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Familial Cancer

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01.01.2016 | Original Article

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

verfasst von: Daniel Robert Quast, Ralph Schneider, Emanuel Burdzik, Steffen Hoppe, Gabriela Möslein

Erschienen in: Familial Cancer | Ausgabe 1/2016

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Abstract

Aim of this study is to evaluate the outcome of long-term conservative treatment with sulindac and high-dose selective estrogen receptor modulators (SERMs) for sporadic and FAP-associated desmoid tumors. Desmoids are very rare tumors in the general population but occur frequently in FAP patients, being encountered in 23–38 %. Treatment of desmoids is still most controversial since response cannot be predicted and they are prone to develop recurrence. This study included all desmoid patients that were treated and followed at our institution and had completed at least 1 year of treatment. Response was defined as stable size or regression of desmoid size between two CT or MRI scans. A total of 134 patients were included. 64 (47.8 %) patients had a confirmed diagnosis of FAP, 69 (51.5 %) patients were sporadic. Overall 114 (85.1 %) patients showed regressive or stable desmoid size. Patients with previous history of multiple desmoid-related surgeries showed less-favorable response. The mean time to reach at least stable size was 14.9 (±9.1) months. After regression or stabilization, medication was tapered in 69 (60.5 %) of the treated patients with only one long-term recurrence after >10 years. The results of this study fortify the role of sulindac and high-dose SERMs as an effective and safe treatment for both, sporadic and FAP-associated desmoid tumors. While invasive treatment frequently results in high recurrence rates, high morbidity and high mortality, this conservative treatment is successful in most patients. The recurrence rate is negligible with no desmoid-related mortality in this large series. Therefore surgical resection, especially for mesenteric desmoids, should be deferred favoring this convincingly effective, well tolerated regimen.

Robanus-Maandag E, Bosch C, Amini-Nik S, Knijnenburg J, Szuhai K, Cervera P et al (2011) Familial adenomatous polyposis-associated desmoids display significantly more genetic changes than sporadic desmoids. PLoS One 6(9):e24354PubMedPubMedCentralCrossRef

Litchman C (2012) Introduction. In: Litchman C (ed) Desmoid tumors. Springer, Dordrecht, pp 1–2CrossRef

Church JM (1995) Desmoid tumors in patients with familial adenomatous polyposis. Semin Colon Rectal Surg 6(1):29–32

Hansmann A, Adolph C, Vogel T, Unger A, Moeslein G (2004) High-dose tamoxifen and sulindac as first-line treatment for desmoid tumors. Cancer 100(3):612–620PubMedCrossRef

Vasen HFA, Möslein G, Alonso A, Aretz S, Bernstein I, Bertario L et al (2008) Guidelines for the clinical management of familial adenomatous polyposis (FAP). Gut 57(5):704–713PubMedCrossRef

Garzón-Benavides M, Pizarro-Moreno A, García-Lozano R, Herrero-Garrido MI, Hervás-Molina AJ, Márquez-Galán JL et al (2010) Andalusian registry for familial adenomatous polyposis. Analysis of patients included. Rev Esp Enferm Dig 102(11):653–657PubMedCrossRef

Friedl W, Caspari R, Sengteller M, Uhlhaas S, Lamberti C, Jungck M et al (2001) Can APC mutation analysis contribute to therapeutic decisions in familial adenomatous polyposis? Experience from 680 FAP families. Gut 48(4):515–521PubMedPubMedCentralCrossRef

Sinha A, Tekkis PP, Gibbons DC, Phillips RK, Clark SK (2011) Risk factors predicting desmoid occurrence in patients with familial adenomatous polyposis: a meta-analysis. Colorectal Dis 13(11):1222–1229PubMedCrossRef

Knudsen AL, Bülow S (2000) Desmoid tumor in familial adenomatous polyposis. Ugeskr Laeg 162(42):5628–5631PubMed

10.

Sturt NJH, Gallagher MC, Bassett P, Philp CR, Neale KF, Tomlinson IPM et al (2004) Evidence for genetic predisposition to desmoid tumours in familial adenomatous polyposis independent of the germline APC mutation. Gut 53(12):1832–1836PubMedPubMedCentralCrossRef

11.

Nieuwenhuis MH, Lefevre JH, Bülow S, Järvinen H, Bertario L, Kernéis S et al (2011) Family history, surgery, and APC mutation are risk factors for desmoid tumors in familial adenomatous polyposis: an international cohort study. Dis Colon Rectum 54(10):1229–1234PubMedCrossRef

12.

Church J (2012) Desmoid disease in familial adenomatous polyposis. In: Litchman C (ed) Desmoid tumors. Springer, Dordrecht, p 147CrossRef

13.

Bertario L, Russo A, Sala P, Varesco L, Giarola M, Mondini P et al (2003) Multiple approach to the exploration of genotype–phenotype correlations in familial adenomatous polyposis. J Clin Oncol 21(9):1698–1707PubMedCrossRef

14.

Lynch HT, Fitzgibbons R Jr (1996) Surgery, desmoid tumors, and familial adenomatous polyposis: case report and literature review. Am J Gastroenterol 91(12):2598–2601PubMed

15.

Zeng W-G, Zhou Z-X, Liang J-W, Hou H-R, Wang Z, Zhou H-T et al (2014) Prognostic factors for desmoid tumor: a surgical series of 233 patients at a single institution. Tumour Biol 35(8):7513–7521PubMedCrossRef

16.

Reitamo JJ, Scheinin TM, Häyry P (1986) The desmoid syndrome. New aspects in the cause, pathogenesis and treatment of the desmoid tumor. Am J Surg 151(2):230–237PubMedCrossRef

17.

Reitamo JJ, Häyry P, Nykyri E, Saxén E (1982) The desmoid tumor. I. Incidence, sex-, age- and anatomical distribution in the Finnish population. Am J Clin Pathol 77(6):665–673PubMed

18.

Posner MC, Shiu MH, Newsome JL, Hajdu SI, Gaynor JJ, Brennan MF (1989) The desmoid tumor. Not a benign disease. Arch Surg 124(2):191–196PubMedCrossRef

19.

Gurbuz AK, Giardiello FM, Petersen GM, Krush AJ, Offerhaus GJ, Booker SV et al (1994) Desmoid tumours in familial adenomatous polyposis. Gut 35(3):377–381PubMedPubMedCentralCrossRef

20.

Nieuwenhuis MH, Cappel WDVTN, Botma A, Nagengast FM, Kleibeuker JH, Mathus-Vliegen EMH et al (2008) Desmoid tumors in a dutch cohort of patients with familial adenomatous polyposis. Clin Gastroenterol Hepatol 6(2):215–219PubMedCrossRef

21.

Bertario L, Russo A, Sala P, Eboli M, Giarola M, D’amico F et al (2001) Genotype and phenotype factors as determinants of desmoid tumors in patients with familial adenomatous polyposis. Int J Cancer 95(2):102–107PubMedCrossRef

22.

Durno C, Monga N, Bapat B, Berk T, Cohen Z, Gallinger S (2007) Does early colectomy increase desmoid risk in familial adenomatous polyposis? Clin Gastroenterol Hepatol 5(10):1190–1194PubMedCrossRef

23.

Schiessling S, Kihm M, Ganschow P, Kadmon G, Büchler MW, Kadmon M (2013) Desmoid tumour biology in patients with familial adenomatous polyposis coli. Br J Surg 100(5):694–703PubMedCrossRef

24.

Nieuwenhuis MH, Casparie M, Mathus-Vliegen LMH, Dekkers OM, Hogendoorn PCW, Vasen HFA (2011) A nation-wide study comparing sporadic and familial adenomatous polyposis-related desmoid-type fibromatoses. Int J Cancer 129(1):256–261PubMedCrossRef

25.

Lewis JJ, Boland PJ, Leung DH, Woodruff JM, Brennan MF (1999) The enigma of desmoid tumors. Ann Surg 229(6):866–872 (discussion 872-873) PubMedPubMedCentralCrossRef

26.

Caspari R, Olschwang S, Friedl W, Mandl M, Boisson C, Böker T et al (1995) Familial adenomatous polyposis: desmoid tumours and lack of ophthalmic lesions (CHRPE) associated with APC mutations beyond codon 1444. Hum Mol Genet 4(3):337–340PubMedCrossRef

27.

Davies DR, Armstrong JG, Thakker N, Horner K, Guy SP, Clancy T et al (1995) Severe Gardner syndrome in families with mutations restricted to a specific region of the APC gene. Am J Hum Genet 57(5):1151–1158PubMedPubMedCentral

28.

Church J, Xhaja X, LaGuardia L, O’Malley M, Burke C, Kalady M (2015) Desmoids and genotype in familial adenomatous polyposis. Dis Colon Rectum 58(4):444–448PubMedCrossRef

29.

Stoeckle E, Coindre JM, Longy M, Binh MBN, Kantor G, Kind M et al (2009) A critical analysis of treatment strategies in desmoid tumours: a review of a series of 106 cases. Eur J Surg Oncol 35(2):129–134PubMedCrossRef

30.

Latchford AR, Sturt NJH, Neale K, Rogers PA, Phillips RKS (2006) A 10-year review of surgery for desmoid disease associated with familial adenomatous polyposis. Br J Surg 93(10):1258–1264PubMedCrossRef

31.

Lahat G, Nachmany I, Itzkowitz E, Abu-Abeid S, Barazovsky E, Merimsky O et al (2009) Surgery for sporadic abdominal desmoid tumor: Is low/no recurrence an achievable goal? ISR Med Assoc J 11(7):398–402PubMed

32.

Berk T, Cohen Z, McLeod RS, Stern HS (1992) Management of mesenteric desmoid tumours in familial adenomatous polyposis. Can J Surg 35(4):393–395PubMed

33.

Fiore M, Rimareix F, Mariani L, Domont J, Collini P, Le Péchoux C et al (2009) Desmoid-type fibromatosis: a front-line conservative approach to select patients for surgical treatment. Ann Surg Oncol 16(9):2587–2593PubMedCrossRef

34.

Shido Y, Nishida Y, Nakashima H, Katagiri H, Sugiura H, Yamada Y et al (2009) Surgical treatment for local control of extremity and trunk desmoid tumors. Arch Orthop Trauma Surg 129(7):929–933PubMedCrossRef

35.

Kasper B, Ströbel P, Hohenberger P (2011) Desmoid tumors: clinical features and treatment options for advanced disease. Oncologist 16(5):682–693PubMedPubMedCentralCrossRef

36.

Nuyttens JJ, Rust PF, Thomas CR Jr, Turrisi AT 3rd (2000) Surgery versus radiation therapy for patients with aggressive fibromatosis or desmoid tumors: a comparative review of 22 articles. Cancer 88(7):1517–1523PubMedCrossRef

37.

Garbay D, Le Cesne A, Penel N, Chevreau C, Marec-Berard P, Blay J-Y et al (2012) Chemotherapy in patients with desmoid tumors: a study from the French Sarcoma Group (FSG). Ann Oncol 23(1):182–186PubMedCrossRef

38.

Chugh R, Wathen JK, Patel SR, Maki RG, Meyers PA, Schuetze SM et al (2010) Efficacy of imatinib in aggressive fibromatosis: results of a phase II multicenter Sarcoma Alliance for Research through Collaboration (SARC) trial. Clin Cancer Res 16(19):4884–4891PubMedCrossRef

39.

Leithner A, Schnack B, Katterschafka T, Wiltschke C, Amann G, Windhager R et al (2000) Treatment of extra-abdominal desmoid tumors with interferon-alpha with or without tretinoin. J Surg Oncol 73(1):21–25PubMedCrossRef

40.

Gounder MM, Lefkowitz RA, Keohan ML, D’Adamo DR, Hameed M, Antonescu CR et al (2011) Activity of Sorafenib against desmoid tumor/deep fibromatosis. Clin Cancer Res 17(12):4082–4090PubMedPubMedCentralCrossRef

41.

Clark SK, Neale KF, Landgrebe JC, Phillips RK (1999) Desmoid tumours complicating familial adenomatous polyposis. Br J Surg 86(9):1185–1189PubMedCrossRef

42.

Bocale D, Rotelli MT, Cavallini A, Altomare DF (2011) Anti-oestrogen therapy in the treatment of desmoid tumours: a systematic review. Colorectal Dis 13(12):e388–e395PubMedCrossRef

43.

Colombo C, Foo WC, Whiting D, Young ED, Lusby K, Pollock RE et al (2012) FAP-related desmoid tumors: a series of 44 patients evaluated in a cancer referral center. Histol Histopathol 27(5):641–649PubMed

44.

Tsukada K, Church JM, Jagelman DG, Fazio VW, McGannon E, George CR et al (1992) Noncytotoxic drug therapy for intra-abdominal desmoid tumor in patients with familial adenomatous polyposis. Dis Colon Rectum 35(1):29–33PubMedCrossRef

45.

Belliveau P, Graham AM (1984) Mesenteric desmoid tumor in Gardner’s syndrome treated by sulindac. Dis Colon Rectum 27(1):53–54PubMedCrossRef

46.

Cohen FJ, Watts S, Shah A, Akers R, Plouffe L Jr (2000) Uterine effects of 3-year raloxifene therapy in postmenopausal women younger than age 60. Obstet Gynecol 95(1):104–110PubMedCrossRef

47.

Shanle EK, Xu W (2010) Selectively targeting estrogen receptors for cancer treatment. Adv Drug Deliv Rev 62(13):1265–1276PubMedPubMedCentralCrossRef

48.

Brooks MD, Ebbs SR, Colletta AA, Baum M (1992) Desmoid tumours treated with triphenylethylenes. Eur J Cancer 28A(6–7):1014–1018PubMedCrossRef

49.

Lackner H, Urban C, Kerbl R, Schwinger W, Beham A (1997) Noncytotoxic drug therapy in children with unresectable desmoid tumors. Cancer 80(2):334–340PubMedCrossRef

50.

Bauernhofer T, Stöger H, Schmid M, Smola M, Gürtl-Lackner B, Höfler G et al (1996) Sequential treatment of recurrent mesenteric desmoid tumor. Cancer 77(6):1061–1065PubMedCrossRef

51.

Kinzbrunner B, Ritter S, Domingo J, Rosenthal CJ (1983) Remission of rapidly growing desmoid tumors after tamoxifen therapy. Cancer 52(12):2201–2204PubMedCrossRef

52.

Tonelli F, Ficari F, Valanzano R, Brandi ML (2003) Treatment of desmoids and mesenteric fibromatosis in familial adenomatous polyposis with raloxifene. Tumori 89(4):391–396PubMed

53.

Leithner A, Gapp M, Radl R, Pascher A, Krippl P, Leithner K et al (2005) Immunohistochemical analysis of desmoid tumours. J Clin Pathol 58(11):1152–1156PubMedPubMedCentralCrossRef

54.

Deyrup AT, Tretiakova M, Montag AG (2006) Estrogen receptor-beta expression in extraabdominal fibromatoses: an analysis of 40 cases. Cancer 106(1):208–213PubMedCrossRef

55.

Santos GAC, Cunha IW, Rocha RM, Mello CAL, Guimarães GC, Fregnani JH et al (2010) Evaluation of estrogen receptor alpha, estrogen receptor beta, progesterone receptor, and cKIT expression in desmoids tumors and their role in determining treatment options. Biosci Trends 4(1):25–30PubMed

56.

Lim CL, Walker MJ, Mehta RR, Gupta TK (1986) Estrogen and antiestrogen binding sites in desmoid tumors. Eur J Cancer Clin Oncol 22(5):583–587PubMedCrossRef

57.

Benson JR, Baum M (1993) Breast cancer, desmoid tumours, and familial adenomatous polyposis—a unifying hypothesis. Lancet 342(8875):848–850PubMedCrossRef

58.

Moeslein G (2006) Invited commentary. Fam Cancer 5(3):287–288CrossRef

59.

Colombo C, Miceli R, Le Péchoux C, Palassini E, Honoré C, Stacchiotti S et al (2015) Sporadic extra abdominal wall desmoid-type fibromatosis: surgical resection can be safely limited to a minority of patients. Eur J Cancer 51(2):186–192PubMedCrossRef

60.

Desurmont T, Lefèvre JH, Shields C, Colas C, Tiret E, Parc Y (2015) Desmoid tumour in familial adenomatous polyposis patients: responses to treatments. Fam Cancer 14(1):31–39PubMedCrossRef

61.

Quintini C, Ward G, Shatnawei A, Xhaja X, Hashimoto K, Steiger E et al (2012) Mortality of intra-abdominal desmoid tumors in patients with familial adenomatous polyposis: a single center review of 154 patients. Ann Surg 255(3):511–516PubMedCrossRef

62.

Bonvalot S, Ternès N, Fiore M, Bitsakou G, Colombo C, Honoré C et al (2013) Spontaneous regression of primary abdominal wall desmoid tumors: more common than previously thought. Ann Surg Oncol 20(13):4096–4102PubMedCrossRef

63.

Quast D, Schneider C, Burdzik E, Möslein G (2011) Abstracts of 2011 InSiGHT-Meeting: long-term outcome of FAP-associated and sporadic desmoid tumours in 154 patients. Fam Cancer 10(4):713–743CrossRef

64.

Skapek SX, Anderson JR, Hill DA, Henry D, Spunt SL, Meyer W, et al. (2012) Safety and efficacy of high-dose tamoxifen and sulindac for desmoid tumor in children: results of a Children’s Oncology Group (COG) Phase II Study. Pediatr Blood Cancer 60(7):1108–1112PubMedPubMedCentralCrossRef

65.

Honeyman JN, Theilen T-M, Knowles MA, McGlynn MM, Hameed M, Meyers P et al (2013) Desmoid fibromatosis in children and adolescents: a conservative approach to management. J Pediatr Surg 48(1):62–66PubMedCrossRef

66.

Burgess A, Xhaja X, Church J (2011) Does intra-abdominal desmoid disease affect patients with an ileal pouch differently than those with an ileorectal anastomosis? Dis Colon Rectum 54(11):1388–1391PubMedCrossRef

67.

Lazar AJF, Tuvin D, Hajibashi S, Habeeb S, Bolshakov S, Mayordomo-Aranda E et al (2008) Specific mutations in the beta-catenin gene (CTNNB1) correlate with local recurrence in sporadic desmoid tumors. Am J Pathol 173(5):1518–1527PubMedPubMedCentralCrossRef

68.

Bo N, Wang D, Wu B, Chen L, Ma R (2011) Analysis of β-catenin expression and exon 3 mutations in pediatric sporadic aggressive fibromatosis. Pediatr Dev Pathol [Internet]. [cited 2012 Aug 9]; http://www.ncbi.nlm.nih.gov/pubmed/21323417

69.

Matono H, Tamiya S, Yokoyama R, Saito T, Iwamoto Y, Tsuneyoshi M et al (2011) Abnormalities of the Wnt/β-catenin signalling pathway induce tumour progression in sporadic desmoid tumours: correlation between β-catenin widespread nuclear expression and VEGF overexpression. Histopathology 59(3):368–375PubMedCrossRef

70.

Kattentidt Mouravieva AA, Geurts-Giele IRR, de Krijger RR, van Noesel MM, van de Ven CP, van den Ouweland AMW et al (2012) Identification of familial adenomatous polyposis carriers among children with desmoid tumours. Eur J Cancer 48(12):1867–1874PubMedCrossRef

71.

Mullen JT, DeLaney TF, Rosenberg AE, Le L, Iafrate AJ, Kobayashi W et al (2013) β-Catenin mutation status and outcomes in sporadic desmoid tumors. Oncologist 18(9):1043–1049PubMedPubMedCentralCrossRef

Titel: 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
verfasst von: Daniel Robert Quast
Ralph Schneider
Emanuel Burdzik
Steffen Hoppe
Gabriela Möslein
Publikationsdatum: 01.01.2016
Verlag: Springer Netherlands
Erschienen in: Familial Cancer / Ausgabe 1/2016
Print ISSN: 1389-9600
Elektronische ISSN: 1573-7292
DOI: https://doi.org/10.1007/s10689-015-9830-z

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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

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