A pilot study evaluating the use of sirolimus in children and young adults with desmoid-type fibromatosis.
children
desmoid tumor
sirolimus
young adults
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
07 Jun 2023
07 Jun 2023
Historique:
revised:
15
04
2023
received:
07
03
2023
accepted:
08
05
2023
pubmed:
7
6
2023
medline:
7
6
2023
entrez:
7
6
2023
Statut:
aheadofprint
Résumé
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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e30466Subventions
Organisme : Desmoid Tumor Research Foundation
Organisme : Pfizer
Informations de copyright
© 2023 Wiley Periodicals LLC.
Références
The management of desmoid tumours: a joint global consensus-based guideline approach for adult and paediatric patients. Eur J Cancer. 2020;127:96-107.
Douglass DP, Navid F, Weiss AR. The role of pharmacotherapeutic agents in children with desmoid tumors. Paediatr Drugs. 2022;24(5):433-445.
Alman BA, Li C, Pajerski ME, Diaz-Cano S, Wolfe HJ. Increased beta-catenin protein and somatic APC mutations in sporadic aggressive fibromatoses (desmoid tumors). Am J Pathol. 1997;151(2):329-334.
Lips DJ, Barker N, Clevers H, Hennipman A. The role of APC and beta-catenin in the aetiology of aggressive fibromatosis (desmoid tumors). Eur J Surg Oncol. 2009;35(1):3-10.
Crago AM, Chmielecki J, Rosenberg M, et al. Near universal detection of alterations in CTNNB1 and Wnt pathway regulators in desmoid-type fibromatosis by whole-exome sequencing and genomic analysis. Genes Chromosomes Cancer. 2015;54(10):606-615.
Fujishita T, Aoki K, Lane HA, Aoki M, Taketo MM. Inhibition of the mTORC1 pathway suppresses intestinal polyp formation and reduces mortality in ApcDelta716 mice. Proc Natl Acad Sci U S A. 2008;105(36):13544-13549.
Pressey JG, Wright JM, Geller JI, Joseph DB, Pressey CS, Kelly DR. Sirolimus therapy for fibromatosis and multifocal renal cell carcinoma in a child with tuberous sclerosis complex. Pediatr Blood Cancer. 2010;54(7):1035-1037.
Lin F, Zhang PL, Yang XJ, Prichard JW, Lun M, Brown RE. Morphoproteomic and molecular concomitants of an overexpressed and activated mTOR pathway in renal cell carcinomas. Ann Clin Lab Sci. 2006;36(3):283-293.
Noske A, Lindenberg JL, Darb-Esfahani S, et al. Activation of mTOR in a subgroup of ovarian carcinomas: correlation with p-eIF-4E and prognosis. Oncol Rep. 2008;20(6):1409-1417.
Chen S, Nakahara T, Uchi H, et al. Immunohistochemical analysis of the mammalian target of rapamycin signalling pathway in extramammary Paget's disease. Br J Dermatol. 2009;161(2):357-363.
Orbach D, Brennan B, Bisogno G, et al. The EpSSG NRSTS 2005 treatment protocol for desmoid-type fibromatosis in children: an international prospective case series. Lancet Child Adolesc Health. 2017;1(4):284-292.
Skapek SX, Ferguson WS, Granowetter L, et al. Vinblastine and methotrexate for desmoid fibromatosis in children: results of a Pediatric Oncology Group Phase II Trial. J Clin Oncol. 2007;25(5):501-506.
Timbergen MJM, Smits R, Grünhagen DJ, Verhoef C, Sleijfer S, Wiemer EAC. Activated Signaling Pathways and Targeted Therapies in Desmoid-Type Fibromatosis: A Literature Review. Front Oncol. 2019;9:397.
Rosenberg L, Yoon CH, Sharma G, Bertagnolli MM, Cho NL. Sorafenib inhibits proliferation and invasion in desmoid-derived cells by targeting Ras/MEK/ERK and PI3K/Akt/mTOR pathways. Carcinogenesis. 2018;39(5):681-688.