TY - JOUR
T1 - Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors)
T2 - An FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up
AU - Penel, N.
AU - Le Cesne, A.
AU - Bui, B. N.
AU - Perol, D.
AU - Brain, E. G.
AU - Ray-Coquard, I.
AU - Guillemet, C.
AU - Chevreau, C.
AU - Cupissol, D.
AU - Chabaud, S.
AU - Jimenez, M.
AU - Duffaud, F.
AU - Piperno-Neumann, S.
AU - Mignot, L.
AU - Blay, J. Y.
PY - 2011/2/1
Y1 - 2011/2/1
N2 - Background: Imatinib evaluated as a new treatment option in patients with recurrent or established progressive aggressive fibromatosis/desmoid tumor (AF/DT). Patients and 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 (P0 = 10%, P1 = 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. Conclusion: 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.
AB - Background: Imatinib evaluated as a new treatment option in patients with recurrent or established progressive aggressive fibromatosis/desmoid tumor (AF/DT). Patients and 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 (P0 = 10%, P1 = 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. Conclusion: 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.
KW - Aggressive fibromatosis
KW - Desmoid tumor
KW - Imatinib
KW - Phase II trial
UR - http://www.scopus.com/inward/record.url?scp=79251560032&partnerID=8YFLogxK
U2 - 10.1093/annonc/mdq341
DO - 10.1093/annonc/mdq341
M3 - Article
C2 - 20622000
AN - SCOPUS:79251560032
SN - 0923-7534
VL - 22
SP - 452
EP - 457
JO - Annals of Oncology
JF - Annals of Oncology
IS - 2
ER -