Join us in Philadelphia from Sept. 25-27th for our "Together We Will" Weekend: - Register today!

The Desmoid Tumor Research Foundation

April 2022

Current management and recent progress in desmoid tumors

Author Information

Zhou MY (1), Bui NQ (2), Charville GW (3), Ghanouni P (4), Ganjoo KN (5).

(1) Department of Medicine, Stanford University School of Medicine, Stanford, CA. (2) Department of Medicine (Oncology), Stanford University School of Medicine, 875 Blake Wilbur Drive, Stanford, CA, 94305, USA. (3) Department of Pathology, Stanford University School of Medicine, Stanford, CA, USA. (4) Department of Radiology, Stanford University School of Medicine, Stanford, CA. (5) Department of Medicine (Oncology), Stanford University School of Medicine, 875 Blake Wilbur Drive, Stanford, CA, 94305, USA. Electronic address: kganjoo@stanford.edu.

Publication

Cancer Treat Res Commun

Summary

Desmoid tumors are rare soft tissue tumors that can have aggressive infiltrative growth and relapse locally. Desmoid tumors can impact functionality and cause treatment-related morbidity and mortality. Here, the authors review current management strategies and avenues for further investigation. As part of the evolution of therapy away from primary surgical approaches to less invasive options, image-guided ablation has been accepted as less morbid and include cryoablation and high-intensity focused ultrasound. Systemic therapy options currently include hormonal agents, nonsteroidal anti-inflammatory drugs, tyrosine kinase inhibitors, and anthracycline-based regimens. Hormonal agents and nonsteroidal anti-inflammatory drugs have benign side effect profiles but generally limited efficacy. Anthracycline-based therapies are limited by the risk of secondary malignancies and cardiomyopathy. Tyrosine kinase inhibitors are well studied, and sorafenib is now one of the most utilized therapies, though limited by its side effect profile. Nirogacestat (PF-0308401) is an investigational small molecule gamma-secretase (GS) inhibitor that has demonstrated efficacy in phase 1 and II trials. A phase III trial investigating patients with desmoid tumors or aggressive fibromatosis is estimated to be completed December 2021 (NCT03785964). In addition to nirogacestat, the gamma-secretase inhibitor AL102 is being investigated for the treatment of patients with progressing desmoid tumors in the phase II/III RINGSIDE trial. Finally, the beta-catenin inhibitor Tegavivint (BC2059) is being investigated in a phase 1 open-label trial in patients with a proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive.

View Abstract