSH publication types

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10.1002/pbc.30466

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Weiss, A. R., Dry, S., Maygar, C., Cutler, A., Lary, C. W., Khoo, C., Fergione, J. E., Hounchell, M. M., Glick, K., Browning, M., Choo, S. H., Hawkins, D. S., Lagmay, J., Manalang, M., Skapek, S. X., Weigel, B., Verwys, S., & Federman, N. (2023). A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis. Pediatric Blood and Cancer, 70(9), Article e30466. https://doi.org/10.1002/pbc.30466

Weiss, AR, Dry, S, Maygar, C, Cutler, A, Lary, CW, Khoo, C, Fergione, JE, Hounchell, MM, Glick, K, Browning, M, Choo, SH, Hawkins, DS, Lagmay, J, Manalang, M, Skapek, SX, Weigel, B, Verwys, S & Federman, N 2023, 'A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis', Pediatric Blood and Cancer, vol. 70, no. 9, e30466. https://doi.org/10.1002/pbc.30466

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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.",

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note = "Funding Information: Aaron R. Weiss is chair of the Desmoid Tumor Research Foundation Medical Advisory Board, is on the medical advisory board for SpringWorks Therapeutics (no honoraria), and is a paid consultant for BioAtla. Noah Federman has received speaking and advisory honoraria from Bayer AG. Noah Federman receives grant support from NCI/NCATS CTSA and CIRM. Funding Information: Funding for this study was provided by a grant from the Desmoid Tumor Research Foundation. Partial drug supply was provided through a grant from Pfizer. We gratefully acknowledge all the patients and their families, care providers, and research personnel who participated in this study, and a special thank you to Clara Maygar for all her tremendous efforts in assisting with the biological aims of the study. Noah Federman receives support from NCI/NCATS CTSA (Grant/Award Number: UL1TR001881) and CIRM (Grant/Award Number: INFRA4‐13685). Publisher Copyright: {\textcopyright} 2023 Wiley Periodicals LLC.",

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AU - Khoo, Carmen

AU - Fergione, Jillian E.

AU - Hounchell, Melanie M.

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AU - Browning, Meghen

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AU - Hawkins, Douglas S.

AU - Lagmay, Joanne

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

Abstract

Bibliographical note

Keywords

PubMed: MeSH publication types

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