title: Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors): an FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up.
abstract: BACKGROUND
  Imatinib evaluated as a new treatment option in patients with recurrent or established progressive aggressive fibromatosis/desmoid tumor (AF/DT).
  
  
  METHODS
  Forty patients with unresectable and progressive symptomatic AF/DT were treated with imatinib (400 mg/day for 1 year) in a Simon's optimal two-stage phase II study (P(0) = 10%, P(1) = 30%, α = 5%, β = 10%). The primary end point was non-progressive at 3 months (RECIST).
  
  
  RESULTS
  The study population consisted of 28 women and 12 men, with a mean age of 41 (range 20-72 years). Most of the primary sites were extra-abdominal (24, 54.5%). Familial adenomatous polyposis was observed in six (15%) cases. The median follow-up was 34 months. Imatinib toxicity was similar to that previously reported in literature. Tumor assessment was validated by a central independent radiology committee for 35 patients At 3 months, one (3%) complete and three (9%) partial confirmed responses were observed. The non-progression rates at 3, 6 and 12 months were, respectively, 91%, 80% and 67%. The 2-year progression-free and overall survival rates were 55% and 95%, respectively. Two patients with mesenteric AF/DT died from progressive disease.
  
  
  CONCLUSIONS
  Imatinib is active in the treatment of recurrent and progressive AF/DT, providing objective response and long-term stable disease in a large proportion of patients.
authors.affiliation: Department of General Cancerology, Oscar Lambret Center, Lille, France. n-penel@o-lambret.fr
authors.forename: N
authors.initials: N
authors.lastname: Penel
authors.forename: A
authors.initials: A
authors.lastname: Le Cesne
authors.forename: B N
authors.initials: BN
authors.lastname: Bui
authors.forename: D
authors.initials: D
authors.lastname: Perol
authors.forename: E G
authors.initials: EG
authors.lastname: Brain
authors.forename: I
authors.initials: I
authors.lastname: Ray-Coquard
authors.forename: C
authors.initials: C
authors.lastname: Guillemet
authors.forename: C
authors.initials: C
authors.lastname: Chevreau
authors.forename: D
authors.initials: D
authors.lastname: Cupissol
authors.forename: S
authors.initials: S
authors.lastname: Chabaud
authors.forename: M
authors.initials: M
authors.lastname: Jimenez
authors.forename: F
authors.initials: F
authors.lastname: Duffaud
authors.forename: S
authors.initials: S
authors.lastname: Piperno-Neumann
authors.forename: L
authors.initials: L
authors.lastname: Mignot
authors.forename: J-Y
authors.initials: JY
authors.lastname: Blay
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001549:Benzamides; D018450:Disease Progression; D005350:Fibroma / Q000188:drug therapy*; D005500:Follow-Up Studies; D006801:Humans; D000068877:Imatinib Mesylate; D008875:Middle Aged; D010879:Piperazines / Q000009:adverse effects / Q000627:therapeutic use*; D011743:Pyrimidines / Q000009:adverse effects / Q000627:therapeutic use*; D012008:Recurrence; D016019:Survival Analysis
chemical_list: D000970:Antineoplastic Agents; D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate