Pazopanib is an active treatment in desmoid tumour/aggressive fibromatosis

Clinical Sarcoma Research

Ian Judson¹  Christina Messiou¹  Khin Thway¹  Heather McCarty²  Charlotte Benson¹  Juan Martin-Liberal¹ 

[1] The Royal Marsden Hospital, Sarcoma Unit, Fulham Road, SW3 6JJ London, UK;Belfast City Hospital, Lisburn Road, BT9 7AB Belfast, UK

关键词: VEGFR;    Sorafenib;    Pegylated doxorubicin;    PDGFR;    Pazopanib;    Imatinib;    Desmoid tumour;    Aggressive fibromatosis;   

Others  :  861366
DOI  :  10.1186/2045-3329-3-13

 received in 2013-11-12, accepted in 2013-11-21,  发布年份 2013

【 摘 要 】

Background

Desmoid tumours/aggressive fibromatosis (DT/AF) are infrequent soft-tissue neoplasms. They usually behave as indolent diseases. However, they may grow locally infiltrating or compressing adjacent structures. The role of local treatment is limited and only a few drugs have shown activity.

Cases presentation

We report the outcome of two patients affected by progressive DT/AF treated with the angiogenesis inhibitor pazopanib in two different institutions. Both patients achieved dramatic improvement in their symptoms and radiological signs of response. The clinical benefit lasted for more than 1 year and it is still ongoing.

Conclusions

Pazopanib is an active treatment in DT/AF. It is the first time this has been reported.

【 授权许可】

   
2013 Martin-Liberal et al.; licensee BioMed Central Ltd.

【 图 表 】

【 参考文献 】

• [1]Alman BA, Pajerski ME, Diaz-Cano S, Corboy K, Wolfe HJ: Aggressive fibromatosis (desmoid tumor) is a monoclonal disorder. Diagn Mol Pathol 1997, 6(2):98-101.
• [2]Biermann JS: Desmoid tumors. Curr Treat Options Oncol 2000, 1(3):262-266.
• [3]Bertagnolli MM, Morgan JA, Fletcher CD, Raut CP, Dileo P, Gill RR, Demetri GD, George S: Multimodality treatment of mesenteric desmoid tumours. Eur J Cancer 2008, 44(16):2404-2410.
• [4]Bonvalot S, Desai A, Coppola S, Le Péchoux C, Terrier P, Dômont J, Le Cesne A: The treatment of desmoid tumors: a stepwise clinical approach. Ann Oncol 2012, 23(Suppl 10):158-166.
• [5]Smith AJ, Lewis JJ, Merchant NB, Leung DH, Woodruff JM, Brennan MF: Surgical management of intra-abdominal desmoid tumours. Br J Surg 2000, 87(5):608-613.
• [6]Ballo MT, Zagars GK, Pollack A, Pisters PW, Pollack RA: Desmoid tumor: prognostic factors and outcome after surgery, radiation therapy, or combined surgery and radiation therapy. J Clin Oncol 1999, 17(1):158-167.
• [7]Hansmann A, Adolph C, Vogel T, Unger A, Moeslein G: High-dose tamoxifen and sulindac as first-line treatment for desmoid tumors. Cancer 2004, 100(3):612-620.
• [8]Deyrup AT, Tretiakova M, Montag AG: Estrogen receptor-beta expression in extraabdominal fibromatoses: an analysis of 40 cases. Cancer 2006, 106(1):208-213.
• [9]Constantinidou A, Jones RL, Scurr M, Al-Muderis O, Judson I: Pegylated liposomal doxorubicin, an effective, well-tolerated treatment for refractory aggressive fibromatosis. Eur J Cancer 2009, 45(17):2930-2934.
• [10]Heinrich MC, McArthur GA, Demetri GD, Joensuu H, Bono P, Herrmann R, Hirte H, Cresta S, Koslin DB, Corless CL, Dirnhofer S, van Oosterom AT, Nikolova Z, Dimitrijevic S, Fletcher JA: Clinical and molecular studies of the effect of imatinib on advanced aggressive fibromatosis (desmoid tumor). J Clin Oncol 2006, 24(7):1195-1203.
• [11]Penel N, Le Cesne A, Bui BN, Perol D, Brain EG, Ray-Coquard I, Guillemet C, Chevreau C, Cupissol D, Chabaud S, Jimenez M, Duffaud F, Piperno-Neumann S, Mignot L, Blay JY: 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.
• [12]Gounder MM, Lefkowitz RA, Keohan ML, D'Adamo DR, Hameed M, Antonescu CR, Singer S, Stout K, Ahn L, Maki RG: Activity of Sorafenib against desmoid tumor/deep fibromatosis. Clin Cancer Res 2011, 17(12):4082-4090.
• [13]Li M, Cordon-Cardo C, Gerald WL, Rosai J: Desmoid fibromatosis is a clonal process. Hum Pathol 1996, 27(9):939-943.
• [14]Martin-Liberal J, Judson I, Benson C: Antiangiogenic approach in soft-tissue sarcomas. Expert Rev Anticancer Ther 2013, 13(8):975-982.
• [15]Potti A, Ganti AK, Tendulkar K, Sholes K, Chitajallu S, Koch M, Kargas S: Determination of vascular endothelial growth factor (VEGF) overexpression in soft tissue sarcomas and the role of overexpression in leiomyosarcoma. J Cancer Res Clin Oncol 2004, 130(1):52-56.
• [16]Graeven U, Andre N, Achilles E, Zornig C, Schmiegel W: Serum levels of vascular endothelial growth factor and basic fibroblast growth factor in patients with soft-tissue sarcoma. J Cancer Res Clin Oncol 1999, 125(10):577-581.
• [17]Hayes AJ, Mostyn-Jones A, Koban MU, A'Hern R, Burton P, Thomas JM: Serum vascular endothelial growth factor as a tumour marker in soft tissue sarcoma. Br J Surg 2004, 91(2):242-247.
• [18]Chao C, Al-Saleem T, Brooks JJ, Rogatko A, Kraybill WG, Eisenberg B: Vascular endothelial growth factor and soft tissue sarcomas: tumor expression correlates with grade. Ann Surg Oncol 2001, 8(3):260-267.
• [19]Iyoda A, Hiroshima K, Baba M, Fujisawa T, Yusa T, Ohwada H: Expression of vascular endothelial growth factor in thoracic sarcomas. Ann Thorac Surg 2001, 71(5):1635-1639.
• [20]George D: Platelet-derived growth factor receptors: a therapeutic target in solid tumors. Semin Oncol 2001, 28(5 Suppl 17):27-33.
• [21]Négrier S, Raymond E: Antiangiogenic treatments and mechanisms of action in renal cell carcinoma. Invest New Drugs 2012, 30(4):1791-1801.
• [22]Savage DG, Antman KH: Imatinib mesylate–a new oral targeted therapy. N Engl J Med 2002, 346(9):683-693.
• [23]van der Graaf WT, Blay JY, Chawla SP, Kim DW, Bui-Nguyen B, Casali PG, Schöffski P, Aglietta M, Staddon AP, Beppu Y, Le Cesne A, Gelderblom H, Judson IR, Araki N, Ouali M, Marreaud S, Hodge R, Dewji MR, Coens C, Demetri GD, Fletcher CD, Dei Tos AP, Hohenberger P, EORTC Soft Tissue and Bone Sarcoma Group: Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet 2012, 379(9829):1879-1886.