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N Engl J Med. 2018 Dec 20;379(25):2417-2428. doi: 10.1056/NEJMoa1805052.

Sorafenib for Advanced and Refractory Desmoid Tumors.

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From Memorial Sloan Kettering Cancer Center and Weill Cornell Medical Center (M.M.G., W.D.T., N.H., N.P.A., R.A.L., Y.M., R.Y.) and Columbia University Vagellos College of Physicians and Surgeons and New York Presbyterian Hospital (L.H.S., G.K.S.), New York, and Northwell Cancer Institute and Cold Spring Harbor Laboratory, Lake Success (R.G.M.) - all in New York; Alliance Statistics and Data Center, Mayo Clinic, Rochester, MN (M.R.M.); Washington University School of Medicine, St. Louis (B.A.V.T.); M.D. Anderson Cancer Center, University of Texas, Houston (V.R.); Mayo Clinic in Florida, Jacksonville (S.A.); Yale University, New Haven, CT (H.A.D.); University Health Network Princess Margaret Cancer Centre, Toronto (A.A.G.); University of Iowa-Holden Comprehensive Cancer Center, Iowa City (M.M.M.); University of Alabama at Birmingham Cancer Center, Birmingham (R.M.C.); Fox Chase Cancer Center, Philadelphia (S.M.); Georgetown University, Lombardi Comprehensive Cancer Center, Washington, DC (M.J.P.); Duke Cancer Institute, Duke University Medical Center, Durham (R.F.R.), and Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill (E.B.) - both in North Carolina; Dayton National Cancer Institute Community Oncology Research Program, Dayton, OH (T.S.); National Cancer Institute, Bethesda, MD (J.J.W.); and the Alliance Statistics and Data Center, Mayo Clinic, Scottsdale, AZ (A.C.D.).



Desmoid tumors (also referred to as aggressive fibromatosis) are connective tissue neoplasms that can arise in any anatomical location and infiltrate the mesentery, neurovascular structures, and visceral organs. There is no standard of care.


In this double-blind, phase 3 trial, we randomly assigned 87 patients with progressive, symptomatic, or recurrent desmoid tumors to receive either sorafenib (400-mg tablet once daily) or matching placebo. Crossover to the sorafenib group was permitted for patients in the placebo group who had disease progression. The primary end point was investigator-assessed progression-free survival; rates of objective response and adverse events were also evaluated.


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


Among patients with progressive, refractory, or symptomatic desmoid tumors, sorafenib significantly prolonged progression-free survival and induced durable responses. (Funded by the National Cancer Institute and others; number, NCT02066181 .).

[Indexed for MEDLINE]

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