Activity of sorafenib against desmoid tumor/deep fibromatosis Journal Article


Authors: 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.
Article Title: Activity of sorafenib against desmoid tumor/deep fibromatosis
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. ©2011 AACR.
Keywords: adolescent; adult; cancer survival; clinical article; human tissue; cancer surgery; fatigue; histopathology; sorafenib; doxorubicin; sunitinib; cancer growth; diarrhea; drug dose reduction; drug efficacy; hypertension; treatment duration; methotrexate; nuclear magnetic resonance imaging; outcome assessment; imatinib; dacarbazine; drug eruption; multiple cycle treatment; image analysis; gastrointestinal symptom; mucosa inflammation; nausea; vomiting; cyclophosphamide; retrospective study; cancer mortality; drug hypersensitivity; leuprorelin; nonsteroid antiinflammatory agent; drug response; brachytherapy; tamoxifen; anthracycline derivative; hand foot syndrome; dry skin; alopecia; congestive heart failure; blurred vision; desmoid tumor; fibromatosis; skin pain; skin bruising; medroxyprogesterone; alveolar bone loss; dry hand; myofibrosis; scotoma; trichodynia
Journal Title: Clinical Cancer Research
Volume: 17
Issue: 12
ISSN: 1078-0432
Publisher: American Association for Cancer Research  
Date Published: 2011-06-15
Start Page: 4082
End Page: 4090
Language: English
DOI: 10.1158/1078-0432.ccr-10-3322
PROVIDER: scopus
PUBMED: 21447727
PMCID: PMC3152981
DOI/URL:
Notes: --- - "Export Date: 17 August 2011" - "CODEN: CCREF" - "Source: Scopus"
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MSK Authors
  1. Meera Hameed
    286 Hameed
  2. David R D'Adamo
    37 D'Adamo
  3. Cristina R Antonescu
    903 Antonescu
  4. Robert Maki
    241 Maki
  5. Mary Louise Keohan
    126 Keohan
  6. Mrinal M Gounder
    230 Gounder
  7. Linda Su Hyun Ahn
    25 Ahn
  8. Samuel Singer
    337 Singer
  9. Katherine Bernadette Ammori
    1 Ammori