A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis.
Publication
, Journal Article
Weiss, AR; Dry, S; Maygar, C; Cutler, A; Lary, CW; Khoo, C; Fergione, JE; Hounchell, MM; Glick, K; Browning, M; Choo, SH; Hawkins, DS ...
Published in: Pediatr Blood Cancer
June 7, 2023
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.
Duke Scholars
Published In
Pediatr Blood Cancer
DOI
EISSN
1545-5017
Publication Date
June 7, 2023
Start / End Page
e30466
Location
United States
Related Subject Headings
- Oncology & Carcinogenesis
- 3213 Paediatrics
- 3211 Oncology and carcinogenesis
- 1114 Paediatrics and Reproductive Medicine
- 1112 Oncology and Carcinogenesis
- 1103 Clinical Sciences
Citation
APA
Chicago
ICMJE
MLA
NLM
Weiss, A. R., Dry, S., Maygar, C., Cutler, A., Lary, C. W., Khoo, C., … Federman, N. (2023). A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis. Pediatr Blood Cancer, e30466. https://doi.org/10.1002/pbc.30466
Weiss, Aaron R., Sarah Dry, Clara Maygar, Anya Cutler, Christine W. Lary, Carmen Khoo, Jillian E. Fergione, et al. “A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis.” Pediatr Blood Cancer, June 7, 2023, e30466. https://doi.org/10.1002/pbc.30466.
Weiss AR, Dry S, Maygar C, Cutler A, Lary CW, Khoo C, et al. A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis. Pediatr Blood Cancer. 2023 Jun 7;e30466.
Weiss, Aaron R., et al. “A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis.” Pediatr Blood Cancer, June 2023, p. e30466. Pubmed, doi: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. A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis. Pediatr Blood Cancer. 2023 Jun 7;e30466.
Published In
Pediatr Blood Cancer
DOI
EISSN
1545-5017
Publication Date
June 7, 2023
Start / End Page
e30466
Location
United States
Related Subject Headings
- Oncology & Carcinogenesis
- 3213 Paediatrics
- 3211 Oncology and carcinogenesis
- 1114 Paediatrics and Reproductive Medicine
- 1112 Oncology and Carcinogenesis
- 1103 Clinical Sciences