Sorafenib was well tolerated with significantly improved progression-free survival in select patients with desmoid tumors, reported Mrinal M. Gounder, MD, of Memorial Sloan-Kettering Cancer Center, New York.
“The study exceeded its primary endpoint for progression-free survival ... Sorafenib may represent a new, first-line or subsequent-line standard of care in select patients with desmoid tumors,” Dr. Gounder said at the annual meeting of the American Society of Clinical Oncology.
For this international prospective study of progression-free survival response to sorafenib, 87 patients were enrolled over 17 months at 25 sites. Patients had unresectable progressive or symptomatic desmoid tumors. Patients were stratified by pain level and disease site and randomized 2:1 to sorafenib 400 mg/day or placebo. Placebo-treated patients were crossed over to sorafenib if they reached RECIST 1.1.
After a median follow up for 26 months, disease had progressed in 22 of 32 patients on placebo and in 7 of 43 patients on sorafenib. One sorafenib-treated patient died. Durable partial responses were seen in 14 of 43 on sorafenib and in 7 of 32 on placebo. At one year, progression-free survival was 43% with placebo (median PFS 9.4 months) and 87% with sorafenib (median PFS not reached [HR = 0.14 (95% CI 0.06-0.33), P less than 0.0001)].
The authors disclosed funding from a wide range of drug companies. Several authors received funding from Bayer, the maker of sorafenib (Nexavar). Clinical trial information: NCT02066181.
SOURCE: Gounder M et al. ASCO 2018 (the annual meeting of the American Society of Clinical Oncology), Abstract 11500.