Treatment Options Evolve for Patients With Desmoid Fibromatosis

Neeta Somaiah, MD, an assistant professor in the Department of Sarcoma Medical Oncology at The University of Texas MD Anderson Cancer Center, discusses the evolution of treatment options for patients with a unique sarcoma.

Over the last year, there has been a number of advances for desmoid fibromatosis, says Somaiah. This disease is not actually a sarcoma because it is not considered malignant, but it falls under the sarcoma category. It’s a morbid disease because patients can have local progression, although these tumors do not metastasize. Patients are usually young and have an altered quality of life due to the morbidity of the disease.

Sorafenib (Nexavar) has shown efficacy in terms of an increased response rate in a phase III clinical trial against placebo. However, the placebo-treated patients did respond in a sense that you do have to understand the biology of disease. Most patients may not need treatment and can be followed, says Somaiah. Only patients who progress require therapy.

Nirogacestat (PF-03084014) is a newer drug, which is a gamma secretase inhibitor. It attacks the basis of these desmoid tumors in decreasing fibroblast deposition, says Somaiah. It has shown responses and is well tolerated among these patients. Other β-catenin inhibitors are also available in this treatment landscape. β-catenin is over expressed in most of these tumors because of the mutation expressed. Another β-catenin inhibitor is under evaluation in a phase I trial of desmoid tumors.

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