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. 2011 Jun 15;17(12):4082-90.
doi: 10.1158/1078-0432.CCR-10-3322. Epub 2011 Mar 29.

Activity of Sorafenib against desmoid tumor/deep fibromatosis

Mrinal M Gounder  1 , Robert A Lefkowitz, Mary Louise Keohan, David R D'Adamo, Meera Hameed, Cristina R Antonescu, Samuel Singer, Katherine Stout, Linda Ahn, Robert G Maki
Affiliations

Activity of Sorafenib against desmoid tumor/deep fibromatosis

Mrinal M Gounder et al. Clin Cancer Res. .

Abstract

Background: Desmoid tumors (deep fibromatoses) are clonal connective tissue malignancies that do not metastasize, but have a significant risk of local recurrence, and are associated with morbidity and occasionally mortality. Responses of desmoid patients to sorafenib on an expanded access program led us to review our experience.

Methods: After Institutional Review Board (IRB) approval, we reviewed data for 26 patients with desmoid tumors treated with sorafenib. Sorafenib was administered at 400 mg oral daily and adjusted for toxicity.

Results: Sorafenib was the first-line therapy in 11/26 patients and the remaining 15/26 had received a median of 2 prior lines of therapy. Twenty-three of 26 patients had shown evidence of progressive disease by imaging, whereas 3 patients had achieved maximum benefit or toxicity with chemotherapy. Sixteen of 22 (∼70%) patients reported significant improvement of symptoms. At a median of 6 months (2-29) of treatment, the best response evaluation criteria in solid tumors (RECIST) 1.1 response included 6/24 (25%) patients with partial response (PR), 17/24 (70%) with stable disease, and 1 with progression and death. Twelve of 13 (92%) patients evaluated by MRI had > 30% decrease in T2 signal intensity, an indirect metric for increased fibrosis and loss of cellularity. Eighty percent of patients with radiological benefit had extra-abdominal desmoids.

Discussion: Sorafenib is active against desmoid tumors. A prospective, randomized clinical trial of sorafenib against other active agents is warranted. Loss of MRI T2 signal may be a useful surrogate for defining responses, but requires validation by examination of tumor pathology.

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Figures

Figure 1
Figure 1
An MRI (with contrast, T2 weighted) of the upper arm depicting a desmoid tumor encasing a neurovascular bundle before (1A) and after six months (1B) of sorafenib.
Figure 2
Figure 2
Waterfall plot of best radiological outcome by size (RECIST 1.1) for individual patients along with duration of response (months) and type of imaging noted below each patient column.
Figure 3
Figure 3
Waterfall plot of best radiological outcome by size (RECIST 1.1) of patients evaluated by MRI, duration of response (months) and change in T2 signal intensity (gray columns).
See this image and copyright information in PMC

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