Targeted therapy of desmoid-type fibromatosis: mechanism, current situation, and future prospects
Zhen Wang, Jianhui Wu, Xiuyun Tian, Chunyi Hao()
Key Laboratory of Carcinogenesis and Translational Research, Ministry of Education, Department of Hepato–Pancreato–Biliary Surgery, Peking University Cancer Hospital and Institute, Beijing 100142, China
Desmoid-type fibromatosis (DF) is a rare monoclonal fibroblastic proliferation that is characterized by locally infiltrative but rarely metastatic lesions. Tyrosine kinase and γ-secretase inhibitors are primarily used in the targeted therapy of DF. The use of these drugs, however, is mainly based on the recommendations of retrospective studies with small sample sizes. Previous studies that focused on the mechanism, efficacy, and safety of targeted therapy for DF were reviewed to provide references for clinical applications and research. The efficacy and safety of targeted therapy were compared with those of other systemic therapy options. Targeted therapy does not provide considerable advantages in efficacy and safety over other medical treatments and is usually applied after the failure of antihormonal therapies, nonsteroidal anti-inflammatory drugs, and chemotherapy. Further studies are required to explore the mechanism, indications, and appropriate drug dosage of the targeted therapy of DF.
Grade 3–4 side effects/dose reduction due to side effects
Characteristics
Imatinib
Mace et al. (2002) [26]
2
100%
100% (FUT: 9 and 11 months)
0%
−
Heinrich et al. (2006) [28]
19
16%
6-M 53%; 12-M 37%; 36-M 12%
Majority
−
Wcislo et al. (2007) [29]
1
100%
100% (FUT 24-M)
0%
−
Chugh et al. (2010) [30]
51
6%
6-M 84%; 12-M 66%; 36-M 58%; 60-M 52%
39%
−
Penel et al. (2011) [20]
40
10%
6-M 80%; 12-M 67%; 24-M 55%; 36-M 40%
45%
−
Kasper et al. (2017) [19]
38
19%
12-M 59%; 24-M 45%
13%
−
Total#
151
13%
−
33%
Nilotinib
Kasper et al. (2017) [19]
8
−
3-M 88%
13%
Had the potential to stabilize DF after the failure of imatinib treatment
Pazopanib
Martin-Liberal et al. (2013) [39]
2
50%
100% *
50%
−
Szucs et al. (2017) [22]
8
38%
6-M 75%; 12-M 75%; 36-M 12.5%
13%
−
Total#
10
40%
−
20%
Sorafenib
Gounder et al. (2011) [40]
26
25%
6-M 95%
15%
Clinical improvement was noted within 2 weeks
Sunitinib
Skubitz et al. (2009) [24]
1
100%
36-M 100%
0%
−
PF-903084014
Messersmith et al. (2015) [53]
7
71%
Response time ranged from 1.74+ months to 24+ months
43%
−
Kummar et al. (2017) [21]
17
29%
94% (median FUT>25 months)
47%
−
Total#
24
42%
−
46%
Antihormone and/or NSAIDS
Hansmann et al. (2003) [70]
27
37%
50%*
0%
30% of the cases developed ovarian cysts; 22% CR
Tanaka et al. (2008) [80]
1
100%
100%*
0%
−
Bocale et al. (2011) [81]
168
50%
80%*
0%
−
Quast et al. (2016) [68]
134
33%
85%*
0%
−
Total#
330
42%
−
0%
Anthracycline-based regimens
Gega et al. [74]
7
100%
Average PFS was 74 months (32.5–107.5)
43%
43% CR
Bertagnolli et al. [79]
10
90%
90%*
−
−
Camargo et al. [73]
35
37%
88%*
6%
−
Garbay et al. [76]
13
54%
100% (median PFS was 40.8 months)
−
−
Total#
65
55%
−
12%
Methotrexate and/or vinblastine
Weisset al. [77]
15
60%
87%*
20%
−
Azzarelli et al. [78]
30
40%
6-M 96%; 12-M 92%; 36-M 80%; 60-M 67%
13%
−
Bertagnolli et al. [79]
4
25%
75%*
−
−
Camargo et al. [73]
22
27%
82%*
−
−
Garbay et al. [76]
27
15%
67% (median PFS 40.8 months)
−
−
Li et al. [82]
71
35%
24-M 80%; 36-M 68%; 60-M 36%
−
−
Total#
169
34%
−
16%
Tab.1
Treatment protocols
Overall response
Grade 3–4 side effects
N
Yes
No
P
N
Yes
No
P
Antihormone and/or NSAIDS
330
139 (42%)
191 (58%)
330
0 (0%)
330 (100%)
Imatinib
151
20 (13%)
131 (87%)
<0.001
132
43 (33%)
89 (67%)
<0.001
Nilotinib
8
−
−
−
8
1 (13%)
7 (87%)
0.024
Pazopanib
10
4 (40%)
6 (60%)
0.893
10
2 (20%)
8 (80%)
0.001
Sorafenib
26
7 (25%)
19 (75%)
0.129
26
4 (15%)
22 (85%)
<0.001
PF-903084014
24
10 (42%)
14 (58%)
0.965
24
11 (46%)
13 (54%)
<0.001
Tab.2
Fig.1
Fig.2
Fig.3
Treatment protocols
Overall response
Grade 3–4 side effects
N
Yes
No
P
N
Yes
No
P
Anthracycline-based regimens
65
36 (55%)
29 (45%)
42
5 (12%)
37 (88%)
Imatinib
151
20 (13%)
131 (87%)
0.001
132
43 (33%)
89 (67%)
0.009
Nilotinib
8
−
−
−
8
1 (13%)
7 (87%)
0.962
Pazopanib
10
4 (40%)
6 (60%)
0.570
10
2 (20%)
8 (80%)
0.874
Sorafenib
26
7 (25%)
19 (75%)
0.014
26
4 (15%)
22 (85%)
0.965
PF-903084014
24
10 (42%)
14 (58%)
0.250
24
11 (46%)
13 (54%)
0.002
Tab.3
Treatment protocols
Overall response
Grade 3–4 side effects
N
Yes
No
P
N
Yes
No
P
Methotrexate and/or vinblastine
169
57 (34%)
112 (66%)
45
7 (16%)
38 (84%)
Imatinib
151
20 (13%)
131 (87%)
<0.001
132
43 (33%)
89 (67%)
0.029
Nilotinib
8
−
−
−
8
1 (13%)
7 (87%)
1.000
Pazopanib
10
4 (40%)
6 (60%)
0.950
10
2 (20%)
8 (80%)
1.000
Sorafenib
26
7 (25%)
19 (75%)
0.492
26
4 (15%)
22 (85%)
1.000
PF-903084014
24
10 (42%)
14 (58%)
0.445
24
11 (46%)
13 (54%)
0.006
Tab.4
1
B Kasper, C Baumgarten, J Garcia, S Bonvalot, R Haas, F Haller, P Hohenberger, N Penel, C Messiou, WT van der Graaf, A Gronchi; Desmoid Working Group. An update on the management of sporadic desmoid-type fibromatosis: a European Consensus Initiative between Sarcoma PAtients EuroNet (SPAEN) and European Organization for Research and Treatment of Cancer (EORTC)/Soft Tissue and Bone Sarcoma Group (STBSG). Ann Oncol 2017; 28(10): 2399–2408 https://doi.org/10.1093/annonc/mdx323
pmid: 28961825
L Bertario, A Russo, P Sala, M Eboli, M Giarola, F D’amico, V Gismondi, L Varesco, MA Pierotti, P; Hereditary Colorectal Tumours Registry. Radice Genotype and phenotype factors as determinants of desmoid tumors in patients with familial adenomatous polyposis. Int J Cancer 2001; 95(2): 102–107 https://doi.org/10.1002/1097-0215(20010320)95:2<102::AID-IJC1018>3.0.CO;2-8
pmid: 11241320
AJ Lazar, D Tuvin, S Hajibashi, S Habeeb, S Bolshakov, E Mayordomo-Aranda, CL Warneke, D Lopez-Terrada, RE Pollock, D Lev. Specific mutations in the β-catenin gene (CTNNB1) correlate with local recurrence in sporadic desmoid tumors. Am J Pathol 2008; 173(5): 1518–1527 https://doi.org/10.2353/ajpath.2008.080475
pmid: 18832571
6
BA Alman, C Li, ME Pajerski, S Diaz-Cano, HJ Wolfe. Increased β-catenin protein and somatic APC mutations in sporadic aggressive fibromatoses (desmoid tumors). Am J Pathol 1997; 151(2): 329–334
pmid: 9250146
7
S Salas, F Chibon, T Noguchi, P Terrier, D Ranchere-Vince, P Lagarde, J Benard, S Forget, C Blanchard, J Dômont, S Bonvalot, L Guillou, A Leroux, A Mechine-Neuville, P Schöffski, M Laë, F Collin, O Verola, A Carbonnelle, L Vescovo, B Bui, V Brouste, H Sobol, A Aurias, JM Coindre. Molecular characterization by array comparative genomic hybridization and DNA sequencing of 194 desmoid tumors. Genes Chromosomes Cancer 2010; 49(6): 560–568 https://doi.org/10.1002/gcc.20766
pmid: 20232483
8
S Le Guellec, I Soubeyran, P Rochaix, T Filleron, A Neuville, I Hostein, JM Coindre. CTNNB1 mutation analysis is a useful tool for the diagnosis of desmoid tumors: a study of 260 desmoid tumors and 191 potential morphologic mimics. Mod Pathol 2012; 25(12): 1551–1558 https://doi.org/10.1038/modpathol.2012.115
pmid: 22766794
9
C Colombo, R Miceli, AJ Lazar, F Perrone, RE Pollock, A Le Cesne, HH Hartgrink, AM Cleton-Jansen, J Domont, JV Bovée, S Bonvalot, D Lev, A Gronchi. CTNNB1 45F mutation is a molecular prognosticator of increased postoperative primary desmoid tumor recurrence: an independent, multicenter validation study. Cancer 2013; 119(20): 3696–3702 https://doi.org/10.1002/cncr.28271
pmid: 23913621
10
DL van Broekhoven, C Verhoef, DJ Grünhagen, JM van Gorp, MA den Bakker, JW Hinrichs, CM de Voijs, T van Dalen. Prognostic value of CTNNB1 gene mutation in primary sporadic aggressive fibromatosis. Ann Surg Oncol 2015; 22(5): 1464–1470 https://doi.org/10.1245/s10434-014-4156-x
pmid: 25341748
11
M Lacroix-Triki, FC Geyer, MB Lambros, K Savage, IO Ellis, AH Lee, JS Reis-Filho. β-catenin/Wnt signalling pathway in fibromatosis, metaplastic carcinomas and phyllodes tumours of the breast. Mod Pathol 2010; 23(11): 1438–1448 https://doi.org/10.1038/modpathol.2010.141
pmid: 20693983
12
J Couture, A Mitri, R Lagace, R Smits, T Berk, HL Bouchard, R Fodde, B Alman, B Bapat. A germline mutation at the extreme 3′ end of the APC gene results in a severe desmoid phenotype and is associated with overexpression of β-catenin in the desmoid tumor. Clin Genet 2000; 57(3): 205–212 https://doi.org/10.1034/j.1399-0004.2000.570306.x
pmid: 10782927
13
JC Harvey, SH Quan, JG Fortner. Gardner’s syndrome complicated by mesenteric desmoid tumors. Surgery 1979; 85(4): 475–477
pmid: 432809
14
C Colombo, R Miceli, C Le Péchoux, E Palassini, C Honoré, S Stacchiotti, O Mir, PG Casali, J Dômont, M Fiore, A Le Cesne, A Gronchi, S Bonvalot. Sporadic extra abdominal wall desmoid-type fibromatosis: surgical resection can be safely limited to a minority of patients. Eur J Cancer 2015; 51(2): 186–192 https://doi.org/10.1016/j.ejca.2014.11.019
pmid: 25500145
15
B Kasper, C Baumgarten, S Bonvalot, R Haas, F Haller, P Hohenberger, G Moreau, WT van der Graaf, A; Desmoid Working Group. Gronchi Management of sporadic desmoid-type fibromatosis: a European consensus approach based on patients’ and professionals’ expertise — a sarcoma patients EuroNet and European Organisation for Research and Treatment of Cancer/Soft Tissue and Bone Sarcoma Group initiative. Eur J Cancer 2015; 51(2): 127–136 https://doi.org/10.1016/j.ejca.2014.11.005
pmid: 25434922
16
S Bonvalot, H Eldweny, V Haddad, F Rimareix, G Missenard, O Oberlin, D Vanel, P Terrier, JY Blay, A Le Cesne, C Le Péchoux. Extra-abdominal primary fibromatosis: aggressive management could be avoided in a subgroup of patients. Eur J Surg Oncol 2008; 34(4): 462–468 https://doi.org/10.1016/j.ejso.2007.06.006
pmid: 17709227
17
M Fiore, F Rimareix, L Mariani, J Domont, P Collini, C Le Péchoux, PG Casali, A Le Cesne, A Gronchi, S Bonvalot. Desmoid-type fibromatosis: a front-line conservative approach to select patients for surgical treatment. Ann Surg Oncol 2009; 16(9): 2587–2593 https://doi.org/10.1245/s10434-009-0586-2
pmid: 19568815
18
S Briand, O Barbier, D Biau, A Bertrand-Vasseur, F Larousserie, P Anract, F Gouin. Wait-and-see policy as a first-line management for extra-abdominal desmoid tumors. J Bone Joint Surg Am 2014; 96(8): 631–638 https://doi.org/10.2106/JBJS.M.00988
pmid: 24740659
19
B Kasper, V Gruenwald, P Reichardt, S Bauer, G Rauch, R Limprecht, M Sommer, A Dimitrakopoulou-Strauss, L Pilz, F Haller, P Hohenberger. Imatinib induces sustained progression arrest in RECIST progressive desmoid tumours: final results of a phase II study of the German Interdisciplinary Sarcoma Group (GISG). Eur J Cancer 2017; 76: 60–67 https://doi.org/10.1016/j.ejca.2017.02.001
pmid: 28282612
20
N Penel, A Le Cesne, BN Bui, D Perol, EG Brain, I Ray-Coquard, C Guillemet, C Chevreau, D Cupissol, S Chabaud, M Jimenez, F Duffaud, S Piperno-Neumann, L Mignot, JY Blay. Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors): an FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up. Ann Oncol 2011; 22(2): 452–457 https://doi.org/10.1093/annonc/mdq341
pmid: 20622000
21
S Kummar, G O’Sullivan Coyne, KT Do, B Turkbey, PS Meltzer, E Polley, PL Choyke, R Meehan, R Vilimas, Y Horneffer, L Juwara, A Lih, A Choudhary, SA Mitchell, LJ Helman, JH Doroshow, AP Chen. Clinical activity of the g-secretase inhibitor PF-03084014 in adults with desmoid tumors (aggressive fibromatosis). J Clin Oncol 2017; 35(14): 1561–1569 https://doi.org/10.1200/JCO.2016.71.1994
pmid: 28350521
22
Z Szucs, C Messiou, HH Wong, H Hatcher, A Miah, S Zaidi, WT van der Graaf, I Judson, RL Jones, C Benson. Pazopanib, a promising option for the treatment of aggressive fibromatosis. Anticancer Drugs 2017; 28(4): 421–426 https://doi.org/10.1097/CAD.0000000000000474
pmid: 28099210
23
C Brogsitter, D Faulhaber, J Kotzerke. Intraarterial treatment of GEP NET: (68)Ga-DOTATOC SUV cannot predict (90)Y-DOTATOC uptake. Clin Cancer Res 2011; 17(7): 2065 https://doi.org/10.1158/1078-0432.CCR-10-1970
pmid: 21447724
24
KM Skubitz, JC Manivel, DR Clohisy, JW Frolich. Response of imatinib-resistant extra-abdominal aggressive fibromatosis to sunitinib: case report and review of the literature on response to tyrosine kinase inhibitors. Cancer Chemother Pharmacol 2009; 64(3): 635–640 https://doi.org/10.1007/s00280-009-1010-0
pmid: 19404642
25
E Buchdunger, CL Cioffi, N Law, D Stover, S Ohno-Jones, BJ Druker, NB Lydon. Abl protein-tyrosine kinase inhibitor STI571 inhibits in vitro signal transduction mediated by c-kit and platelet-derived growth factor receptors. J Pharmacol Exp Ther 2000; 295(1): 139–145
pmid: 10991971
26
J Mace, J Sybil Biermann, V Sondak, C McGinn, C Hayes, D Thomas, L Baker. Response of extraabdominal desmoid tumors to therapy with imatinib mesylate. Cancer 2002; 95(11): 2373–2379 https://doi.org/10.1002/cncr.11029
pmid: 12436445
27
M Carroll, S Ohno-Jones, S Tamura, E Buchdunger, J Zimmermann, NB Lydon, DG Gilliland, BJ Druker. CGP 57148, a tyrosine kinase inhibitor, inhibits the growth of cells expressing BCR-ABL, TEL-ABL, and TEL-PDGFR fusion proteins. Blood 1997; 90(12): 4947–4952
pmid: 9389713
28
MC Heinrich, GA McArthur, GD Demetri, H Joensuu, P Bono, R Herrmann, H Hirte, S Cresta, DB Koslin, CL Corless, S Dirnhofer, AT van Oosterom, Z Nikolova, S Dimitrijevic, JA Fletcher. Clinical and molecular studies of the effect of imatinib on advanced aggressive fibromatosis (desmoid tumor). J Clin Oncol 2006; 24(7): 1195–1203 https://doi.org/10.1200/JCO.2005.04.0717
pmid: 16505440
29
G Wcislo, K Szarlej-Wcislo, C Szczylik. Control of aggressive fibromatosis by treatment with imatinib mesylate. A case report and review of the literature. J Cancer Res Clin Oncol 2007; 133(8): 533–538 https://doi.org/10.1007/s00432-007-0198-9
pmid: 17453242
30
R Chugh, JK Wathen, SR Patel, RG Maki, PA Meyers, SM Schuetze, DA Priebat, DG Thomas, JA Jacobson, BL Samuels, RS Benjamin, LH; Sarcoma Alliance for Research through Collaboration (SARC). Baker Efficacy of imatinib in aggressive fibromatosis: results of a phase II multicenter Sarcoma Alliance for Research through Collaboration (SARC) trial. Clin Cancer Res 2010; 16(19): 4884–4891 https://doi.org/10.1158/1078-0432.CCR-10-1177
pmid: 20724445
31
J Verweij, PG Casali, J Zalcberg, A LeCesne, P Reichardt, JY Blay, R Issels, A van Oosterom, PC Hogendoorn, M Van Glabbeke, R Bertulli, I Judson. Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: randomised trial. Lancet 2004; 364(9440): 1127–1134 https://doi.org/10.1016/S0140-6736(04)17098-0
pmid: 15451219
32
JR Zalcberg, J Verweij, PG Casali, A Le Cesne, P Reichardt, JY Blay, M Schlemmer, M Van Glabbeke, M Brown, IR Judson; EORTC Soft Tissue and Bone Sarcoma Group, the Italian Sarcoma Group; Australasian Gastrointestinal Trials Group. Outcome of patients with advanced gastro-intestinal stromal tumours crossing over to a daily imatinib dose of 800 mg after progression on 400 mg. Eur J Cancer 2005; 41(12): 1751–1757 https://doi.org/10.1016/j.ejca.2005.04.034
pmid: 16098458
33
CD Blanke, C Rankin, GD Demetri, CW Ryan, M von Mehren, RS Benjamin, AK Raymond, VH Bramwell, LH Baker, RG Maki, M Tanaka, JR Hecht, MC Heinrich, CD Fletcher, JJ Crowley, EC Borden. Phase III randomized, intergroup trial assessing imatinib mesylate at two dose levels in patients with unresectable or metastatic gastrointestinal stromal tumors expressing the kit receptor tyrosine kinase: S0033. J Clin Oncol 2008; 26(4): 626–632 https://doi.org/10.1200/JCO.2007.13.4452
pmid: 18235122
34
E Weisberg, P Manley, J Mestan, S Cowan-Jacob, A Ray, JD Griffin. AMN107 (nilotinib): a novel and selective inhibitor of BCR-ABL. Br J Cancer 2006; 94(12): 1765–1769 https://doi.org/10.1038/sj.bjc.6603170
pmid: 16721371
PW Manley, N Stiefl, SW Cowan-Jacob, S Kaufman, J Mestan, M Wartmann, M Wiesmann, R Woodman, N Gallagher. Structural resemblances and comparisons of the relative pharmacological properties of imatinib and nilotinib. Bioorg Med Chem 2010; 18(19): 6977–6986 https://doi.org/10.1016/j.bmc.2010.08.026
pmid: 20817538
37
PW Manley, P Drueckes, G Fendrich, P Furet, J Liebetanz, G Martiny-Baron, J Mestan, J Trappe, M Wartmann, D Fabbro. Extended kinase profile and properties of the protein kinase inhibitor nilotinib. Biochim Biophys Acta 2010; 1804(3): 445–453 https://doi.org/10.1016/j.bbapap.2009.11.008
pmid: 19922818
38
WT van der Graaf, JY Blay, SP Chawla, DW Kim, B Bui-Nguyen, PG Casali, P Schöffski, M Aglietta, AP Staddon, Y Beppu, A Le Cesne, H Gelderblom, IR Judson, N Araki, M Ouali, S Marreaud, R Hodge, MR Dewji, C Coens, GD Demetri, CD Fletcher, AP Dei Tos, P Hohenberger; EORTC Soft Tissue and Bone Sarcoma Group; PALETTE Study Group. Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet 2012; 379(9829): 1879–1886 https://doi.org/10.1016/S0140-6736(12)60651-5
pmid: 22595799
39
J Martin-Liberal, C Benson, H McCarty, K Thway, C Messiou, I Judson. Pazopanib is an active treatment in desmoid tumour/aggressive fibromatosis. Clin Sarcoma Res 2013; 3(1): 13 https://doi.org/10.1186/2045-3329-3-13
pmid: 24279994
40
MM Gounder, RA Lefkowitz, ML Keohan, DR D’Adamo, M Hameed, CR Antonescu, S Singer, K Stout, L Ahn, RG Maki. Activity of sorafenib against desmoid tumor/deep fibromatosis. Clin Cancer Res 2011; 17(12): 4082–4090 https://doi.org/10.1158/1078-0432.CCR-10-3322
pmid: 21447727
41
GT Wong, D Manfra, FM Poulet, Q Zhang, H Josien, T Bara, L Engstrom, M Pinzon-Ortiz, JS Fine, HJ Lee, L Zhang, GA Higgins, EM Parker. Chronic treatment with the γ-secretase inhibitor LY-411,575 inhibits β-amyloid peptide production and alters lymphopoiesis and intestinal cell differentiation. J Biol Chem 2004; 279(13): 12876–12882 https://doi.org/10.1074/jbc.M311652200
pmid: 14709552
42
SJ Pollack, H Lewis. Secretase inhibitors for Alzheimer’s disease: challenges of a promiscuous protease. Curr Opin Investig Drugs 2005; 6(1): 35–47
pmid: 15675602
43
DB Henley, KL Sundell, G Sethuraman, SA Dowsett, PC May. Safety profile of semagacestat, a γ-secretase inhibitor: IDENTITY trial findings. Curr Med Res Opin 2014; 30(10): 2021–2032 https://doi.org/10.1185/03007995.2014.939167
pmid: 24983746
44
LW Ellisen, J Bird, DC West, AL Soreng, TC Reynolds, SD Smith, J Sklar. TAN-1, the human homolog of the Drosophila notch gene, is broken by chromosomal translocations in T lymphoblastic neoplasms. Cell 1991; 66(4): 649–661 https://doi.org/10.1016/0092-8674(91)90111-B
pmid: 1831692
45
AP Weng, AA Ferrando, W Lee, JP Morris 4th, LB Silverman, C Sanchez-Irizarry, SC Blacklow, AT Look, JC Aster. Activating mutations of NOTCH1 in human T cell acute lymphoblastic leukemia. Science 2004; 306(5694): 269–271 https://doi.org/10.1126/science.1102160
pmid: 15472075
46
P Zagouras, S Stifani, CM Blaumueller, ML Carcangiu, S Artavanis-Tsakonas. Alterations in Notch signaling in neoplastic lesions of the human cervix. Proc Natl Acad Sci USA 1995; 92(14): 6414–6418 https://doi.org/10.1073/pnas.92.14.6414
pmid: 7604005
47
Y Miyamoto, A Maitra, B Ghosh, U Zechner, P Argani, CA Iacobuzio-Donahue, V Sriuranpong, T Iso, IM Meszoely, MS Wolfe, RH Hruban, DW Ball, RM Schmid, SD Leach. Notch mediates TGF α-induced changes in epithelial differentiation during pancreatic tumorigenesis. Cancer Cell 2003; 3(6): 565–576 https://doi.org/10.1016/S1535-6108(03)00140-5
pmid: 12842085
48
V Rodilla, A Villanueva, A Obrador-Hevia, A Robert-Moreno, V Fernández-Majada, A Grilli, N López-Bigas, N Bellora, MM Albà, F Torres, M Duñach, X Sanjuan, S Gonzalez, T Gridley, G Capella, A Bigas, L Espinosa. Jagged1 is the pathological link between Wnt and Notch pathways in colorectal cancer. Proc Natl Acad Sci USA 2009; 106(15): 6315–6320 https://doi.org/10.1073/pnas.0813221106
pmid: 19325125
49
HA Kim, BK Koo, JH Cho, YY Kim, J Seong, HJ Chang, YM Oh, DE Stange, JG Park, D Hwang, YY Kong. Notch1 counteracts WNT/b-catenin signaling through chromatin modification in colorectal cancer. J Clin Invest 2012; 122(9): 3248–3259 https://doi.org/10.1172/JCI61216
pmid: 22863622
J Yao, C Qian, T Shu, X Zhang, Z Zhao, Y Liang. Combination treatment of PD98059 and DAPT in gastric cancer through induction of apoptosis and downregulation of WNT/b-catenin. Cancer Biol Ther 2013; 14(9): 833–839 https://doi.org/10.4161/cbt.25332
pmid: 23792588
52
JJ Arcaroli, KS Quackenbush, A Purkey, RW Powell, TM Pitts, S Bagby, AC Tan, B Cross, K McPhillips, EK Song, WM Tai, RA Winn, K Bikkavilli, M Vanscoyk, SG Eckhardt, WA Messersmith. Tumours with elevated levels of the Notch and Wnt pathways exhibit efficacy to PF-03084014, a g-secretase inhibitor, in a preclinical colorectal explant model. Br J Cancer 2013; 109(3): 667–675 https://doi.org/10.1038/bjc.2013.361
pmid: 23868008
53
WA Messersmith, GI Shapiro, JM Cleary, A Jimeno, A Dasari, B Huang, MN Shaik, R Cesari, X Zheng, JM Reynolds, PA English, KR McLachlan, KA Kern, PM LoRusso. A Phase I, dose-finding study in patients with advanced solid malignancies of the oral g-secretase inhibitor PF-03084014. Clin Cancer Res 2015; 21(1): 60–67 https://doi.org/10.1158/1078-0432.CCR-14-0607
pmid: 25231399
54
S Roussin, C Mazouni, F Rimareix, C Honoré, P Terrier, O Mir, J Dômont, C Le Péchoux, A Le Cesne, S Bonvalot. Toward a new strategy in desmoid of the breast? Eur J Surg Oncol 2015; 41(4): 571–576 https://doi.org/10.1016/j.ejso.2015.01.001
pmid: 25639193
D Lev, D Kotilingam, C Wei, MT Ballo, GK Zagars, PW Pisters, AA Lazar, SR Patel, RS Benjamin, RE Pollock. Optimizing treatment of desmoid tumors. J Clin Oncol 2007; 25(13): 1785–1791 https://doi.org/10.1200/JCO.2006.10.5015
pmid: 17470870
MT Ballo, GK Zagars, A Pollack, PW Pisters, RA Pollack. Desmoid tumor: prognostic factors and outcome after surgery, radiation therapy, or combined surgery and radiation therapy. J Clin Oncol 1999; 17(1): 158–167 https://doi.org/10.1200/JCO.1999.17.1.158
pmid: 10458229
59
A Gronchi, PG Casali, L Mariani, S Lo Vullo, M Colecchia, L Lozza, R Bertulli, M Fiore, P Olmi, M Santinami, J Rosai. Quality of surgery and outcome in extra-abdominal aggressive fibromatosis: a series of patients surgically treated at a single institution. J Clin Oncol 2003; 21(7): 1390–1397 https://doi.org/10.1200/JCO.2003.05.150
pmid: 12663732
SA Leibel, WM Wara, DR Hill, EG Bovill Jr, AA de Lorimier, JH Beckstead, TL Phillips. Desmoid tumors: local control and patterns of relapse following radiation therapy. Int J Radiat Oncol Biol Phys 1983; 9(8): 1167–1171 https://doi.org/10.1016/0360-3016(83)90175-X
pmid: 6409853
62
G Pignatti, G Barbanti-Bròdano, D Ferrari, F Gherlinzoni, F Bertoni, P Bacchini, E Barbieri, A Giunti, M Campanacci. Extraabdominal desmoid tumor. A study of 83 cases. Clin Orthop Relat Res 2000; (375): 207–213 https://doi.org/10.1097/00003086-200006000-00025
pmid: 10853171
A Sørensen, J Keller, OS Nielsen, OM Jensen. Treatment of aggressive fibromatosis: a retrospective study of 72 patients followed for 1−27 years. Acta Orthop Scand 2002; 73(2): 213–219 https://doi.org/10.1080/000164702753671830
pmid: 12079022
66
AM Crago, B Denton, S Salas, A Dufresne, JJ Mezhir, M Hameed, M Gonen, S Singer, MF Brennan. A prognostic nomogram for prediction of recurrence in desmoid fibromatosis. Ann Surg 2013; 258(2): 347–353 https://doi.org/10.1097/SLA.0b013e31828c8a30
pmid: 23532110
67
M Fiore, C Colombo, S Radaelli, D Callegaro, E Palassini, M Barisella, C Morosi, GG Baldi, S Stacchiotti, PG Casali, A Gronchi. Hormonal manipulation with toremifene in sporadic desmoid-type fibromatosis. Eur J Cancer 2015; 51(18): 2800–2807 https://doi.org/10.1016/j.ejca.2015.08.026
pmid: 26602014
68
DR Quast, R Schneider, E Burdzik, S Hoppe, G Möslein. Long-term outcome of sporadic and FAP-associated desmoid tumors treated with high-dose selective estrogen receptor modulators and sulindac: a single-center long-term observational study in 134 patients. Fam Cancer 2016; 15(1): 31–40 https://doi.org/10.1007/s10689-015-9830-z
pmid: 26275868
69
T Desurmont, JH Lefèvre, C Shields, C Colas, E Tiret, Y Parc. Desmoid tumour in familial adenomatous polyposis patients: responses to treatments. Fam Cancer 2015; 14(1): 31–39 https://doi.org/10.1007/s10689-014-9760-1
pmid: 25315103
70
A Hansmann, C Adolph, T Vogel, A Unger, G Moeslein. High-dose tamoxifen and sulindac as first-line treatment for desmoid tumors. Cancer 2004; 100(3): 612–620 https://doi.org/10.1002/cncr.11937
pmid: 14745880
B Fisher, JP Costantino, DL Wickerham, CK Redmond, M Kavanah, WM Cronin, V Vogel, A Robidoux, N Dimitrov, J Atkins, M Daly, S Wieand, E Tan-Chiu, L Ford, N Wolmark. Tamoxifen for prevention of breast cancer: report of the National Surgical Adjuvant Breast and Bowel Project P-1 Study. J Natl Cancer Inst 1998; 90(18): 1371–1388 https://doi.org/10.1093/jnci/90.18.1371
pmid: 9747868
73
VP de Camargo, ML Keohan, DR D’Adamo, CR Antonescu, MF Brennan, S Singer, LS Ahn, RG Maki. Clinical outcomes of systemic therapy for patients with deep fibromatosis (desmoid tumor). Cancer 2010; 116(9): 2258–2265 https://doi.org/10.1002/cncr.25089
pmid: 20187095
74
M Gega, H Yanagi, R Yoshikawa, M Noda, H Ikeuchi, K Tsukamoto, T Oshima, Y Fujiwara, N Gondo, K Tamura, J Utsunomiya, T Hashimoto-Tamaoki, T Yamamura. Successful chemotherapeutic modality of doxorubicin plus dacarbazine for the treatment of desmoid tumors in association with familial adenomatous polyposis. J Clin Oncol 2006; 24(1): 102–105 https://doi.org/10.1200/JCO.2005.02.1923
pmid: 16382119
75
A Constantinidou, RL Jones, M Scurr, O Al-Muderis, I Judson. Pegylated liposomal doxorubicin, an effective, well-tolerated treatment for refractory aggressive fibromatosis. Eur J Cancer 2009; 45(17): 2930–2934 https://doi.org/10.1016/j.ejca.2009.08.016
pmid: 19767198
76
D Garbay, A Le Cesne, N Penel, C Chevreau, P Marec-Berard, JY Blay, M Debled, N Isambert, A Thyss, E Bompas, O Collard, S Salas, JM Coindre, B Bui, A Italiano. Chemotherapy in patients with desmoid tumors: a study from the French Sarcoma Group (FSG). Ann Oncol 2012; 23(1): 182–186 https://doi.org/10.1093/annonc/mdr051
pmid: 21444357
A Azzarelli, A Gronchi, R Bertulli, JD Tesoro, D Baratti, E Pennacchioli, P Dileo, A Rasponi, A Ferrari, S Pilotti, PG Casali. Low-dose chemotherapy with methotrexate and vinblastine for patients with advanced aggressive fibromatosis. Cancer 2001; 92(5): 1259–1264 https://doi.org/10.1002/1097-0142(20010901)92:5<1259::AID-CNCR1446>3.0.CO;2-Y
pmid: 11571741
79
MM Bertagnolli, JA Morgan, CD Fletcher, CP Raut, P Dileo, RR Gill, GD Demetri, S George. Multimodality treatment of mesenteric desmoid tumours. Eur J Cancer 2008; 44(16): 2404–2410 https://doi.org/10.1016/j.ejca.2008.06.038
pmid: 18706807
80
K Tanaka, R Yoshikawa, H Yanagi, M Gega, Y Fujiwara, T Hashimoto-Tamaoki, S Hirota, T Tsujimura, N Tomita. Regression of sporadic intra-abdominal desmoid tumour following administration of non-steroidal anti-inflammatory drug. World J Surg Oncol 2008; 6(1): 17 https://doi.org/10.1186/1477-7819-6-17
pmid: 18257933
81
D Bocale, MT Rotelli, A Cavallini, DF Altomare. Anti-oestrogen therapy in the treatment of desmoid tumours: a systematic review. Colorectal Dis 2011; 13(12): e388–e395 https://doi.org/10.1111/j.1463-1318.2011.02758.x
pmid: 21831172
82
S Li, Z Fan, Z Fang, J Liu, C Bai, R Xue, L Zhang, T Gao. Efficacy of vinorelbine combined with low-dose methotrexate for treatment of inoperable desmoid tumor and prognostic factor analysis. Chin J Cancer Res 2017; 29(5): 455–462 https://doi.org/10.21147/j.issn.1000-9604.2017.05.10
pmid: 29142465