Gounder MM, et al. - Researchers assessed the efficacy of sorafenib as a treatment option in patients with progressive, symptomatic, or recurrent desmoid tumors, compared with placebo. Treatment with sorafenib offered significantly prolonged progression-free survival and induced durable responses in patients with progressive, refractory, or symptomatic desmoid tumors.

Methods

• This was a double-blind, phase 3 trial.
• Participants were 87 patients with progressive, symptomatic, or recurrent desmoid tumors.
• Researchers randomly assigned participants to receive either sorafenib (400-mg tablet once daily) or matching placebo.
• For patients in the placebo group who had disease progression, crossover to the sorafenib group was allowed.
• Investigator-assessed progression-free survival was primary end point.
• They also assessed rates of objective response and adverse events.

Results

• During a median follow-up of 27.2 months, the sorafenib group and the placebo group demonstrated the 2-year progression-free survival rate of 81% (95% confidence interval [CI], 69 to 96) and 36% (95% CI, 22 to 57), respectively (hazard ratio for progression or death, 0.13; 95% CI, 0.05 to 0.31; P<0.001).
• In the sorafenib group and in the placebo group, the reported objective response rate was 33% (95% CI, 20 to 48) and 20% (95% CI, 8 to 38), respectively, before crossover.
• In the sorafenib group and in the placebo group, the reported median time to an objective response among patients who had a response was 9.6 months (interquartile range, 6.6 to 16.7) and 13.3 months (interquartile range, 11.2 to 31.1), respectively.
• The objective responses are ongoing.
• Grade 1 or 2 events of rash (73%), fatigue (67%), hypertension (55%), and diarrhea (51%) were most frequently experienced adverse events among patients who received sorafenib.