Activity of Sorafenib against desmoid tumor/deep fibromatosis

Gounder, M. M., Lefkowitz, R. A., Keohan, M. L., D'Adamo, D. R., Hameed, M., Antonescu, C. R., Singer, S., Stout, K., Ahn, L., Maki, R. G. (June 2011) Activity of Sorafenib against desmoid tumor/deep fibromatosis. Clin Cancer Res, 17 (12). pp. 4082-90. ISSN 1078-0432 (Print)1078-0432 (Linking)

DOI: 10.1158/1078-0432.CCR-10-3322


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 ( approximately 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.

Item Type: Paper
Uncontrolled Keywords: Adolescent Adult Benzenesulfonates/administration & dosage/*therapeutic use Female Fibromatosis, Aggressive/diagnosis/*drug therapy/mortality/pathology/surgery Humans Magnetic Resonance Imaging Male Middle Aged Niacinamide/analogs & derivatives Phenylurea Compounds Protein Kinase Inhibitors/administration & dosage/*therapeutic use Pyridines/administration & dosage/*therapeutic use Retrospective Studies Treatment Outcome Watchful Waiting Young Adult
Subjects: diseases & disorders > cancer
diseases & disorders > cancer > drugs and therapies
CSHL Authors:
Communities: CSHL labs > Maki lab
Depositing User: Matt Covey
Date: 15 June 2011
Date Deposited: 21 Oct 2016 19:44
Last Modified: 21 Oct 2016 19:44
PMCID: PMC3152981
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