Wednesday, March 28, 2018 Interim results from a randomized clinical trial for patients with desmoid tumors or aggressive fibromatosis (DT/DF) show that the drug sorafenib tosylate (Nexavar) extended progression-free survival compared with a placebo. Progression-free survival is the length of time patients lived before their disease worsened. Based on these interim results, the data & safety monitoring board overseeing the trial recommended that the primary results of the study be released.
The trial was sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH); designed & conducted by researchers with the Alliance for Clinical Trials in Oncology (Alliance); & supported by Bayer HealthCare AG, which provided the study drug.
“Sorafenib is a book way of treating this rare cancer,” said lead investigator and study chair Mrinal M. Gounder, M.D., sarcoma medical oncologist at Memorial Sloan Kettering Cancer Center in New York City. “The promising results of this phase 3 trial represent a paradigm shift in the approach to treatment of patients with desmoid tumors.”
DT/DF are rare sarcomas estimated to occur in approximately 1,000 people each year in the United States, many of them relatively young. The sarcomas usually arise in the extremities or the abdomen & occasionally are associated with familial adenomatous polyposis or Gardner Syndrome. DT/DF are locally aggressive & can result in pain & decreased mobility, & they can invade vital organs or structures resulting in bowel obstructions & other serious complications.
“Currently, there is no standard treatment for this rare disease, & the effectiveness…