Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors): an FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up.
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.
Penel
N
N
Department of General Cancerology, Oscar Lambret Center, Lille, France. n-penel@o-lambret.fr
Le Cesne
A
A
Bui
B N
BN
Perol
D
D
Brain
E G
EG
Ray-Coquard
I
I
Guillemet
C
C
Chevreau
C
C
Cupissol
D
D
Chabaud
S
S
Jimenez
M
M
Duffaud
F
F
Piperno-Neumann
S
S
Mignot
L
L
Blay
J-Y
JY
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
D000970:Antineoplastic Agents; D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate