A pilot study evaluating the use of sirolimus in children and young adults with desmoid‐type fibromatosis-Reference-Cited by-同舟云学术

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

Published:2023-06-07 Issue:9 Volume:70 Page:
ISSN:1545-5009
Container-title:Pediatric Blood & Cancer
language:en
Short-container-title:Pediatric Blood & Cancer

Author:

Weiss Aaron R.¹^ORCID,Dry Sarah²,Maygar Clara²,Cutler Anya³,Lary Christine W.⁴,Khoo Carmen³,Fergione Jillian E.¹,Hounchell Melanie M.¹,Glick Kathleen¹,Browning Meghen⁵,Choo Sun Ha⁶^ORCID,Hawkins Douglas S.⁷^ORCID,Lagmay Joanne⁸,Manalang Michelle⁹,Skapek Stephen X.¹⁰,Weigel Brenda¹¹,Verwys Stephanie¹,Federman Noah¹²^ORCID

Affiliation:

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

Abstract

AbstractDeregulation 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.

Funder

Desmoid Tumor Research Foundation

Pfizer

Publisher

Wiley

Subject

Oncology,Hematology,Pediatrics, Perinatology and Child Health

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/pbc.30466

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