Sorafenib or anthracycline‐based chemotherapy for progressive desmoid tumors.

Background: Desmoid tumors can cause morbidity due to local invasion, potentially being fatal when fast growth compromises vital structures. In this context, a timely treatment response is required. This study aims to compare the activity of sorafenib and anthracycline‐containing regimens during the first year of treatment. Methods: The authors conducted a multi‐institutional retrospective analysis of desmoid tumor patients treated with either sorafenib or an anthracycline‐containing regimen over 1 year. The primary end point was the overall response rate (ORR). The secondary end points were time to response (TTR), progression‐free survival (PFS), and adverse events. Results: From 2005 to 2022, 80 patients received sorafenib and 51 received an anthracycline‐containing regimen with similar baseline characteristics. The 1‐year ORR was 37% for anthracycline and 13% for sorafenib (p =.016). Median best response was –9% (range, –73 to 51) for anthracycline and –4% (range, –69 to 126) for sorafenib. Median TTR was 5.6 months (95% confidence interval [CI], 3.4–7.8) for anthracycline and 8.7 months (95% CI, 6.3–11.1) for sorafenib (p =.2). One‐year PFS was 73% (95% CI, 60–86) for anthracycline and 59% (95% CI, 47–71) for sorafenib (p =.3). Common grade 1–2 adverse events for sorafenib were hand‐foot syndrome (40%), diarrhea (25%), and fatigue (22%); for anthracycline, they were nausea (31%), fatigue (16%), and rash (14%). Grade 3 events were higher in the anthracycline group, 27% versus 14% (p <.05). Conclusion: Anthracycline‐based therapy provided a greater 1‐year response rate than sorafenib but was associated with a higher rate of serious adverse events. Higher‐risk desmoid tumors, which need a more timely response, might benefit from anthracycline‐based therapies, whereas average‐risk tumors could benefit from sorafenib. This article reports a multi‐institutional retrospective comparison of the activity between sorafenib and anthracycline‐based chemotherapies for desmoid tumors. It shows that anthracycline‐based chemotherapies can produce a greater proportion of responses in the first year of treatment at the expense of greater toxicity. [ABSTRACT FROM AUTHOR]