Effective Management of Desmoid Fibromatosis With Sorafenib in Children

Abstract

Purpose: Desmoid fibromatosis is a locally aggressive neoplasm with high recurrence rate after surgery and no well-defined treatment guidelines. In this paper, we evaluate the effectiveness of sorafenib on tumor response. Methods: We identified all pediatric patients with desmoid tumors treated with sorafenib in our institution. We then completed a thorough retrospective chart review and evaluated treatment response based on clinical changes. Descriptive statistics were used to determine efficacy of sorafenib. Results: Four patients were studied. Sorafenib was dosed starting at 200mg daily and raised to 400mg daily. Median duration of treatment was 12 months. Side effects included fatigue, nausea, skin rash, hypertension, weight loss and neuropathy. In all patients, treatment with sorafenib resulted in significant clinical improvement within months of treatment and no evidence of disease progression from 2 to 13 months from discontinuation of therapy. Conclusions: Based on the promising results on our patients, we conclude that sorafenib is active agent in desmoid fibromatosis and its use should be strongly considered in the first line treatment in cases of unresectable disease. However, further prospective studies should be conducted to further define the role of sorafenib in this treatment.