Exploring Current U.S. Physician Preferences for the Treatment of Patients with Desmoid Tumors

Summary

Desmoid tumors are rare, locally invasive soft-tissue tumors characterized by a heterogeneous clinical course. Currently, there are no FDA approved therapies for the treatment of desmoid tumors, but a number of agents are used off-label along with locoregional interventions, such as surgery. The identification of physicians who treat these patients and the therapies used is rendered challenging by the lack of a current dedicated ICD-10 code for desmoid tumors. This prevents the utilization of resources, such as claims data, that are typically used to characterize treatment preferences.  Recent joint global consensus guidelines recommend an initial active surveillance period followed by a surgical intervention for abdominal wall tumors and systemic therapies for other locations upon progression. However, in practice, U.S. physician treatment preferences remain unclear. SpringWorks Therapeutics, which is developing a gamma secretase inhibitor for the treatment of desmoid tumors, sought to better understand how U.S. physicians currently treat patients with desmoid tumors. We designed a physician survey to assess past and current therapies used along with a series of hypothetical case studies to evaluate future treatment preferences. The goals of the survey included understanding whether physicians preferred a watchful waiting approach to an active intervention as a front-line therapy, which active treatments (i.e., locoregional or systemic therapies) physicians preferred, and whether patient presentation and etiology altered treatment preferences. The results of the case study portion of the survey were presented at the 2020 American Association for Cancer Research Virtual Annual Meeting II and are described below.